Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
rAAV9-DES-hGAA Re-administration in Late-Onset Pompe Disease
NCT02240407Phase 1Completed
- Sponsor
- University of Florida
- Start
- Oct 2017
- Primary completion
- Aug 2021
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
Phase 1, randomised, double-blind trial of AAV9-DES-coGAA in Pompe disease (2 arms), sponsored by University of Florida. Primary endpoint: Safety of rAAV9-DES-hGAA vector at 520 days. Started Oct 2017; status: completed.
02
Drugs
AAV9-DES-coGAA
03
Conditions
Pompe disease
Beyond the preview
Go deeper on NCT02240407.
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 NCT02240407.