Contact SCGE




Gene Therapy Trial Report

Summary

A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy


NCTID NCT07137338 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication 🔄 BAG3-Associated Dilated Cardiomyopathy (DCM)
Disease Ontology Term 🔄 DOID:0110448
Drug Product Name 🔄 RP-A701
Sponsor Rocket Pharmaceuticals Inc.
Funder Type Industry
Recruitment Status 🔄
Enrollment Count 8 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 BAG3
Therapeutic Modality 🔄 Gene transfer
Therapy Route 🔄 in vivo
Mechanism of Action 🔄 Functional gene replacement
Route of Administration 🔄 Intravenous
Drug Product Type 🔄 Viral vector
Gene Delivery System Type 🔄 Viral transduction
Vector Type 🔄 Adeno-associated virus
Viral Vector Subtype AAVrh74
Dose 1 🔄 Undisclosed

Study Record Dates


Current Phase Phase1
Submit Date 2025-07-25
Completion Date 2029-06
Last Update 🔄 2026-06-30

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
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 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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 🔄 3
Locations 🔄 United States

Regulatory Information


Has US IND True
FDA Designations 🔄 Fast Track
Recent Updates First patient dosing in Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy anticipated in mid-2026

Resources/Links