3 completed, 2 active not recruiting, 1 recruiting. 6 Phase 1, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 6 | Completed 3, Active not recruiting 2, Recruiting 1 |
| Phase 2 | 2 | Active not recruiting 1, Recruiting 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 |
|---|---|---|---|---|---|---|
| A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD) AB-1009 (GAA Gene) - Pompe Disease (Late-onset), Pompe Disease Late-Onset | Phase 1 / Phase 2 | Recruiting | AskBio Inc | 12 | 2026-04-15 | 2026-10-13 |
| A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) AB-1003 dose level 1, AB-1003 dose level 2, Placebo - Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2 | Phase 1 / Phase 2 | Active not recruiting | AskBio Inc | 9 | 2023-05-15 | 2026-08-26 |
| GDNF Gene Therapy for Parkinson's Disease AAV2-GDNF - Parkinson's Disease | Phase 1 | Active not recruiting | Brain Neurotherapy Bio, Inc. | 11 | 2020-04-01 | 2025-09-19 |
| AB-1002 in Patients With Class III Heart Failure 3 x 10e13vg AB-1002 - Congestive Heart Failure, Heart Failure | Phase 1 | Completed | AskBio Inc | 11 | 2019-11-20 | 2026-08-06 |
| AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease ACTUS-101 - Pompe Disease | Phase 1 | Completed | AskBio Inc | 7 | 2018-12-17 | 2026-04-13 |
| Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy rAAV2.5-CMV-minidystrophin (d3990) - Duchenne Muscular Dystrophy | Phase 1 | Completed | Nationwide Children's Hospital | 6 | 2006-03-01 | 2013-02-05 |
Glycogen Storage Disease Type II (2) · Muscular Dystrophies (2) · Arrhythmias, Cardiac (1) · Cardiovascular Diseases (1) · Glycogen Storage Disease (1) · Heart Failure (1) · Heart Failure, Diastolic (1) · Heart Failure, Systolic (1) · Limb-girdle muscular dystrophy type 2A (1) · Lysosomal Storage Diseases (1) · Muscular Dystrophies, Limb-Girdle (1) · Muscular Dystrophy, Duchenne (1) · Muscular Dystrophy, Limb-Girdle, Type 2I (1) · Myocardial Ischemia (1) · Neuromuscular Diseases (1)
United States (6)