Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen (APOLLO-LOPD)

  • STATUS
    Recruiting
  • End date
    Dec 10, 2024
  • participants needed
    40
  • sponsor
    Genzyme, a Sanofi Company
Updated on 10 May 2022
beta-human chorionic gonadotropin
deficiency
alglucosidase alfa
myozyme
gaa gene

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 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.

Description

Study duration per participants is approximatively 56 weeks including a 52-week treatment period.

Details
Condition Pompe's Disease
Treatment alglucosidase alfa
Clinical Study IdentifierNCT04676373
SponsorGenzyme, a Sanofi Company
Last Modified on10 May 2022

Eligibility

Yes No Not Sure

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
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
The patient, if female and of childbearing potential, must have a negative pregnancy test (beta-human chorionic gonadotropin) at baseline
local regulation) must have the ability to comply with the clinical protocol

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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