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

Outcome

Mixed primary results

paper The primary efficacy measure was mean change from baseline using the Hammersmith Functional Motor Scale version appropriate for each cohort. PMID 38330285 ↗

paper At month 12, the mean change from baseline in Hammersmith scale score was -0.3 points (95% CI -2.1 to 1.4) in cohort 1 PMID 38330285 ↗

paper 0.6 points (-1.4 to 2.7) in cohort 2 (n = 15), PMID 38330285 ↗

paper 7.1 (1.8 to 12.5) for the 2-mg/kg (n = 8) and 20-mg/kg (n = 9) arms, respectively. PMID 38330285 ↗

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