ONYX
Research type
Research Study
Full title
An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab
IRAS ID
1006172
Contact name
Desiree Warne
Contact email
Sponsor organisation
Scholar Rock, Inc.
Eudract number
2022-001771-14
Research summary
‘ONYX’ is a study of an investigational drug (also known as the “study drug”) called apitegromab as a possible treatment for spinal muscular atrophy or ‘SMA’ for short.
Apitegromab is a protein that acts upon a muscle protein called myostatin. Myostatin is one of the factors that control the size of muscles and motor function (how well muscles work). Once patients develop symptoms of SMA, they progressively lose the use of their muscles over time, patients’ muscles can become so weak that moving, breathing, and eating become difficult.
Apitegromab has been evaluated in healthy adults and in children and adults who have symptoms of later onset SMA. These studies have shown that apitegromab may help to improve or stabilise motor function in patients who cannot walk, when combined with another treatment called nusinersen. To date no safety concerns have been detected in these studies.
During the ONYX study, participants will continue to receive their regular medication for their SMA whether it is nusinersen (Spinraza) or Risdiplam (Evrysdi). Both of these medications are approved by the Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of SMA but and are only available via managed access agreements. However, the use of these medications with apitegromab is not an approved combination for the treatment of SMA.
This study is an extension to another study already reviewed and approved in UK by MHRA, HRA and East Midlands - Derby Research Ethics Committee called ‘SAPPHIRE’. Participants who completed the SAPPHIRE study will be asked if they would like to participate in ONYX.
The main goal of this study is to learn how well the study drug works and how safe the study drug is when taken long-term. Some SAPPHIRE participants will have been on placebo (an inactive drug) but ONYX gives all participants (including those who received placebo in SAPPHIRE) the opportunity to be treated with active study drug.REC name
Wales REC 5
REC reference
23/WA/0161
Date of REC Opinion
14 Aug 2023
REC opinion
Further Information Favourable Opinion