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• Glycemic Characterization and Pancreatic Imaging Correlates in Cystic Fibrosis

  NCT03961516

  Conditions:   Cystic Fibrosis;   Cystic Fibrosis-related Diabetes;   Cystic Fibrosis Liver Disease;   Pancreatic Steatosis;   Hepatic Steatosis
  Interventions:   Device: Frequently Sampled Oral Glucose Tolerance Test and CGM;   Radiation: MRI Pancreas and Liver
  Sponsor:   University Hospitals Cleveland Medical Center
  Recruiting
• CyberCycling to Reduce Therapeutic Side Effects and Improve Quality of Life in AYA Cancers

  NCT02916472

  Condition:   Adolescent and Young Adult Cancers
  Interventions:   Behavioral: CyberCycling;   Behavioral: Stretching;   Behavioral: Resistance Bands
  Sponsor:   Case Comprehensive Cancer Center
  Recruiting
• Restoring High Dimensional Hand Function to Persons With Chronic High Tetraplegia

  NCT03482310

  Conditions:   Spinal Cord Injuries;   Tetraplegia;   Quadriplegia
  Intervention:   Device: Using the Neuroport cortical recording array to determine the desired grasp pattern for a functional electrical stimulation (FES) system
  Sponsors:   VA Office of Research and Development;   Case Western Reserve University
  Recruiting
• Getting Ahead for Cancer Control

  NCT03247634

  Condition:   Cancer Prevention
  Intervention:   Behavioral: Getting Ahead in a Just-Gettin'-By World
  Sponsor:   Case Comprehensive Cancer Center
  Recruiting
• Comparing the Efficacy of Epidural and Quadratus Lumborum Analgesia After Open Nephrectomy Surgery

  NCT03110081

  Condition:   Open Nephrectomy Surgery
  Interventions:   Procedure: Quadratus lumborum block;   Procedure: Epidural analgesia
  Sponsor:   The Cleveland Clinic
  Recruiting
• ACTHAR Therapy for Central Nervous System Sarcoidosis

  NCT02920710

  Condition:   Sarcoidosis
  Intervention:   Drug: Repository Corticotropin Injection
  Sponsors:   The Cleveland Clinic;   Mallinckrodt
  Recruiting
• Enlarged Vestibular Aqueduct Registry

  NCT02798783

  Conditions:   Enlarged Vestibular Aqueduct;   Sensorineural Hearing Loss
  Intervention:  
  Sponsor:   Todd Otteson, MD, MPH, FAAP, FACS
  Recruiting
• Genetics of Uveitis

  NCT02357238

  Conditions:   Uveitis;   Infectious Uveitis
  Intervention:  
  Sponsor:   The Cleveland Clinic
  Recruiting
• Analysis of Anterior Chamber Inflammation by Optical Coherence Tomography

  NCT01746537

  Condition:   Uveitis
  Intervention:  
  Sponsor:   The Cleveland Clinic
  Recruiting
• Inotuzumab Ozogamicin Post-Transplant For Acute Lymphocytic Leukemia

  NCT03104491

  Condition:   Cluster of Differentiation Antigen (CD22)-Positive Acute Lymphoblastic Leukemia
  Intervention:   Drug: Inotuzumab Ozogamicin
  Sponsor:   Leland Metheny
  Recruiting
• Tac, Mini-MTX, MMF Versus Tac, MTX for GVHD Prevention

  NCT01951885

  Conditions:   Chronic Myelogenous Leukemia;   Acute Lymphoblastic Leukemia;   Acute Myeloid Leukemia;   Acute Biphenotypic Leukemia;   Myelodysplastic Syndrome;   Myeloproliferative Neoplasm;   Non-Hodgkin Lymphoma;   Hodgkins Disease;   Chronic Lymphocytic Leukemia;   Multiple Myeloma
  Interventions:   Drug: tacrolimus;   Drug: methotrexate;   Drug: Mycophenolate mofetil;   Drug: Methotrexate (low dose)
  Sponsors:   Case Comprehensive Cancer Center;   National Cancer Institute (NCI)
  Recruiting
• Project Guard: Reducing Alcohol Misuse/Abuse in the National Guard

  NCT02860442

  Condition:   Alcohol Use
  Interventions:   Behavioral: Smartphone brief intervention (SP-BI);   Other: Enhanced Usual Care (EUC)
  Sponsors:   Joseph Calabrese, MD;   Case Western Reserve University;   University of Michigan;   University of Toledo Health Science Campus
  Recruiting
• RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome

  NCT02991807

  Conditions:   PTEN Gene Mutation;   PTEN Hamartoma Tumor Syndrome
  Interventions:   Drug: RAD001;   Drug: Placebo
  Sponsors:   Boston Children’s Hospital;   National Institutes of Health (NIH);   National Institute of Neurological Disorders and Stroke (NINDS);   Office of Rare Diseases (ORD);   National Center for Advancing Translational Science (NCATS);   Novartis Pharmaceuticals;   PTEN Research;   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  Recruiting
• Comparative Effectiveness of EIBI and Adaptive ABA for Children With Autism

  NCT04078061

  Condition:   Autism Spectrum Disorder
  Interventions:   Behavioral: MABA;   Behavioral: EIBI
  Sponsors:   University of Rochester;   The Cleveland Clinic;   May Institute;   Nationwide Children's Hospital;   Vanderbilt University Medical Center;   University of Michigan;   University of Virginia
  Recruiting
• Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome

  NCT03383575

  Conditions:   Acute Myeloid Leukemia;   Blasts 20-30 Percent of Bone Marrow Nucleated Cells;   Chronic Myelomonocytic Leukemia;   IDH2 Gene Mutation;   Myelodysplastic Syndrome With Excess Blasts;   Recurrent High Risk Myelodysplastic Syndrome;   Refractory High Risk Myelodysplastic Syndrome
  Interventions:   Drug: Azacitidine;   Drug: Enasidenib;   Other: Quality-of-Life Assessment
  Sponsors:   M.D. Anderson Cancer Center;   National Cancer Institute (NCI);   Celgene
  Recruiting
• Tumor Necrosis Factor Inhibition in Focal Segmental Glomerulosclerosis and Treatment Resistant Minimal Change Disease

  NCT04009668

  Conditions:   FSGS;   MCD;   Focal Segmental Glomerulosclerosis;   Minimal Change Disease
  Intervention:   Drug: adalimumab
  Sponsors:   University of Michigan;   New York University
  Recruiting
• Sickle Cell Disease, Hemechip

  NCT03948516

  Condition:   Sickle Cell Disease
  Intervention:   Device: HemeChip
  Sponsors:   University Hospitals Cleveland Medical Center;   US federal government;   Amino Kano Teaching Hospital;   Murtala Mohammed Specialist Hospital;   Hasiya Bayero Pediatric Hospital;   University of Nebraska;   University of North Carolina, Chapel Hill;   Case Western Reserve University
  Recruiting
• A Study to Evaluate the Safety and Efficacy of MEDI8897 for the Prevention of Medically Attended RSV LRTI in Healthy Late Preterm and Term Infants

  NCT03979313

  Condition:   Respiratory Syncytial Virus Infections
  Interventions:   Drug: MEDI8897;   Drug: Placebo
  Sponsor:   MedImmune LLC
  Recruiting
• Safety and Efficacy of Itacitinib in Combination With Corticosteroids for Treatment of Graft-Versus-Host Disease in Pediatric Subjects

  NCT03721965

  Condition:   Acute Graft-versus-host Disease
  Interventions:   Drug: Itacitinib;   Drug: Corticosteroids
  Sponsor:   Incyte Corporation
  Recruiting
• National Collaborative to Improve Care of Children With Complex Congenital Heart Disease

  NCT02852031

  Condition:   Hypoplastic Left Heart Syndrome (HLHS)
  Intervention:   Other: Collaborative Learning Network
  Sponsor:   Children's Hospital Medical Center, Cincinnati
  Recruiting
• Dextromethorphan Pediatric Acute Cough Study

  NCT02651116

  Condition:   Cough
  Interventions:   Drug: Dextromethorphan Hydrobromide;   Drug: Placebo;   Device: Cough recording device
  Sponsor:   Pfizer
  Recruiting
• Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome

  NCT02521493

  Conditions:   Acute Myeloid Leukemia;   Blasts 5 Percent or More of Bone Marrow Nucleated Cells;   Cytopenia;   Down Syndrome;   Myelodysplastic Syndrome;   Myeloid Leukemia Associated With Down Syndrome;   Myeloproliferative Neoplasm;   Trisomy 21;   Trisomy 21 Mosaicism
  Interventions:   Drug: Asparaginase;   Drug: Asparaginase Erwinia chrysanthemi;   Drug: Cytarabine;   Drug: Daunorubicin;   Drug: Daunorubicin Hydrochloride;   Drug: Etoposide;   Other: Laboratory Biomarker Analysis;   Drug: Mitoxantrone;   Drug: Mitoxantrone Hydrochloride;   Drug: Thioguanine
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• Apixaban for the Acute Treatment of Venous Thromboembolism in Children

  NCT02464969

  Condition:   Venous Thromboembolism
  Interventions:   Drug: Apixaban;   Drug: Standard of Care
  Sponsors:   Pfizer;   Bristol-Myers Squibb
  Recruiting
• A Study of the Safety and Effectiveness of Apixaban in Preventing Blood Clots in Children With Leukemia Who Have a Central Venous Catheter and Are Treated With Asparaginase

  NCT02369653

  Conditions:   Lymphoma;   Acute Lymphoblastic Leukemia
  Interventions:   Drug: Apixaban;   Other: No systemic anticoagulant prophylaxis
  Sponsors:   Bristol-Myers Squibb;   Pfizer
  Recruiting
• Response and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma

  NCT02176967

  Conditions:   Ganglioneuroblastoma;   Localized Resectable Neuroblastoma;   Localized Unresectable Neuroblastoma;   Neuroblastoma
  Interventions:   Drug: Carboplatin;   Other: Clinical Observation;   Drug: Cyclophosphamide;   Drug: Doxorubicin Hydrochloride;   Drug: Etoposide;   Other: Laboratory Biomarker Analysis;   Other: Pharmacological Study
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• Healthy Sleep for Children With Down Syndrome

  NCT03922165

  Conditions:   Down Syndrome;   Sleep Disordered Breathing
  Intervention:   Procedure: Adenotonsillectomy
  Sponsors:   Brigham and Women's Hospital;   Children's Hospital of Philadelphia;   University Hospitals Cleveland Medical Center;   Children's Hospital Medical Center, Cincinnati;   University of Michigan;   University of Texas Southwestern Medical Center;   University of Rochester;   Children's Hospital of The King's Daughters
  Recruiting
• Active Surveillance, Bleomycin, Carboplatin, Etoposide, or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors

  NCT03067181

  Conditions:   Adult Germ Cell Tumor;   Childhood Extracranial Germ Cell Tumor;   Childhood Germ Cell Tumor;   Extragonadal Embryonal Carcinoma;   Grade 2 Ovarian Teratoma;   Malignant Germ Cell Tumor;   Malignant Ovarian Teratoma;   Stage I Ovarian Choriocarcinoma;   Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7;   Stage I Ovarian Teratoma AJCC v6 and v7;   Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7;   Stage I Testicular Choriocarcinoma AJCC v6 and v7;   Stage I Testicular Embryonal Carcinoma AJCC v6 and v7;   Stage I Testicular Yolk Sac Tumor AJCC v6 and v7;   Stage II Ovarian Choriocarcinoma;   Stage II Ovarian Embryonal Carcinoma AJCC v6 and v7;   Stage II Ovarian Yolk Sac Tumor AJCC v6 and v7;   Stage II Testicular Choriocarcinoma AJCC v6 and v7;   Stage II Testicular Embryonal Carcinoma AJCC v6 and v7;   Stage II Testicular Yolk Sac Tumor AJCC v6 and v7;   Stage III Ovarian Choriocarcinoma;   Stage III Ovarian Embryonal Carcinoma AJCC v6 and v7;   Stage III Ovarian Yolk Sac Tumor AJCC v6 and v7;   Stage III Testicular Choriocarcinoma AJCC v6 and v7;   Stage III Testicular Embryonal Carcinoma AJCC v6 and v7;   Stage III Testicular Yolk Sac Tumor AJCC v6 and v7;   Stage IV Ovarian Choriocarcinoma;   Stage IV Ovarian Embryonal Carcinoma AJCC v6 and v7;   Stage IV Ovarian Yolk Sac Tumor AJCC v6 and v7;   Testicular Mixed Choriocarcinoma and Embryonal Carcinoma;   Testicular Mixed Choriocarcinoma and Teratoma;   Testicular Mixed Choriocarcinoma and Yolk Sac Tumor
  Interventions:   Other: Best Practice;   Drug: Bleomycin;   Biological: Bleomycin Sulfate;   Drug: Carboplatin;   Drug: Cisplatin;   Drug: Etoposide;   Drug: Etoposide Phosphate;   Other: Laboratory Biomarker Analysis;   Other: Pharmacogenomic Study;   Other: Quality-of-Life Assessment;   Other: Questionnaire Administration
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors

