Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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19 February 2015 |
Main ID: |
NCT00763932 |
Date of registration:
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30/09/2008 |
Prospective Registration:
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No |
Primary sponsor: |
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Public title:
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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
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Scientific title:
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A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid a-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies |
Date of first enrolment:
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April 2003 |
Target sample size:
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7 |
Recruitment status: |
Completed |
URL:
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http://clinicaltrials.gov/show/NCT00763932 |
Study type:
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Interventional |
Study design:
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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Phase 2
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Countries of recruitment
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France
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South Africa
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United States
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Contacts
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Name:
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Medical Monitor |
Address:
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Telephone:
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Email:
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Affiliation:
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Genzyme, a Sanofi Company |
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Key inclusion & exclusion criteria
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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
Age minimum:
18 Years
Age maximum:
N/A
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Glycogen Storage Disease Type II
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Pompe Disease Infantile-Onset
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Intervention(s)
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Biological: Myozyme
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Primary Outcome(s)
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The objective of this extension study was to monitor the long-term safety and efficacy
[Time Frame: 3 years]
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Secondary ID(s)
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AGLU02003
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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