NCT06666413招募中4 期
A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)
Genzyme, a Sanofi Company1 个研究点 分布在 1 个国家目标入组 13 人开始时间: 2025年5月7日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- 入组人数
- 13
- 试验地点
- 1
- 主要终点
- Incidence of AEs, SAEs, AESIs including IARs during the TE period
研究概览
简要总结
This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT.
Study details include:
-
The study duration: total study duration is approximately 64 weeks.
-
Screening period of up to 8 weeks
-
Treatment period of 52 weeks
-
Follow-up period of 4 weeks. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 weeks)
-
The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.
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
- — 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be <18 years of age, at the time of signing the informed consent.
- •Participants have documented onset of Pompe disease symptoms before 12 months of age (corrected for gestation if born before 40 weeks); and diagnosis of IOPD is confirmed by GAA enzyme deficiency from any tissue source and GAA gene pathogenic mutations.
- •Participants must have documented cardiomyopathy at the time of diagnosis.
- •Contraceptive use should be consistent with local regulations Participant's parent/legally authorized representative (LAR) must be capable of giving signed informed consent.
排除标准
- •Participants are excluded from the study if any of the following criteria apply:
- •Participants with major congenital abnormality that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.
- •Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).
- •Participants who have received an ERT other than alglucosidase alfa or avalglucosidase alfa, or any other treatment for Pompe disease, including gene therapy prior to the enrollment.
- •Participants who have received alglucosidase alfa or avalglucosidase alfa less than 1 week prior to the first dose of avalglucosidase alfa given as IMP Participants who are anticipated to take prohibited therapy (ie, any other treatment for Pompe disease) during this study.
- •Participants who have taken other investigational drugs (not Pompe disease specific) within 30 days or 5 elimination half-lives in blood of that drug before enrollment, whichever is longer, or are anticipated to take any other concurrent investigational treatments.
- •Participants not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.
- •The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
研究组 & 干预措施
avalglucosidase alfa
Experimental
Avalglucosidase alfa will be administered by IV infusion following reconstitution and dilution at a dose of 20 mg/kg body weight qow
干预措施: Avalglucosidase alfa (Drug)
结局指标
主要结局
Incidence of AEs, SAEs, AESIs including IARs during the TE period
时间窗: From baseline to Week 56
Abnormality in clinical laboratory, vital signs, and ECG parameters during the TE period
时间窗: From baseline to Week 56
次要结局
- Proportion of participants who are alive and free of invasive ventilation at Week 52(At Week 52)
- Change from baseline to Week 52 in LVM Z score evaluated by echocardiography(From baseline to Week 52)
- The absolute change in body growth Z scores from baseline to Week 52(From baseline to Week 52)
- The percentiles of the body growth Z scores changes from baseline to Week 52(From baseline to Week 52)
- Change from baseline to Week 52 in distance walked during six-minute walk test (6MWT) (in applicable participants)(From baseline to Week 52)
- Change from baseline to Week 52 in quick motor function test (QMFT) score (in applicable participants)(From baseline to Week 52)
- Number of motor development milestones achieved as evaluated by Motor Milestone Checklist at Week 52(At Week 52)
研究者
研究点 (1)
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