  NCT02867592

  Conditions:   Adrenal Cortex Carcinoma;   Alveolar Soft Part Sarcoma;   Central Nervous System Neoplasm;   Childhood Clear Cell Sarcoma of Soft Parts;   Clear Cell Sarcoma of Soft Tissue;   Ewing Sarcoma;   Hepatoblastoma;   Hepatocellular Carcinoma;   Osteosarcoma;   Recurrent Adrenal Cortex Carcinoma;   Recurrent Alveolar Soft Part Sarcoma;   Recurrent Ewing Sarcoma;   Recurrent Hepatoblastoma;   Recurrent Hepatocellular Carcinoma;   Recurrent Malignant Central Nervous System Neoplasm;   Recurrent Malignant Solid Neoplasm;   Recurrent Osteosarcoma;   Recurrent Renal Cell Carcinoma;   Recurrent Rhabdomyosarcoma;   Recurrent Soft Tissue Sarcoma;   Recurrent Thyroid Gland Medullary Carcinoma;   Refractory Ewing Sarcoma;   Refractory Malignant Central Nervous System Neoplasm;   Refractory Malignant Solid Neoplasm;   Refractory Osteosarcoma;   Refractory Rhabdomyosarcoma;   Refractory Soft Tissue Sarcoma;   Renal Cell Carcinoma;   Rhabdomyosarcoma;   Solid Neoplasm;   Thyroid Gland Medullary Carcinoma;   Wilms Tumor
  Interventions:   Drug: Cabozantinib;   Drug: Cabozantinib S-malate;   Other: Pharmacological Study
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• DOSE Trial of Opioid Sparing Effect

  NCT03938857

  Conditions:   Dexmedetomidine;   Mechanical Ventilation Complication;   Critically Ill
  Interventions:   Drug: Fentanyl;   Drug: Dexmedetomidine
  Sponsors:   Duke University;   National Institutes of Health (NIH);   Johns Hopkins University;   Intermountain Health Care, Inc.;   Vanderbilt University Medical Center
  Recruiting
• A Study to Determine the Outcomes of Patients With Localized B Cell Lymphoblastic Lymphoma (B-LLy) When Treated With Standard Risk B-ALL Therapy

  NCT03914625

  Conditions:   B Acute Lymphoblastic Leukemia;   B Lymphoblastic Lymphoma;   Down Syndrome
  Interventions:   Drug: Asparaginase Erwinia chrysanthemi;   Biological: Blinatumomab;   Drug: Cyclophosphamide;   Drug: Cytarabine;   Drug: Dexamethasone;   Drug: Doxorubicin;   Drug: Doxorubicin Hydrochloride;   Drug: Leucovorin;   Drug: Leucovorin Calcium;   Drug: Mercaptopurine;   Drug: Mercaptopurine Oral Suspension;   Drug: Methotrexate;   Drug: Prednisolone;   Drug: Prednisone;   Drug: Thioguanine;   Drug: Vincristine;   Drug: Vincristine Sulfate
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• Hydroxyurea Optimization Through Precision Study

  NCT03789591

  Conditions:   Sickle Cell Disease;   Sickle Cell Anemia
  Intervention:   Drug: Hydroxyurea
  Sponsors:   Children's Hospital Medical Center, Cincinnati;   Doris Duke Charitable Foundation
  Recruiting
• Veliparib, Radiation Therapy, and Temozolomide in Treating Patients With Newly Diagnosed Malignant Glioma Without H3 K27M or BRAFV600 Mutations

  NCT03581292

  Conditions:   Anaplastic Astrocytoma;   Glioblastoma;   Malignant Glioma
  Interventions:   Radiation: Radiation Therapy;   Drug: Temozolomide;   Drug: Veliparib
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• Cisplatin and Combination Chemotherapy in Treating Children and Young Adults With Hepatoblastoma or Liver Cancer After Surgery

  NCT03533582

  Conditions:   Childhood Hepatocellular Carcinoma;   Childhood Malignant Liver Neoplasm;   Elevated Alpha-Fetoprotein;   Hepatoblastoma;   Hepatocellular Malignant Neoplasm, Not Otherwise Specified;   SMARCB1 Gene Mutation
  Interventions:   Drug: Carboplatin;   Drug: Cisplatin;   Drug: Doxorubicin;   Drug: Etoposide;   Drug: Fluorouracil;   Drug: Gemcitabine;   Drug: Irinotecan;   Other: Laboratory Biomarker Analysis;   Drug: Oxaliplatin;   Other: Patient Observation;   Drug: Sorafenib;   Drug: Vincristine Sulfate
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• Larotrectinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With NTRK Fusions (A Pediatric MATCH Treatment Trial)

  NCT03213704

  Conditions:   Advanced Malignant Solid Neoplasm;   Recurrent Childhood Ependymoma;   Recurrent Childhood Malignant Germ Cell Tumor;   Recurrent Childhood Medulloblastoma;   Recurrent Childhood Non-Hodgkin Lymphoma;   Recurrent Childhood Rhabdomyosarcoma;   Recurrent Childhood Soft Tissue Sarcoma;   Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor;   Recurrent Glioma;   Recurrent Hepatoblastoma;   Recurrent Langerhans Cell Histiocytosis;   Recurrent Malignant Glioma;   Recurrent Malignant Solid Neoplasm;   Recurrent Neuroblastoma;   Recurrent Osteosarcoma;   Recurrent Rhabdoid Tumor;   Refractory Central Nervous System Neoplasm;   Refractory Childhood Malignant Germ Cell Tumor;   Refractory Ependymoma;   Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor;   Refractory Glioma;   Refractory Hepatoblastoma;   Refractory Langerhans Cell Histiocytosis;   Refractory Malignant Glioma;   Refractory Malignant Solid Neoplasm;   Refractory Neuroblastoma;   Refractory Non-Hodgkin Lymphoma;   Refractory Osteosarcoma;   Refractory Rhabdoid Tumor;   Refractory Rhabdomyosarcoma;   Wilms Tumor
  Interventions:   Drug: Larotrectinib;   Drug: Larotrectinib Sulfate
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• PI3K/mTOR Inhibitor LY3023414 in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial)

  NCT03213678

  Conditions:   Advanced Malignant Solid Neoplasm;   Ann Arbor Stage III Non-Hodgkin Lymphoma;   Ann Arbor Stage IV Non-Hodgkin Lymphoma;   Malignant Glioma;   Recurrent Central Nervous System Neoplasm;   Recurrent Ependymoma;   Recurrent Ewing Sarcoma;   Recurrent Glioma;   Recurrent Hepatoblastoma;   Recurrent Langerhans Cell Histiocytosis;   Recurrent Malignant Germ Cell Tumor;   Recurrent Malignant Solid Neoplasm;   Recurrent Medulloblastoma;   Recurrent Neuroblastoma;   Recurrent Non-Hodgkin Lymphoma;   Recurrent Osteosarcoma;   Recurrent Peripheral Primitive Neuroectodermal Tumor;   Recurrent Rhabdomyosarcoma;   Recurrent Soft Tissue Sarcoma;   Refractory Central Nervous System Neoplasm;   Refractory Langerhans Cell Histiocytosis;   Refractory Malignant Germ Cell Tumor;   Refractory Malignant Solid Neoplasm;   Refractory Neuroblastoma;   Refractory Non-Hodgkin Lymphoma;   Rhabdoid Tumor;   Stage III Osteosarcoma AJCC v7;   Stage III Soft Tissue Sarcoma AJCC v7;   Stage IV Osteosarcoma AJCC v7;   Stage IV Soft Tissue Sarcoma AJCC v7;   Stage IVA Osteosarcoma AJCC v7;   Stage IVB Osteosarcoma AJCC v7;   Wilms Tumor
  Interventions:   Other: Laboratory Biomarker Analysis;   Other: Pharmacological Study;   Drug: Samotolisib
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)

  NCT03155620

  Conditions:   Advanced Malignant Solid Neoplasm;   Ann Arbor Stage III Non-Hodgkin Lymphoma;   Ann Arbor Stage IV Non-Hodgkin Lymphoma;   Histiocytic Sarcoma;   Juvenile Xanthogranuloma;   Langerhans Cell Histiocytosis;   Malignant Glioma;   Recurrent Central Nervous System Neoplasm;   Recurrent Childhood Rhabdomyosarcoma;   Recurrent Ependymoma;   Recurrent Ewing Sarcoma;   Recurrent Glioma;   Recurrent Hepatoblastoma;   Recurrent Langerhans Cell Histiocytosis;   Recurrent Malignant Germ Cell Tumor;   Recurrent Malignant Solid Neoplasm;   Recurrent Medulloblastoma;   Recurrent Neuroblastoma;   Recurrent Non-Hodgkin Lymphoma;   Recurrent Osteosarcoma;   Recurrent Peripheral Primitive Neuroectodermal Tumor;   Recurrent Rhabdoid Tumor;   Recurrent Soft Tissue Sarcoma;   Refractory Central Nervous System Neoplasm;   Refractory Ewing Sarcoma;   Refractory Glioma;   Refractory Hepatoblastoma;   Refractory Langerhans Cell Histiocytosis;   Refractory Malignant Germ Cell Tumor;   Refractory Malignant Solid Neoplasm;   Refractory Medulloblastoma;   Refractory Neuroblastoma;   Refractory Non-Hodgkin Lymphoma;   Refractory Osteosarcoma;   Refractory Peripheral Primitive Neuroectodermal Tumor;   Refractory Rhabdoid Tumor;   Refractory Rhabdomyosarcoma;   Rhabdoid Tumor;   Stage III Osteosarcoma AJCC v7;   Stage III Soft Tissue Sarcoma AJCC v7;   Stage IV Osteosarcoma AJCC v7;   Stage IV Soft Tissue Sarcoma AJCC v7;   Stage IVA Osteosarcoma AJCC v7;   Stage IVB Osteosarcoma AJCC v7;   Wilms Tumor
  Interventions:   Procedure: Biopsy;   Procedure: Biospecimen Collection;   Drug: Ensartinib;   Drug: Erdafitinib;   Other: Laboratory Biomarker Analysis;   Drug: Larotrectinib;   Procedure: Mutation Carrier Screening;   Drug: Olaparib;   Drug: Palbociclib;   Other: Pharmacological Study;   Drug: Samotolisib;   Drug: Selumetinib Sulfate;   Drug: Tazemetostat;   Drug: Ulixertinib;   Drug: Vemurafenib
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• Iobenguane I-131 or Crizotinib and Standard Therapy in Treating Younger Patients With Newly-Diagnosed High-Risk Neuroblastoma or Ganglioneuroblastoma

