2024-513859-33-00招募中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)
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 8
- 试验地点
- 6
- 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
To determine the effect of avalglucosidase alfa treatment on survival and invasive ventilator-free survival of IOPD participants ≤ 6 months of age after 52 weeks of treatment.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 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 ERT gene therapy.
- •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.
结局指标
主要结局
Proportion of participants who are alive and free of invasive ventilation at Week 52
Proportion of participants who are alive and free of invasive ventilation at Week 52
次要结局
- Proportion of participants who are alive and free of invasive ventilation at 12 and 18 months of age
- Proportion of participants who are alive at Week 52
- Proportion of participants who are alive 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
- Proportion of participants who are free of supplemental oxygen use at Week 52
- Change from baseline to Week 52 in left ventricular mass (LVM)-Z score
- Change from baseline to Week 52 in Alberta Infant Motor Scale (AIMS) score
- Change from baseline to Week 52 in body length Z-scores
- Change from baseline to Week 52 in body weight Z-scores
- Change from baseline to Week 52 in head circumference Z-scores
- Change from baseline to Week 52 in body length percentiles
- Change from baseline to Week 52 in body weight percentiles
- Change from baseline to Week 52 in head circumference percentiles
- Change from baseline to Week 52 in urinary Hex4
- Number of participants experiencing at least 1 treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR)
- Number of participants with abnormalities in physical examinations
- Number of participants with potentially clinically significant abnormality (PCSA) in clinical laboratory results
- Number of participants with PCSA in vital signs measurements
- Number of participants with PCSA in 12-lead electrocardiogram (ECG)
- Incidence of treatment-emergent anti-drug antibodies (ADA)
- Plasma concentration of avalglucosidase alfa
研究者
Clinical Sciences and Operations
Scientific
Sanofi-Aventis Recherche & Developpement
研究点 (6)
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