A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa Treatment - APOLLO-IOPD

Sanofi(China) Investment Co Ltd, Shanghai Branch0 个研究点开始时间: 2021年7月27日最近更新: 2021年8月2日

试验速览

暂无简介。

•-Subject’s parents or legal guardians must provide written informed consent prior to any study-related procedures.
•-Documented onset of Pompe disease symptoms up to 12 months of age (corrected for gestation if born before 40 weeks); diagnosis of Pompe disease confirmed by acid alpha-glucosidase enzyme deficiency from any tissue source and acid alpha-glucosidase gene mutations.
•-Age 0-12 months at enrollment, defined as at the time of providing written informed consent.
•-Cardiomyopathy (abnormal left ventricular mass indices [LVMIs], measured by echocardiography, abnormal value is defined as =65 g/m2 for patients up to 12 months old) confirmed by cardiologist at study site.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•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

•-Patient who has previously been treated with acid alpha-glucosidase.
•-Patient who is participating in another clinical study using any investigational therapy.
•-Conditions/situations such as:
•-Clinical signs of cardiac failure with ejection fraction < 40%.
•-Respiratory insufficiency (oxygen saturation < 90% or carbon dioxide partial pressure > 55 mm Hg [venous] or > 40 mm hydrargyrum [arterial] in room air or any ventilator use).
•-Patients who are dependent on invasive or non-invasive ventilator support.
•-Patients with major congenital anomaly or clinically significant intercurrent organic disease unrelated to Pompe disease.
•-Patients not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.

发起方

Sanofi(China) Investment Co Ltd, Shanghai Branch