  NCT03126916

  Conditions:   Ganglioneuroblastoma;   INRG Stage L2;   INRG Stage M;   INRG Stage MS;   Neuroblastoma;   NMYC Gene Amplification;   Recurrent Neuroblastoma
  Interventions:   Biological: Aldesleukin;   Procedure: Autologous Hematopoietic Stem Cell Transplantation;   Drug: Busulfan;   Drug: Carboplatin;   Drug: Cisplatin;   Drug: Crizotinib;   Drug: Cyclophosphamide;   Drug: Dexrazoxane Hydrochloride;   Biological: Dinutuximab;   Drug: Doxorubicin Hydrochloride;   Drug: Etoposide Phosphate;   Radiation: External Beam Radiation Therapy;   Radiation: Iobenguane I-131;   Drug: Isotretinoin;   Other: Laboratory Biomarker Analysis;   Drug: Melphalan;   Other: Pharmacological Study;   Biological: Sargramostim;   Procedure: Therapeutic Conventional Surgery;   Drug: Thiotepa;   Drug: Topotecan Hydrochloride;   Drug: Vincristine Sulfate
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• Imatinib Mesylate and Combination Chemotherapy in Treating Patients With Newly Diagnosed Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia

  NCT03007147

  Conditions:   B Acute Lymphoblastic Leukemia;   BCR-ABL1 Fusion Protein Expression;   Minimal Residual Disease;   Philadelphia Chromosome Positive;   T Acute Lymphoblastic Leukemia;   Untreated Adult Acute Lymphoblastic Leukemia;   Untreated Childhood Acute Lymphoblastic Leukemia
  Interventions:   Procedure: Allogeneic Hematopoietic Stem Cell Transplantation;   Drug: Cyclophosphamide;   Drug: Cytarabine;   Drug: Daunorubicin Hydrochloride;   Drug: Dexamethasone;   Drug: Dexrazoxane Hydrochloride;   Drug: Doxorubicin;   Drug: Etoposide;   Biological: Filgrastim;   Drug: Ifosfamide;   Drug: Imatinib Mesylate;   Other: Laboratory Biomarker Analysis;   Drug: Leucovorin Calcium;   Drug: Mercaptopurine;   Drug: Methotrexate;   Drug: Methylprednisolone;   Drug: Pegaspargase;   Drug: Prednisolone;   Other: Questionnaire Administration;   Drug: Therapeutic Hydrocortisone;   Drug: Thioguanine;   Drug: Vincristine Sulfate
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI);   University of Milano Bicocca
  Recruiting
• Reduced Craniospinal Radiation Therapy and Chemotherapy in Treating Younger Patients With Newly Diagnosed WNT-Driven Medulloblastoma

  NCT02724579

  Conditions:   CTNNB1 Gene Mutation;   Medulloblastoma;   Wnt Positive
  Interventions:   Drug: Cisplatin;   Drug: Cyclophosphamide;   Other: Laboratory Biomarker Analysis;   Drug: Lomustine;   Radiation: Radiation Therapy;   Drug: Vincristine;   Drug: Vincristine Sulfate
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• Combination Chemotherapy With or Without Temsirolimus in Treating Patients With Intermediate Risk Rhabdomyosarcoma

  NCT02567435

  Conditions:   Alveolar Rhabdomyosarcoma;   Botryoid-Type Embryonal Rhabdomyosarcoma;   Embryonal Rhabdomyosarcoma;   Rhabdomyosarcoma;   Sclerosing Rhabdomyosarcoma;   Spindle Cell Rhabdomyosarcoma
  Interventions:   Drug: Cyclophosphamide;   Biological: Dactinomycin;   Drug: Irinotecan Hydrochloride;   Other: Laboratory Biomarker Analysis;   Other: Questionnaire Administration;   Radiation: Radiation Therapy;   Drug: Temsirolimus;   Drug: Vincristine Sulfate;   Drug: Vinorelbine
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• An Open-label, Ascending, Repeated Dose-finding Study of Sarilumab in Children and Adolescents With Polyarticular-course Juvenile Idiopathic Arthritis (pcJIA)

  NCT02776735

  Condition:   Juvenile Idiopathic Arthritis
  Intervention:   Drug: Sarilumab
  Sponsors:   Sanofi;   Regeneron Pharmaceuticals
  Recruiting
• A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Balovaptan in Children With Autism Spectrum Disorder

  NCT04049578

  Condition:   Autism Spectrum Disorder
  Intervention:   Drug: Balovaptan
  Sponsor:   Hoffmann-La Roche
  Recruiting
• Pediatric Adenotonsillectomy Trial for Snoring

  NCT02562040

  Condition:   Sleep-Disordered Breathing
  Interventions:   Procedure: Early Adenotonsillectomy (eAT);   Behavioral: Watchful Waiting with Supportive Care (WWSC)
  Sponsors:   Brigham and Women's Hospital;   Children's Hospital of Philadelphia;   University Hospitals Cleveland Medical Center;   Children's Hospital Medical Center, Cincinnati;   University of Michigan;   University of Texas Southwestern Medical Center;   University of Rochester;   Children's Hospital of The King's Daughters;   Boston Children’s Hospital
  Recruiting
• Study Evaluating PK/PD and Safety of Betrixaban in Pediatric Patients

  NCT03346083

  Condition:   VTE Prophylaxis
  Intervention:   Drug: Betrixaban
  Sponsor:   Portola Pharmaceuticals
  Recruiting
• A Safety and PK Study of IV Eravacycline

  NCT03696550

  Condition:   Bacterial Infections
  Intervention:   Drug: Eravacycline (TP-434)
  Sponsor:   Tetraphase Pharmaceuticals, Inc.
  Recruiting
• Respiratory Syncytial Virus (RSV) Investigational Vaccine in Infants Aged 6 and 7 Months Likely to be Unexposed to RSV

  NCT03636906

  Condition:   Respiratory Syncytial Virus Infections
  Interventions:   Biological: Respiratory syncytial virus (RSV) vaccine GSK3389245A;   Biological: Bexsero;   Biological: Nimenrix;   Biological: Menveo;   Biological: Synflorix;   Drug: Placebo
  Sponsor:   GlaxoSmithKline
  Recruiting
• A Study to Evaluate the Safety of MEDI8897 for the Prevention of Medically Attended Respiratory Syncytial Virus(RSV) Lower Respiratory Track Infection (LRTI) in High-risk Children

  NCT03959488

  Condition:   Respiratory Syncytial Virus Infections
  Interventions:   Drug: MEDI8897;   Drug: Palivizumab
  Sponsor:   MedImmune LLC
  Recruiting
• Efficacy and Safety of Erenumab in Pediatric Subjects With Episodic Migraine

  NCT03836040

  Condition:   Migraine
  Interventions:   Drug: Erenumab Dose 1;   Drug: Erenumab Dose 2;   Drug: Erenumab Dose 3;   Other: Placebo
  Sponsor:   Amgen
  Recruiting
• Efficacy and Safety of Erenumab in Pediatric Subjects With Chronic Migraine

  NCT03832998

  Condition:   Migraine
  Interventions:   Drug: Erenumab Dose 1;   Drug: Erenumab Dose 2;   Drug: Erenumab Dose 3;   Other: Placebo
  Sponsor:   Amgen
  Recruiting
• Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

  NCT02616484

  Condition:   Pyruvate Dehydrogenase Complex Deficiency
  Interventions:   Drug: Dichloroacetate (DCA);   Other: Placebo;   Genetic: Genotype
  Sponsors:   University of Florida;   Columbia University;   Medosome Biotec LLC;   Saol Therapeutics
  Recruiting
• Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age

  NCT03691779

  Condition:   Cystic Fibrosis
  Interventions:   Drug: VX-445;   Drug: TEZ;   Drug: IVA
  Sponsor:   Vertex Pharmaceuticals Incorporated
  Recruiting
• Trial to Evaluate the Long-term Efficacy of Oral Aripiprazole in the Treatment of Pediatric Subjects With Tourette's Disorder

  NCT03661983

  Condition:   Tourette's Disorder (TD)
  Interventions:   Drug: Aripiprazole (OPC-14597);   Drug: Placebo
  Sponsor:   Otsuka Pharmaceutical Development & Commercialization, Inc.
  Recruiting
• Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)

  NCT03614663

  Condition:   Fragile X Syndrome
  Interventions:   Drug: ZYN002 - CBD Transdermal Gel;   Other: Placebo Transdermal Gel
  Sponsor:   Zynerba Pharmaceuticals, Inc.
  Recruiting
• Diabetes Study of Linagliptin and Empagliflozin in Children and Adolescents (DINAMO)TM

  NCT03429543

  Condition:   Diabetes Mellitus, Type 2
  Interventions:   Drug: Linagliptin;   Drug: Empagliflozin;   Drug: Placebo
  Sponsor:   Boehringer Ingelheim
  Recruiting
• Moderately Preterm Infants With Caffeine at Home for Apnea (MoCHA) Trial

  NCT03340727

  Condition:   Apnea of Prematurity
  Interventions:   Drug: Caffeine Citrate;   Drug: Placebo
  Sponsors:   NICHD Neonatal Research Network;   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  Recruiting
• Study to Explore the Therapeutic Effect of Eluxadoline in Treating Irritable Bowel Syndrome With Diarrhea in Children.

  NCT03339128

  Condition:   Irritable Bowel Syndrome
  Interventions:   Drug: Eluxadoline;   Drug: Placebo
  Sponsor:   Allergan
  Recruiting
• Web-Based Physical Activity Intervention in Improving Long Term Health in Children and Adolescents With Newly Diagnosed Acute Lymphoblastic Leukemia in First Remission

  NCT03223753

  Condition:   Acute Lymphoblastic Leukemia in Remission
  Interventions:   Other: Educational Intervention;   Other: Internet-Based Intervention;   Other: Laboratory Biomarker Analysis;   Device: Monitoring Device;   Other: Quality-of-Life Assessment;   Other: Questionnaire Administration
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• Safety and Efficacy Study of Viaskin Peanut in Peanut-allergic Young Children 1-3 Years of Age

  NCT03211247

  Condition:   Peanut Allergy
  Interventions:   Biological: Viaskin Peanut 250 mcg;   Biological: Viaskin Peanut 100 mcg;   Biological: Placebo
  Sponsor:   DBV Technologies
  Recruiting
• Study to Evaluate the Efficacy and Safety of 3 Fixed Doses of Intranasal Esketamine in Addition to Comprehensive Standard of Care for the Rapid Reduction of the Symptoms of Major Depressive Disorder, Including Suicidal Ideation, in Pediatric Participants Assessed to be at Imminent Risk for Suicide

  NCT03185819

  Condition:   Depressive Disorder, Major
  Interventions:   Drug: Intranasal Placebo;   Drug: Midazolam Placebo Solution;   Drug: Midazolam;   Drug: Esketamine
  Sponsor:   Janssen Research & Development, LLC
  Recruiting
• Bezlotoxumab (MK-6072) Versus Placebo in Children With Clostridium Difficile Infection (CDI) (MK-6072-001)

  NCT03182907

  Condition:   Clostridium Difficile Infection
  Interventions:   Biological: Bezlotoxumab;   Drug: Placebo
  Sponsor:   Merck Sharp & Dohme Corp.
  Recruiting
• A Study to Investigate the Efficacy and Safety of Canagliflozin in Children and Adolescents (>=10 to <18 Years) With Type 2 Diabetes Mellitus

  NCT03170518

  Condition:   Diabetes Mellitus, Type 2
  Interventions:   Drug: Canagliflozin 100 mg;   Drug: Canagliflozin 300 mg;   Drug: Placebo
  Sponsor:   Janssen Research & Development, LLC
  Recruiting
• Vedolizumab IV in Pediatric Participants With Ulcerative Colitis (UC) or Crohn's Disease (CD)

  NCT03138655

  Conditions:   Ulcerative Colitis;   Crohn's Disease
  Intervention:   Drug: Vedolizumab
  Sponsor:   Takeda
  Recruiting
• BAX 111 rVWF in Pediatrics

  NCT02932618

  Condition:   Von Willebrand Disease
  Interventions:   Biological: von Willebrand factor (Recombinant);   Biological: Antihemophilic Factor (Recombinant)
  Sponsors:   Baxalta now part of Shire;   Baxalta Innovations GmbH, now part of Shire
  Recruiting
• A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (PAH)

  NCT02932410

  Condition:   Pulmonary Arterial Hypertension
  Interventions:   Drug: Macitentan;   Other: Standard-of-care
  Sponsor:   Actelion
  Recruiting
• A Study to Investigate the Efficacy and Safety of Balovaptan (RO5285119) in Participants With Autism Spectrum Disorder (ASD)

