BridgeMD
Tests treatment safety and results for Dilated Cardiomyopathy (DCM)
Official title A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
ClinicalTrials.gov ID: NCT07137338
What this study is testing
What is 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) is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for dilated cardiomyopathy (dcm).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65
You may be able to join if
- people 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.
You likely can't join if
- 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
See the full eligibility criteria
- people 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
- 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
The study team makes the final eligibility decision.
Where it's taking place
- San Diego, California, United States
- Rochester, Minnesota, United States
- Charleston, South Carolina, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Diego, California, United States; Rochester, Minnesota, United States; Charleston, South Carolina, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.