12 completed, 4 terminated, 3 withdrawn, 1 active not recruiting. 22 Phase 2, 5 Phase 1, 5 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 22 | Completed 12, Terminated 4, Withdrawn 3, Active not recruiting 1, Recruiting 1, Unknown 1 |
| Phase 1 | 5 | Terminated 2, Completed 1, Unknown 1, Withdrawn 1 |
| Phase 3 | 5 | Completed 5 |
Follow Peroxisomal Disorders: new trials by email Literature Review of Peroxisomal Disorders trials Research Report on Peroxisomal Disorders
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(AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) PXL065 - ALD (Adrenoleukodystrophy) | Phase 2 | Withdrawn | Poxel SA | 2023-09-01 | 2024-12-16 | |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(AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) PXL770 - Adrenomyeloneuropathy | Phase 2 | Withdrawn | Poxel SA | 2023-09-01 | 2024-12-16 | |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 |
| Hydroxychloroquine Administration for Reduction of Pexophagy Hydroxychloroquine, Placebo - Zellweger Syndrome, Peroxisome Biogenesis Disorders | Phase 2 | Completed | The Hospital for Sick Children | 3 | 2019-01-11 | 2020-12-17 |
| 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Male Subjects With Adrenoleukodystrophy Sobetirome (NV1205) - X-Linked Adrenoleukodystrophy | Phase 1 / Phase 2 | Withdrawn | NeuroVia, Inc. | 2018-08-01 | 2019-03-07 | |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 |
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome | Phase 2 | Active not recruiting | Masonic Cancer Center, University of Minnesota | 149 | 2014-07-10 | 2026-11-03 |
| 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 |
| Canakinumab in Patients With Active Hyper-IgD Syndrome Canakinumab - Mevalonate Kinase Deficiency | Phase 2 | Completed | Novartis Pharmaceuticals | 9 | 2011-03-01 | 2015-11-05 |
| 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 |
| Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Stem Cell Transplantation, Cyclophosphamide, Campath-1H - Hurler's Syndrome, Maroteaux-Lamy Syndrome | Phase 2 | Terminated | Masonic Cancer Center, University of Minnesota | 18 | 2008-03-01 | 2017-12-28 |
| 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 |
Showing 20 of 22. Sign in to see all of them.
Adrenoleukodystrophy (18) · Leukodystrophy, Metachromatic (8) · Leukodystrophy, Globoid Cell (7) · Mucopolysaccharidosis I (6) · alpha-Mannosidosis (5) · Fucosidosis (4) · Metabolism, Inborn Errors (4) · Mucopolysaccharidosis II (4) · Sphingolipidoses (4) · Aspartylglucosaminuria (3) · Mucopolysaccharidosis VI (3) · Mucopolysaccharidosis VII (3) · Sandhoff Disease (3) · Tay-Sachs Disease (3)
Brain Diseases (22) · Central Nervous System Diseases (22) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (22) · Genetic Diseases, Inborn (22) · Nervous System Diseases (22) · Nutritional and Metabolic Diseases (22) · Brain Diseases, Metabolic (21) · Brain Diseases, Metabolic, Inborn (21) · Metabolic Diseases (21) · Demyelinating Diseases (19) · Genetic Diseases, X-Linked (19) · Hereditary Central Nervous System Demyelinating Diseases (19) · Intellectual Disability (19) · Leukoencephalopathies (19) · Neurobehavioral Manifestations (19)
Stem Cell Transplantation (5) · Alemtuzumab (4) · Busulfan (3) · Cyclophosphamide (3) · Bone Marrow Transplantation (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) · Clofarabine (1) · Cyclosporine (1) · GC 1 compound (1) · Hematopoietic Stem Cell Transplantation (1)
Masonic Cancer Center, University of Minnesota (5) · 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) · Novartis Pharmaceuticals (1) · Paul Szabolcs (1) · Shenzhen Second People's Hospital (1) · SwanBio Therapeutics, Inc. (1) · Talaris Therapeutics Inc. (1) · The Hospital for Sick Children (1) · University of Florida (1)
United States (12) · Spain (6) · France (4) · Germany (3) · United Kingdom (3) · Argentina (2) · Australia (2) · Netherlands (2) · Canada (1) · Chile (1) · China (1) · Colombia (1) · Hungary (1) · Italy (1) · Russia (1)
Paul Orchard, MD (5) · 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 28482144, PMID 28976817, PMID 31077039, PMID 32634238, PMID 32750127, PMID 35778568, PMID 36681445, PMID 39383458, PMID 39383459