  NCT02901431

  Condition:   Autism Spectrum Disorder
  Interventions:   Drug: Placebo;   Drug: RO5285119
  Sponsor:   Hoffmann-La Roche
  Recruiting
• Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease

  NCT02850406

  Condition:   Sickle Cell Disease
  Intervention:   Drug: GBT440
  Sponsor:   Global Blood Therapeutics
  Recruiting
• Azacitidine and Combination Chemotherapy in Treating Infants With Acute Lymphoblastic Leukemia and KMT2A Gene Rearrangement

  NCT02828358

  Conditions:   Acute Leukemia of Ambiguous Lineage;   B Acute Lymphoblastic Leukemia;   Mixed Phenotype Acute Leukemia
  Interventions:   Drug: Azacitidine;   Drug: Cyclophosphamide;   Drug: Cytarabine;   Drug: Daunorubicin Hydrochloride;   Drug: Dexamethasone;   Drug: Hydrocortisone Sodium Succinate;   Other: Laboratory Biomarker Analysis;   Drug: Leucovorin Calcium;   Drug: Mercaptopurine;   Drug: Methotrexate;   Drug: Pegaspargase;   Other: Pharmacological Study;   Drug: Prednisolone;   Drug: Thioguanine;   Drug: Vincristine Sulfate
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• Pediatric ACL: Understanding Treatment Options

  NCT02772770

  Condition:   Anterior Cruciate Ligament Reconstruction
  Interventions:   Other: Non-operative treatment group will undergo standardized treatment which will include rehabilitation, bracing and certain activity restrictions;   Procedure: Surgical technique: Transphyseal reconstruction;   Procedure: Surgical technique: Partial transphyseal reconstruction;   Procedure: Surgical technique: Physeal sparing reconstruction by the Anderson method;   Procedure: Surgical technique: Physeal sparing reconstruction by the Micheli/Kocher method
  Sponsors:   Boston Children’s Hospital;   Children's Hospital of Philadelphia;   Children's Hospital Medical Center, Cincinnati;   Rady Children's Hospital, San Diego;   Washington University School of Medicine;   Children's Healthcare of Atlanta;   The Cleveland Clinic;   Tennessee Orthopedic Alliance;   Hospital for Special Surgery, New York;   Stanford University
  Recruiting
• Active Reference (Fluoxetine) Fixed-dose Study of Vortioxetine in Paediatric Patients Aged 7 to 11 Years With Major Depressive Disorder (MDD)

  NCT02709655

  Condition:   Depressive Disorder, Major
  Interventions:   Drug: Vortioxetine 10 mg/day;   Drug: Vortioxetine 20 mg/day;   Drug: Fluoxetine 20mg/day;   Other: Placebo
  Sponsors:   H. Lundbeck A/S;   Takeda
  Recruiting
• Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LCZ696 Followed by a 52-week Study of LCZ696 Compared With Enalapril in Pediatric Patients With Heart Failure

  NCT02678312

  Condition:   Pediatric Heart Failure
  Interventions:   Drug: LCZ696;   Drug: Enalapril;   Drug: Placebo of LCZ696;   Drug: Placebo of Enalapril
  Sponsor:   Novartis Pharmaceuticals
  Recruiting
• BAX 855 Previously Untreated Patient (PUP)

  NCT02615691

  Condition:   Hemophilia A
  Intervention:   Biological: PEGylated Recombinant Factor VIII
  Sponsor:   Baxalta now part of Shire
  Recruiting
• Efficacy and Safety of Lacosamide as Adjunctive Therapy in Subjects ≥1 Month to <4 Years With Partial-onset Seizures

  NCT02477839

  Condition:   Epilepsy With Partial-onset Seizures
  Interventions:   Drug: Lacosamide;   Other: Placebo
  Sponsor:   UCB BIOSCIENCES, Inc.
  Recruiting
• Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B

  NCT02141074

  Conditions:   Congenital Bleeding Disorder;   Haemophilia B
  Intervention:   Drug: nonacog beta pegol
  Sponsor:   Novo Nordisk A/S
  Recruiting
• Efficacy and Safety Trial of Flexible Doses of Oral Ziprasidone in Children and Adolescents With Bipolar I Disorder

  NCT02075047

  Condition:   Bipolar Disorder
  Interventions:   Drug: placebo oral capsules;   Drug: ziprasidone oral capsules
  Sponsor:   Pfizer
  Recruiting
• Generic Database of Very Low Birth Weight Infants

  NCT00063063

  Conditions:   Infant, Newborn;   Infant, Low Birth Weight;   Infant, Small for Gestational Age;   Infant, Premature
  Intervention:  
  Sponsors:   NICHD Neonatal Research Network;   National Center for Research Resources (NCRR);   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  Recruiting
• Follow-up Visit of High Risk Infants

  NCT00009633

  Conditions:   Infant, Newborn;   Infant, Low Birth Weight;   Infant, Small for Gestational Age;   Infant, Premature
  Intervention:  
  Sponsors:   NICHD Neonatal Research Network;   National Center for Research Resources (NCRR);   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  Recruiting
• Axitinib and Nivolumab in Treating Patients With Unresectable or Metastatic TFE/Translocation Renal Cell Carcinoma

  NCT03595124

  Conditions:   Metastatic Renal Cell Carcinoma;   Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3 Gene Fusions;   Stage III Renal Cell Cancer AJCC v8;   Stage IV Renal Cell Cancer AJCC v8;   Unresectable Renal Cell Carcinoma
  Interventions:   Drug: Axitinib;   Biological: Nivolumab
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• Nephrotic Syndrome Study Network

  NCT01209000

  Conditions:   Minimal Change Disease (MCD);   Membranous Nephropathy;   Glomerulosclerosis, Focal Segmental
  Intervention:   Procedure: Kidney Biopsy
  Sponsors:   University of Michigan;   National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);   The NephCure Foundation
  Recruiting
• Irinotecan Hydrochloride, Temozolomide, and Dinutuximab With or Without Eflornithine in Treating Patients With Relapsed or Refractory Neuroblastoma

  NCT03794349

  Conditions:   Ganglioneuroblastoma;   Recurrent Neuroblastoma;   Refractory Neuroblastoma
  Interventions:   Biological: Dinutuximab;   Drug: Eflornithine;   Drug: Irinotecan;   Biological: Sargramostim;   Drug: Temozolomide
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• A Study of NKTR-214 Combined With Nivolumab vs Nivolumab Alone in Participants With Previously Untreated Inoperable or Metastatic Melanoma

  NCT03635983

  Condition:   Melanoma
  Interventions:   Biological: NKTR-214;   Biological: Nivolumab
  Sponsors:   Bristol-Myers Squibb;   Nektar Therapeutics
  Recruiting
• Study to Evaluate the Efficacy and Safety of Perampanel as Monotherapy or First Adjunctive Therapy in Subjects With Partial Onset Seizures With or Without Secondarily Generalized Seizures or With Primary Generalized Tonic-Clonic Seizures

  NCT03288129

  Conditions:   Partial Onset Seizures;   Secondarily Generalized Seizures;   Primary Generalized Tonic-Clonic Seizures
  Intervention:   Drug: Perampanel
  Sponsor:   Eisai Inc.
  Recruiting
• A Study of Ruxolitinib vs Best Available Therapy (BAT) in Patients With Steroid-refractory Chronic Graft vs. Host Disease (GvHD) After Bone Marrow Transplantation (REACH3)

  NCT03112603

  Condition:   Graft-versus-host Disease (GVHD)
  Interventions:   Drug: Ruxolitinib;   Drug: Extracorporeal photopheresis (ECP);   Drug: Low-dose methotrexate (MTX);   Drug: Mycophenolate mofetil (MMF);   Drug: mechanistic Target of Rapamycin (mTOR) inhibitors (everolimus or sirolimus);   Drug: Infliximab;   Drug: Rituximab;   Drug: Pentostatin;   Drug: Imatinib;   Drug: Ibrutinib
  Sponsor:   Incyte Corporation
  Recruiting
• Study Comparing Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients

  NCT02851407

  Condition:   Veno-occlusive Disease
  Interventions:   Drug: Defibrotide;   Other: Best Supportive Care
  Sponsor:   Jazz Pharmaceuticals
  Recruiting
• Laser Ablation of Abnormal Neurological Tissue Using Robotic NeuroBlate System

  NCT02392078

  Conditions:   Metastatic Brain Tumor;   Primary Brain Tumor;   Epileptic/Seizure Foci
  Intervention:   Device: NeuroBlate System
  Sponsor:   Monteris Medical
  Recruiting
• Chronic Hypertension and Pregnancy (CHAP) Project

  NCT02299414

  Condition:   Hypertension
  Interventions:   Drug: Anti-hypertensive therapy;   Other: No anti-hypertensive therapy (unless BP is severe)
  Sponsors:   University of Alabama at Birmingham;   Columbia University;   Drexel University College of Medicine;   Rutgers, The State University of New Jersey;   Lehigh Valley Hospital;   Saint Peters University Hospital;   Christiana Care Health Services;   Washington University School of Medicine;   Duke University;   University of Texas Southwestern Medical Center;   The University of Texas Health Science Center, Houston;   Stanford University;   University of Pennsylvania;   The University of Texas Medical Branch, Galveston;   University of Utah;   Intermountain Health Care, Inc.;   University of California, San Francisco;   Johns Hopkins University;   University of Pittsburgh;   Ochsner Health System;   University of North Carolina, Chapel Hill;   National Heart, Lung, and Blood Institute (NHLBI);   WakeMed Health and Hospitals;   San Francisco General Hospital;   McKay-Dee Hospital;   Winthrop University Hospital;   New York Hospital Queens;   Latter Day Saints Hospital;   Lyndon B Johnson General Hospital;   Virtua Medical Group;   Bayview Medical Center;   Duke Regional Hospital;   Utah Valley Regional Medical Center;   Northwestern;   Brown (WIHRI);   Baylor College of Medicine;   Case Western/Metro Health;   Ohio State University;   University of Iowa;   University of California, San Diego;   Indiana University;   Unity Point Health-Meriter Hospital WI;   Weill Medical College of Cornell University;   University of Oklahoma;   Medical University of South Carolina;   Beaumont Hospital;   University of Colorado, Denver;   University of Kansas Medical Center;   Denver Health and Hospital Authority;   Gundersen Health System;   Aurora Health Care;   Oregon Health and Science University;   Medical College of Wisconsin;   Temple University;   New Jersey Medical School;   University of South Alabama;   Vanderbilt University;   University of Arkansas;   Miami Valley Hospital;   Emory University;   St. Luke's Hospital and Health Network, Pennsylvania;   Cleveland Clinic Fairview Hospital;   University of Tennessee Health Science Center;   TriHealth Inc.;   Cleveland Clinic Hillcrest Hospital;   Tulane University;   Yale University;   Arrowhead Regional Medical Center
  Recruiting
• Prospective Global Registry for the Study of Chronic Total Occlusion Intervention

  NCT02061436

  Condition:   Coronary Artery Disease
  Intervention:  
  Sponsor:   Minneapolis Heart Institute Foundation
  Recruiting
• North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

  NCT01694940

  Conditions:   Mitochondrial Disorders;   Mitochondrial Genetic Disorders;   Mitochondrial Diseases;   Disorder of Mitochondrial Respiratory Chain Complexes;   Deletion and Duplication of Mitochondrial DNA
  Intervention:  
  Sponsors:   Columbia University;   National Institute of Neurological Disorders and Stroke (NINDS)
  Recruiting
• Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant

