1 active not recruiting, 1 completed, 1 recruiting. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Active not recruiting 1 |
| Phase 2 | 1 | Active not recruiting 1 |
Follow TUBB4A-Related Leukodystrophy: new trials by email Literature Review of TUBB4A-Related Leukodystrophy trials Research Report on TUBB4A-Related Leukodystrophy
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 |
|---|---|---|---|---|---|---|
| An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy nL-TUBB4-001 - Genetic Disease, TUBB4A-Related Leukodystrophy | Phase 1 / Phase 2 | Active not recruiting | University of California, San Diego | 1 | 2025-09-19 | 2025-10-29 |
| 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 |
Adrenoleukodystrophy (2) · Aicardi-Goutieres syndrome (2) · Alexander Disease (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) · Gangliosidoses (2) · Gangliosidoses, GM2 (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Hypomyelinating, 5 (2) · Leukodystrophy, Hypomyelinating, 6 (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Abnormalities, Multiple (2) · Adrenal Gland Diseases (2) · Adrenal Insufficiency (2) · Arthritis (2) · Arthritis, Rheumatoid (2) · Attention Deficit and Disruptive Behavior Disorders (2) · Autoimmune Diseases (2) · Bone Diseases (2) · Bone Diseases, Developmental (2) · Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Brain Infarction (2) · Brain Ischemia (2)
Children's Hospital of Philadelphia (2) · University of California, San Diego (1)
United States (3)
U01NS106845 (1), U54NS115052 (1)