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

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

Category:

Neurological Diseases

Diseases Treated:

Spinal Muscular Atrophy Types 2 and 3

Eligibility Overview:

  • Between 2 and 21 years old
  • Diagnosed with SMA type 2 or type 3 and not able to walk
  • Those taking Spinraza must have taken it for at least 10 months.
  • Those taking Evrsysdi must have taken it for at least 6 months.

Eligibility exclusions:

  • Those who have previously received Zolgensma gene therapy

  1. Brief Summary

    The SAPPHIRE study will assess the safety of apetigromab compared to placebo for improving motor function in patients with later-onset spinal muscular atrophy (SMA) who are receiving either Evrysdi® (risdiplam) or Spinraza® (nusinersen). Apetigromab is a monoclonal antibody that selectively inhibits myostatin and works to address muscle atrophy in SMA patients. Therefore, it potentially complements the mechanisms of SMN-directed therapies such as Evrysdi and Spinraza.

    The study drug is given via IV every 4 weeks. The treatment period is 1 year (52 weeks) with a follow-up period of 20 weeks.

    Primary Objectives

    To determine the safety and efficacy of apetigromab in improving motor function in patients with later-onset spinal muscular atrophy who are already receiving Evrysdi or Spinraza

    Eligibility criteria

    Inclusion criteria include:

    • Between 2 and 21 years old
    • Diagnosed with SMA types 2 or 3 and not able to walk
    • Those taking Spinraza must have taken it for at least 10 months.
    • Those taking Evrysdi must have taken it for at least 6 months.

    Exclusion criteria include:

    • Those who have already had Zolgensma gene therapy

    Study Sites

    St. Jude Children’s Research Hospital, Memphis, Tennessee
    Collaborating sites globally

  2. 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.

    Purpose of this clinical trial

    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.

    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.

  3. SAPPHIRE Quick View
    Sponsor Scholar Rock, Inc.
    ClinicalTrials.gov identifier NCT05156320
    Trial start date August 2022
    Estimated enrollment 204
    Study type Interventional
    Phase Phase 3
    Conditions

    Neurological disease; spinal muscular atrophy, types 2 and 3
    Ages 2 to 21 years old
    Principal investigator Richard S. Finkel, MD
    Study site St. Jude Children’s Research Hospital
    For a consultation or to discuss SAPPHIRE St. Jude Physician/Patient Referral Office
    1-888-226-4343
    referralinfo@stjude.org

Contact

Richard S. Finkel, MD

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334
Email: referralinfo@stjude.org

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.

Close
Close