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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
One-time treatment with a single ascending dose
University of California, San Diego
San Diego, California, United States
RECRUITINGMayo Clinic
Rochester, Minnesota, United States
NOT_YET_RECRUITINGMedical University of South Carolina
Charleston, South Carolina, United States
NOT_YET_RECRUITINGIncidence of Treatment-emergent Adverse Events (TEAE)
Number of participants with Adverse Events following a single IV dose of RP-A701
Time frame: Baseline up to End of Study (up to 24 months post-infusion)
Incidence of Treatment-emergent Serious Adverse Events (SAE).
Number of participants with Serious Adverse Events (SAE) following a single IV dose of RP-A701
Time frame: Baseline up to End of Study (up to 24 months post-infusion)
Incidence of Dose Limiting Toxicities (DLT).
Number of participants with Dose Limiting Toxicities (DLT) following a single IV dose of RP-A701
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.
Change in measures of LV systolic function, including LV ejection fraction (LVEF), LV global longitudinal strain.
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.
Change in peak oxygen consumption (VO2) or ventilatory efficiency (VE/VCO2 slope) by cardiopulmonary exercise test (CPET)
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.
Change in 6-minute walk distance.
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.
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Change in BAG3 myocardial 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).
Change in symptoms of HF assessed by New York Heart Association (NYHA) class.
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.
Change in Kansas City Cardiomyopathy Questionnaire (KCCQ-12) Overall Summary Score.
Time frame: Baseline up to End of Study (up to 24 months post-infusion)