8 Participants NeededMy employer runs this trial
Gene Therapy for Dilated Cardiomyopathy
Recruiting at 2 trial locations
CI
Overseen ByClinical Information
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: Rocket Pharmaceuticals Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
What You Need to Know Before You Apply
What is the purpose of 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.
Are You a Good Fit for This Trial?
Inclusion Criteria
I am between 18 and 65 years old.
I have stable heart failure and have been on a steady treatment plan for over 30 days.
I am willing and able to sign the consent form.
See 5 more
What Are the Treatments Tested in This Trial?
Interventions
- RP-A701
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Group I: Single ascending dose of RP-A701 in up to 2 consecutive cohortsExperimental Treatment1 Intervention
Participants will receive a single intravenous dose of RP-A701 on Day 0 and will be followed for up to two years
Find a Clinic Near You
Who Is Running the Clinical Trial?
Rocket Pharmaceuticals Inc.
Lead Sponsor
Trials
17
Recruited
430+
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