ong-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab
- Conditions
- Spinal Muscular Atrophy (SMA)MedDRA version: 20.1Level: LLTClassification code 10041583Term: Spinal muscular atrophy, unspecifiedSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2022-001771-14-PL
- Lead Sponsor
- Scholar Rock, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 260
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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 235
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 27
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
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 SMN-targeted 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 clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA;Secondary Objective: Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points<br><br>Further evaluate the immunogenicity of apitegromab;Primary end point(s): Incidence of TEAEs and SAEs by severity<br><br>;Timepoint(s) of evaluation of this end point: Evaluted at timepoints as described in the protocol
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - HFMSE total score at prespecified time points (excludes TOPAZ Cohort 1 patients)<br>- RULM total score at prespecified time points (excludes TOPAZ Cohort 1 patients)<br>- Number of WHO motor development milestones attained at prespecified time points (excludes TOPAZ Cohort 1 patients)<br>- RHS total score and results for 6-Minute Walk Test, 30-Second Sit-to-Stand, 10-Meter Walk/Run (from the RHS), and timed rise from floor (from the RHS) at prespecified time points (TOPAZ Cohort 1 patients only);Timepoint(s) of evaluation of this end point: Evaluted at timepoints as described in the protocol