  NCT03779854

  Conditions:   Acute Biphenotypic Leukemia;   Acute Leukemia;   Acute Leukemia of Ambiguous Lineage;   Acute Lymphoblastic Leukemia;   Acute Undifferentiated Leukemia;   Allogeneic Hematopoietic Stem Cell Transplantation Recipient;   Blastic Plasmacytoid Dendritic Cell Neoplasm;   Blasts Under 25 Percent of Bone Marrow Nucleated Cells;   Blasts Under 5 Percent of Bone Marrow Nucleated Cells;   Mixed Phenotype Acute Leukemia;   Myelodysplastic Syndrome With Excess Blasts-1;   Myelodysplastic Syndrome With Excess Blasts-2
  Interventions:   Radiation: Total-Body Irradiation;   Drug: Thiotepa;   Drug: Fludarabine;   Drug: Cyclophosphamide;   Drug: Busulfan;   Biological: Allogeneic Hematopoietic Stem Cell Transplantation (unmanipulated T cell replete BM);   Drug: Tacrolimus;   Drug: Methotrexate;   Biological: Allogeneic Hematopoietic Stem Cell Transplantation (naive T cell-depleted PBSC)
  Sponsors:   Fred Hutchinson Cancer Research Center;   National Cancer Institute (NCI)
  Recruiting
• Study of Etelcalceide in Pediatric Subjects With Secondary Hyperparathyroidism and CKD on Hemodialysis

  NCT03633708

  Conditions:   Secondary Hyperparathyroidism;   Chronic Kidney Disease
  Intervention:   Drug: Etelcalcetide
  Sponsor:   Amgen
  Recruiting
• Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

  NCT03802799

  Condition:   Fragile X Syndrome
  Intervention:   Drug: ZYN002 - CBD Transdermal Gel
  Sponsor:   Zynerba Pharmaceuticals, Inc.
  Recruiting
• Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome

  NCT03649477

  Condition:   Prader-Willi Syndrome
  Interventions:   Drug: intranasal carbetocin Dose 1;   Drug: intranasal carbetocin Dose 2;   Drug: placebo
  Sponsor:   Levo Therapeutics, Inc.
  Recruiting
• A Study to Evaluate Scales for Repetitive and Restricted Behaviors in Children, Adolescents, and Adults With Autism Spectrum Disorder (ASD)

  NCT03611075

  Condition:   Autism Spectrum Disorder
  Intervention:   Other: No Intervention
  Sponsor:   Hoffmann-La Roche
  Recruiting
• Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder

  NCT03572933

  Condition:   CDKL5 Deficiency Disorder
  Interventions:   Drug: ganaxolone;   Drug: Placebo
  Sponsor:   Marinus Pharmaceuticals
  Recruiting
• This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC 1 or 2

  NCT03566238

  Conditions:   PFIC1;   PFIC2
  Interventions:   Drug: A4250 (odevixibat);   Drug: Placebo
  Sponsor:   Albireo
  Recruiting
• Web Based Orthopaedic Sports Medicine Registry

  NCT01191151

  Conditions:   Degenerative and Traumatic Pathology of the Knee;   Degenerative and Traumatic Pathology of the Shoulder and Elbow;   Degenerative and Traumatic Pathology of the Foot and Ankle;   Degenerative and Traumatic Pathology of the Hand and Wrist;   Degenerative and Traumatic Pathology of the Hip
  Intervention:  
  Sponsor:   Arthrex, Inc.
  Recruiting
• A Pilot Study to Evaluate the Hypoglossal Nerve Stimulator in Adolescents With Down Syndrome and Obstructive Sleep Apnea

  NCT02344108

  Conditions:   Sleep Apnea, Obstructive;   Down Syndrome
  Intervention:   Device: Inspire® Upper Airway Simulation System (Model 3028 IPG )
  Sponsors:   Christopher Hartnick, M.D.;   Massachusetts General Hospital;   University Hospitals Cleveland Medical Center;   Emory University;   Children's Hospital Medical Center, Cincinnati;   Inspire Medical Systems, Inc.;   University of Pittsburgh;   Children's Hospital of The King's Daughters;   University of Texas Southwestern Medical Center;   Texas Children's Hospital;   Oregon Health and Science University;   C.S. Mott Children's Hospital;   Seattle Children's Hospital;   Children's Hospital of Philadelphia
  Recruiting
• A Study to Investigate the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) as an Adjunctive Therapy in Children and Adults With Lennox-Gastaut Syndrome

  NCT03355209

  Condition:   Lennox Gastaut Syndrome
  Interventions:   Drug: ZX008 0.2 or 0.8 mg/kg/day;   Drug: Matching Placebo
  Sponsor:   Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
  Recruiting
• Olaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Defects in DNA Damage Repair Genes (A Pediatric MATCH Treatment Trial)

  NCT03233204

  Conditions:   Advanced Malignant Solid Neoplasm;   Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma;   Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma;   Low Grade Glioma;   Malignant Glioma;   Recurrent Childhood Central Nervous System Neoplasm;   Recurrent Childhood Ependymoma;   Recurrent Childhood Malignant Germ Cell Tumor;   Recurrent Childhood Non-Hodgkin Lymphoma;   Recurrent Childhood Rhabdomyosarcoma;   Recurrent Childhood Soft Tissue Sarcoma;   Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor;   Recurrent Glioma;   Recurrent Hepatoblastoma;   Recurrent Langerhans Cell Histiocytosis;   Recurrent Malignant Solid Neoplasm;   Recurrent Medulloblastoma;   Recurrent Neuroblastoma;   Recurrent Osteosarcoma;   Refractory Central Nervous System Neoplasm;   Refractory Childhood Malignant Germ Cell Tumor;   Refractory Ependymoma;   Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor;   Refractory Glioma;   Refractory Hepatoblastoma;   Refractory Langerhans Cell Histiocytosis;   Refractory Malignant Glioma;   Refractory Malignant Solid Neoplasm;   Refractory Medulloblastoma;   Refractory Neuroblastoma;   Refractory Non-Hodgkin Lymphoma;   Refractory Osteosarcoma;   Refractory Rhabdomyosarcoma;   Refractory Soft Tissue Sarcoma;   Rhabdoid Tumor;   Wilms Tumor
  Intervention:   Drug: Olaparib
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)

  NCT03220035

  Conditions:   Advanced Malignant Solid Neoplasm;   Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma;   Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma;   Ependymoma;   Ewing Sarcoma;   Hepatoblastoma;   Langerhans Cell Histiocytosis;   Malignant Germ Cell Tumor;   Malignant Glioma;   Osteosarcoma;   Peripheral Primitive Neuroectodermal Tumor;   Recurrent Childhood Central Nervous System Neoplasm;   Recurrent Childhood Non-Hodgkin Lymphoma;   Recurrent Malignant Solid Neoplasm;   Recurrent Neuroblastoma;   Refractory Central Nervous System Neoplasm;   Refractory Malignant Solid Neoplasm;   Refractory Neuroblastoma;   Refractory Non-Hodgkin Lymphoma;   Rhabdoid Tumor;   Rhabdomyosarcoma;   Soft Tissue Sarcoma;   Wilms Tumor
  Interventions:   Other: Laboratory Biomarker Analysis;   Drug: Vemurafenib
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• Safety and Efficacy of 2 Treatment Regimens of Aztreonam for Inhalation Solution in Children With Cystic Fibrosis and New Onset Pseudomonas Aeruginosa Infection

  NCT03219164

  Conditions:   Pseudomonas Aeruginosa Respiratory Tract Infection;   Cystic Fibrosis
  Interventions:   Drug: AZLI;   Drug: Placebo
  Sponsor:   Gilead Sciences
  Recruiting
• Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial)

  NCT03213665

  Conditions:   Advanced Malignant Solid Neoplasm;   Ann Arbor Stage III Hodgkin Lymphoma;   Ann Arbor Stage III Non-Hodgkin Lymphoma;   Ann Arbor Stage IV Hodgkin Lymphoma;   Ann Arbor Stage IV Non-Hodgkin Lymphoma;   Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor;   EZH2 Gene Mutation;   Low Grade Glioma;   Recurrent Central Nervous System Neoplasm;   Recurrent Ependymoma;   Recurrent Ewing Sarcoma;   Recurrent Glioma;   Recurrent Hepatoblastoma;   Recurrent Hodgkin Lymphoma;   Recurrent Langerhans Cell Histiocytosis;   Recurrent Malignant Germ Cell Tumor;   Recurrent Malignant Glioma;   Recurrent Malignant Solid Neoplasm;   Recurrent Medulloblastoma;   Recurrent Neuroblastoma;   Recurrent Non-Hodgkin Lymphoma;   Recurrent Osteosarcoma;   Recurrent Peripheral Primitive Neuroectodermal Tumor;   Recurrent Rhabdoid Tumor;   Recurrent Rhabdomyosarcoma;   Recurrent Soft Tissue Sarcoma;   Refractory Hodgkin Lymphoma;   Refractory Langerhans Cell Histiocytosis;   Refractory Malignant Germ Cell Tumor;   Refractory Malignant Glioma;   Refractory Malignant Solid Neoplasm;   Refractory Medulloblastoma;   Refractory Neuroblastoma;   Refractory Non-Hodgkin Lymphoma;   Refractory Osteosarcoma;   Refractory Peripheral Primitive Neuroectodermal Tumor;   Refractory Rhabdoid Tumor;   Refractory Soft Tissue Sarcoma;   Rhabdoid Tumor;   SMARCA4 Gene Inactivation;   SMARCB1 Gene Inactivation;   Stage III Soft Tissue Sarcoma AJCC v7;   Stage IV Soft Tissue Sarcoma AJCC v7;   Wilms Tumor
  Interventions:   Other: Laboratory Biomarker Analysis;   Drug: Tazemetostat
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)

  NCT03213652

  Conditions:   Advanced Malignant Solid Neoplasm;   ALK Fusion Protein Expression;   ALK Gene Mutation;   Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma;   Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma;   Recurrent Childhood Central Nervous System Neoplasm;   Recurrent Childhood Non-Hodgkin Lymphoma;   Recurrent Malignant Solid Neoplasm;   Recurrent Neuroblastoma;   Refractory Central Nervous System Neoplasm;   Refractory Malignant Solid Neoplasm;   Refractory Neuroblastoma;   Refractory Non-Hodgkin Lymphoma;   ROS1 Fusion Positive;   ROS1 Gene Mutation
  Interventions:   Drug: Ensartinib;   Other: Laboratory Biomarker Analysis;   Other: Pharmacological Study
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)

  NCT03210714

  Conditions:   Advanced Malignant Solid Neoplasm;   Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma;   Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma;   Low Grade Glioma;   Malignant Glioma;   Recurrent Central Nervous System Neoplasm;   Recurrent Childhood Ependymoma;   Recurrent Childhood Malignant Germ Cell Tumor;   Recurrent Childhood Medulloblastoma;   Recurrent Childhood Non-Hodgkin Lymphoma;   Recurrent Childhood Rhabdomyosarcoma;   Recurrent Childhood Soft Tissue Sarcoma;   Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor;   Recurrent Hepatoblastoma;   Recurrent Langerhans Cell Histiocytosis;   Recurrent Malignant Solid Neoplasm;   Recurrent Neuroblastoma;   Recurrent Osteosarcoma;   Refractory Central Nervous System Neoplasm;   Refractory Langerhans Cell Histiocytosis;   Refractory Malignant Solid Neoplasm;   Refractory Neuroblastoma;   Refractory Non-Hodgkin Lymphoma;   Rhabdoid Tumor;   Stage III Soft Tissue Sarcoma AJCC v7;   Stage IV Soft Tissue Sarcoma AJCC v7;   Wilms Tumor
  Interventions:   Drug: Erdafitinib;   Other: Laboratory Biomarker Analysis;   Other: Pharmacological Study
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• A Trial to Describe the Immunogenicity and Safety of 2 Doses of Bivalent rLP2086 (Trumenba) and a Pentavalent Meningococcal Vaccine in Healthy Subjects >=10 to <26 Years of Age.

