2 completed, 2 terminated, 1 recruiting. 2 Phase 2, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Completed 1, Terminated 1 |
| Phase 1 | 1 | Terminated 1 |
| Phase 3 | 1 | Completed 1 |
Follow AMN: new trials by email Literature Review of AMN trials Research Report on AMN
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 |
|---|---|---|---|---|---|---|
| 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 |
| Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia Natural History Observation - AMN, AMN Gene Mutation | Terminated | SwanBio Therapeutics, Inc. | 65 | 2021-06-21 | 2025-10-30 | |
| 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 | |
| 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 |
Adrenoleukodystrophy (5) · Leukoencephalopathies (4) · Peroxisomal Disorders (4) · Adrenal Insufficiency (2) · Aicardi-Goutieres syndrome (2) · Alexander Disease (2) · Alexanders leukodystrophy (2) · Allan-Herndon-Dudley syndrome (2) · Attention Deficit Disorder with Hyperactivity (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · CADASIL (2) · Canavan Disease (2) · Charcot-Marie-Tooth Disease (2) · Cockayne Syndrome (2)
Adrenal Gland Diseases (5) · Brain Diseases (5) · Central Nervous System Diseases (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Endocrine System Diseases (5) · Genetic Diseases, Inborn (5) · Genetic Diseases, X-Linked (5) · Intellectual Disability (5) · Nervous System Diseases (5) · Neurobehavioral Manifestations (5) · Neurologic Manifestations (5) · Nutritional and Metabolic Diseases (5) · X-Linked Intellectual Disability (5) · Congenital Abnormalities (4) · Hereditary Sensory and Motor Neuropathy (4)
Biotin (1)
Children's Hospital of Philadelphia (2) · SwanBio Therapeutics, Inc. (2) · MedDay Pharmaceuticals SA (1)
United States (4) · Germany (2) · Netherlands (2) · France (1) · Spain (1)
Adeline Vanderver, MD (2) · Frederic Sedel, MD (1) · Patrick Aubourg, MD (1)
U01NS106845 (1), U54NS115052 (1)