6 completed, 4 recruiting, 2 not yet recruiting, 2 withdrawn. 4 Not applicable, 3 Phase 1, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 4 | Completed 3, Not yet recruiting 1 |
| Phase 1 | 3 | Withdrawn 2, Completed 1 |
| Phase 2 | 2 | Completed 1, Withdrawn 1 |
Follow X-linked Adrenoleukodystrophy: new trials by email Literature Review of X-linked Adrenoleukodystrophy trials Research Report on X-linked Adrenoleukodystrophy
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| IT and IV Lentiviral Gene Therapy for X-ALD Intrathecal and intravenous LV gene therapy - X-linked Adrenoleukodystrophy | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 30 | 2027-06-01 | 2026-09-01 |
| Disease Progression in Women With X-linked Adrenoleukodystrophy X-linked Adrenoleukodystrophy | Recruiting | Minoryx Therapeutics, S.L. | 40 | 2024-01-02 | 2024-01-31 | |
| Validation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked Adrenoleukodystrophy brainQuant module of brainTale-care platform - X-linked Adrenoleukodystrophy | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 200 | 2023-09-01 | 2023-07-03 | |
| Registry of X-linked Adrenoleukodystrophy X-linked Adrenoleukodystrophy | Recruiting | Beijing Tiantan Hospital | 200 | 2023-07-20 | 2023-07-11 | |
| SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD) SMART-ALD, Waiting list - X-linked Adrenoleukodystrophy | Not applicable | Completed | Leipzig University Medical Center | 68 | 2022-05-20 | 2024-03-13 |
| Quality of Life in Women With X-linked Adrenoleukodystrophy X-linked Adrenoleukodystrophy | Recruiting | Leipzig University Medical Center | 200 | 2019-12-01 | 2026-08-27 | |
| Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy Sobetirome (NV1205) - X-Linked Adrenoleukodystrophy | Phase 1 / Phase 2 | Withdrawn | NeuroVia, Inc. | 2018-08-01 | 2019-03-07 | |
| A Study to Prospectively Assess Disease Progression in Male Children With X-ALD X-Linked Adrenoleukodystrophy | Unknown | NeuroVia, Inc. | 100 | 2018-02-16 | 2018-08-08 | |
| LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies Leukodystrophy, White Matter Disease | Completed | Children's Hospital of Philadelphia | 236 | 2017-01-06 | 2025-11-10 | |
| The Myelin Disorders Biorepository Project Leukodystrophy, White Matter Disease | Recruiting | Children's Hospital of Philadelphia | 12000 | 2016-12-08 | 2025-10-23 | |
| A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy vitamin D3 - X-linked Adrenoleukodystrophy | Phase 1 | Completed | Stanford University | 21 | 2016-11-21 | 2022-06-09 |
| Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy Pioglitazone - Adrenomyeloneuropathy, X-linked Adrenoleukodystrophy | Phase 2 | Completed | Pujol, Aurora, M.D. | 18 | 2016-01-01 | 2019-09-10 |
| Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) Sobetirome - X-Linked Adrenoleukodystrophy, Adrenomyeloneuropathy | Phase 1 | Withdrawn | Thomas S. Scanlan | 2013-04-01 | 2014-02-14 | |
| Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy exercise training - X-linked Adrenoleukodystrophy | Not applicable | Completed | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | 31 | 2012-05-01 | 2018-08-29 |
| The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD) Bezafibrate - X-linked Adrenoleukodystrophy, Adrenomyeloneuropathy | Not applicable | Completed | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | 10 | 2010-07-01 | 2011-08-10 |
| Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy Lorenzo's Oil - X-linked Adrenoleukodystrophy | No longer available | University of Minnesota | 2018-01-23 |
Adrenoleukodystrophy (16) · Peroxisomal Disorders (4) · Aicardi-Goutieres syndrome (2) · Alexander Disease (2) · Alexanders leukodystrophy (2) · Allan-Herndon-Dudley syndrome (2) · Attention Deficit Disorder with Hyperactivity (2) · CADASIL (2) · Canavan Disease (2) · Central Nervous System Diseases (2) · Charcot-Marie-Tooth Disease (2) · Cockayne Syndrome (2) · Demyelinating Diseases (2) · Gangliosidoses (2) · Gangliosidoses, GM2 (2)
Adrenal Gland Diseases (16) · Adrenal Insufficiency (16) · Brain Diseases (16) · Brain Diseases, Metabolic (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Endocrine System Diseases (16) · Genetic Diseases, Inborn (16) · Hereditary Central Nervous System Demyelinating Diseases (16) · Heredodegenerative Disorders, Nervous System (16) · Intellectual Disability (16) · Metabolism, Inborn Errors (16) · Nervous System Diseases (16) · Neurobehavioral Manifestations (16) · Neurologic Manifestations (16) · Nutritional and Metabolic Diseases (16)
GC 1 compound (2) · Bezafibrate (1) · Cholecalciferol (1) · Exercise (1) · Lorenzo's oil (1) · Pioglitazone (1) · Resistance Training (1) · Vitamin D (1) · Waiting Lists (1) · trierucate (1)
Children's Hospital of Philadelphia (2) · Leipzig University Medical Center (2) · NeuroVia, Inc. (2) · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) (1) · Assistance Publique - Hôpitaux de Paris (1) · Beijing Tiantan Hospital (1) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (1) · Minoryx Therapeutics, S.L. (1) · Pujol, Aurora, M.D. (1) · Shenzhen Geno-Immune Medical Institute (1) · Stanford University (1) · Thomas S. Scanlan (1) · University of Minnesota (1)
United States (7) · China (2) · France (2) · Germany (2) · Argentina (1) · Australia (1) · Chile (1) · Colombia (1) · Netherlands (1) · Russia (1) · Spain (1) · Ukraine (1) · United Kingdom (1)
Adeline Vanderver, MD (2) · John Henderson, MD (2) · David Koeller, MD (1) · Gerald V Raymond, MD (1) · Kathleen M Zackowski, Ph.D. (1) · Keith Van Haren, MD (1) · Yilong Wang, MD,PhD (1)
PMID 22911730, PMID 32342562, PMID 32749716, PMID 41030609
K23NS087151 (1), U01NS106845 (1), U54NS115052 (1)