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

Purpose

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

Eligibility

Eligible Ages
Between 18 Years and 55 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

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

Exclusion Criteria

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

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
AFTX-201 		Participants will receive a single intravenous dose of AFTX-201
  • 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

Recruiting Locations

Houston Methodist Hospital
Houston, Texas 77030
Contact:
Jennifer (Garrett) Medley Sr. Clinical Trials Manager
346-238-4516
jmgarrett@houstonmethodist.org

More Details

Status
Recruiting
Sponsor
Affinia Therapeutics

Study Contact

Jennifer (Garrett) Medley Clinical Trials Information
(617) 430-7817
clinicaltrials@affiniatx.com

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