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Monoclonal Antibodies

Long-Term Apitegromab for Spinal Muscular Atrophy (ONYX Trial)

Phase 3

Waitlist Available

Research Sponsored by Scholar Rock, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients have completed the Phase 2 TOPAZ (Study SRK-015-002) trial or the Phase 3 SAPPHIRE (Study SRK-015-003) trial (For TOPAZ, completed is defined as completion of Visit EC14 in Extension Period C or participating in TOPAZ at the time the trial is ended. For SAPPHIRE, completed is defined as completion of Visit 14 or participating in SAPPHIRE at the time the trial is ended)

Able to receive study drug infusions and provide blood samples through the use of a peripheral IV or a long-term IV access device that the patient has placed for reasons independent from the trial

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 6 years

Awards & highlights

ONYX Trial Summary

This trial will study the long-term safety and effectiveness of a drug to treat Type 2 and 3 Spinal Muscular Atrophy.

Who is the study for?

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.Check my eligibility

What is being tested?

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.See study design

What are the potential side effects?

While specific side effects aren't listed here, common ones may include reactions at the infusion site, potential allergic responses if there's a history of hypersensitivity to similar treatments, and general drug-related adverse effects.

ONYX Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have finished the Phase 2 TOPAZ or Phase 3 SAPPHIRE trial.

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I can receive medication through an IV and give blood samples.

ONYX Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 6 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 6 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 6 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA

Secondary outcome measures

Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points

Further evaluate the immunogenicity of apitegromab

Other outcome measures

Further characterize the PK of apitegromab

Further evaluate the pharmacodynamic (PD) effects of apitegromab

To further evaluate the effect of apitegromab on patient/caregiver-reported disability, fatigability, and suicidal ideation and behavior

ONYX Trial Design

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

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Scholar Rock, Inc.Lead Sponsor

3 Previous Clinical Trials

358 Total Patients Enrolled

Media Library

Apitegromab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT05626855 — Phase 3

Spinal Muscular Atrophy Research Study Groups: Treatment Period

Spinal Muscular Atrophy Clinical Trial 2023: Apitegromab Highlights & Side Effects. Trial Name: NCT05626855 — Phase 3

Apitegromab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05626855 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has the federal agency granted sanction to this particular treatment protocol?

"Treatment Period has been found to have a high level of safety due its Phase 3 status, which signals the presence of data both for efficacy and multiple rounds backing up its security; thus, it scored an impressive three."

Answered by AI

Is enrollment in this research still open?

"Per the clinicaltrials.gov website, this medical investigation is not presently recruiting participants. The trial was initially posted on April 24th 2023 and last modified on November 15th 2022. Although no longer enrolling patients, 234 other studies are actively soliciting candidates at present."

Answered by AI

In what geographic regions is this experiment taking place?

"Currently, 19 healthcare centres are taking part in this medical trial. These sites encompass cities such as Saint Paul, Phoenix and Chicago along with 16 additional locations. To reduce travelling demands associated with the study's participation, it is advised that patients choose a site closest to them for enrollment."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX

2023. "Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05626855.

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