Study design & results
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Trial intelligence / Profile preview
RP-A501 gene therapy in male patients with Danon disease
2023-506480-34-00Phase 2Authorised
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Jun 2024
- Modality
- Gene Therapies
- Countries
- 5
01
Overview
Phase 2, open-label, single-arm trial of RP-A501 in Danon disease, sponsored by Rocket Pharmaceuticals, Inc. Primary endpoint: Myocardial LAMP2 protein expression and decrease in left ventricular mass index. Planned start Jun 2024; status: authorised.
02
Drugs
RP-A501
03
Conditions
Danon disease
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Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
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