An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab - SRK-015-004

Scholar Rock Inc.0 个研究点目标入组 6 人开始时间: 待定最近更新: 2024年4月9日

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•1. Informed consent document signed by the patient if the patient is legally an
•adult. If the patient is legally a minor, informed consent document signed by
•the patient's parent or legal guardian and patient's oral or written assent
•obtained, if applicable and in accordance with the regulatory and legal
•requirements of the participating location.
•2. Patients who have completed the Phase 2 TOPAZ (Study SRK-015-002) trial or
•the Phase 3 SAPPHIRE (Study SRK-015-003) trial.
•3. Estimated life expectancy >2 years from Baseline (Day 1).
•4. 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 (i.e., for background medical
•care and not for the purpose of receiving apitegromab in the trial), throughout
•5. Able to adhere to the requirements of the protocol.
•6. Females of childbearing potential must have a negative pregnancy test at
•Baseline and agree to use at least 1 acceptable method of contraception
•throughout the trial and for 20 weeks after the last dose of apitegromab.
•Female patients who are expected to have reached reproductive maturity by the
•end of the trial must agree to adhere to trial-specific contraception
•requirements.

•1. Patient permanently discontinued study treatment during the feeder trial
•(i.e., TOPAZ or SAPPHIRE).
•2. 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.
•3. Patient is currently enrolled in any investigational drug trial other than
•TOPAZ or SAPPHIRE.
•4. Prior history of severe hypersensitivity reaction or intolerance to
•SMNtargeted therapies.
•5. Prior history of severe hypersensitivity reaction or intolerance to
•apitegromab.
•6. 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.
•7. 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.
•8. Pregnant or breastfeeding.
•9. 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.

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