1 enrolling by invitation, 1 recruiting. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Enrolling by invitation 1 |
| Phase 2 | 1 | Enrolling by invitation 1 |
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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 |
|---|---|---|---|---|---|---|
| rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease rAAV-Olig001-ASPA, Levetiracetam, Prednisone - Canavan Disease | Phase 1 / Phase 2 | Enrolling by invitation | Myrtelle Inc. | 24 | 2021-04-01 | 2025-11-14 |
| The Myelin Disorders Biorepository Project Leukodystrophy, White Matter Disease | Recruiting | Children's Hospital of Philadelphia | 12000 | 2016-12-08 | 2025-10-23 |
Canavan Disease (2) · Adrenoleukodystrophy (1) · Aicardi-Goutieres syndrome (1) · Alexander Disease (1) · Alexanders leukodystrophy (1) · Allan-Herndon-Dudley syndrome (1) · Attention Deficit Disorder with Hyperactivity (1) · CADASIL (1) · Charcot-Marie-Tooth Disease (1) · Cockayne Syndrome (1) · Gangliosidoses (1) · Gangliosidoses, GM2 (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Hypomyelinating, 5 (1) · Leukodystrophy, Hypomyelinating, 6 (1)
Levetiracetam (1) · Prednisone (1)
United States (2)
Adeline Vanderver, MD (1) · Robert Lober, MD, PhD (1)
U01NS106845 (1), U54NS115052 (1)