Status | Study |
Available |
Study Name: Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy Condition: X-linked Adrenoleukodystrophy Date: 2014-05-05 Interventions: Drug: Lorenzo's Oil Oral use of Lorenzo's oil to lower very long chain fatty acids |
Recruiting |
Study Name: Reduced Intensity Conditioning in Patients Aged ≤35 With Non-Malignant Disorders Undergoing UCBT, BMT, or PBSCT Condition: Primary Immunodeficiency Syndromes Congenital Bone Marrow Failure Syndromes Date: 2013-10-10 Interventions: Drug: Hydroxyurea Hydroxyurea |
Available |
Study Name: Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants Condition: Hematologic Malignancies Inborn Errors of Metabolism Disorders Date: 2013-06-17 Interventions: Biological: CliniMACS CD34 Reagent System The CliniMACS CD34 Reagent System is a medical device that is |
Completed |
Study Name: Betaine and Peroxisome Biogenesis Disorders Condition: Peroxisome Biogenesis Disorders Date: 2013-03-29 Interventions: Drug: Betaine Betaine will be given orally (mixed with foo |
Recruiting |
Study Name: A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD) Condition: Cerebral Adrenoleukodystrophy (CALD) Date: 2013-03-22 Interventions: Genetic: Lenti-D Drug Product Lenti-D Drug Product (autologous CD34+ cell-enriched population that conta |
Withdrawn |
Study Name: Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) Condition: X-Linked Adrenoleukodystrophy Adrenomyelo Date: 2013-02-06 Interventions: Drug: Sobetirome 50 mcg or 100 mcg once-daily oral O |
Completed |
Study Name: Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders Condition: Lysosomal Storage Disease Peroxisomal Disorder Date: 2012-06-20 Interventions: Drug: Campath-1H A daily dose |
Completed |
Study Name: Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy Condition: X-linked Adrenoleukodystrophy Date: 2012-05-07 Interventions: Behavioral: exercise training The exercise program uses a local Curves® gym to provide an individua |
Recruiting |
Study Name: Human Placental-Derived Stem Cell Transplantation Condition: Mucopolysaccharidosis I Mucopolysaccharidosis VI Date: 2012-04-25 Interventions: Drug: Human Placental Derived Stem Cell Infusions of thawed HPDSC to be given following UCB infusion. |
Completed |
Study Name: A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants Condition: Adrenomyeloneuropathy Date: 2011-11-28 Interventions: Drug: N-acetylcysteine N-acety |