Status:
COMPLETED
A Study of rhGAA 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
5-18 years
Phase:
PHASE2
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's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related procedures; patient's signature required if patient understands informed consent
- patient must have a diagnosis of Pompe disease based on deficient endogenous GAA activity or GAA gene mutations
- patient must have demonstrable muscle weakness
- patient must be greater than or equal to five years of age and younger than eighteen years of age
- patient must be able to provide 3 reproducible FVC tests in sitting position during screening
- patient must perform muscle function testing
- patient must ambulate 10 meters (assistive devices permitted)
- patient and legal guardian must comply with the clinical protocol
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 clinical or observational study
- patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities
- Female patients pregnant, lactating or unwilling to practice birth control methods during study
- Male patients unwilling to use barrier contraceptives during study
Key Trial Info
Start Date :
February 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 1 2006
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT00250939
Start Date
February 1 2005
End Date
November 1 2006
Last Update
February 6 2014
Active Locations (1)
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1
Sophia Kinderziekenhuis, Erasmus MC
Rotterdam, Netherlands, 3015