Trial record 20 of 50 for:
"Glycogen storage disease type 2"
Extension Study of Long-Term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies
This study has been completed.
Sponsor:
Genzyme
Information provided by:
Genzyme
ClinicalTrials.gov Identifier:
NCT00763932
First received: September 30, 2008
Last updated: July 7, 2009
Last verified: September 2008
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Purpose
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line
| Condition | Intervention | Phase |
|---|---|---|
|
Pompe Disease Infantile-Onset Glycogen Storage Disease Type II |
Biological: Myozyme |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies |
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:
- The objective of this extension study was to monitor the long-term safety and efficacy [ Time Frame: 3 years ] [ Designated as safety issue: No ]
| Enrollment: | 7 |
| Study Start Date: | April 2003 |
| Study Completion Date: | July 2006 |
| Primary Completion Date: | June 2006 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: 1 |
Biological: Myozyme
10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow
Other Name: Alglucosidase alfa
|
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- The patient was enrolled in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502
- The patient's legal guardian(s) provided written informed consent prior to any study related procedures being performed
- The patient and his/her guardian(s) were able to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.
Exclusion Criteria:
- Patients were excluded from this study if they did not meet the specific inclusion criteria, or if the patient experienced any unmanageable AE in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502 (as determined and agreed upon by the Principal Investigator and Genzyme Corporation), due to Synpac rhGAA therapy, that would preclude continuing therapy
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00763932
Locations
| United States, California | |
| Children's Hospital of Oakland | |
| Oakland, California, United States | |
| Children's Hospital of Orange Country | |
| Orange, California, United States | |
| United States, Nebraska | |
| University of Nebraska Medical Center | |
| Omaha, Nebraska, United States | |
| United States, New Jersey | |
| Institute for Genetic Medicine, Saint Peter's University Hospital | |
| New Brunswick, New Jersey, United States | |
| United States, New York | |
| New York University [NYU] School of Medicine | |
| New York, New York, United States | |
| France | |
| Pediatrique Hospital Debrousse | |
| Lyon, France | |
| Hôpital Porte Madeleine | |
| Orleans, France | |
| South Africa | |
| The Morningside Clinic | |
| Johannesburg, South Africa | |
Sponsors and Collaborators
Genzyme
Investigators
| Study Director: | Medical Monitor | Genzyme |
More Information
Additional Information:
No publications provided
| Responsible Party: | Medical Monitor, Genzyme Corporation |
| ClinicalTrials.gov Identifier: | NCT00763932 History of Changes |
| Other Study ID Numbers: | AGLU02003 |
| Study First Received: | September 30, 2008 |
| Last Updated: | July 7, 2009 |
| Health Authority: | United States: Food and Drug Administration |
Additional relevant MeSH terms:
|
Glycogen Storage Disease Type II Glycogen Storage Disease 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 23, 2013