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Tracking Information | |||||
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First Received Date † | December 23, 2003 | ||||
Last Updated Date | September 30, 2008 | ||||
Start Date † | December 2003 | ||||
Current Primary Outcome Measures † |
Provide ERT with MZ on an expanded access basis to severely affected patients with infantile-onset Pompe disease for whom there were no alternative treatments [ Time Frame: 52 weeks ] [ Designated as safety issue: No ] | ||||
Original Primary Outcome Measures † | Same as current | ||||
Change History | Complete list of historical versions of study NCT00074919 on ClinicalTrials.gov Archive Site | ||||
Current Secondary Outcome Measures † | |||||
Original Secondary Outcome Measures † | |||||
Descriptive Information | |||||
Brief Title † | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-Onset Pompe Disease | ||||
Official Title † | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-Onset Pompe Disease | ||||
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 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 objective of this protocol is to provide enzyme replacement therapy with rhGAA on an expanded access basis, to severely affected patients with infantile-onset Pompe disease for whom there is no alternative treatment and who do not meet the clinical characteristics described in the inclusion criteria for participation in other Genzyme Corporation-sponsored study currently enrolling patients with infantile-onset Pompe disease. |
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Detailed Description | |||||
Study Phase | |||||
Study Type † | Interventional | ||||
Study Design † | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study | ||||
Condition † |
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Intervention † | Biological: Myozyme | ||||
Study Arms / Comparison Groups | |||||
Publications * | |||||
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline. |
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Recruitment Information | |||||
Recruitment Status † | Completed | ||||
Enrollment † | 33 | ||||
Completion Date | February 2007 | ||||
Primary Completion Date | July 2006 (final data collection date for primary outcome measure) | ||||
Eligibility Criteria † | Inclusion Criteria:
Exclusion Criteria:
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Gender | Both | ||||
Ages | |||||
Accepts Healthy Volunteers | No | ||||
Contacts †† | |||||
Location Countries † | United States | ||||
Expanded Access Status | |||||
Administrative Information | |||||
NCT ID † | NCT00074919 | ||||
Responsible Party | Medical Monitor, Genzyme Corporation | ||||
Secondary IDs †† | |||||
Study Sponsor † | Genzyme | ||||
Collaborators †† | |||||
Investigators † |
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Information Provided By | Genzyme | ||||
Verification Date | April 2007 | ||||
† Required WHO trial registration data element. †† WHO trial registration data element that is required only if it exists. |