NCT05626855进行中（未招募）3 期
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
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 238
- 试验地点
- 96
- 主要终点
- Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Treatment Period
Experimental
Patients who are ≥2 years of age with Type 2 and Type 3 SMA will receive apitegromab 20 mg/kg every 4 weeks by intravenous (IV) infusion during the 104-week Treatment Period
干预措施: Apitegromab (Drug)
结局指标
主要结局
Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA
时间窗: Up to 6 years
Incidence of TEAEs and SAEs by severity
次要结局
- Further evaluate the immunogenicity of apitegromab(Up to 6 years)
- Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points(Up to 6 years)
研究者
研究点 (96)
Loading locations...
相似试验
终止
3 期
Evaluation of the Long-term Safety, Tolerability, and Efficacy of Perampanel (E2007) as an Adjunctive Therapy in Levodopa Treated Parkinson's Disease Subjects With Motor FluctuationsParkinson's DiseaseNCT00505622Eisai Limited328
已完成
3 期
A Study to Evaluate the Long-Term Safety and Tolerability of Faricimab in Participants With Diabetic Macular EdemaDiabetic Macular EdemaNCT04432831Hoffmann-La Roche1,479
已完成
3 期
A Study to Evaluate the Long-Term Safety and Tolerability of Faricimab in Participants With Neovascular Age-Related Macular DegenerationNeovascular Age-related Macular DegenerationNCT04777201Hoffmann-La Roche1,036
终止
3 期
Extension Study of the Long-term Safety and Tolerability of Octreotide Acetate in Patients With Moderately Severe or Severe Non-proliferative Diabetic Retinopathy or Low Risk Proliferative Diabetic RetinopathyDiabetic RetinopathyNCT00248157Novartis Pharmaceuticals105
终止
3 期
Extension Study of Long-term Safety and Tolerability of Octreotide Acetate in Patients With Moderately Severe or Severe Non-proliferative Diabetic Retinopathy or Low Risk Proliferative Diabetic RetinopathyDiabetic RetinopathyNCT00248131Novartis Pharmaceuticals260