Status:
COMPLETED
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Pompe Disease (Late-onset)
Glycogen Storage Disease Type II (GSD-II)
Eligibility:
All Genders
8+ years
Phase:
PHASE3
Brief Summary
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells...
Eligibility Criteria
Inclusion
- Patient must provide signed, informed consent prior to performing any study-related procedures.
- Patient must have a diagnosis of Pompe disease based on deficient endogenous GAA activity in cultured skin fibroblasts of less than or equal to 40% of the normal mean of the testing laboratory and 2 confirmed GAA gene mutations;
- Patient must be greater than or equal to 8 years of age at the time of enrollment;
- Patient must be able to ambulate 40 meters (approximately 130 feet) in 6 minutes on each test performed on two consecutive days (use of assistive devices such as a walker, cane, or crutches, is permitted);
- Patient must have an FVC of greater than or equal to 30% and \< 80% predicted in the upright position;
- Patient must have a postural drop in FVC (liters) of at least 10% from the upright to the supine position;
- Patient must have proximal muscle weakness in the lower limbs based on unilateral QMT of the knee extensors defined as \< 80% of the predicted value based on age, gender and body size
- Patient must be able to tolerate pulmonary function testing (PFT) and muscle testing in the supine position;
- Patient must have testable muscle in bilateral knee flexors and knee extensors, and testable muscle in bilateral elbow flexors and elbow extensors;
- Patient must be able to provide reproducible muscle and pulmonary function test results;
- Patient (and patient's legal guardian if patient is \< 18 years of age) must have the ability to comply with the clinical protocol;
- A female patient of childbearing potential must have a negative pregnancy test (urine) at Baseline. Note: All female patients of childbearing potential and sexually mature males must use a medically accepted method of contraception throughout the study.
Exclusion
- Patient requires the use of invasive ventilatory support;
- Patient requires the use of noninvasive ventilatory support while awake and in an upright position;
- Patient has received enzyme replacement therapy with GAA from any source;
- Patient has used an investigational product within 30 days prior to study enrollment, or is currently enrolled in another study which involves clinical evaluations, unless prior approval is given by Genzyme;
- Patient has a major congenital anomaly, medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities;
Key Trial Info
Start Date :
September 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2007
Estimated Enrollment :
90 Patients enrolled
Trial Details
Trial ID
NCT00158600
Start Date
September 1 2005
End Date
September 1 2007
Last Update
April 28 2015
Active Locations (8)
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1
Tower Hematology Oncology Medical Group
Beverly Hills, California, United States, 90211
2
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
3
Washington University Medical Center
St Louis, Missouri, United States, 63110
4
Mount Sinai School of Medicine
New York, New York, United States, 10029