| Status | Study |
| Active, not recruiting |
Study Name: MD1003-AMN MD1003 in Adrenomyeloneuropathy Condition: Adrenomyeloneuropathy Adrenoleukodystroph Date: 2016-11-09 Interventions: Drug: MD1003 100 mg capsule Ot |
| Recruiting |
Study Name: Newborn Screening for Adrenoleukodystrophy Condition: Adrenoleukodystrophy Date: 2016-10-31 Interventions: Procedure: newborns testing for ALD Routine newborn screening dried blood spots sample is used to test i |
| Not yet recruiting |
Study Name: Early Diagnosis Of Childhood Cerebral ALD Condition: Adrenoleukodystrophy Date: 2016-10-25 |
| Enrolling by invitation |
Study Name: Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product Condition: Cerebral Adrenoleukodystrophy (CALD) Adre Date: 2016-02-17 Interventions: Other: Safety and efficacy assessments Vector copy number (VCN) measurement, safety evaluations, disease |
| Recruiting |
Study Name: A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy Condition: X-linked Adrenoleukodystrophy Date: 2015-10-29 Interventions: Dietary Supplement: vitamin D3 Daily oral supplement provided by study investigators |
| Recruiting |
Study Name: Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy Condition: Metachromatic Leukodystrophy Adrenoleukodystrophy Date: 2015-08-12 Interventions: Genetic: transduced CD34+ hematopoietic stem cell Autologous hematopoietic stem cells (HSCs) collected f |
| Withdrawn |
Study Name: MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD) Condition: Cerebral Adrenoleukodystrophy Date: 2015-03-27 Interventions: Biological: Mesenchymal Stem Cells |
| Recruiting |
Study Name: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Condition: Adrenoleukodystrophy Batten Disease Date: 2014-09-23 Interventions: Biological: DUOC-01 Intrathecal administration of DUOC-01 |
| Recruiting |
Study Name: Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD) Condition: X-Linked Adrenoleukodystrophy (X-ALD) Date: 2014-07-28 Interventions: Genetic: Allo-HSCT Allogeneic Hematopoietic Stem Cell Transplantation |
| Recruiting |
Study Name: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Condition: Mucopolysaccharidosis Disorders Hurler Syndrome Hunter Syn Date: 2014-06-20 Interventions: Biological: Stem Cell Transplantation |