Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa Treatment

Phase 1

• Conditions: Infantile-Onset Pompe Disease; MedDRA version: 20.1Level: PTClassification code 10053185Term: Glycogen storage disease type IISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2021-004047-25-Outside-EU/EEA
• Lead Sponsor: Sanofi(China) Investment Co Ltd, Shanghai Branch

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-07-27
• Last Update Posted: 2 August 2021

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

-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

Exclusion Criteria

-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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified