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  • Lentiviral Gene Therapy for X-ALD
    NCT03727555
    Condition:   X-linked Adrenoleukodystrophy
    Intervention:   Genetic: Intracerebral LV gene therapy
    Sponsor:   Shenzhen Geno-Immune Medical Institute
    Recruiting
  • Minnesota Adrenoleukodystrophy Registry Study (MARS) and Biobank
    NCT03789721
    Condition:   ALD (Adrenoleukodystrophy)
    Interventions:   Other: Medical Record Abstraction;   Other: Biospecimen Sample Collection
    Sponsor:   Masonic Cancer Center, University of Minnesota
    Recruiting
  • A Study to Prospectively Assess Disease Progression in Male Children With X-ALD
    NCT03278899
    Condition:   X-Linked Adrenoleukodystrophy
    Intervention:  
    Sponsor:   NeuroVia, Inc.
    Recruiting
  • Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
    NCT02204904
    Conditions:   X-Linked Adrenoleukodystrophy (X-ALD);   Cerebral Adrenoleukodystrophy (CALD);   Adrenoleukodystrophy (ALD)
    Intervention:   Genetic: Allo-HSCT
    Sponsor:   bluebird bio
    Recruiting
  • Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
    NCT02559830
    Conditions:   Metachromatic Leukodystrophy;   Adrenoleukodystrophy
    Intervention:   Genetic: transduced CD34+ hematopoietic stem cell
    Sponsors:   Shenzhen Second People's Hospital;   Shenzhen University;   Guangzhou Women and Children's Medical Center
    Recruiting
  • Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children
    NCT03649919
    Conditions:   Xlsma;   DMD;   X-ALD;   TSC
    Intervention:  
    Sponsors:   Children's Hospital of Fudan University;   Children's Hospital of Shanghai Jiaotong University;   Children's Medical Center of Shanghai Jiaotong University;   Xinhua Hospital of Shanghai Jiaotong University
    Not yet recruiting
  • Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
    NCT03367546
    Conditions:   Sickle Cell Disease;   Thalassemia;   High Risk Hematologic Disorders;   Cerebral Adrenoleukodystrophy;   Inherited Metabolic Disorders
    Intervention:   Procedure: Blood and Marrow Transplant
    Sponsor:   Masonic Cancer Center, University of Minnesota
    Recruiting
  • UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
    NCT02254863
    Conditions:   Adrenoleukodystrophy;   Batten Disease;   Mucopolysaccharidosis II;   Leukodystrophy, Globoid Cell;   Leukodystrophy, Metachromatic;   Neimann Pick Disease;   Pelizaeus-Merzbacher Disease;   Sandhoff Disease;   Tay-Sachs Disease;   Brain Diseases, Metabolic, Inborn;   Alpha-Mannosidosis;   Sanfilippo Mucopolysaccharidoses
    Intervention:   Biological: DUOC-01
    Sponsors:   Joanne Kurtzberg, MD;   The Marcus Foundation
    Recruiting
  • MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
    NCT02171104
    Conditions:   Mucopolysaccharidosis Disorders;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux Lamy Syndrome;   Sly Syndrome;   Alpha-Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Glycoprotein Metabolic Disorders;   Sphingolipidoses;   Recessive Leukodystrophies;   Globoid Cell Leukodystrophy;   Metachromatic Leukodystrophy;   Niemann-Pick B;   Niemann-Pick C Subtype 2;   Sphingomyelin Deficiency;   Peroxisomal Disorders;   Adrenoleukodystrophy With Cerebral Involvement;   Zellweger Syndrome;   Neonatal Adrenoleukodystrophy;   Infantile Refsum Disease;   Acyl-CoA Oxidase Deficiency;   D-Bifunctional Enzyme Deficiency;   Multifunctional Enzyme Deficiency;   Alpha-methylacyl-CoA Racmase Deficiency;   Mitochondrial Neurogastrointestingal Encephalopathy;   Severe Osteopetrosis;   Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);   Inherited Metabolic Disorders
    Interventions:   Biological: Stem Cell Transplantation;   Drug: IMD Preparative Regimen;   Drug: Osteopetrosis Only Preparative Regimen;   Drug: Osteopetrosis Haploidentical Only Preparative Regimen;   Drug: cALD SR-A (Standard-Risk, Regimen A);   Drug: cALD SR-B (Standard-Risk, Regimen B);   Drug: cALD HR-D (High-Risk, Regimen C);   Drug: cALD HR-D (High-Risk, Regimen D)
    Sponsor:   Masonic Cancer Center, University of Minnesota
    Recruiting
  • Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
    NCT03513328
    Conditions:   Bone Marrow Failure Syndrome;   Thalassemia;   Sickle Cell Disease;   Diamond Blackfan Anemia;   Acquired Neutropenia in Newborn;   Acquired Anemia Hemolytic;   Acquired Thrombocytopenia;   Hemophagocytic Lymphohistiocytoses;   Wiskott-Aldrich Syndrome;   Chronic Granulomatous Disease;   Common Variable Immunodeficiency;   X-linked Lymphoproliferative Disease;   Severe Combined Immunodeficiency;   Hurler Syndrome;   Mannosidosis;   Adrenoleukodystrophy
    Interventions:   Drug: Thiotepa--single daily dose;   Drug: Thiotepa--escalated dose
    Sponsors:   University of Florida;   Live Like Bella Pediatric Cancer Research
    Recruiting
  • Precision Exercise in Children With Malignant Hemopathies
    NCT04090268
    Conditions:   Leukemia, Myeloid, Acute;   Leukemia, B-cell;   Leukemia, T Cell;   Lymphoma, Hodgkin;   Lymphoma, Non-Hodgkin;   Drepanocytosis;   Thalassemia Major;   Adrenoleukodystrophy
    Intervention:   Other: Sport Therapy
    Sponsors:   University of Milano Bicocca;   Comitato Maria Letizia Verga,Italy
    Recruiting
  • MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
    NCT03406962
    Condition:   Inherited Metabolic Disorders (IMD)
    Intervention:   Drug: MGTA-456
    Sponsor:   Magenta Therapeutics, Inc.
    Recruiting
  • Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
    NCT01962415
    Conditions:   Primary Immunodeficiency (PID);   Congenital Bone Marrow Failure Syndromes;   Inherited Metabolic Disorders (IMD);   Hereditary Anemias;   Inflammatory Conditions
    Interventions:   Drug: Hydroxyurea;   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Melphalan;   Drug: Thiotepa
    Sponsor:   Paul Szabolcs
    Recruiting

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