Dosing of patients in Phase 2 Trial of SRK-015 in Type 2 and Typ 3 of Spinal Muscular Atrophy

Scholar Rock Announces the Phase 2 clinical trial of SRK-015 in TOPAZ study .

TOPAZ Phase 2 Trial Design

The Phase 2 proof-of-concept trial will evaluate the safety and efficacy of SRK-015 dosed intravenously every four weeks (Q4W) over a 12-month treatment period. The trial is anticipated to enroll approximately 55 patients with Type 2 or Type 3 SMA in the U.S., Canada, and Europe across three distinct and parallel cohorts.

Clinical Trials
NCT03921528