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Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC

ConditionArrhythmogenic Right Ventricular Cardiomyopathy
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-65
SponsorTenaya Therapeutics

About this trial

This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

PKP2 mutation (pathogenic or likely pathogenic)

Arrhythmogenic Right Ventricular Cardiomyopathy as defined by the 2010 revised Task Force Criteria

Left Ventricular Ejection Fraction ≥50%

Functioning Implantable Cardiac Defibrillator with remote integration capabilities at least 9 months prior to Screening

Disqualifiers

Ventricular tachycardia (VT) ablation within 6 months of Screening or planned VT ablation within 6 months after Screening

High AAV9 neutralizing antibody titer

Prior myocardial infarction

Right Ventricular Heart Failure

Trial design

Design model

Sequential

Treatments tested in this trial

  • TN-401

    Genetic

    TN-401 is a recombinant adeno-associated virus serotype 9 (AAV9) gene therapy containing Plakophilin-2 (PKP2) transgene. It is a single (one-time) intravenous dose.

Treatment groups

15 Participants
are divided into 2 treatment groups
Group A: Cohort 1Experimental treatment 1 intervention
Group B: Cohort 2Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number and severity of Adverse Events over the course of the study.

Time frame
52 weeks
2

Number of Serious Adverse Events related to study drug.

Time frame
5 years

Secondary outcomes

1

To assess changes in daily PVC and NSVT counts

Time frame
Week 52
2

To assess frequency of ICD therapy administration

Time frame
Week 52
3

To assess frequency of sustained VT

Time frame
Week 52

Other outcomes

Sponsors and contacts

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

Tenaya Therapeutics

Lead sponsor

Source ClinicalTrials.gov
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