CompletedPhase 3

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

United States188 participantsStarted 2022-04-14

Plain-language 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.

Who can participate

Age range2 Years – 21 Years

SexALL

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Inclusion criteria

✓. If receiving the SMN upregulator therapy nusinersen, must have completed at least 10 months of dosing (i.e., completed the loading regimen and at least 2 maintenance doses) before Screening;
✓. If receiving the SMN upregulator therapy risdiplam, must have completed at least 6 months of dosing before Screening.

What they're measuring

Main Efficacy Population: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score.

Timeframe: Baseline up to 12 months.

Trial details

NCT IDNCT05156320

SponsorScholar Rock, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-12-18

View on ClinicalTrials.gov More Spinal Muscular Atrophy trials