An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa in Treatment naïve Pediatric Participants withInfantile-Onset Pompe Disease (IOPD) - Baby-COMET

Sanofi-Aventis Recherche & Développement0 个研究点目标入组 18 人开始时间: 2021年1月21日最近更新: 2024年7月22日

适应症

试验速览

暂无简介。

•- Participants must have confirmed diagnosis of infantile-onset Pompe disease defined as: the presence of 2 lysosomal acid a-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, 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
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 20
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Participants are excluded from the study if any of the following criteria apply:
•- 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.

发起方