A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
Recruiting · NCT07137338 · Interventional (participants receive a specific treatment) · Lead sponsor: Rocket Pharmaceuticals Inc.
View the official record on ClinicalTrials.gov →Interventions studied
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)
What this trial is about
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 take part
Age range
18 Years to 65 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 1
Study type
Interventional (participants receive a specific treatment)
Inclusion criteria
- Subjects are eligible for inclusion into the study only if all the following criteria apply:
- 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
- NYHA Class II or III HF symptoms with stable HF therapeutic guideline-directed medical regimen for 30 days 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
- Severe RV dysfunction assessed by echocardiogram or CMR ≤ 12 months prior to screening
- LVEF \< 25% by echocardiogram or CMR at ≤ 3 months prior to screening
- NYHA Class I or IV HF
Where it is running
3 locations listed across 3 US states.
- University of California, San Diego - San Diego, California, United States
- Mayo Clinic - Rochester, Minnesota, United States
- Medical University of South Carolina - Charleston, South Carolina, United States