Status:
COMPLETED
Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Pompe Disease
Eligibility:
All Genders
8+ years
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
- Must provide written informed consent prior to any study-related procedures being performed
- Clinical presentation consistent with late-onset Pompe disease, or a current clinical diagnosis of late-onset Pompe disease
- Must be greater than or equal to 8 years of age
- Must be able to ambulate (use of assistive devices, such as a walker, cane or crutches, is permitted)
Exclusion
- Requires the use of invasive ventilatory support
- Requires the use of noninvasive ventilatory support while awake and in an upright position
- Use of any investigational product within 30 days prior to study enrollment
- Unwillingness to comply with protocol requirements
- Has clinically significant organic disease, unstable medical condition, serious or intercurrent illness
- Is pregnant or lactating
- Has participated in the Prospective Observational Study in Patients with Late-Onset Pompe Disease (AGLU02303, "LOPOS")
Key Trial Info
Start Date :
May 1 2005
Trial Type :
OBSERVATIONAL
End Date :
October 1 2005
Estimated Enrollment :
60 Patients enrolled
Trial Details
Trial ID
NCT00113035
Start Date
May 1 2005
End Date
October 1 2005
Last Update
May 5 2015
Active Locations (5)
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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