Long-Term Apitegromab for Spinal Muscular Atrophy
(ONYX Trial)
Recruiting at 67 trial locations
DW
PM
Overseen ByPaul Miller
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: Scholar Rock, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Trial Summary
What is the purpose of this trial?
This trial is testing a medication called Apitegromab to see if it is safe and effective for people with certain types of spinal muscular atrophy (SMA). It focuses on patients who have already participated in previous research. The goal is to see if the medication can help improve their muscle function over time.
Eligibility Criteria
This trial is for patients with Type 2 or Type 3 Spinal Muscular Atrophy (SMA) who completed the TOPAZ or SAPPHIRE trials. They must be able to receive infusions, travel to the site, and have a life expectancy over two years. Women of childbearing age need a negative pregnancy test and must use contraception.Inclusion Criteria
I have finished the Phase 2 TOPAZ or Phase 3 SAPPHIRE trial.
I am not pregnant and agree to use birth control during and for 20 weeks after the trial.
Able to adhere to the requirements of the protocol, including travel to the trial site and completing all trial procedures and trial visits
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Exclusion Criteria
You had a bad reaction or couldn't tolerate apitegromab before.
I don't have any current health issues that would affect my participation.
Pregnant or breastfeeding
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Treatment Details
Interventions
- Apitegromab
Trial OverviewThe ONYX study tests the long-term safety and effectiveness of Apitegromab in SMA patients from previous studies. It's an open-label extension study, meaning all participants know they are receiving Apitegromab and are monitored over time.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Treatment PeriodExperimental Treatment1 Intervention
Patients who are ≥2 years of age with Type 2 and Type 3 SMA will receive apitegromab 20 mg/kg every 4 weeks by intravenous (IV) infusion during the 104-week Treatment Period
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Who Is Running the Clinical Trial?
Scholar Rock, Inc.
Lead Sponsor
Trials
7
Recruited
710+
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