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RP-A501 Gene Therapy in Male Patients With Danon Disease
NCT03882437Phase 1Completed
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Apr 2019
- Primary completion
- Jul 2025
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of RP-A501 in Danon disease, sponsored by Rocket Pharmaceuticals, Inc. Primary endpoints: Number of participants with treatment-related adverse events as assessed by NCI CTCAE at 3 years, Evaluation of cardiomyocyte histologic correction following administration of RP-A501 via endomyocardial biopsy at 3 years and 1 more. Started Apr 2019; status: completed.
02
Drugs
RP-A501
03
Conditions
Danon disease
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