A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment

ALGMYL09010

Primary Objective: To evaluate the effect of one-year Alglucosidase alfa treatment on motor function \[Six-minute walk test (6MWT) and lung function predicted Forced vital capacity (FVC)\] among Chinese Late Onset Pompe Disease patients above 5 years old. To evaluate the safety of Myozyme 20mg/kg, IV biweekly in Chinese LOPD patients above 3 years old. Secondary Objective: To evaluate the effect of one-year treatment with Alglucosidase alfa on improvement of manual muscle test (MMT), Maximal inspiratory and expiratory pressure (MIP and MEP)\], Quick Motor Function Test scores, and health-related quality of life (SF-12) among LOPD patients over 5 years old.

2021-03-10

2024-07-25

2024-07-25

Active (not recruiting)

Early phase I

41

Inclusion criteria : * Patients (or and patient's legal guardian) must provide written informed consent prior to any study-related procedures * The patient must be ≥ 3 years of age at the time of enrollment. 1. For patient ≥ 3-year and \< 5-year old: must be able to walk 10 meters or climb 4-step stairs independently. 2. For patients ≥5-year old i. Must be able to ambulate 40 meters in 6 minutes without assistance ii. Must be able to successfully perform repeated forced vital capacity (VC) measurements in upright position of ≥ 30% predicted and ≤85% predicted. * The patient has confirmed Pompe's Disease with at least 2 of the following condition, 1. GAA enzyme deficiency from any tissue source. 2. 2 confirmed GAA gene mutations. 3. muscle pathology meet the diagnosis of Pompe disease. * The patient (and patient's legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol. * The patient, if female and of childbearing potential, must have a negative pregnancy test (beta-human chorionic gonadotropin) at baseline. Exclusion criteria: * Use of invasive ventilatory support (Invasive ventilation is defined as any form of ventilatory support applied with the use of an endotracheal tube.) * Use of non-invasive ventilatory support while awake and in an upright position. (Non-invasive ventilation is defined as any form of ventilatory support applied without the use of an endotracheal tube.) * Previously treated with Enzyme Replacement Treatment. * A Female patient of childbearing potential with a positive pregnancy test. * Wheelchair dependent. * The patient has a major congenital anomaly. * The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities. * The patients with ≥5-year old are unable to ambulate 40 meters without assistance or unable to successfully perform repeated FVC of \>30% and \<85% predicted (upright). The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

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2024-03-08