This is a registry record, not a result. A trial being listed, recruiting or completed says nothing by itself about whether the compound works or is safe.
| Registry number | NCT05156320 |
|---|---|
| Status | Completed |
| Phase | Phase 3 |
| Sponsor | Scholar Rock, Inc. |
| Enrollment | 188 participants |
| Design | Interventional, Randomized, Parallel, Masking: Quadruple |
| 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; Anti-myostatin |
| Interventions | Apitegromab; Placebo |
| Start | Apr 14, 2022 |
| Primary completion | Dec 18, 2024 |
| Study completion | Dec 18, 2024 |
| Results posted | Not yet |
| Registry last updated | Jan 22, 2026 |
Compounds on this site that cite this trial
What the registry says the trial is about
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.
Primary outcomes
These are what the trial was designed to measure first. They are registered in advance, which is what makes a later result checkable.
| Outcome measure | Time frame |
|---|---|
| Main Efficacy Population: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score. | Baseline up to 12 months. |
7 secondary outcomes also registered. See the registry record for the full list.
Source: ClinicalTrials.gov (U.S. National Library of Medicine). Last checked against the registry on Oct 3, 2026. See the trial changes feed for what has changed recently.
