NCT07426419

An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)

A Phase 1/2 Multicenter, Open-Label, Dose-Escalation and Dose-Expansion Trial to Assess the Safety, Tolerability, Pharmacodynamics, and Preliminary Efficacy of AFTX-201 Administered to Adult Participants With BCL2-Associated Athanogene 3 (BAG3) Mutation-Associated Dilated Cardiomyopathy: UPBEAT Trial

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 22 (estimated)
• Sponsor: Affinia Therapeutics · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 1/2, open-label, dose-exploration and dose-expansion, clinical trial evaluating the safety, tolerability, pharmacodynamics, and preliminary efficacy of a single intravenous infusion of AFTX-201 in adults with dilated cardiomyopathy caused by a BAG3 gene mutation

Detailed description

Participants will receive a single intravenous infusion of AFTX-201 and will be followed for safety and clinical assessments. The study includes short-term safety monitoring and long-term follow-up for up to 60 months

Conditions

• Dilated Cardiomyopathy (DCM)
• BAG3 Mutation Associated Dilated Cardiomyopathy

Interventions

Type	Name	Description
GENETIC	AFTX-201	AFTX-201 is a gene therapy product consisting of the ATC-0187 capsid containing the BCL2-associated Athanogene 3 (BAG3) transgene. It is administered as a single intravenous infusion

Timeline

• Start date: 2026-03-01
• Primary completion: 2028-12-01
• Completion: 2032-12-01
• First posted: 2026-02-23
• Last updated: 2026-02-23

Regulatory

• FDA-regulated drug study