A Study to Investigate Safety and Tolerability of Higher Infusion Rate to shORten the duraTion of FabrazymE Infusion
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Sanofi
- 入组人数
- 8
- 试验地点
- 5
- 主要终点
- Percent Reduction in Shortest Tolerated Infusion Duration From Pre-study Average of Recent 3 Infusions
研究概览
简要总结
This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume. This study aims to generate data to provide the guidance on how infusion rate can be safely increased and minimize the burden of the life-long treatment with Fabrazyme.
详细描述
The total duration will be up to 7 months
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 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •- Participants with confirmed diagnosis of FD who are ≥2 and ≤65 years of age at the time of signing the informed consent form (ICF) or assent, if applicable.
- •Cohort 1: female participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- •Cohort 2: non-classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- •Cohort 3: classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- •Cohort 4: participants with body weight <30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- •Women of childbearing potential must use a highly effective method of contraception through the study.
排除标准
- •Female participants who are pregnant or breastfeeding.
- •History of significant allergic disease or hypersensitivity to Fabrazyme or other medicinal products.
- •Contraindication to Fabrazyme or any of the premedications or rescue medications (diphenhydramine, loratadine, cetirizine, fexofenadine, acetaminophen, montelukast, dexamethasone).
- •Any other medical condition considered to make the increased infusion rate not tolerable at the Investigator's discretion.
- •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
agalsidase beta
agalsidase beta 1 mg/kg infusion once every other week
干预措施: AGALSIDASE BETA (GZ419828) (Drug)
agalsidase beta
agalsidase beta 1 mg/kg infusion once every other week
干预措施: Acetaminophen (Drug)
agalsidase beta
agalsidase beta 1 mg/kg infusion once every other week
干预措施: Diphenhydramine (Drug)
agalsidase beta
agalsidase beta 1 mg/kg infusion once every other week
干预措施: Dexamethasone (Drug)
agalsidase beta
agalsidase beta 1 mg/kg infusion once every other week
干预措施: Montelukast (Drug)
agalsidase beta
agalsidase beta 1 mg/kg infusion once every other week
干预措施: Loratadine (Drug)
agalsidase beta
agalsidase beta 1 mg/kg infusion once every other week
干预措施: Cetirizine (Drug)
agalsidase beta
agalsidase beta 1 mg/kg infusion once every other week
干预措施: Fexofenadine (Drug)
结局指标
主要结局
Percent Reduction in Shortest Tolerated Infusion Duration From Pre-study Average of Recent 3 Infusions
时间窗: Up to 16 weeks
Percent reduction was with respect to the shortest tolerated infusion for the participant. The shortest infusion duration tolerated was the actual shortest duration (highest infusion rate) of a completed infusion tolerated by the participant without experiencing a second infusion associated reaction (IAR). The pre-study infusion duration was derived as the average of the duration of 3 most recent infusions the participant received prior to participating to this study. Percent reduction of infusion duration was calculated as: 100 x (average of pre-study infusion duration \[minutes\] - actual shortest infusion duration tolerated \[minutes\]) / average of pre-study infusion duration (minutes).
Shortest Actual Tolerated Infusion Duration
时间窗: Up to 16 weeks
The shortest infusion duration tolerated was the actual shortest duration (highest infusion rate) of a completed infusion tolerated by the participant without experiencing a second IAR. IARs were events recorded as adverse event of special interest (AESI) and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun.
次要结局
- Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing Any Infusion Associated Reaction(Up to 16 weeks)
- Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing a Second Infusion Associated Reaction(Up to 16 weeks)
- Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing Any Infusion Associated Reaction(Up to 16 weeks)
- Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing a Second Infusion Associated Reaction(Up to 16 weeks)
- Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing Any Infusion Associated Reaction(Up to 16 weeks)
- Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing a Second Infusion Associated Reaction(Up to 16 weeks)