A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subject… (NCT07137338) | Clinical Trial Compass
RecruitingPhase 1
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
United States8 participantsStarted 2026-06
Plain-language summary
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.
Who can participate
Age range
18 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male or female between 18 and 65 years of age at the time of signing the informed consent
. Capable of and willing to provide signed informed consent
. Clinical diagnosis of DCM defined as and requiring each of the following:
. Mild to moderate systolic dysfunction (LVEF ≥ 25% and ≤ 45%) by echocardiography or CMR performed within 3 months of enrollment.
. Absence of severe coronary artery disease (\>70% stenosis) or active myocardial ischemia as the etiology of LV systolic dysfunction
. Absence of uncontrolled hypertension, significant cardiac valve disease (i.e., greater than moderate in severity), infiltrative disorder, or systemic disease known to cause cardiomyopathy.
. Documentation of a pathogenic or likely pathogenic variant in BAG3
. History of ICD implantation ≥ 3 months prior to enrollment
Exclusion criteria
. CV disease that may be related to a genetic etiology other than a BAG3 pathogenic or likely pathogenic variant.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of Treatment-emergent Adverse Events (TEAE)
Timeframe: Baseline up to End of Study (up to 24 months post-infusion)
2
Incidence of Treatment-emergent Serious Adverse Events (SAE).
Timeframe: Baseline up to End of Study (up to 24 months post-infusion)
3
Incidence of Dose Limiting Toxicities (DLT).
Timeframe: Baseline up to End of Study (up to 24 months post-infusion)