Status | Study |
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 |
Active, not recruiting |
Study Name: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders Condition: Hurler Syndrome (MPS I) Hurler-Scheie Syndrome With Early Neurologic Involvement and/or Sens Date: 2011-06-10 Interventions: Biological: FCRx infusion Enriched hematopoetic stem cell infusion |
Completed |
Study Name: The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD) Condition: X-linked Adrenoleukodystrophy Adrenomyelo Date: 2010-07-13 Interventions: Drug: Bezafibrate Week 0 to 12: 400 mg once daily 1 tablet. Week 13 to 24: 400 mg once daily 2 tablets. |
Active, not recruiting |
Study Name: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Condition: Mucopolysaccharidosis Hurler Syndrome Hunter Syndrome Date: 2010-01-05 Interventions: Drug: Campath-1H Administered |
Terminated |
Study Name: ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases Condition: Inherited Metabolic Diseases Lysosomal Storage Disorders P Date: 2008-04-03 Interventions: Biological: ALD-101 A subpopulation of cord blood cells composed of cells that express a high level of t |
Terminated |
Study Name: A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy Condition: Adrenomyeloneuropathy Adrenoleukodystroph Date: 2007-10-16 Interventions: Drug: Lorenzo's oil 4:1 glyceryl trioleate-glyceryl trierucate (Lorenzo's oil)given orally, daily as app |
Completed |
Study Name: HSCT for High Risk Inherited Inborn Errors Condition: Adrenoleukodystrophy Metachromatic Leukodystrophy Date: 2006-09-29 Interventions: Drug: Clofarabine days -7 thro |
Available |
Study Name: Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children Condition: Adrenoleukodystrophy Date: 2006-01-16 Interventions: Procedure: blood sampling and medical records |
Completed |
Study Name: Stem Cell Transplant for Inborn Errors of Metabolism Condition: Adrenoleukodystrophy Metachromatic Leukodystrophy Date: 2005-09-12 Interventions: Procedure: Stem Cell Transplant |
Completed |
Study Name: Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid Condition: Infantile Refsum's Disease Zellweger Syndrome Date: 2000-12-06 Interventions: Drug: Cholic Acids 10-15 mg/kg body weight/day taken orally. |