3 recruiting, 1 active not recruiting, 1 approved for marketing, 1 completed. 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 1 | Active not recruiting 1 |
Follow Alexander Disease: new trials by email Literature Review of Alexander Disease trials Research Report on Alexander Disease
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 the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD) zilganersen, Placebo - Alexander Disease | Phase 3 | Active not recruiting | Ionis Pharmaceuticals, Inc. | 54 | 2021-06-01 | 2026-08-21 |
| Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases. collection of data - Inherited Disease, Rare Diseases | Recruiting | Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta | 100 | 2021-03-01 | 2024-11-19 | |
| 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 | |
| Evaluation of Outcome Metrics in Alexander Disease Alexander Disease | Recruiting | Children's Hospital of Philadelphia | 200 | 2016-01-26 | 2026-01-15 | |
| Zilganersen Expanded Access Program for Individuals With Alexander Disease zilganersen - Alexander Disease | Approved for marketing | Ionis Pharmaceuticals, Inc. | 2026-09-29 |
Adrenoleukodystrophy (3) · Leukodystrophy, Globoid Cell (3) · Leukodystrophy, Metachromatic (3) · Leukoencephalopathies (3) · Pelizaeus-Merzbacher Disease (3) · Xanthomatosis, Cerebrotendinous (3) · Aicardi-Goutieres syndrome (2) · Alexanders leukodystrophy (2) · Allan-Herndon-Dudley syndrome (2) · Attention Deficit Disorder with Hyperactivity (2) · CADASIL (2) · Canavan Disease (2) · Charcot-Marie-Tooth Disease (2) · Cockayne Syndrome (2)
Brain Diseases (6) · Brain Diseases, Metabolic (6) · Brain Diseases, Metabolic, Inborn (6) · Central Nervous System Diseases (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Demyelinating Diseases (6) · Hereditary Central Nervous System Demyelinating Diseases (6) · Heredodegenerative Disorders, Nervous System (6) · Metabolism, Inborn Errors (6) · Neurodegenerative Diseases (6) · Nutritional and Metabolic Diseases (6) · Genetic Diseases, Inborn (5) · Metabolic Diseases (5) · Nervous System Diseases (5) · Adrenal Gland Diseases (3)
Children's Hospital of Philadelphia (3) · Ionis Pharmaceuticals, Inc. (2) · Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (1)
United States (4) · Italy (2) · Australia (1) · Canada (1) · Israel (1) · Japan (1) · Netherlands (1) · United Kingdom (1)
Adeline Vanderver, MD (2) · Amy Waldman, MD (1)
PMID 32342562, PMID 32749716, PMID 37782421, PMID 37920915, PMID 41667276
U01NS106845 (1), U54NS115052 (1)