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Home > "S" Clinical Trials Conditions > Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease  Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
For Condition: Pompe Disease,Glycogenosis 2,Acid Maltase Deficiency Disease,Glycogen Storage Disease Type II
Status: Completed
Sponsor(s): Genzyme ,
Synopsis: Pompe disease 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 to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In infants with severe cases of Pompe disease (called Classical Infantile Pompe disease), an excessive amount of glycogen accumulates and is stored in various tissues, especially heart, skeletal muscle, and liver, which prevents their normal function. This study being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with Classical Infantile Pompe disease who have a small, but inactive, amount of natural GAA enzyme present in their bodies (called Cross-Reacting Immunologic Material-Positive or "CRIM (+)" patients), will be studied.
Details:
Eligibility:
Study Type: Interventional, Treatment, Non-Randomized, Open Label, Historical Control, Single Group Assignment, Safety/Efficacy Study
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: Inclusion Criteria: - Clinical diagnosis of Classical Infantile Pompe Disease - endogenous GAA activity < 1.0% - cardiomegaly - cardiomyopathy - CRIM (+) - ability to comply with the clinical protocol which will require extensive clinical evaluations Exclusion Criteria: - respiratory insufficiency - cardiac failure - major congenital abnormality - any other medical condition that could potentially decrease survival - CRIM (-)
Total Enrollment: 8
Location and Contact Information:
Duke University Medical Center
Durham, North Carolina, 27710
United States
Additional Information:
Study ID Numbers: AGLU-001-00;
Study Start Date: May 2001
Record last reviewed: March 2004
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00025896
Other Acid Maltase Deficiency Disease Studies:
1. A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II
2. A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease)
3. Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
4. A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease
Related Studies:
Other Acid Maltase Deficiency Disease Clinical Trials
Other North Carolina Clinical Trials
Other Durham Clinical Trials
Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
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