RIDGE-1

• Research type

  Research Study

• Full title

  First-in-Human, Open-Label, Safety, Tolerability, Dose Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-401, a Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Plakophilin-2 (PKP2) Transgene, in Adults with PKP2 Mutation-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

• IRAS ID

  1009325

• Contact name

  Matthew Pollman

• Contact email

  mpollman@tenayathera.com

• Sponsor organisation

  Tenaya Therapeutics, Inc.

• Clinicaltrials.gov Identifier

  NCT06228924

• Research summary

  This first-in-human, dose finding clinical trial is designed to evaluate the safety, tolerability, and pharmacodynamics (PD) (its molecular, biochemical, and physiologic effects or actions) of TN-401 in adult patients with symptomatic PKP2 mutation-associated arrhythmogenic right ventricular cardiomyopathy (ARVC).

  There are currently no approved therapies for ARVC and none which address the underlying genetic and pathogenic cause of the PKP2 mutation associated with ARVC. Unlike the palliative therapies that constitute the current ARVC standard of care, a single intravenous dose of TN401 may provide a durable, disease modifying therapy. TN401 gene therapy has the potential to directly restore the PKP2 gene product in the cardiomyocyte whose deficit is the biological basis of this genetic cardiomyopathy.

  The trial will consist of 2 escalating dose cohorts. All patients in each cohort will receive a single IV infusion of TN-401.

  The trial period comprises 2 stages: treatment and long-term follow-up, respectively.
  This trial will be conducted at approximately 18 trial centers globally and trial participation is expected to be approximately 5.5 years in duration.

  Patients will be required to attend clinic/hospital visits whereby study procedures will be performed. TN-401 is at a research stage, so it may cause side effects that are not known at this time. Patients will be monitored throughout the study; participation is voluntary and informed consent is mandatory.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  24/LO/0241

• Date of REC Opinion

  10 Jun 2024

• REC opinion

  Further Information Favourable Opinion