NCT03921528

An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy

Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 58 (actual)
• Sponsor: Scholar Rock, Inc. · Industry
• Sex: All
• Age: 2 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.

Conditions

• Spinal Muscular Atrophy
• Spinal Muscular Atrophy Type 3
• Spinal Muscular Atrophy Type 2
• SMA
• Neuromuscular Diseases
• Muscular Atrophy
• Atrophy
• Muscular Atrophy, Spinal
• Neuromuscular Manifestations

Interventions

Type	Name	Description
BIOLOGICAL	SRK-015	SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 will be administered every 4 weeks by intravenous infusion.

Timeline

• Start date: 2019-04-22
• Primary completion: 2021-01-19
• Completion: 2024-02-28
• First posted: 2019-04-19
• Last updated: 2024-12-02
• Results posted: 2022-11-17

Locations

16 sites across 4 countries: United States, Italy, Netherlands, Spain

Regulatory

• FDA-regulated drug study