NCT04910776进行中（未招募）3 期
An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- Sanofi
- 入组人数
- 17
- 试验地点
- 16
- 主要终点
- Proportion of participants who are alive and free of invasive ventilation at Week 52
研究概览
简要总结
This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD.
Study details include:
- Study duration: Screening - up to 4 weeks;
- Primary Analysis Period (PAP) - 52 weeks;
- Extended Treatment Period (ETP) - 52 weeks;
- Extended Long term Treatment Period (ELTP) - 104 weeks; 4-week follow-up period for a total study duration - up to 4.08 years.
- Treatment duration: Up to 4 years
- Visit frequency: every other week and potentially every week
详细描述
Study duration may be variable by country, including at least completion of the PAP and ETP, and up to 4.08 years.
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
- 0 Days 至 12 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must have confirmed diagnosis of infantile-onset Pompe disease defined as: the presence of 2 lysosomal acid α-glucosidase (GAA) pathogenic variants and a documented GAA deficiency from blood, skin, or muscle tissue; or the presence of 1 GAA pathogenic variant and a documented GAA deficiency from blood, skin and muscle tissue in 2 separate samples (from either 2 different tissues or from the same tissue but at 2 different sampling dates).
- •Participants must have established cross-reactive immunological material (CRIM) status available prior to enrollment.
- •Participants must have cardiomyopathy at the time of diagnosis: ie, left ventricular mass index (LVMI) equivalent to mean age specific LVMI
- •+1 standard deviation for participants diagnosed by newborn screening or sibling screening;
- •+2 standard deviation for participants diagnosed by clinical evaluation.
- •Parents or legally authorized representative(s) must be capable of giving signed informed consent.
排除标准
- •Participants with symptoms of respiratory insufficiency, including any ventilation use (invasive or noninvasive) at the time of enrollment.
- •Participants with major congenital abnormality.
- •Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).
- •Participant received any Pompe disease specific treatment, eg enzyme-replacement gene therapy (ERT).
- •Participant who has previously been treated in any clinical trial of avalglucosidase alfa.
- •Participant 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
Administered intravenously every 2 weeks
干预措施: avalglucosidase alfa (Drug)
结局指标
主要结局
Proportion of participants who are alive and free of invasive ventilation at Week 52
时间窗: Week 52
次要结局
- Change from baseline to Week 52 in left ventricular mass (LVM)-Z score(Week 52)
- Proportion of participants who are alive and free of invasive ventilation at 12 and 18 months of age(at 12 and 18 months of age)
- Proportion of participants who are alive at Week 52(Week 52)
- Proportion of participants who are alive at 12 and 18 months of age(at 12 and 18 months of age)
- Proportion of participants who are free of ventilator use (invasive and non-invasive separate and combined) at Week 52(Week 52)
- Proportion of participants who are free of supplemental oxygen use at Week 52(Week 52)
- Change from baseline to Week 52 in Alberta Infant Motor Scale (AIMS) score(Week 52)
- Change from baseline to Week 52 in body length Z-scores(Week 52)
- Change from baseline to Week 52 in head circumference percentiles(Week 52)
- Change from baseline to Week 52 in urinary Hex4(Week 52)
- Number of participants with potentially clinically significant abnormality (PCSA) in clinical laboratory results(Week 52, Week 208)
- Change from baseline to Week 52 in body weight Z-scores(Week 52)
- Change from baseline to Week 52 in head circumference Z-scores(Week 52)
- Change from baseline to Week 52 in body length percentiles(Week 52)
- Change from baseline to Week 52 in body weight percentiles(Week 52)
- Number of participants experiencing at least 1 treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR)(Week 52, Week 212)
- Number of participants with abnormalities in physical examinations(Week 52, Week 208)
- Number of participants with PCSA in vital signs measurements(Week 52, Week 208)
- Number of participants with PCSA in 12-lead electrocardiogram (ECG)(Week 52, Week 208)
- Incidence of treatment-emergent anti-drug antibodies (ADA)(Week 52, Week 208)
- Plasma concentration of avalglucosidase alfa(at Day 1, Week 12, and Week 52)
研究者
研究点 (16)
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