9 completed, 2 terminated. 11 Phase 3, 5 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 11 | Completed 9, Terminated 2 |
| Phase 2 | 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 |
|---|---|---|---|---|---|---|
| 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 |
| A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD) Lenti-D - Cerebral Adrenoleukodystrophy (CALD) | Phase 3 | Completed | Genetix Biotherapeutics Inc. | 35 | 2019-01-24 | 2024-05-24 |
| 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 |
| A Study of Canakinumab in Patients With Systemic Juvenile Idiopathic Arthritis or Hereditary Periodic Fevers Who Participated in the CACZ885G2301E1, CACZ885G2306 or CACZ885N2301 Studies canakinumab - Systemic Juvenile Idiopathic Arthritis, Hereditary Periodic Fevers | Phase 3 | Completed | Novartis Pharmaceuticals | 31 | 2014-11-03 | 2020-05-19 |
| 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 |
| 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 |
| Betaine and Peroxisome Biogenesis Disorders Betaine - Peroxisome Biogenesis Disorders | Phase 3 | Completed | McGill University Health Centre/Research Institute of the McGill University Health Centre | 12 | 2013-03-01 | 2016-06-28 |
| ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases ALD-101 - Inherited Metabolic Diseases, Lysosomal Storage Disorders | Phase 3 | Terminated | Aldagen | 40 | 2008-03-01 | 2014-07-08 |
| A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy Lorenzo's oil - Adrenomyeloneuropathy, Adrenoleukodystrophy | Phase 3 | Terminated | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 240 | 2005-03-01 | 2012-03-02 |
| 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 |
| Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid Cholic Acids - Infantile Refsum's Disease, Zellweger Syndrome | Phase 3 | Completed | Mirum Pharmaceuticals, Inc. | 85 | 1992-01-01 | 2023-10-03 |
Adrenoleukodystrophy (9) · Fucosidosis (2) · Gangliosidosis, GM1 (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Metachromatic (2) · Refsum Disease, Infantile (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · Zellweger Syndrome (2) · Arthralgia (1) · Arthritis, Juvenile (1) · Bronchiolitis Obliterans Syndrome (1) · Canavan Disease (1) · Cholestasis (1)
Brain Diseases (11) · Brain Diseases, Metabolic (11) · Brain Diseases, Metabolic, Inborn (11) · Central Nervous System Diseases (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Genetic Diseases, Inborn (11) · Metabolic Diseases (11) · Nervous System Diseases (11) · Nutritional and Metabolic Diseases (11) · Metabolism, Inborn Errors (10) · Neurologic Manifestations (10) · Adrenal Gland Diseases (9) · Adrenal Insufficiency (9) · Demyelinating Diseases (9) · Endocrine System Diseases (9)
Antilymphocyte Serum (1) · Betaine (1) · Biotin (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cholic Acid (1) · Cholic Acids (1) · Cyclophosphamide (1) · Lorenzo's oil (1) · Plasma Exchange (1) · Stem Cell Transplantation (1) · canakinumab (1) · leriglitazone (1)
Genetix Biotherapeutics Inc. (2) · Aldagen (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · Masonic Cancer Center, University of Minnesota (1) · McGill University Health Centre/Research Institute of the McGill University Health Centre (1) · MedDay Pharmaceuticals SA (1) · Minoryx Therapeutics, S.L. (1) · Mirum Pharmaceuticals, Inc. (1) · Novartis Pharmaceuticals (1) · Pujol, Aurora, M.D. (1)
United States (7) · France (5) · Germany (4) · Spain (3) · United Kingdom (3) · Italy (2) · Netherlands (2) · Argentina (1) · Australia (1) · Canada (1) · Hungary (1)
Paul Orchard, MD (2) · Adrian Thrasher, MD, PhD (1) · Christine Duncan, MD (1) · David Williams, MD (1) · Florian Eichler, MD (1) · Frederic Sedel, MD (1) · Gerald V Raymond, M.D. (1) · Hernan Amartino, MD (1) · Himal Lal Thakar, MD (1) · Jakob Sieker, MD. (1) · James Heubi, MD (1) · James Hinson, MD (1) · Joanne Kurtzberg, MD (1) · Jorn-Sven Kuhl, MD (1) · Kenneth Setchell, PhD (1)
PMID 39383458, PMID 21586746, PMID 28644367, PMID 28976817, PMID 36681445, PMID 39383459
2014 (1), 2018 (1)