  NCT03135834

  Condition:   Meningococcal Vaccine
  Interventions:   Biological: MenABCWY;   Biological: Saline;   Biological: rLP2086;   Biological: MenACWY-CRM
  Sponsor:   Pfizer
  Recruiting
• Late Effects After Treatment in Patients With Previously Diagnosed High-Risk Neuroblastoma

  NCT03057626

  Conditions:   Recurrent Neuroblastoma;   Stage 2A Neuroblastoma;   Stage 2B Neuroblastoma;   Stage 3 Neuroblastoma;   Stage 4 Neuroblastoma;   Stage 4S Neuroblastoma
  Interventions:   Other: Cytology Specimen Collection Procedure;   Other: Laboratory Biomarker Analysis;   Other: Quality-of-Life Assessment
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• Lactobacillus Plantarum in Preventing Acute Graft Versus Host Disease in Children Undergoing Donor Stem Cell Transplant

  NCT03057054

  Conditions:   Hematopoietic Cell Transplant Recipient;   Malignant Neoplasm
  Interventions:   Procedure: Allogeneic Hematopoietic Stem Cell Transplantation;   Biological: Lactobacillus plantarum strain 299;   Biological: Lactobacillus plantarum strain 299v;   Other: Placebo
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia

  NCT03025698

  Condition:   Aplastic Anemia
  Interventions:   Drug: Eltrombopag;   Drug: hATG;   Drug: CsA
  Sponsor:   Novartis Pharmaceuticals
  Recruiting
• Rituximab and LMP-Specific T-Cells in Treating Pediatric Solid Organ Recipients With EBV-Positive, CD20-Positive Post-Transplant Lymphoproliferative Disorder

  NCT02900976

  Conditions:   CD20 Positive;   EBV-Related Post-Transplant Lymphoproliferative Disorder;   Epstein-Barr Virus Positive;   Monomorphic Post-Transplant Lymphoproliferative Disorder;   Polymorphic Post-Transplant Lymphoproliferative Disorder;   Recurrent Monomorphic Post-Transplant Lymphoproliferative Disorder;   Recurrent Polymorphic Post-Transplant Lymphoproliferative Disorder;   Refractory Monomorphic Post-Transplant Lymphoproliferative Disorder;   Refractory Polymorphic Post-Transplant Lymphoproliferative Disorder;   Solid Organ Transplant Recipient
  Interventions:   Biological: Allogeneic LMP1/LMP2-Specific Cytotoxic T-Lymphocytes;   Biological: Rituximab
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• Neuropsychological and Behavioral Testing in Younger Patients With Cancer

  NCT00772200

  Conditions:   Childhood Malignant Neoplasm;   Cognitive Side Effects of Cancer Therapy
  Interventions:   Procedure: Cognitive Assessment;   Other: Quality-of-Life Assessment
  Sponsors:   Children's Oncology Group;   National Cancer Institute (NCI)
  Recruiting
• Low Dose Oral Methotrexate in Pediatric Crohn's Disease Patients Initiating Anti-Tumor Necrosis Factor (Anti-TNF) Therapy

  NCT02772965

  Condition:   Pediatric Crohn's Disease
  Interventions:   Drug: Methotrexate;   Other: Sugar pill (placebo)
  Sponsors:   University of North Carolina, Chapel Hill;   Patient-Centered Outcomes Research Institute;   ImproveCareNow (ICN);   The Leona M. and Harry B. Helmsley Charitable Trust;   Children's Hospital Medical Center, Cincinnati;   Grifols Diagnostics Solutions, Inc;   National Institutes of Health (NIH);   National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
  Recruiting
• Neuroblastoma Maintenance Therapy Trial

  NCT02679144

  Condition:   Neuroblastoma
  Intervention:   Drug: Difluoromethylornithine (DFMO)
  Sponsors:   Giselle Sholler;   KC Pharma;   Beat NB Cancer Foundation
  Recruiting
• Phase 1 Pediatric Pharmacokinetics/Pharmacodynamics (PK/PD) Study

  NCT02303431

  Conditions:   Deep Vein Thrombosis;   Venous Thromboembolism
  Interventions:   Drug: Edoxaban low dose;   Drug: Edoxaban high dose
  Sponsor:   Daiichi Sankyo, Inc.
  Recruiting
• BIOPIC: Fungal Biomarkers for Diagnosis and Response to Therapy for Pediatric Candidemia

  NCT02220790

  Condition:   Invasive Candidiasis
  Intervention:  
  Sponsors:   Duke University;   Children's Hospital of Philadelphia
  Recruiting
• LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell Histiocytosis

  NCT02205762

  Condition:   Langerhans Cell Histiocytosis
  Interventions:   Drug: Prednisone;   Drug: Vinblastine;   Drug: mercaptopurine;   Drug: INDOMETHACIN;   Drug: Methotrexate;   Drug: Cytosine Arabinoside;   Drug: 2-chlorodeoxyadenosine;   Procedure: hematopoietic stem cell transplantation (RIC-HSCT);   Biological: Intravenous immunoglobulin
  Sponsors:   North American Consortium for Histiocytosis;   Histiocyte Society
  Recruiting
• A Trial of Temsirolimus With Etoposide and Cyclophosphamide in Children With Relapsed Acute Lymphoblastic Leukemia and Non-Hodgkins Lymphoma

  NCT01614197

  Conditions:   Lymphoblastic Leukemia, Acute, Childhood;   Lymphoblastic Lymphoma;   Peripheral T-cell Lymphoma
  Interventions:   Drug: Temsirolimus;   Drug: Etoposide;   Drug: Cyclophosphamide;   Drug: Methotrexate;   Drug: Hydrocortisone;   Drug: Cytarabine
  Sponsors:   Therapeutic Advances in Childhood Leukemia Consortium;   Pfizer
  Recruiting
• Evaluation of the Duration of Therapy for Thrombosis in Children

  NCT00687882

  Condition:   Venous Thrombosis
  Interventions:   Other: Shortened duration (6 weeks) of anticoagulant therapy;   Other: Conventional duration (3 months) of anticoagulant therapy;   Other: No Intervention
  Sponsors:   Johns Hopkins All Children's Hospital;   National Heart, Lung, and Blood Institute (NHLBI)
  Recruiting
• The Pediatric Anesthesia Quality Improvement Project

  NCT00674388

  Conditions:   Surgery;   Anesthesia;   Children
  Intervention:  
  Sponsors:   The Society for Pediatric Anesthesia;   Children's Hospital Medical Center, Cincinnati;   Monroe Carell Jr. Children's Hospital at Vanderbilt;   Seattle Children's Hospital;   Boston Children’s Hospital;   Emory-Children's Center;   Texas Children's Hospital;   Johns Hopkins University;   Children's Hospital Los Angeles;   Morgan Stanley Children's Hospital of New York;   Children's Hospital of Philadelphia;   Ann & Robert H Lurie Children's Hospital of Chicago;   University of Pittsburgh;   Phoenix Children's Hospital;   University of Michigan;   Children's National Health System;   Nationwide Children's Hospital;   Arkansas Children's Hospital Research Institute;   Children's Hospital Colorado;   St. Jude Children's Research Hospital;   Cook Children's Health Care System;   SSM Cardinal Glennon Children's Medical Center;   The Children's Hospital at Montefiore;   Children's Medical Center Dallas;   Lucile Packard Children's Hospital Stanford;   Alfred I. duPont Hospital for Children;   Johns Hopkins All Children's Hospital;   Children's Hospitals and Clinics of Minnesota;   Oakland Medical Center;   Children's Hospital and Medical Center, Omaha, Nebraska;   Medical University of South Carolina;   Akron Children's Hospital;   Women & Children's Hospital of Buffalo;   Dayton Children's Hospital;   Riley Children's Hospital;   Cleveland Clinic Children's Hospital;   Penn State Hershey Children's Hospital
  Recruiting
• Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders

  NCT03335488

  Condition:   Urea Cycle Disorder
  Interventions:   Drug: RAVICTI;   Drug: NaPBA
  Sponsor:   Horizon Therapeutics, LLC
  Recruiting
• Prevention of Urinary Stones With Hydration

  NCT03244189

  Condition:   Urinary Stones
  Interventions:   Behavioral: Fluid prescription;   Behavioral: Financial incentive;   Behavioral: Structured Problem Solving
  Sponsors:   Duke University;   National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  Recruiting
• International BPA Registry

  NCT03245268

  Conditions:   Chronic Thromboembolic Pulmonary Hypertension;   CTEPH
  Intervention:   Procedure: Balloon pulmonary angioplasty
  Sponsor:   International CTEPH Association
  Recruiting
• Zoliflodacin in Uncomplicated Gonorrhoea

  NCT03959527

  Condition:   Gonorrhea
  Interventions:   Drug: zoliflodacin;   Drug: ceftriaxone;   Drug: azithromycin
  Sponsor:   Global Antibiotics Research and Development Partnership
  Recruiting
• Pravastatin to Prevent Preeclampsia

  NCT03944512

  Conditions:   Preeclampsia;   Obstetric Labor Complications;   Hypertension in Pregnancy
  Interventions:   Drug: Pravastatin;   Other: Placebo
  Sponsors:   The George Washington University Biostatistics Center;   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);   National Heart, Lung, and Blood Institute (NHLBI)
  Recruiting
• A Randomized Trial of Pessary in Singleton Pregnancies With a Short Cervix

  NCT02901626

  Conditions:   Short Cervix;   Preterm Delivery
  Intervention:   Device: Pessary
  Sponsors:   The George Washington University Biostatistics Center;   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  Recruiting
• NeuMoDx PrEDiCTiNG Study Evaluation Plan

  NCT03970850

  Conditions:   Chlamydia Trachomatis Infection;   Neisseria Gonorrheae Infection
  Interventions:   Diagnostic Test: NeuMoDx CT/NG Assay;   Diagnostic Test: FDA-cleared NAATs
  Sponsors:   NeuMoDx Molecular, Inc.;   NAMSA
  Recruiting
• Immunotherapy (Nivolumab or Brentuximab Vedotin) Plus Combination Chemotherapy in Treating Patients With Newly Diagnosed Stage III-IV Classic Hodgkin Lymphoma

  NCT03907488

  Conditions:   Ann Arbor Stage III Hodgkin Lymphoma;   Ann Arbor Stage III Lymphocyte-Depleted Classic Hodgkin Lymphoma;   Ann Arbor Stage III Mixed Cellularity Classic Hodgkin Lymphoma;   Ann Arbor Stage III Nodular Sclerosis Classic Hodgkin Lymphoma;   Ann Arbor Stage IIIA Hodgkin Lymphoma;   Ann Arbor Stage IIIB Hodgkin Lymphoma;   Ann Arbor Stage IV Hodgkin Lymphoma;   Ann Arbor Stage IV Lymphocyte-Depleted Classic Hodgkin Lymphoma;   Ann Arbor Stage IV Mixed Cellularity Classic Hodgkin Lymphoma;   Ann Arbor Stage IV Nodular Sclerosis Classic Hodgkin Lymphoma;   Ann Arbor Stage IVA Hodgkin Lymphoma;   Ann Arbor Stage IVB Hodgkin Lymphoma;   Classic Hodgkin Lymphoma;   Lymphocyte-Rich Classic Hodgkin Lymphoma
  Interventions:   Drug: Brentuximab Vedotin;   Drug: Dacarbazine;   Drug: Doxorubicin;   Drug: Doxorubicin Hydrochloride;   Biological: Filgrastim;   Biological: Nivolumab;   Biological: Pegfilgrastim;   Other: Quality-of-Life Assessment;   Other: Questionnaire Administration;   Radiation: Radiation Therapy;   Drug: Vinblastine;   Drug: Vinblastine Sulfate
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation

  NCT03904134

  Conditions:   Acute Myeloid Leukemia;   Acute Lymphoblastic Leukemia;   Myelodysplastic Syndromes;   Non-hodgkin Lymphoma;   Hodgkin Lymphoma;   Acquired Aplastic Anemia;   Sickle Cell Disease
  Intervention:   Other: Donor Search Prognosis Score
  Sponsors:   Center for International Blood and Marrow Transplant Research;   National Heart, Lung, and Blood Institute (NHLBI);   National Cancer Institute (NCI);   Blood and Marrow Transplant Clinical Trials Network;   National Marrow Donor Program;   Medical College of Wisconsin
  Recruiting
• TK216 in Patients With Relapsed or Refractory Ewing Sarcoma

  NCT02657005

  Condition:   Sarcoma, Ewing
  Intervention:   Drug: TK216
  Sponsor:   Oncternal Therapeutics, Inc
  Recruiting
• RHAPSODY Phase 3 Study to Assess the Efficacy and Safety of Rilonacept Treatment in Subjects With Recurrent Pericarditis

