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| Sponsored by: |
Genzyme |
| Information provided by: | Genzyme |
| ClinicalTrials.gov Identifier: | NCT00053573 |
Purpose
Glycogen Storage Disease Type II ("GSD-II"; also known as 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 patients with GSD-II, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for GSD-II. Patients diagnosed with infantile-onset GSD-II who are greater than 6 months old, but less than or equal to 36 months old will be studied.
| Condition | Intervention | Phase |
|
Glycogen Storage Disease Type II Pompe Disease Acid Maltase Deficiency Disease Glycogenosis 2 |
Drug: recombinant human acid alpha-glucosidase (rhGAA) |
Phase I Phase II |
| Genetics Home Reference related topics: | Pompe disease |
| ChemIDplus related topics: | Alglucosidase Alfa Glucan 1,4-alpha-Glucosidase |
| Study Type: | Interventional |
| Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
| Official Title: | An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-II |
Eligibility
| Ages Eligible for Study: | 6 Months to 36 Months |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| United States, Florida | |||||
| University of Florida College of Medicine | |||||
| Gainesville, Florida, United States, 32610 | |||||
| United States, North Carolina | |||||
| Duke University Medical Center | |||||
| Durham, North Carolina, United States, 27710 | |||||
| United States, Ohio | |||||
| Children's Hospital Medical Center | |||||
| Cincinnati, Ohio, United States, 45229 | |||||
| France | |||||
| Pediatrique Hopital de Brousse | |||||
| Lyon, France | |||||
| Israel | |||||
| Rambam Medical Center | |||||
| Haifa, Israel, 31096 | |||||
| United Kingdom | |||||
| Royal Manchester Children's Hospital | |||||
| Manchester, United Kingdom, M27 4 HA | |||||
| Genzyme |
More Information
US FDA Approved Full Prescribing Information for Myozyme® 
  |
| Study ID Numbers: | AGLU01702 |
| First Received: | January 31, 2003 |
| Last Updated: | July 16, 2007 |
| ClinicalTrials.gov Identifier: | NCT00053573 |
| Health Authority: | United States: Food and Drug Administration |
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