Pompe's disease
Conditions
Interventions
Trade Name: MYOZYME
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Alglucosidase alfa
CAS Number: 420784-05-0
Concentration unit: mg milligram(s)
Concentration type: equal
Concentrati
Sponsors
Genzyme
Eligibility
Sex/Gender
All
Inclusion criteria
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. a. For patient = 3-year and =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
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).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main 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. ;Primary end point(s): 1 Change from baseline in Six-minute walk test (6MWT) for the patients =5-year old 2 - Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients =5-year old 3 - Number of participants with adverse events Number of AEs ;Timepoint(s) of evaluation of this end point: 1 and 2: Baseline to 12 months 3: From signing the ICF to the 30th day after the last dosage of the study medications | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Change from baseline in maximal inspiratory pressure (MIP) in upright position for the patients =5-year old Change from baseline in maximal expiratory pressure (MEP) in upright position for the patients =5-year old Change from baseline in manual muscle test (MMT) for deltoid muscle, quadriceps femoris, iliopsoas, neck stretch flexor for the patients =5-year old Change from baseline in Quick Motor Function Test scores for the patients =5-year old Change from baseline in 12- Item Short-form health survey scores for the patients =5-year old ;Timepoint(s) of evaluation of this end point: Week 52 | — |
Countries
China
Contacts
Public Contact-
Genzyme
Outcome results
None listed