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Clinical Trials/NCT07426419
NCT07426419
Not yet recruiting
Phase 1

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

Affinia Therapeutics0 sites22 target enrollmentStarted: March 1, 2026Last updated:
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InterventionsAFTX-201

Overview

Phase
Phase 1
Status
Not yet recruiting
Sponsor
Affinia Therapeutics
Enrollment
22
Primary Endpoint
Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsRecords & IdentifiersSimilar Trials

Overview

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

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 Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
18 Years to 55 Years (Adult)
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

Arms & Interventions

AFTX-201

Experimental

Participants will receive a single intravenous dose of AFTX-201

Intervention: AFTX-201 (Genetic)

Outcomes

Primary Outcomes

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

  • 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)
  • Number of participants with adverse events during long-term follow-up(Baseline through 60 months)

Investigators

Sponsor
Affinia Therapeutics
Sponsor Class
Industry
Responsible Party
Sponsor

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