SAPPHIRE

• Research type

  Research Study

• 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

• IRAS ID

  1004906

• Contact name

  Stephanie Howard

• Contact email

  showard@scholarrock.com

• Sponsor organisation

  Scholar Rock, Inc.

• Eudract number

  2021-005314-34

• Clinicaltrials.gov Identifier

  NCT05156320

• Research summary

  Scholar Rock, Inc. is sponsoring a study of an investigational drug called apitegromab (SRK 015) as a possible treatment for later-onset spinal muscular atrophy (SMA).
  Apitegromab is a protein that acts upon a muscle protein called myostatin. Myostatin is one of the factors that control the size and function of muscles.
  The main goal of this study is to learn how well apitegromab (SRK-015) works and how safe apitegromab (SRK-015) is compared with placebo when added to treatments with nusinersen and risdiplam. A placebo is an inactive material that looks like the study drug but does not have any active drug, in this case apitegromab (SRK-015). Researchers use a placebo to see if apitegromab (SRK-015) works better or is safer than taking nothing.
  This study will take place at about 55 to 60 study centres globally and will include about 204 patients with SMA (approximately 156 patients who are 2 to 12 years of age and 48 patients who are 13 to 21 years of age). The study is divided into 3 periods: a screening period (up to 4 weeks), a treatment period (52 weeks), and a follow-up period (20 weeks). Participants will be in this study for approximately 76 weeks (about 18 months), and participants will need to come to the study centre 18 times over this period. If the study drug helps the participants' condition, participants may be offered the option to enter an extension research study after they complete 52 weeks (12 months) of treatment.
  Participants will have a 67% (2 in 3) chance of receiving apitegromab and a 33% (1 in 3) chance of receiving placebo. Neither participants nor the study doctor can choose which study drug participants receive. Participants, the Sponsor studying the drug, the study doctor, and almost all other people involved in the study will not know whether participants are receiving apitegromab or placebo.
  A description of this clinical study is available on http://www.clinicaltrials.gov. ClinicalTrials.gov

• REC name

  East Midlands - Derby Research Ethics Committee

• REC reference

  22/EM/0054

• Date of REC Opinion

  8 Apr 2022

• REC opinion

  Further Information Favourable Opinion