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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
• Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

  NCT03852498

  Condition:   Cerebral Adrenoleukodystrophy (CALD)
  Intervention:   Drug: Genetic
  Sponsor:   bluebird bio
  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
• A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy

  NCT02595489

  Condition:   X-linked Adrenoleukodystrophy
  Intervention:   Dietary Supplement: vitamin D3
  Sponsors:   Stanford University;   National Institute of Neurological Disorders and Stroke (NINDS);   Hugo W. Moser Research Institute at Kennedy Krieger, Inc.;   ALD Connect, 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
• 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