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RecruitingPhase 1

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

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)

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years to 65 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This early study tests RP-A701, a gene therapy, in high-risk adults with BAG3 dilated cardiomyopathy, a condition that makes the heart enlarged and weak. It checks safety, side effects, and early signs of benefit after one dose given into a vein.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be 18 to 65 years old.
  • Be able and willing to sign a form agreeing to join the study.
  • Have an enlarged, weak heart that pumps out 25% to 45% of its blood with each beat, shown by a heart scan within 3 months of joining.
  • The weak heart must not be caused by severely blocked heart arteries or an active lack of blood flow to the heart.
  • Have no uncontrolled high blood pressure, more than moderate heart valve disease, abnormal buildup in heart tissue, or whole-body disease known to weaken the heart.
  • Have a confirmed BAG3 gene change that causes or likely causes disease.
  • Have had an implanted defibrillator, a device that treats dangerous heart rhythms, for at least 3 months.
  • Have heart failure symptoms that limit ordinary or lighter activity, but be comfortable at rest. Be on stable, recommended heart failure treatment for at least 30 days.

Who can't join

  • Have heart or blood vessel disease that may be caused by a gene change other than BAG3.
  • Have taken part in a gene transfer or gene editing study before.
  • Have received medicine into a vein to strengthen heart pumping, widen blood vessels, or remove extra fluid within 30 days of joining.
  • Have had a blood clot inside the heart or a clot that traveled to an artery.
  • Have severe weakness of the heart’s right pumping chamber on a heart scan within 12 months before screening.
  • Have a heart scan showing that the left pumping chamber pumps out less than 25% of its blood per beat within 3 months before screening.
  • Have heart failure with no limits on ordinary activity, or symptoms even at rest.

Simplified for readability. The official criteria below are what the study team uses.

Official 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

Study sites (3)

CA

MN

SC