  NCT03737110

  Condition:   Recurrent Pericarditis
  Interventions:   Drug: Rilonacept;   Drug: Placebo
  Sponsor:   Kiniksa Pharmaceuticals, Ltd.
  Recruiting
• ACURATE IDE: Safety and Efficacy Study of Acurate Valve for Transcatheter Aortic Valve Replacement

  NCT03735667

  Condition:   Aortic Stenosis
  Interventions:   Device: ACURATE neo2™ Transfemoral TAVR System;   Device: Medtronic CoreValve TAVR System;   Device: Edwards SAPIEN 3 TAVR System
  Sponsor:   Boston Scientific Corporation
  Recruiting
• PF-06651600 for the Treatment of Alopecia Areata

  NCT03732807

  Condition:   Alopecia Areata
  Interventions:   Drug: PF-06651600 Induction Dose;   Drug: PF-06651600 Maintenance Dose #1;   Drug: PF-06651600 Maintenance Dose #2;   Drug: PF-06651600 Maintenance Dose #3;   Drug: Placebo
  Sponsor:   Pfizer
  Recruiting
• Open-Label Extension Study of Diazoxide Choline in Patients With Prader-Willi Syndrome

  NCT03714373

  Condition:   Prader-Willi Syndrome
  Intervention:   Drug: DCCR
  Sponsor:   Soleno Therapeutics, Inc.
  Recruiting
• Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

  NCT03316560

  Condition:   X-Linked Retinitis Pigmentosa
  Intervention:   Biological: rAAV2tYF-GRK1-RPGR
  Sponsor:   Applied Genetic Technologies Corp
  Recruiting
• Surveillance HeartCare® Outcomes Registry

  NCT03695601

  Condition:   Heart Transplant Rejection
  Intervention:   Other: Standard of Care
  Sponsor:   CareDx
  Recruiting
• A Phase III Study Assessing the Efficacy and Safety of DE-117 Ophthalmic Solution Compared With Timolol Maleate Ophthalmic Solution 0.5% in Subjects With Glaucoma or Ocular Hypertension - Spectrum 4 Study

  NCT03691662

  Condition:   Glaucoma and Ocular Hypertension
  Interventions:   Drug: DE-117 Ophthalmic Solution;   Drug: Timolol Maleate Ophthalmic Solution 0.5%
  Sponsor:   Santen Inc.
  Recruiting
• Clinical Trial of Brentuximab Vedotin in Classical Hodgkin Lymphoma

  NCT03646123

  Condition:   Hodgkin Lymphoma
  Interventions:   Drug: brentuximab vedotin;   Drug: doxorubicin;   Drug: vinblastine;   Drug: dacarbazine;   Drug: G-CSF;   Drug: nivolumab
  Sponsor:   Seattle Genetics, Inc.
  Recruiting
• APOL1 Long-term Kidney Transplantation Outcomes Network (APOLLO)

  NCT03615235

  Conditions:   Kidney Diseases;   Kidney Failure;   Kidney Disease, Chronic
  Intervention:  
  Sponsors:   Wake Forest University Health Sciences;   National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);   National Institute on Minority Health and Health Disparities (NIMHD);   National Institute of Allergy and Infectious Diseases (NIAID)
  Recruiting
• A Study to Evaluate Upadacitinib in Adolescent and Adult Subjects With Moderate to Severe Atopic Dermatitis

  NCT03607422

  Condition:   Atopic Dermatitis
  Interventions:   Drug: Upadacitinib;   Drug: Placebo for Upadacitinib
  Sponsor:   AbbVie
  Recruiting
• Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)

  NCT03603288

  Condition:   Duchenne Muscular Dystrophy
  Intervention:   Drug: idebenone 150 mg film-coated tablets
  Sponsor:   Santhera Pharmaceuticals
  Recruiting
• Evaluation of Upadacitinib in Adolescent and Adult Patients With Moderate to Severe Atopic Dermatitis (Eczema)- Measure Up 1

  NCT03569293

  Condition:   Atopic Dermatitis
  Interventions:   Drug: Upadacitinib;   Drug: Placebo for Upadacitinib
  Sponsor:   AbbVie
  Recruiting
• A Study to Evaluate the Safety, Tolerability, and Immunogenicity of V114 in Allogeneic Hematopoietic Stem Cell Transplant Recipients (V114-022/PNEU-STEM)

  NCT03565900

  Condition:   Pneumococcal Infections
  Interventions:   Biological: V114;   Biological: Prevnar 13™;   Biological: PNEUMOVAX™23
  Sponsor:   Merck Sharp & Dohme Corp.
  Recruiting
• Vigil + Irinotecan and Temozolomide in Ewing's Sarcoma

  NCT03495921

  Conditions:   Ewing Sarcoma;   Ewing Family of Tumors;   Ewing's Tumor Metastatic;   Ewing's Sarcoma Metastatic;   Ewing's Tumor Recurrent;   Rare Diseases;   Sarcoma;   Neoplasms, Connective and Soft Tissue;   Neoplasms by Histologic Type;   Neoplasms, Bone Tissue;   Neoplasms, Connective Tissue;   Sarcoma, Ewing;   Neoplasms
  Interventions:   Biological: Vigil;   Drug: Irinotecan;   Drug: Temozolomide
  Sponsor:   Gradalis, Inc.
  Recruiting
• Study of Sparsentan in Patients With Primary Focal Segmental Glomerulosclerosis (FSGS)

  NCT03493685

  Condition:   Focal Segmental Glomerulosclerosis
  Interventions:   Drug: sparsentan;   Drug: irbesartan
  Sponsor:   Retrophin, Inc.
  Recruiting
• An Extension Study of Oral Ozanimod for Moderately to Severely Active Crohn's Disease

  NCT03467958

  Condition:   Crohn Disease
  Intervention:   Drug: Ozanimod
  Sponsor:   Celgene
  Recruiting
• A Placebo-Controlled Study of Oral Ozanimod as Maintenance Therapy for Moderately to Severely Active Crohn's Disease

  NCT03464097

  Condition:   Crohn Disease
  Interventions:   Drug: Ozanimod;   Other: Placebo
  Sponsor:   Celgene
  Recruiting
• Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis

  NCT03451045

  Condition:   Cystic Fibrosis
  Interventions:   Drug: Lenabasum 20 mg;   Drug: Lenabasum 5 mg;   Other: Placebo
  Sponsors:   Corbus Pharmaceuticals Inc.;   Cystic Fibrosis Foundation
  Recruiting
• Evaluation of Safety and Efficacy of Brimonidine Tartrate Ophthalmic Suspension

  NCT03450629

  Conditions:   Open Angle Glaucoma;   Ocular Hypertension
  Interventions:   Drug: Brimonidine Tartrate Ophthalmic Suspension;   Drug: Brimonidine Tartrate Ophthalmic Solution
  Sponsor:   Sun Pharma Advanced Research Company Limited
  Recruiting
• Safety and Efficacy of TRx0237 in Subjects With Early Alzheimer's Disease

  NCT03446001

  Condition:   Alzheimer Disease
  Interventions:   Drug: TRx0237 8 mg/day;   Drug: Placebo;   Drug: TRx0237 16 mg/day
  Sponsor:   TauRx Therapeutics Ltd
  Recruiting
• A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome

  NCT03440814

  Condition:   Prader-Willi Syndrome
  Interventions:   Drug: DCCR;   Drug: Placebo for DCCR
  Sponsor:   Soleno Therapeutics, Inc.
  Recruiting
• Induction Study #1 of Oral Ozanimod as Induction Therapy for Moderately to Severely Active Crohn's Disease

  NCT03440372

  Condition:   Crohn Disease
  Interventions:   Drug: Ozanimod;   Other: Placebo
  Sponsor:   Celgene
  Recruiting
• Study to Evaluate Efficacy and Safety of PF-04965842 With or Without Topical Medications in Subjects Aged 12 Years and Older With Moderate to Severe Atopic Dermatitis

  NCT03422822

  Condition:   Dermatitis, Atopic
  Interventions:   Drug: PF-04965842 100 mg;   Drug: PF-04965842 200 mg;   Drug: Placebo
  Sponsor:   Pfizer
  Recruiting
• A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

  NCT03417245

  Conditions:   Hemophilia A;   Hemophilia B
  Interventions:   Drug: fitusiran;   Drug: factor VIII or factor IX
  Sponsor:   Genzyme, a Sanofi Company
  Recruiting
• A Multicenter, Randomized, Double-Blind, Placebo Controlled Induction Study to Evaluate the Efficacy and Safety of Risankizumab in Subjects With Moderately to Severely Active Ulcerative Colitis Who Have Failed Prior Biologic Therapy

  NCT03398148

  Condition:   Ulcerative Colitis (UC)
  Interventions:   Drug: risankizumab IV;   Drug: placebo for risankizumab;   Drug: risankizumab SC
  Sponsor:   AbbVie
  Recruiting
• A Study to Assess the Efficacy and Safety of Risankizumab in Subjects With Ulcerative Colitis Who Responded to Induction Treatment in M16-067 or M16-065

  NCT03398135

  Condition:   Ulcerative Colitis (UC)
  Interventions:   Drug: risankizumab;   Drug: placebo for risankizumab
  Sponsor:   AbbVie
  Recruiting
• CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes

  NCT03350828

  Condition:   Cystic Fibrosis
  Intervention:  
  Sponsors:   Nicole Hamblett;   Cystic Fibrosis Foundation
  Recruiting
• A 5-year Longitudinal Observational Study of Patients Undergoing Therapy for Inflammatory Bowel Disease

  NCT03251118

  Conditions:   Inflammatory Bowel Diseases;   Crohn's Disease;   Ulcerative Colitis;   Indeterminate Colitis
  Intervention:  
  Sponsor:   Target PharmaSolutions, Inc.
  Recruiting
• A Study of AeroVanc for the Treatment of MRSA Infection in CF Patients

  NCT03181932

  Conditions:   MRSA;   Cystic Fibrosis
  Interventions:   Drug: Vancomycin inhalation powder;   Drug: Placebo inhalation powder
  Sponsor:   Savara Inc.
  Recruiting
• Phase 1/2 Study of LOXO-292 in Patients With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer

  NCT03157128

  Conditions:   Non-Small Cell Lung Cancer;   Medullary Thyroid Cancer;   Colon Cancer;   Any Solid Tumor
  Intervention:   Drug: LOXO-292
  Sponsor:   Loxo Oncology, Inc.
  Recruiting
• A Study in Participants Previously Enrolled in a Genentech− and/or F. Hoffmann-La Roche Ltd-Sponsored Atezolizumab Study (IMbrella A)

  NCT03148418

  Condition:   Cancer
  Intervention:   Drug: Atezolizumab
  Sponsor:   Hoffmann-La Roche
  Recruiting
• Trametinib in Treating Patients With Epithelioid Hemangioendothelioma That Is Metastatic, Locally Advanced, or Cannot Be Removed by Surgery

  NCT03148275

  Conditions:   Epithelioid Hemangioendothelioma;   Locally Advanced Epithelioid Hemangioendothelioma;   Metastatic Epithelioid Hemangioendothelioma
  Interventions:   Other: Questionnaire Administration;   Drug: Trametinib
  Sponsor:   National Cancer Institute (NCI)
  Recruiting
• A Study of the Efficacy and Safety of Risankizumab in Subjects With Moderately to Severely Active Crohn's Disease

  NCT03105128

  Condition:   Crohn's Disease
  Interventions:   Drug: placebo for risankizumab;   Drug: risankizumab IV;   Drug: risankizumab SC
  Sponsor:   AbbVie
  Recruiting
• A Study to Assess the Efficacy and Safety of Risankizumab in Subjects With Moderately to Severely Active Crohn's Disease Who Failed Prior Biologic Treatment

  NCT03104413

  Condition:   Crohn's Disease
  Interventions:   Drug: placebo for risankizumab IV;   Drug: risankizumab SC;   Drug: risankizumab IV
  Sponsor:   AbbVie
  Recruiting
• A Study of Repotrectinib (TPX-0005) in Patients With Advanced Solid Tumors Harboring ALK, ROS1, or NTRK1-3 Rearrangements

