Trials / Completed
CompletedNCT01976091
A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)
Phase I/IIA Gene Transfer Clinical Trial for LGMD2D (Alpha-Sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 6 (actual)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
- Sex
- All
- Age
- 7 Years
- Healthy volunteers
- Not accepted
Summary
This is an open-label, dose escalation gene transfer therapy study evaluating the safety of SRP-9004 (patidistrogene bexoparvovec) via isolated limb infusion (ILI) administration in approximately 6 participants with LGMD2D.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | SRP-9004 | Isolated Limb Infusion (ILI) |
Timeline
- Start date
- 2015-02-01
- Primary completion
- 2019-03-14
- Completion
- 2019-03-14
- First posted
- 2013-11-05
- Last updated
- 2023-06-15
- Results posted
- 2022-04-01
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT01976091. Inclusion in this directory is not an endorsement.