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NCT Number: NCT07426419

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

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

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Key information

Conditions

Dilated Cardiomyopathy (DCM) BAG3 Mutation Associated Dilated Cardiomyopathy Cardiomegaly Cardiomyopathies Cardiomyopathy, Dilated Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heart Diseases Laminopathies

Age range

18 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

University of Miami Hospital and Clinics, Miami, Florida, United States

Loading trial locations.

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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

Primary outcomes

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

    Time frame: Baseline through 12 months

    Safety and tolerability of AFTX-201 over 12 months.

Secondary outcomes

  1. Change from baseline in pharmacodynamic biomarkers

    Time frame: Baseline through 12 months

    Assessment of the extent of AFTX-201 transduction and myocardial BAG3 protein expression after a single IV dose

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

    Time frame: Baseline through 12 months

    Assessment of the impact of a single IV dose of AFTX-201 on measurement of cardiovascular function

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

    Time frame: Baseline through 12 months

    Assessment of the impact of a single IV dose of AFTX-201 on measurement of cardiovascular function

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

    Time frame: Baseline through 12 months

    Assessment of the impact of a single IV dose of AFTX-201 on features of heart failure (HF) including functional classification by New York Heart Association (NYHA) class

  5. Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)

    Time frame: Baseline through 12 months

    Assessment of the impact of a single IV dose of AFTX-201 on quality of life

  6. Number of participants with adverse events during long-term follow-up

    Time frame: Baseline through 60 months

    Safety and tolerability of AFTX-201 over 60 months

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Trials Information

CONTACT

[email protected]

(617) 430-7817 ext. 2

Sponsors and collaborators

Lead sponsor

Affinia Therapeutics

Industry

Registry information

Official study 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

Acronym: UPBEAT

Important dates

Study start
2026
Primary completion
2028
Study completion
2032
First posted
Feb 23, 2026
Registry last updated
Jul 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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