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.
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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…
Contacts

Clinical Information

646-627-0033

clinicaltrials@rocketpharma.com

CONTACT