RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)
RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3): One-time treatment with a single ascending dose
Study 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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
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 defined as and requiring each of the following:
1. Mild to moderate systolic dysfunction (LVEF ≥ 25% and ≤ 45%) by echocardiography or CMR performed within 3 months of enrollment.
2. Absence of severe coronary artery disease (\>70% stenosis) or active myocardial ischemia as the etiology of LV systolic dysfunction
3. Absence of uncontrolled hypertension, significant cardiac valve disease (i.e., greater than moderate in severity), infiltrative disorder, or systemic disease known to cause cardiomyopathy.
4. Documentation of a pathogenic or likely pathogenic variant in BAG3
5. History of ICD implantation ≥ 3 months prior to enrollment
6. NYHA Class II or III HF symptoms with stable HF therapeutic guideline-directed medical regimen for 30 days prior to enrollment
Exclusion Criteria:
1. CV disease that may be related to a genetic etiology other than a BAG3 pathogenic or likely pathogenic variant.
2. Previous participation in a study of gene transfer or gene editing.
3. I.V. inotropic, vasodilator, or diuretic therapy ≤ 30 days prior to enrollment.
4. History of intracardiac thrombosis or arterial thromboembolic events
5. Severe RV dysfunction assessed by echocardiogram or CMR ≤ 12 months prior to screening
6. LVEF \< 25% by echocardiogram or CMR at ≤ 3 months prior to screening
7. NYHA Class I or IV HF
Primary outcome measure(s)
Incidence of Treatment-emergent Adverse Events (TEAE) — Baseline up to End of Study (up to 24 months post-infusion) Number of participants with Adverse Events following a single IV dose of RP-A701
Incidence of Treatment-emergent Serious Adverse Events (SAE). — Baseline up to End of Study (up to 24 months post-infusion) Number of participants with Serious Adverse Events (SAE) following a single IV dose of RP-A701
Incidence of Dose Limiting Toxicities (DLT). — Baseline up to End of Study (up to 24 months post-infusion) Number of participants with Dose Limiting Toxicities (DLT) following a single IV dose of RP-A701
Trial sites (3)
Facility
City
Region
Status
University of California, San Diego
San Diego
California
Recruiting
Mayo Clinic
Rochester
Minnesota
Recruiting
Medical University of South Carolina
Charleston
South Carolina
Recruiting
More Rocket Pharmaceuticals Inc. trials in the USA
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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