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Abstract Details

A Phase 2 Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy (TOPAZ): An Introduction
Neuromuscular and Clinical Neurophysiology (EMG)
P8 - Poster Session 8 (8:00 AM-9:00 AM)
1-012
N/A
SRK-015 is a fully human anti-proMyostatin monoclonal antibody that’s being developed and investigated for the treatment of later-onset SMA.  This Phase 2 study will be conducted in approximately 25 study sites across United States, Canada and Europe to evaluate the safety and efficacy of SRK-015 on motor function in Types 2 and 3 SMA patients, aged 2 through 21 years old for 52 weeks.  
Approximately 55 male and female later-onset SMA patients will be enrolled across 3 separate parallel Cohorts.  All patients will receive SRK-015 every 4 weeks via intravenous infusion. Patients in Cohorts 1 and 2 will be directly assigned to a 20 mg/kg SRK-015 dose and patients in Cohort 3 randomized 1:1 in a double-blind manner to either 2 mg/kg or 20 mg/kg SRK-015.  Cohort 1 will enroll ambulatory Type 3 patients at least some of whom are not receiving an approved SMA treatment and patients already receiving an approved SMA treatment that had been started after the patient turned 5 years old.  Cohort 2 will enroll Type 2 and non-ambulatory Type 3 patients, aged 5-21 years old, already receiving an approved SMA treatment that had been started after the patient turned 5 years old. Cohort 3 will enroll Type 2 patients, already receiving an approved SMA treatment that was started before the patient turned 5 years old. The primary efficacy endpoint for Cohort 1 is the change from baseline in the Revised Hammersmith Scale.  The primary efficacy endpoint for Cohorts 2 and 3 is change from baseline in Hammersmith Functional Motor Scale Expanded.  Safety will be assessed throughout the trial by the Safety Surveillance Team. Blood samples for the measurement of SRK-015 concentrations, circulating latent myostatin concentrations, and anti-SRK-015 antibodies will be obtained. Baseline patient characteristics, demographics and disease history will be presented.   
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Authors/Disclosures
Amy Place
PRESENTER 		Amy Place has received personal compensation for serving as an employee of Scholar Rock. Amy Place has received personal compensation in the range of $0-$499 for serving as an Expert Witness for Tegus. Amy Place has stock in Scholar Rock.