Long-Term Apitegromab for Spinal Muscular Atrophy

(ONYX Trial)

Recruiting at 67 trial locations

Overseen By Paul Miller

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Scholar Rock, Inc.

No Placebo Group All 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 Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

238 Participants Needed

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

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

Treatment Details

Interventions

• Apitegromab

Trial Overview The 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

Group I: Treatment Period Experimental Treatment 1 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

Who Is Running the Clinical Trial?

Scholar Rock, Inc.

Lead Sponsor

Trials

7

Recruited

710+