APOLLO-LOPD: Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
Study Details
Study Description
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 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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 4 |
Detailed Description
Study duration per participants is approximatively 56 weeks including a 52-week treatment period.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Alglucosidase Alfa A dose of 20 mg/kg body weight once every 2 weeks for a minimum of 52 weeks |
Drug: ALGLUCOSIDASE ALFA
Pharmaceutical form:solution for infusion
Route of administration: intravenous
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old [Baseline to 12 months]
This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes.
- Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients ≥5-year old [Baseline to 12 months]
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
- Number of participants with adverse events Number of AEs [From signing the ICF to the 30th day after the last dosage of the study medications]
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Secondary Outcome Measures
- Change from baseline in maximal inspiratory pressure (MIP) in upright position for the patients ≥5-year old [Week 52]
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
- Change from baseline in maximal expiratory pressure (MEP) in upright position for the patients ≥5-year old [Week 52]
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
- Change from baseline in manual muscle test (MMT) for deltoid muscle, quadriceps femoris, iliopsoas, neck stretch flexor for the patients ≥5-year old [Week 52]
MMT has been reported most often as a summary score of a total number of proximal, distal, and axial muscle groups tested bilaterally or as a proximal score that sums a number of proximal muscle groups from the upper and lower extremities.
- Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old [Week 52]
The Quick Motor Function Test is a reliable and valid test for assessing motor function in patients with Pompe's disease.
- Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old [Week 52]
The 12-Item Short Form Health Survey (SF-12) was developed for the Medical Outcomes evaluation of patients with chronic conditions.
Eligibility Criteria
Criteria
Inclusion criteria :
-
Patients (or and patient's legal guardian) must provide written informed consent prior to any study-related procedures
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The patient must be ≥ 3 years of age at the time of enrollment.
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For patient ≥ 3-year and < 5-year old: must be able to walk 10 meters or climb 4-step stairs independently.
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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.
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2 confirmed GAA gene mutations.
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muscle pathology meet the diagnosis of Pompe disease.
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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.
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The patient, if female and of childbearing potential, must have a negative pregnancy test (beta-human chorionic gonadotropin) at baseline.
Exclusion criteria:
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Use of invasive ventilatory support (Invasive ventilation is defined as any form of ventilatory support applied with the use of an endotracheal tube.)
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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.)
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Previously treated with Enzyme Replacement Treatment.
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A Female patient of childbearing potential with a positive pregnancy test.
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Wheelchair dependent.
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The patient has a major congenital anomaly.
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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.
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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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Investigational Site | China | China |
Sponsors and Collaborators
- Genzyme, a Sanofi Company
Investigators
- Study Director: Clinical Sciences & Operations, Sanofi
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- ALGMYL09010
- U1111-1238-1267