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

February 16, 2026 updated by: Affinia Therapeutics

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

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

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

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

Study Type

Interventional

Enrollment (Estimated)

22

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline through 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Baseline through 12 months
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
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
Assessment of the impact of a single IV dose of AFTX-201 on measurement of cardiovascular function
Baseline through 12 months
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
Baseline through 12 months
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
Baseline through 12 months
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
Baseline through 60 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Affinia Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

March 1, 2026

Primary Completion (Estimated)

December 1, 2028

Study Completion (Estimated)

December 1, 2032

Study Registration Dates

First Submitted

February 3, 2026

First Submitted That Met QC Criteria

February 16, 2026

First Posted (Actual)

February 23, 2026

Study Record Updates

Last Update Posted (Actual)

February 23, 2026

Last Update Submitted That Met QC Criteria

February 16, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AFTX-201-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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