Status:
COMPLETED
Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Pompe Disease (Late-onset)
Glycogen Storage Disease Type II (GSD-II)
Eligibility:
All Genders
18+ 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
- male or female aged greater than or equal to 18 years
- patient's legally authorized guardian(s) must provide signed, informed consent prior to initiation of study; patient's signature required if patient understands informed consent
- patient must have a documented deficit in acid alpha-glucosidase (GAA) activity , corresponding to the diagnosis of Pompe disease confirmed by documented genotyping
- patient presents with advanced documented symptoms of the disease defined as follows: patient is in a wheel chair and presents diaphragmatic dysfunction and requires invasive ventilation or non invasive ventilation (12 or more hours daily)
Exclusion
- patient has received enzyme replacement therapy with GAA from any source
- patient has taken an experimental drug in the 30 days prior to study enrollment, or is currently included in another study involving clinical evaluations; If this is the case, inclusion of the patient in the present study will be subject to prior agreement by Genzyme
- major congenital anomaly
- clinically important organic disease (except for symptoms related to Pompe disease) or any other medical condition, serious intercurrent illness, or other extenuating circumstance that, in the physician's opinion should preclude the patient's participation in the study or may reduce survival
- pregnancy and breastfeeding (women of childbearing age must use a medically accepted method of contraception throughout the entire duration of the trial. Male patients must use a medically accepted birth control method throughout the entire duration of the study)
Key Trial Info
Start Date :
December 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 1 2007
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT00268944
Start Date
December 1 2005
End Date
June 1 2007
Last Update
February 5 2014
Active Locations (1)
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1
Hopital Raymond Poincare
Garches, France, 92380