On Sunday, Dyne Therapeutics Said It Initiated Phase 3 HARMONIA Trial Of Zeleciment Basivarsen (Z-basivarsen, DYNE-101) For Myotonic Dystrophy Type 1
Benzinga Newsdesk 8 months ago
BiotechHealth CareGeneral
HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1.48-week trial will enroll approximately 150 individuals, and first sites are now open for enrollment.Primary endpoint is
- HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1.
- 48-week trial will enroll approximately 150 individuals, and first sites are now open for enrollment.
- Primary endpoint is the five times sit to stand (5xSTS) test; secondary and exploratory endpoints will assess muscle function, CNS manifestations, and patient- and clinician-reported outcomes.
- HARMONIA trial design and protocol aligned with FDA; trial intended to serve as confirmatory trial for traditional approval in the U.S. and support ex-U.S. marketing applications