2024-515304-39-00已完成2 期
A Long-Term follow-up Study to evaluate safety and tolerability of olipudase alfa in patients who completed the DFI12712 or the LTS13632 Study in France - PTA17397
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 3
- 试验地点
- 2
- 主要终点
- Number of adverse events (AEs) / serious adverse events (SAEs)
研究概览
简要总结
The objective of this study is to assess safety and tolerability of olipudase alfa in patients with ASMD who have completed the DFI12712 or LTS13632 study in France until olipudase alfa reimbursement is granted in France or until 5 years whichever comes first.
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •The patient has completed Study the DFI12712 (ASCEND) or LTS13632 in France
- •The patient must provide signed, informed consent prior to performing any study related procedures.
- •The patient is willing to comply with the clinical protocol.
- •The patient, if female and of childbearing potential, must have a negative pregnancy test result [urine beta-human chorionic gonadotropin (β-HCG)] at enrollment.
- •Sexually active female patients of childbearing potential and male patients are required to practice true abstinence in line with their preferred and usual lifestyle or to use 2 acceptable effective methods of contraception for the entire duration of the treatment period and for at least 28 days after receiving the last study drug dose.
排除标准
- •Any patient who has not participated in the Study DFI12712 or the Study LTS13632
- •A patient who experienced any systemic hypersensitivity reactions to olipudase alfa in Study DFI12712 or Study LTS13632 which, in the opinion of the Investigator, could indicate that treatment continuation may present an unreasonable risk.
- •The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
- •The patient is unwilling or unable to abstain from alcohol for 1 day prior to and 3 days after each olipudase alfa infusion for the duration of the treatment period.
- •Individuals accommodated in an institution because of regulatory or legal order; prisoners or participants who are legally institutionalized
- •The patient is concurrently participating in another clinical study of investigational treatment
- •Any of the following medical conditions: - The patient has any new condition or worsening of an existing condition which, in the opinion of the Investigator, would make the patient unsuitable for enrollment or could interfere with the patient’s participating in or completing the study. - Requirement for recurrent dose adjustment of anticoagulation treatment over the last 6 months. - Pregnancy or breastfeeding.
研究组 & 干预措施
Xenpozyme (olipudase alfa,GZ402665)
Test
干预措施: Xenpozyme (olipudase alfa,GZ402665) (Drug)
结局指标
主要结局
Number of adverse events (AEs) / serious adverse events (SAEs)
Number of adverse events (AEs) / serious adverse events (SAEs)
次要结局
未报告次要终点
研究者
Clinical Sciences and Operations
Scientific
Sanofi Winthrop Industrie
研究点 (2)
Loading locations...
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