Recruiting NCT07137338
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: 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).
- Who it may be relevant to
- Registry conditions: Dilated Cardiomyopathy (DCM). Basic parameters: 18 years — 65 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human BCL2-associated Athanogene 3 (BAG3) Gene Coding Sequence (RP-A701) in Subjects With Dilated Cardiomyopathy Arising From Pathogenic BAG3 Variants (BAG3-DCM)
Overview
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.
Interventions
- Genetic 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
Primary outcome measures
- Incidence of Treatment-emergent Adverse Events (TEAE) [Time frame: Baseline up to End of Study (up to 24 months post-infusion)]
- Incidence of Treatment-emergent Serious Adverse Events (SAE). [Time frame: Baseline up to End of Study (up to 24 months post-infusion)]
- Incidence of Dose Limiting Toxicities (DLT). [Time frame: Baseline up to End of Study (up to 24 months post-infusion)]
Secondary outcome measures (6)
- To assess the impact of RP-A701 on features of cardiovascular function. [Time frame: Baseline up to End of Study (up to 24 months post-infusion)]
- To assess the impact of RP-A701 on features of cardiovascular function. [Time frame: Baseline up to End of Study (up to 24 months post-infusion)]
- To assess the impact of RP-A701 on features of cardiovascular function. [Time frame: Baseline up to End of Study (up to 24 months post-infusion)]
- To assess the extent of RP-A701 transduction and protein expression. [Time frame: Baseline up to End of Study (up to 24 months post-infusion)]
- To assess the impact of RP-A701 on features of heart failure (HF). [Time frame: Baseline up to End of Study (up to 24 months post-infusion)]
- To assess the impact of RP-A701 on quality of life. [Time frame: Baseline up to End of Study (up to 24 months post-infusion)]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 3 centers
- University of California, San Diego — San Diego
- Mayo Clinic — Rochester
- Medical University of South Carolina — Charleston
Identifiers
NCT: NCT07137338 · RP-A701-0125