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
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Sanofi
- 入组人数
- 3
- 试验地点
- 4
- 主要终点
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
This was an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingomyelinase deficiency (ASMD) who completed the DFI12712 or the LTS13632 Study in France until olipudase alfa reimbursement was granted in France.
Study and treatment duration:
The period between the participant's completion of Study DFI12712 or LTS13632 and olipudase alfa reimbursement was available in France.
In case reimbursement was not obtained, this study ended 5 years after starting.
Visit frequency: every 2 weeks.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant has completed Study DFI12712 (ASCEND) or LTS13632 in France
- •The participant must provide signed, informed consent prior to performing any study-related procedures.
- •The participant was willing to comply with the clinical protocol.
- •The participant, if female and of childbearing potential, must have had a negative pregnancy test result [urine beta-human chorionic gonadotropin (β-HCG)] at enrollment.
- •Sexually active female participants of childbearing potential and male participants were 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
- •Participants are excluded from the study if any of the following criteria apply:
- •Any participant who has not participated in the Study DFI12712 or the Study LTS13632
- •A participant 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 participant, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
- •The participant was 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 were legally institutionalized.
- •The participant was concurrently participating in another clinical study of investigational treatment.
- •Any of the following medical conditions:
- •The participant has had any new condition or worsening of an existing condition which, in the opinion of the Investigator, would make the participant unsuitable for enrollment or could interfere with the participant's participating in or completing the study.
- •Requirement for recurrent dose adjustment of anticoagulation treatment over the last 6 months.
- •Pregnancy or breastfeeding.
- •The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
研究组 & 干预措施
GZ402665
Olipudase alfa administered intravenously every 2 weeks
干预措施: Olipudase alfa (Drug)
结局指标
主要结局
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months
An AE was any untoward medical occurrence in participant or clinical study participant temporally associated with the use of study drug, whether or not considered related to the study drug. An SAE was any AE, that at any dose: resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was a medically important event.
Number of adverse events (AEs) / serious adverse events (SAEs)
时间窗: Baseline to approximately 5 years
次要结局
未报告次要终点