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Life sciences · Phase 1 Trial
ClinicalTrials.gov · October 1, 2026
No summary has been generated for this record yet. What follows is drawn from its source metadata only.
Phase 1 Trial.
No findings were extractable from the material analysed.
Safety was not reported in the material analysed. Check the source before drawing any conclusion about harm.
The source did not state who this applies to in practice.
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Registry record from ClinicalTrials.gov (NCT07137338). This is a study registration, not published results. Lead sponsor: Rocket Pharmaceuticals Inc.. Recruitment status: RECRUITING. Phase: PHASE1. Study type: INTERVENTIONAL. Enrollment: 8 participants (ESTIMATED). Conditions: Dilated Cardiomyopathy (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). Primary outcome measures: Incidence of Treatment-emergent Adverse Events (TEAE) , Baseline up to End of Study (up to 24 months post-infusion); Incidence of Treatment-emergent Serious Adverse Events (SAE). , Baseline up to End of Study (up to 24 months post-infusion); Incidence of Dose Limiting Toxicities (DLT). , Baseline up to End of Study (up to 24 months post-infusion). Brief summary: 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.