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

Phase 2 Completed 58 enrolled Scholar Rock, Inc.

RandomizedParallel-groupOpen-labelTreatment

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.

Timeline

Start: 2019-04-22
Primary completion: 2021-01-19
Completion: 2024-02-28

Publications

Crawford TO, Darras BT, Day JW, Dunaway Young S, Duong T, Nelson LL, Barrett D, Song G, Bilic S, Cote S, Sadanowicz M, Iarrobino R, Xu TJ, O'Neil J, Rossello J, Place A, Kertesz N, Nomikos G, Chyung Y. Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study. Neurology. 2024 Mar 12;102(5):e209151. doi: 10.1212/WNL.0000000000209151. Epub 2024 Feb 8.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Apitegromab	Monoclonal antibody	2 mg/kg	Intravenous
Subject	Apitegromab	Monoclonal antibody	20 mg/kg	Intravenous

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

Summary

Timeline

Publications

Drugs

Indications