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SAPPHIRE: Efficacy and Safety of Apitegromab in Patients with Spinal Muscular Atrophy Receiving Nusinersen or Risdiplam Therapy

About this study

This study will test how well the drug apetigromab works for children with spinal muscular atrophy. Researchers want to find out if it is safe and helps improve muscle and motor function. They will compare improvements in those taking the study drug to those taking a placebo. Those who take part in this study will continue taking their Evrysdi® (risdiplam) or Spinraza® (nusinersen).

Apetigromab works by stopping muscles from getting smaller and weaker. This is called muscle atrophy. Researchers want to find out if apetigromab helps muscle function improve or stay the same in those who cannot walk.

Patients in this study will take the study drug or placebo for 1 year while they stay on their prescribed Spinraza or Evrysdi. The study drug is given by IV infusion once a month.

Eligibility overview

  • Between 2 and 21 years old
  • A diagnosis of SMA type 2 or 3, and not able to walk
  • Those taking Spinraza must have taken it for at least 10 months.
  • Those taking Evrsydi must have taken it for at least 6 months.

Those who have taken Zolgensma cannot take part in this study.

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Overview

Full title:

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

Study goal:

This study aims to find out if the drug apetigromab is a safe and effective treatment when used along with the standard treatment for children with spinal muscular atrophy type 2 or 3.

Diagnosis:

Spinal Muscular Atrophy Types 2 and 3

Age:

Between 2 and 21 years old

Clinical trials categories:

Neurological Disorders Spinal Muscular Atrophy (SMA)

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

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