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: Recruiting
Location: See all (4) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 70
Healthy Volunteers: f
View:
• Male or female, 18 to 70 years of age
• Truncating mutation in BAG3
• Dilated cardiomyopathy with left ventricular ejection fraction (LVEF) \< 45%
• NYHA Class II or III heart failure symptoms
• NT-proBNP ≥ 300 pg/mL for subjects in sinus rhythm or paced rhythm, or ≥ 600 pg/mL for subjects in atrial fibrillation
• Willing and able to sign informed consent and comply with study procedures
Locations
United States
Florida
University of Miami Hospital and Clinics
RECRUITING
Miami
Missouri
St. Lukes Hospital
RECRUITING
Kansas City
Texas
Houston Methodist Hospital
RECRUITING
Houston
Other Locations
Canada
Institut de Cardiologie de Montréal / Montreal Heart Institute
RECRUITING
Montreal
Contact Information
Primary
Clinical Trials Information
clinicaltrials@affiniatx.com
(617) 430-7817
Time Frame
Start Date: 2026-09
Estimated Completion Date: 2032-12
Participants
Target number of participants: 22
Treatments
Experimental: AFTX-201
Participants will receive a single intravenous dose of AFTX-201
Related Therapeutic Areas
Sponsors
Leads: Affinia Therapeutics