RECRUITING

Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM

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Conditions

Hypertrophic CardiomyopathyHypertrophic Cardiomyopathy (HCM)Myosin Binding Protein C3 (MYBPC3)Nonobstructive HCMGenetic HCMFamilial HCMAdenoassociated Virus (AAV)Gene TherapyObstructive HCMnHCMoHCM

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is a first-in-human, non-randomized, open-label study designed to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-201 in adult patients with symptomatic hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.

Official Title

First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-201, a Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Myosin Binding Protein C Transgene, in Adults With MYBPC3 Mutation-associated Hypertrophic Cardiomyopathy (HCM)

Quick Facts

Study Start:2023-08-10
Study Completion:2032-08
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05836259

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.

Ages Eligible for Study:18 Years to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * MYBPC3 mutation
  2. * Hypertrophic Cardiomyopathy (obstructive and nonobstructive)
  3. * Left Ventricular Ejection Fraction ≥45%
  4. * NYHA Functional Class II or III symptoms
  5. * NT-proBNP ≥160pg/ml
  1. * High AAV9 neutralizing antibody titer

Contacts and Locations

Study Contact

Matt Pollman, M.D.
CONTACT
650-209-8092
mpollman@tenayathera.com
LaTanya Tomlinson, RN, MHSA
CONTACT
650-825-6990
clinical.trials@tenayathera.com

Study Locations (Sites)

UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research
La Jolla, California, 92093
United States
University of California San Francisco
San Francisco, California, 94117
United States
Emory University
Atlanta, Georgia, 30322
United States
Brigham and Women's Hospital
Boston, Massachusetts, 02115
United States
Mayo Clinic
Rochester, Minnesota, 55905
United States
The Christ Hospital Physicians - The Ohio Heart and Vascular Center
Cincinnati, Ohio, 45219
United States
Cleveland Clinic
Cleveland, Ohio, 44195
United States
Oregon Health & Science University
Portland, Oregon, 97239
United States
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
United States
Houston Methodist Hospital
Houston, Texas, 77030
United States

Collaborators and Investigators

Sponsor: Tenaya 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 Registration Dates

Study Start Date2023-08-10
Study Completion Date2032-08

Study Record Updates

Study Start Date2023-08-10
Study Completion Date2032-08

Terms related to this study

Keywords Provided by Researchers

  • Hypertrophic Cardiomyopathy (HCM)
  • Myosin Binding Protein C3 (MYBPC3)
  • Nonobstructive HCM
  • Genetic HCM
  • Familial HCM
  • Adenoassociated Virus (AAV)
  • Gene Therapy
  • Obstructive HCM
  • nHCM
  • oHCM

Additional Relevant MeSH Terms

  • Hypertrophic Cardiomyopathy