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

Running, not enrolling · Phase 3

Conditions studied: Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin

In brief

The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.

Key facts

Study ID

NCT05626855

Run by

Scholar Rock, Inc.

People needed

238

Starts

2023-04-17

Expected to finish

2029-05-02

Last updated by the study team

2025-05-25

Who can join

Age: 2 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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)
• Estimated life expectancy >2 years from the Baseline Visit (Day 1)
• 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
• Able to adhere to the requirements of the protocol, including travel to the trial site and completing all trial procedures and trial visits
• Females of childbearing potential must have a negative pregnancy test at the Baseline Visit and agree to use at least 1 highly effective method of contraception throughout the trial and for 20 weeks after the last dose of apitegromab

You may not qualify if…

• Patient permanently discontinued study treatment during the feeder trial (i.e., TOPAZ or SAPPHIRE)
• Nutritional status that was not stable over the past 6 months and is not anticipated to be stable throughout the trial or medical necessity for a gastric/nasogastric feeding tube, where the majority of feeds are given by this route, as assessed by the Investigator
• Patient is currently enrolled in any investigational drug trial other than TOPAZ or SAPPHIRE
• Prior history of severe hypersensitivity reaction or intolerance to SMN-targeted therapies
• Prior history of severe hypersensitivity reaction or intolerance to apitegromab
• Use of chronic daytime noninvasive ventilatory support for >16 hours daily in the 2 weeks before dosing, or anticipated to regularly receive such daytime ventilator support chronically throughout the trial
• Any acute or comorbid condition interfering with the well-being of the patient at the patient's last visit in TOPAZ or SAPPHIRE, (including active systemic infection, the need for acute treatment, or inpatient observation due to any reason). After resolution of the condition, the patient can be enrolled in the trial if they meet all the other eligibility criteria.
• Pregnant or breastfeeding
• Any other condition or clinically significant laboratory result or ECG value that, in the opinion of the Investigator, may compromise safety or compliance, would preclude the patient from successful completion of the trial, or interfere with the interpretation of the results

Where it is running

• Phoenix Childrens Hospital — Phoenix, Arizona, United States
• UCSD Altman Clinical and Translational Research — La Jolla, California, United States
• Children's Hospital Los Angeles — Los Angeles, California, United States
• Stanford Neuroscience Health Center — Palo Alto, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Nemours Biomedical Research — Orlando, Florida, United States
• Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• University of Iowa — Iowa City, Iowa, United States
• University of Kansas Medical Center — Fairway, Kansas, United States
• Johns HopkinsHospital — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States
• Gillette Children's Specialty Healthcare — Saint Paul, Minnesota, United States
• Washington University Medical Campus — St Louis, Missouri, United States
• Columbia University Medical Center — New York, New York, United States
• Wake Forest University School of Medicine — Winston-Salem, North Carolina, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Oregon Health & Science University — Portland, Oregon, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• St. Jude Children's Research Hospital — Memphis, Tennessee, United States
• University of Texas Southwestern - Pediatric Neurology — Dallas, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• Children's Specialty Group PLLC (Children's Hospital of The King's Daughters) — Newport News, Virginia, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• University of Wisconsin Hospital — Madison, Wisconsin, United States

Full record on ClinicalTrials.gov

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