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
RECRUITINGMedical University of South Carolina
Charleston, South Carolina, United States
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)