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A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

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NCT07047144 · readout ≤ 833 d

Sponsored by Scholar Rock, Inc. (industry) · SRRK — their whole pipeline →.

Phase
Phase 2
Status
Recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
52estimated
Sites
25
Countries
Belgium, France, Italy +4

Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →

Source: ClinicalTrials.gov, retrieved 2026-08-19

Dates

each axis at the precision it was filed, with the registry's own basis
DateFiledBasisWhat it is
Start2025-09-15actualWhen the study began enrolling
Primary completionNov 2028estimatedThe date the last participant is measured for the primary outcome — the readout window
Study completionMar 2029estimatedThe whole study's end, after follow-up
First posted2025-07-02actualWhen this record first appeared on the registry
Results postedWhen the sponsor posted results to the registry
Record updated2026-05-01actualThe sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Conditions

  • Spinal Muscular Atrophy
  • SMA
  • Spinal Muscular Atrophy Type 2
  • Spinal Muscular Atrophy Type 3
  • Neuromuscular Manifestations
  • Anti-myostatin

Interventions

  • Drug: Apitegromab — also filed as SRK-015
  • Drug: Nusinersen — also filed as Spinraza
  • Drug: Risdiplam — also filed as Evrysdi

Primary outcomes

what the primary-completion date above is the date OF
Evaluate the PK of apitegromab in subjects <2 years old with SMA
measured 52 Weeks
Evaluate the PD of apitegromab in subjects <2 years old with SMA
measured 52 Weeks
Evaluate the motor function outcomes (ie, efficacy) due to apitegromab treatment
measured 48 Weeks

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