Trials / Completed

CompletedNCT05156320

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

Status: Completed
Phase: Phase 3
Study type: Interventional
Enrollment: 188 (actual)

Sponsor: Scholar Rock, Inc. · Industry
Sex: All
Age: 2 Years – 21 Years
Healthy volunteers: Not accepted

Summary

This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved survival motor neuron (SMN) upregulator therapy (i.e., either nusinersen or risdiplam), to confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.

Conditions

Interventions

Type	Name	Description
DRUG	Apitegromab	Apitegromab is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that specifically binds to human pro/latent myostatin with high affinity inhibiting myostatin activation. SRK-015 was administered every 4 weeks by intravenous (IV) infusion.
DRUG	Placebo	Placebo was administered every 4 weeks by intravenous (IV) infusion.

Timeline

Start date: 2022-04-14
Primary completion: 2024-12-18
Completion: 2024-12-18
First posted: 2021-12-14
Last updated: 2026-01-22

Locations

50 sites across 9 countries: United States, Belgium, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom

Regulatory

FDA-regulated drug study

Source: ClinicalTrials.gov record NCT05156320. Inclusion in this directory is not an endorsement.