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

Conditions

• Hypertrophic Cardiomyopathy

Eligibility Criteria

Sex

ALL

Age

18 Years - 75 Years

Healthy Volunteers

Not accepted

Interventions

• TN-201 — GENETIC
  TN-201 is a recombinant adeno-associated virus serotype 9 (AAV9) containing Myosin Binding Protein C (MYBPC3) transgene. It is a single (one-time) intravenous dose.

Study Details

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.

Key Dates

Start date

Aug 10, 2023

Status verified

Mar 2026

Primary completion

Aug 31, 2027

Completion

Aug 31, 2032

Study Design

Enrollment

30 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1
  Dose for Cohort 1 will be 3E13 vg/kg
• Experimental: Cohort 2
  Dose for Cohort 2 will be 6E13 vg/kg

Primary Outcome Measure

Number and severity of Adverse Events over the course of the study. [ Time Frame: 5 Years ]

Central Contacts

• Matt Pollman, M.D.
  650-209-8092
  [email protected]
• LaTanya Tomlinson, RN, MHSA
  650-825-6990
  [email protected]

Locations (10)

Find similar trials in La Jolla, CA

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