NCT03614234已完成3 期
Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 29
- 试验地点
- 27
- 主要终点
- Evaluation of treatment-related adverse events
研究概览
简要总结
The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.
详细描述
This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of treatment will be until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completion of study PB-102-F
- •The patient signs informed consent.
- •Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, effective contraception method. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination.
排除标准
- •Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.
研究组 & 干预措施
Experimental open label
Experimental
Pegunigalsidase alfa
干预措施: pegunigalsidase alfa (Drug)
结局指标
主要结局
Evaluation of treatment-related adverse events
时间窗: Throughout the study, 364 weeks
CTCAE v4.03
次要结局
- Pain assessment(Every 6 months throughout the duration of the study, 364 weeks)
- Cardiac assessment(Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364)
- Biomarkers for Fabry disease(Every 6 months throughout the duration of the study, 364 weeks)
- Kidney function 1(Every 6 months throughout the duration of the study, 364 weeks)
- Kidney function 2(Every 6 months throughout the duration of the study, 364 weeks)
- Clinical assessment(Every four weeks throughout the duration of the study, 364 weeks)
- Symptom assessment(Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364)
- Quality of life assessment(Every 6 months throughout the duration of the study, 364 weeks)
研究者
研究点 (27)
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