Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of … (NCT05626855) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
United States238 participantsStarted 2023-04-17
Plain-language summary
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.
Who can participate
Age range2 Years
SexALL
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Inclusion Criteria:
* 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
Exclusion Criteria:
* 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 dru…
What they're measuring
1
Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA