7 completed, 2 terminated. 9 Phase 3, 5 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 9 | Completed 7, Terminated 2 |
| Phase 2 | 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
Fucosidosis (2) · Gangliosidosis, GM1 (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Metachromatic (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · Canavan Disease (1) · Cholestasis (1) · Gaucher Disease (1) · Lysosomal Storage Diseases (1) · Metabolism, Inborn Errors (1) · Mucolipidoses (1) · Mucopolysaccharidoses (1) · Mucopolysaccharidosis I (1)
Adrenal Gland Diseases (9) · Adrenal Insufficiency (9) · Brain Diseases (9) · Brain Diseases, Metabolic (9) · Brain Diseases, Metabolic, Inborn (9) · Central Nervous System Diseases (9) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Demyelinating Diseases (9) · Endocrine System Diseases (9) · Genetic Diseases, Inborn (9) · Genetic Diseases, X-Linked (9) · Hereditary Central Nervous System Demyelinating Diseases (9) · Heredodegenerative Disorders, Nervous System (9) · Intellectual Disability (9) · Leukoencephalopathies (9)
Antilymphocyte Serum (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) · 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) · MedDay Pharmaceuticals SA (1) · Minoryx Therapeutics, S.L. (1) · Mirum Pharmaceuticals, Inc. (1) · Pujol, Aurora, M.D. (1)
United States (7) · France (4) · Germany (4) · Spain (3) · United Kingdom (3) · Italy (2) · Netherlands (2) · Argentina (1) · Australia (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
2018 (1)