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

ConditionsDilated Cardiomyopathy (DCM)BAG3 Mutation Associated Dilated Cardiomyopathy
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-70
SponsorAffinia Therapeutics

About this 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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

None

Disqualifiers

None

Trial design

Design model

Single group

Treatments tested in this trial

  • 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

Treatment groups

22 Participants
are divided into 1 treatment group
Group A: AFTX-201Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

Safety and tolerability of AFTX-201 over 12 months.

Time frame
Baseline through 12 months

Secondary outcomes

1

Change from baseline in pharmacodynamic biomarkers

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

Time frame
Baseline through 12 months
2

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

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

Time frame
Baseline through 12 months
3

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

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

Time frame
Baseline through 12 months
4

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

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

Time frame
Baseline through 12 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Affinia Therapeutics

Lead sponsor

Source ClinicalTrials.gov
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