Status:
COMPLETED
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Pompe's Disease
Eligibility:
All Genders
3+ years
Phase:
PHASE4
Brief Summary
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 Chin...
Detailed Description
Study duration per participants is approximatively 56 weeks including a 52-week treatment period.
Eligibility Criteria
Inclusion
- 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.
- For patient ≥ 3-year and \< 5-year old: must be able to walk 10 meters or climb 4-step stairs independently.
- 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,
- GAA enzyme deficiency from any tissue source.
- 2 confirmed GAA gene mutations.
- 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.
Exclusion
Key Trial Info
Start Date :
March 10 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 25 2024
Estimated Enrollment :
41 Patients enrolled
Trial Details
Trial ID
NCT04676373
Start Date
March 10 2021
End Date
July 25 2024
Last Update
February 10 2025
Active Locations (1)
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1
Investigational Site
China, China