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Phase I Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.Micro-dystrophin
The proposed phase I clinical trial is a pilot study to evaluate safety and biological activity of the rAAVrh74.MCK.micro-Dystrophin vector administered by an intramuscular route. This study will evaluated the micro-Dystrophin vector as a potential dystrophin replacement mechanism for Duchenne Muscular Dystrophy. Two cohorts will undergo gene transfer in a standard three-six dose escalation scheme to establish maximum tolerated dose (MTD) using toxicity. A minimum of three subjects will be enrolled into each cohort. The first cohort will receive a total dose of 3E11 vg. The second cohort will receive 1E12 vg total dose.
Details
| Lead sponsor | Jerry R. Mendell |
|---|---|
| Phase | Phase 1 |
| Status | COMPLETED |
| Enrolment | 2 |
| Start date | 2015-03 |
| Completion | 2017-09 |
Conditions
- Duchenne Muscular Dystrophy
Interventions
- rAAVrh74.MCK.micro-Dystrophin
Primary outcomes
- Safety based on number of participants with adverse events — 2 years
AEs will be monitored and scored for severity and relatedness to the study article.
Countries
United States