Ignite Creation Date:
2024-05-06 @ 6:55 PM
Last Modification Date:
2024-10-26 @ 2:57 PM
Study NCT ID:
NCT05836259
Status:
RECRUITING
Last Update Posted:
2024-05-21
First Post:
2023-04-18
Brief Title:
Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
Sponsor:
Tenaya Therapeutics
Organization:
Tenaya Therapeutics
Study Overview
Official Title:
First-in-Human Open-Label Safety Tolerability Dose-Finding Pharmacodynamic and Cardiac Transgene Expression Study of TN-201 a Recombinant Adenoassociated Virus Serotype 9 AAV9 Containing Myosin Binding Protein C Transgene in Adults With MYBPC3 Mutation-Associated Hypertrophic Cardiomyopathy HCM
Status:
RECRUITING
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
MyPEAK-1
Brief Summary:
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 MYBPC3 mutation-associated nonobstructive hypertrophic cardiomyopathy nHCM
Detailed Description:
The study will consist of 2 escalating dose cohorts The study will enroll at least 6 and as many as 15 patients All patients will receive active drug The study will follow patients for 5 years following a single dose of TN-201
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None