A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
Recruiting
Phase 1
Interventional Study
Dilated Cardiomyopathy (DCM)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 18 – 65
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 8 (estimated)
- Sponsor
- Rocket Pharmaceuticals Inc. · Industry
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About This Trial
This is a Phase 1, open-label, dose-escalation trial to characterize the safety, tolerability, and preliminary efficacy of RP-A701 following a single IV administration in high-risk adult patients with BAG3-DCM.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
Subjects are eligible for inclusion into the study only if all the following criteria apply:
1. Male or female between 18 and 65 years of age at the time of signing the informed consent
2. Capable of and willing to provide signed informed consent
3. Clinical diagnosis of DCM de…
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