Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Phase I Intramuscular Gene Transfer for Dysferlin Deficiency
NCT02710500Phase 1Completed
- Sponsor
- Sarepta Therapeutics
- Start
- Mar 2016
- Primary completion
- Jul 2019
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
Phase 1, randomised, double-blind trial of MYO-201 in dysferlinopathy (2 cohorts), sponsored by Sarepta Therapeutics. Primary endpoint: Determination of safety based on the development of unacceptable toxicity at 2 years. Started Mar 2016; status: completed.
02
Drugs
MYO-201
03
Conditions
dysferlinopathy
Beyond the preview
Go deeper on NCT02710500.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on NCT02710500.