  NCT03093116

  Conditions:   Locally Advanced Solid Tumors;   Metastatic Solid Tumors
  Intervention:   Drug: Oral repotrectinib (TPX-0005)
  Sponsor:   Turning Point Therapeutics, Inc.
  Recruiting
• DCM Precision Medicine Study

  NCT03037632

  Condition:   Idiopathic Dilated Cardiomyopathy
  Intervention:   Behavioral: Family Heart Talk Booklet
  Sponsors:   Ray Hershberger;   National Heart, Lung, and Blood Institute (NHLBI);   National Human Genome Research Institute (NHGRI)
  Recruiting
• 3T MRI CIED Post-Approval Study

  NCT02969395

  Condition:   Cardiac Rhythm Disorder
  Intervention:  
  Sponsor:   Medtronic
  Recruiting
• Efficacy and Safety Study of Maribavir Treatment Compared to Investigator-assigned Treatment in Transplant Recipients With Cytomegalovirus (CMV) Infections That Are Refractory or Resistant to Treatment With Ganciclovir, Valganciclovir, Foscarnet, or Cidofovir

  NCT02931539

  Condition:   Cytomegalovirus (CMV)
  Interventions:   Drug: Maribavir;   Drug: Ganciclovir;   Drug: Valganciclovir;   Drug: Foscarnet;   Drug: Cidofovir
  Sponsor:   Shire
  Recruiting
• Mino-Lok Therapy (MLT) for the Treatment of CRBSI/CLABSI

  NCT02901717

  Condition:   Catheter-related Infections
  Interventions:   Drug: Mino-Lok;   Drug: Antibiotic lock
  Sponsor:   Leonard-Meron Biosciences, Inc.
  Recruiting
• Study of Perampanel as Adjunctive Treatment for Inadequately Controlled Seizures Associated With Lennox-Gastaut Syndrome

  NCT02834793

  Condition:   Lennox-Gastaut Syndrome (LGS)
  Interventions:   Drug: Placebo;   Drug: Perampanel
  Sponsor:   Eisai Inc.
  Recruiting
• A 5-year Longitudinal Observational Study of Patients With Nonalcoholic Fatty Liver (NAFL) or Nonalcoholic Steatohepatitis (NASH)

  NCT02815891

  Conditions:   Nonalcoholic Fatty Liver;   Nonalcoholic Steatohepatitis
  Intervention:  
  Sponsor:   Target PharmaSolutions, Inc.
  Recruiting
• An Study of Patients With Ph- Chromosome-negative Relapsed or Refractory Acute Lymphoblastic Leukemia in the US

  NCT02783651

  Condition:   Relapsed/Refractory Acute Lymphoblastic Leukemia
  Intervention:   Other: Other
  Sponsor:   Amgen
  Recruiting
• Natural History of Rett Syndrome & Related Disorders

  NCT02738281

  Conditions:   Rett Syndrome;   MECP2 Duplication dIsorder;   CDKL5 Disorder;   FOXG1 Syndrome
  Intervention:  
  Sponsors:   University of Alabama at Birmingham;   National Institutes of Health (NIH);   National Center for Advancing Translational Science (NCATS);   Rare Diseases Clinical Research Network;   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);   National Institute of Neurological Disorders and Stroke (NINDS)
  Recruiting
• Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard Umbilical Cord Blood in Patients With Leukemia, Lymphoma, and Myelodysplastic Syndrome (MDS)

  NCT02730299

  Conditions:   Hematological Malignancies;   Acute Lymphoblastic Leukemia (ALL);   Acute Myelogenous Leukemia (AML);   Chronic Myelogenous Leukemia (CML);   Myelodysplastic Syndrome (MDS);   Lymphoma;   Acute Leukemia
  Interventions:   Drug: NiCord® (omidubicel);   Other: Cord Blood Unit
  Sponsor:   Gamida Cell ltd
  Recruiting
• Once-Nightly Sodium Oxybate for Treatment of Excessive Daytime Sleepiness and Cataplexy in Narcolepsy

  NCT02720744

  Conditions:   Excessive Daytime Sleepiness;   Cataplexy;   Narcolepsy
  Interventions:   Drug: Sodium Oxybate;   Drug: Placebo
  Sponsor:   Avadel
  Recruiting
• Testing the Effect of Adding Chronic Oral Azithromycin to Inhaled Tobramycin in People With CF

  NCT02677701

  Condition:   Cystic Fibrosis
  Interventions:   Drug: azithromycin;   Drug: placebo (for azithromycin);   Drug: inhaled tobramycin
  Sponsors:   Seattle Children's Hospital;   National Heart, Lung, and Blood Institute (NHLBI);   Cystic Fibrosis Foundation;   CF Therapeutics Development Network Coordinating Center
  Recruiting
• Longitudinal Study for Eosinophilic Granulomatosis With Polyangiitis (Churg-Strauss)

  NCT00315380

  Conditions:   Eosinophilic Granulomatosis With Polyangiitis;   Churg-Strauss Syndrome
  Intervention:  
  Sponsors:   University of Pennsylvania;   Office of Rare Diseases (ORD);   National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);   Rare Diseases Clinical Research Network
  Recruiting
• A Study to Test the Effect of the Drug Larotrectinib in Adults and Children With NTRK-fusion Positive Solid Tumors

  NCT02576431

  Condition:   Solid Tumors Harboring NTRK Fusion
  Intervention:   Drug: BAY2757556 (Larotrectinib, Vitrakvi)
  Sponsor:   Bayer
  Recruiting
• A Trial of Pessary and Progesterone for Preterm Prevention in Twin Gestation With a Short Cervix

  NCT02518594

  Condition:   Short Cervical Length
  Interventions:   Drug: Vaginal progesterone;   Drug: Placebo;   Device: Arabin Pessary
  Sponsors:   The George Washington University Biostatistics Center;   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  Recruiting
• Safety and Efficacy Trial of RPC1063 for Moderate to Severe Ulcerative Colitis

  NCT02435992

  Condition:   Ulcerative Colitis
  Interventions:   Drug: RPC1063;   Drug: Placebo
  Sponsor:   Celgene
  Recruiting
• Phenotype, Genotype & Biomarkers in ALS and Related Disorders

  NCT02327845

  Conditions:   Amyotrophic Lateral Sclerosis;   Frontotemporal Dementia;   Primary Lateral Sclerosis;   Hereditary Spastic Paraplegia;   Progressive Muscular Atrophy;   Multisystem Proteinopathy
  Intervention:  
  Sponsors:   University of Miami;   National Institute of Neurological Disorders and Stroke (NINDS);   National Center for Advancing Translational Science (NCATS);   St. Jude Children's Research Hospital;   ALS Association
  Recruiting
• Safety and Efficacy Study of Lotus Valve for Transcatheter Aortic Valve Replacement

  NCT02202434

  Condition:   Aortic Stenosis
  Interventions:   Device: Lotus Valve System;   Device: CoreValve/Evolut R Transcatheter Aortic Valve Replacement System;   Device: LOTUS Edge Valve System
  Sponsor:   Boston Scientific Corporation
  Recruiting
• Abatacept for the Treatment of Relapsing, Non-Severe, Granulomatosis With Polyangiitis (Wegener's)

  NCT02108860

  Conditions:   Granulomatosis With Polyangiitis (Wegener's);   Granulomatosis With Polyangiitis;   Wegener's Granulomatosis;   ANCA-Associated Vasculitis
  Interventions:   Drug: Abatacept;   Drug: placebo
  Sponsors:   University of South Florida;   The Cleveland Clinic;   Bristol-Myers Squibb;   University of Pennsylvania;   National Institutes of Health (NIH);   National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
  Recruiting
• A Comparison of Intra-operative Radiotherapy Boost With External Beam Radiotherapy Boost in Early Breast Cancer.

  NCT01792726

  Condition:   Early Breast Cancer
  Intervention:   Radiation: Boost to the tumour bed
  Sponsors:   University College, London;   National Institute for Health Research, United Kingdom
  Recruiting
• A Registry for Patients With Chronic Hypoparathyroidism

  NCT01922440

  Condition:   Chronic Hypoparathyroidism
  Intervention:   Drug: Combination product (Natpara) and drug or supplements [Disease and drug registry]
  Sponsor:   Shire
  Recruiting
• Longitudinal Study of the Porphyrias

  NCT01561157

  Conditions:   Acute Porphyrias;   Cutaneous Porphyrias
  Intervention:  
  Sponsor:   Icahn School of Medicine at Mount Sinai
  Recruiting
• Registry of Avance® Nerve Graft's Utilization and Recovery Outcomes Post Peripheral Nerve Reconstruction

  NCT01526681

  Condition:   Peripheral Nerve Injuries
  Interventions:   Other: Processed Human Nerve Graft;   Other: Standard Treatment, Autogenous Nerve Graft, Direct Suture, etc.;   Other: Autogenous Nerve Graft;   Device: Nerve Tube Conduit;   Procedure: Autologous Breast Reconstruction with Neurotization;   Procedure: Autologous Breast Reconstruction without Neurotization
  Sponsor:   Axogen Corporation
  Recruiting
• Product Surveillance Registry

  NCT01524276

  Conditions:   Cardiac Rhythm Disorders;   Urological Disorders;   Neurological Disorders;   Cardiovascular Disorders;   Digestive Disorders;   Intracranial Aneurysm;   Mechanical Circulatory Support
  Intervention:  
  Sponsor:   Medtronic
  Recruiting
• One-Time DNA Study for Vasculitis

  NCT01241305

  Conditions:   Eosinophilic Granulomatosis With Polyangiitis (Churg-Strauss);   Giant Cell Arteritis;   Granulomatosis With Polyangiitis (Wegener's);   Microscopic Polyangiitis;   Polyarteritis Nodosa;   Takayasu's Arteritis
  Intervention:  
  Sponsors:   University of Pennsylvania;   National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);   Office of Rare Diseases (ORD);   Rare Diseases Clinical Research Network
  Recruiting
• Natural History Study of SCID Disorders

  NCT01186913

  Conditions:   Severe Combined Immunodeficiency (SCID);   Leaky SCID;   Omenn Syndrome;   Reticular Dysgenesis;   ADA SCID;   XSCID
  Intervention:  
  Sponsors:   National Institute of Allergy and Infectious Diseases (NIAID);   Primary Immune Deficiency Treatment Consortium (PIDTC);   Office of Rare Diseases (ORD);   National Center for Advancing Translational Science (NCATS)
  Recruiting
• A Safety Extension Study of Trastuzumab Emtansine in Participants Previously Treated With Trastuzumab Emtansine Alone or in Combination With Other Anti-Cancer Therapy in One of the Parent Studies

  NCT00781612

  Condition:   Neoplasm Metastasis
  Interventions:   Drug: Docetaxel;   Drug: Paclitaxel;   Drug: Pertuzumab;   Drug: Trastuzumab;   Drug: Trastuzumab Emtansine
  Sponsors:   Genentech, Inc.;   Hoffmann-La Roche
  Recruiting
• Closure of Muscular Ventricular Septal Defects (VSDs) With the AMPLATZER Muscular VSD (MuVSD) Occluder - Post Approval Study

  NCT00647387

  Condition:   Ventricular Septal Defects
  Intervention:   Device: Muscular VSD Occluder device implantation (AMPLATZER Muscular VSD Occluder)
  Sponsor:   Abbott Medical Devices
  Recruiting
• International Collaborative Gaucher Group (ICGG) Gaucher Registry

  NCT00358943

  Conditions:   Gaucher Disease;   Cerebroside Lipidosis Syndrome;   Glucocerebrosidase Deficiency Disease;   Glucosylceramide Beta-Glucosidase Deficiency Disease
  Intervention:  
  Sponsor:   Genzyme, a Sanofi Company
  Recruiting
• Pompe Disease Registry

  NCT00231400

  Conditions:   Glycogen Storage Disease Type II;   Pompe Disease
  Intervention:  
  Sponsor:   Genzyme, a Sanofi Company
  Recruiting
• Mucopolysaccharidosis I (MPS I) Registry

  NCT00144794

  Condition:   Mucopolysaccharidosis I (MPS I)
  Intervention:  
  Sponsor:   Genzyme, a Sanofi Company
  Recruiting