Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602
This study has been completed.
Sponsor:
Genzyme
Information provided by:
Genzyme
ClinicalTrials.gov Identifier:
NCT00125879
First received: August 1, 2005
Last updated: July 7, 2009
Last verified: April 2007
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Purpose
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 to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective of this study is to evaluate the long-term safety and efficacy of Myozyme treatment in patients with infantile-onset Pompe disease.
| Condition | Intervention | Phase |
|---|---|---|
|
Glycogen Storage Disease Type II |
Biological: Myozyme |
Phase 2 Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Long-Term Continuation Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 |
Resource links provided by NLM:
Genetics Home Reference related topics:
glycogen storage disease type IX
Pompe disease
Schindler disease
succinic semialdehyde dehydrogenase deficiency
Drug Information available for:
Alglucosidase Alfa
U.S. FDA Resources
Further study details as provided by Genzyme:
Primary Outcome Measures:
- Long-term Safety and Efficacy [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
| Enrollment: | 16 |
| Study Start Date: | June 2005 |
| Study Completion Date: | December 2006 |
| Primary Completion Date: | June 2006 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: 1 |
Biological: Myozyme
20 mg/kg qow or 40 mg/kg qow
Other Name: alglucosidase alfa
|
Eligibility| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- The patient's legal guardian(s) must provide written informed consent prior to any study-related procedures being performed
- The patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol
- The patient must have completed Protocol AGLU01602.
Exclusion Criteria:
- Patient has experienced any unmanageable adverse event (AE) in Protocol AGLU01602 due to Myozyme that would preclude continuing treatment with Myozyme
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00125879
Locations
| United States, Alabama | |
| University of Alabama | |
| Birmingham, Alabama, United States, 35233 | |
| United States, Florida | |
| Shands Hospital at the University of Florida | |
| Gainesville, Florida, United States, 32610 | |
| Miami Children's Hospital | |
| Miami, Florida, United States, 33155 | |
| United States, Georgia | |
| Emory University Medical Genetics | |
| Decatur, Georgia, United States, 30033 | |
| 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 | |
| CHU Amiens | |
| Amiens, France, 80054 | |
| CHU Cote de Nacre | |
| Caen, France, 14033 | |
| Germany | |
| Universitats-Kinderklinik Mainz | |
| Mainz, Germany, 55131 | |
| Israel | |
| Rambam Medical Center | |
| Haifa, Israel, 35254 | |
| Italy | |
| San Gerardo Hospital | |
| Monza, Italy, 20052 | |
| Netherlands | |
| Erasmus MC University | |
| Rotterdam, Netherlands, 3015 GJ | |
| Taiwan | |
| Tzu-Chi General Hospital | |
| Hua-lien, Taiwan, 970 | |
| Chi-Mei Medical Center Dept of Pediatrics | |
| Tainan, Taiwan, 710 | |
Sponsors and Collaborators
Genzyme
Investigators
| Study Director: | Medical Monitor | Genzyme |
More Information
No publications provided by Genzyme
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Medical Monitor, Genzyme Corporation |
| ClinicalTrials.gov Identifier: | NCT00125879 History of Changes |
| Other Study ID Numbers: | AGLU02403 |
| Study First Received: | August 1, 2005 |
| Last Updated: | July 7, 2009 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Genzyme:
|
Glycogen Storage Disease Type II GSD-II Pompe Disease Pompe Disease (Late-onset) |
Acid Maltase Deficiency Disease Glycogenosis 2 Glycogen Storage Disease Type II (GSD-II) |
Additional relevant MeSH terms:
|
Glycogen Storage Disease Glycogen Storage Disease Type II Metabolic Diseases Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Lysosomal Storage Diseases, Nervous System |
Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Lysosomal Storage Diseases |
ClinicalTrials.gov processed this record on May 16, 2013