A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Replacement Therapy in Participants With Mucopolysaccharidosis I (MPS I) in China
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 12
- 试验地点
- 5
- 主要终点
- Participants with adverse events (AEs)
研究概览
简要总结
This is a single treatment arm study that is open-label to be conducted in Chinese participants with MPS I.
Trial Objectives are to evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants, and to evaluate the efficacy of Aldurazyme on the percent change of urinary glycosaminoglycans (uGAGs) from baseline to Week 26. The study will also evaluate the effect on uGAG level and liver volume (hepatomegaly) after 26 weeks, with Aldurazyme treatment in Chinese MPS I participants.
Treatment duration will include: 2 weeks of screening, 26 weeks of treatment and 1 week of follow-up period. During the treatment period, weekly visits are designed to accommodate weekly administration of Aldurazyme (laronidase).
详细描述
Study duration for each participant will be a total of 29 weeks which will include 2 weeks of screening, 26 weeks of treatment period and 1 week of follow-up period.
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
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Chinese participants have a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and fibroblast or leukocyte IDUA (iduronidase) activity <10% of normal.
- •Participants have to be able to stand independently and walk a minimum of 5 meters in 6 minutes.
- •A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies:
- •Is not a woman of childbearing potential (WOCBP).
- •Is a WOCBP and agrees to use an acceptable contraceptive method during the intervention period and at a minimum until 7 days after the last dose of study intervention.
- •A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) within the screening period before the first dose of study intervention.
- •If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.
- •Contraceptive/barrier method is not applicable for male participants.
排除标准
- •Prior tracheostomy or bone marrow transplantation or hematopoietic stem cell transplantation.
- •Have a plan to undergo bone marrow transplantation or hematopoietic stem cell transplantation within half a year after enrollment.
- •Received an investigational drug, or device, other than Aldurazyme, within 30 days prior to study enrollment.
- •Received an investigational gene therapy.
- •The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
研究组 & 干预措施
Aldurazyme (laronidase)
Aldurazyme (laronidase) treatment at approved dose and regimen, administered every week as an IV infusion
干预措施: Laronidase (Drug)
结局指标
主要结局
Participants with adverse events (AEs)
时间窗: Baseline to Week 27
Incidence of AEs, serious adverse events (SAEs) and adverse events of special interest (AESIs) including infusion associated reactions (IARs) during the treatment emergent (TE) period
The incidence of Potentially clinically significant abnormality (PCSA) analyses for clinical laboratory, vital signs, and ECG parameters during the TE period
时间窗: Baseline to Week 27
The percent change of uGAGs
时间窗: Baseline to Week 26
次要结局
- The percent change of uGAGs(Baseline to Week 2, Week 4, Week 8, Week 12 and Week 20)
- The absolute change of uGAGs(Baseline to Week 2, Week 4, Week 8, Week 12, Week 20 and Week 26)
- The percent change of liver volume (Abdominal B type ultrasound examination)(Baseline to Week 26)