10 completed, 3 terminated, 3 withdrawn, 1 recruiting. 18 Phase 2, 5 Phase 1, 5 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 18 | Completed 10, Terminated 3, Withdrawn 3, Recruiting 1, Unknown 1 |
| Phase 1 | 5 | Terminated 2, Completed 1, Unknown 1, Withdrawn 1 |
| Phase 3 | 5 | Completed 5 |
Follow Adrenoleukodystrophy: new trials by email Literature Review of Adrenoleukodystrophy trials Research Report on 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 |
|---|---|---|---|---|---|---|
| Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) PXL065 - ALD (Adrenoleukodystrophy) | Phase 2 | Withdrawn | Poxel SA | 2023-09-01 | 2024-12-16 | |
| Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) PXL770 - Adrenomyeloneuropathy | Phase 2 | Withdrawn | Poxel SA | 2023-09-01 | 2024-12-16 | |
| A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) SBT101 Treatment - AMN, AMN Gene Mutation | Phase 1 / Phase 2 | Terminated | SwanBio Therapeutics, Inc. | 9 | 2022-11-17 | 2026-08-26 |
| Plasma Exchange With Albumin in AMN Patients Albumin solution - Adrenomyeloneuropathy, Adrenoleukodystrophy | Phase 2 / Phase 3 | Completed | Pujol, Aurora, M.D. | 5 | 2020-03-09 | 2022-09-22 |
| 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Male Subjects With Adrenoleukodystrophy Sobetirome (NV1205) - X-Linked Adrenoleukodystrophy | Phase 1 / Phase 2 | Withdrawn | NeuroVia, Inc. | 2018-08-01 | 2019-03-07 | |
| Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation Thiotepa--single daily dose, Thiotepa--escalated dose - Bone Marrow Failure Syndrome, Thalassemia | Phase 1 / Phase 2 | Completed | University of Florida | 6 | 2018-06-15 | 2023-09-15 |
| A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients Leriglitazone, Placebo - Adrenoleukodystrophy | Phase 2 / Phase 3 | Completed | Minoryx Therapeutics, S.L. | 116 | 2017-12-08 | 2026-10-28 |
| 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 |
| Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy transduced CD34+ hematopoietic stem cell - Metachromatic Leukodystrophy, Adrenoleukodystrophy | Phase 1 / Phase 2 | Unknown | Shenzhen Second People's Hospital | 50 | 2015-01-01 | 2022-05-31 |
| MD1003-AMN MD1003 in Adrenomyeloneuropathy MD1003 100 mg capsule, Placebo - Adrenomyeloneuropathy, Adrenoleukodystrophy | Phase 2 / Phase 3 | Completed | MedDay Pharmaceuticals SA | 67 | 2014-10-01 | 2017-10-09 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
| A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD) Lenti-D Drug Product (eli-cel) - Cerebral Adrenoleukodystrophy (CALD) | Phase 2 / Phase 3 | Completed | Genetix Biotherapeutics Inc. | 32 | 2013-08-21 | 2022-04-25 |
| A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants N-acetylcysteine, lipoic acid, vitamin E - Adrenomyeloneuropathy | Phase 2 | Completed | Pujol, Aurora, M.D. | 13 | 2011-09-01 | 2019-03-11 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
| Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Campath-1H, Cyclophosphamide, Busulfan - Mucopolysaccharidosis, Hurler Syndrome | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 46 | 2009-12-01 | 2018-02-05 |
| HSCT for High Risk Inherited Inborn Errors Clofarabine, Total body Irradiation, Melphalan - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 38 | 2006-09-01 | 2019-07-11 |
| Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy glyceryl trierucate/glyceryl trioleate - Adrenoleukodystrophy | Phase 2 | Terminated | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | 126 | 1998-04-01 | 2020-10-23 |
| Stem Cell Transplant for Inborn Errors of Metabolism Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 135 | 1995-01-01 | 2017-12-28 |
Leukodystrophy, Metachromatic (6) · Leukodystrophy, Globoid Cell (5) · Mucopolysaccharidosis I (4) · Metabolism, Inborn Errors (3) · Mucopolysaccharidosis II (3) · Sandhoff Disease (3) · Tay-Sachs Disease (3) · alpha-Mannosidosis (3) · Anemia, Diamond-Blackfan (2) · Anemia, Sickle Cell (2) · Fucosidosis (2) · Gangliosidosis, GM1 (2) · Gaucher Disease (2) · Granulomatous Disease, Chronic (2)
Adrenal Gland Diseases (18) · Brain Diseases (18) · Central Nervous System Diseases (18) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (18) · Endocrine System Diseases (18) · Genetic Diseases, Inborn (18) · Genetic Diseases, X-Linked (18) · Intellectual Disability (18) · Nervous System Diseases (18) · Neurobehavioral Manifestations (18) · Neurologic Manifestations (18) · Nutritional and Metabolic Diseases (18) · X-Linked Intellectual Disability (18) · Adrenal Insufficiency (17) · Brain Diseases, Metabolic (17)
Alemtuzumab (3) · Stem Cell Transplantation (3) · Busulfan (2) · Cyclophosphamide (2) · Hydroxyurea (2) · Melphalan (2) · 2-chloro-3-(1-hydroxy-5,6,7,8-tetrahydronaphthalen-2-yl)-6-oxo-5-phenyl-7H-thieno)(2,3-b)pyridin-4-olate potassium (1) · Acetylcysteine (1) · Antilymphocyte Serum (1) · Biotin (1) · Bone Marrow Transplantation (1) · Clofarabine (1) · Cyclosporine (1) · GC 1 compound (1) · Hematopoietic Stem Cell Transplantation (1)
Masonic Cancer Center, University of Minnesota (3) · Pujol, Aurora, M.D. (3) · Poxel SA (2) · Genetix Biotherapeutics Inc. (1) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (1) · MedDay Pharmaceuticals SA (1) · Minoryx Therapeutics, S.L. (1) · NeuroVia, Inc. (1) · Paul Szabolcs (1) · Shenzhen Second People's Hospital (1) · SwanBio Therapeutics, Inc. (1) · Talaris Therapeutics Inc. (1) · University of Florida (1)
United States (10) · Spain (5) · France (4) · Germany (3) · United Kingdom (3) · Argentina (2) · Australia (2) · Netherlands (2) · Chile (1) · China (1) · Colombia (1) · Hungary (1) · Italy (1) · Russia (1) · Ukraine (1)
Paul Orchard, MD (4) · Adrian Thrasher, MD, PhD (1) · Biljana Horn, MD (1) · Christine Duncan, MD (1) · David Williams, MD (1) · Florian Eichler, MD (1) · Frederic Sedel, MD (1) · Gerald V Raymond, M.D. (1) · GuangFu Chen, M.D.,Ph.D (1) · Hernan Amartino, MD (1) · Hua Jiang, M.D,Ph.D (1) · Jakob Sieker, MD. (1) · Jiacai Zhuo (1) · John Henderson, MD (1) · Jorn-Sven Kuhl, MD (1)
PMID 21586746, PMID 11231629, PMID 11399225, PMID 19892975, PMID 23845948, PMID 23966770, PMID 28976817, PMID 31077039, PMID 32634238, PMID 32750127, PMID 35778568, PMID 36681445, PMID 39383458, PMID 39383459