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 range18 Years – 65 Years
SexALL
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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.
✕. Previous participation in a study of gene transfer or gene editing.
✕. I.V. inotropic, vasodilator, or diuretic therapy ≤ 30 days prior to enrollment.
✕. History of intracardiac thrombosis or arterial thromboembolic events
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)