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Recruiting NCT07426419

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

Phase I / Phase II Interventional Dilated Cardiomyopathy (DCM) BAG3 Mutation Associated Dilated Cardiomyopathy

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: AFTX-201.
Who it may be relevant to
Registry conditions: Dilated Cardiomyopathy (DCM), BAG3 Mutation Associated Dilated Cardiomyopathy. Basic parameters: 18 years — 55 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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

Interventions

  • 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

Primary outcome measures

  • Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) [Time frame: Baseline through 12 months]
Secondary outcome measures (6)
  • Change from baseline in pharmacodynamic biomarkers [Time frame: Baseline through 12 months]
  • Change from baseline in measurement of cardiac function including left ventricular ejection fraction (LVEF) [Time frame: Baseline through 12 months]
  • Change from baseline in measurement of cardiac function including peak oxygen consumption (pVO2) by cardiopulmonary exercise test (CPET) [Time frame: Baseline through 12 months]
  • Change from baseline in New York Heart Association (NYHA) functional class [Time frame: Baseline through 12 months]
  • Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) [Time frame: Baseline through 12 months]
  • Number of participants with adverse events during long-term follow-up [Time frame: Baseline through 60 months]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 3 centers
  • University of Miami Hospital and Clinics — Miami
  • St. Lukes Hospital — Kansas City
  • Houston Methodist Hospital — Houston

Identifiers

NCT: NCT07426419 · AFTX-201-001

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