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

Phase 3

Not yet recruiting

• Conditions: neuromuscular disease; SMA; 10029317

• Registration Number: NL-OMON53608
• Lead Sponsor: Scholar Rock Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 9 April 2024

Recruitment & Eligibility

• Status: Pending
• Sex: Not specified
• Target Recruitment: 6

Inclusion Criteria

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
the trial.
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.

Exclusion Criteria

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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified