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

NCT07426419 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: AFTX-201-001
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

Brief 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

Conditions

Dilated Cardiomyopathy (DCM); BAG3 Mutation Associated Dilated Cardiomyopathy

Keywords

AFTX-201; Gene Therapy; AAV; BAG3; Dilated Cardiomyopathy; DCM; ATC-0187

Outcome Measures

Primary Outcomes (1)

• Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

  Safety and tolerability of AFTX-201 over 12 months.

  Baseline through 12 months

Secondary Outcomes (6)

• Change from baseline in pharmacodynamic biomarkers

  Baseline through 12 months

• Change from baseline in measurement of cardiac function including left ventricular ejection fraction (LVEF)

  Baseline through 12 months

• Change from baseline in measurement of cardiac function including peak oxygen consumption (pVO2) by cardiopulmonary exercise test (CPET)

  Baseline through 12 months

• Change from baseline in New York Heart Association (NYHA) functional class

  Baseline through 12 months

• Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)

  Baseline through 12 months

Study Arms (1)

AFTX-201

EXPERIMENTAL

Participants will receive a single intravenous dose of AFTX-201

Genetic: AFTX-201

Interventions

AFTX-201 GENETIC

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

AFTX-201

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Male or female, 18 to 55 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

You may not qualify if:

• Prior myocardial infarction, heart transplant, or presence/requirement of a left ventricular assist device (LVAD)
• IV therapy with positive inotropes, vasodilators, or diuretics for heart failure within 30 days prior to enrollment
• Positive neutralizing antibodies to ATC-0187
• Any condition that, in the investigator's opinion, would place the subject at undue risk or interfere with study participation or interpretation of results

Sponsors & Collaborators

• Affinia Therapeutics: lead

Study Sites (1)

Houston Methodist Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Cardiomyopathy, Dilated

Condition Hierarchy (Ancestors)

Cardiomegaly; Heart Diseases; Cardiovascular Diseases; Cardiomyopathies; Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Clinical Trials Information

CONTACT

(617) 430-7817; clinicaltrials@affiniatx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 3, 2026
• First Posted: February 23, 2026
• Study Start: June 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2032
• Last Updated: June 9, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)