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: |
NCT00025896 |
Date of registration:
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31/10/2001 |
Prospective Registration:
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No |
Primary sponsor: |
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Public title:
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Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
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Scientific title:
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A Prospective Multinational, Multicenter, Clinical Trial of the Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) in Cross-Reacting Immunologic Material-Positive Patients With Classical Infantile Pompe Disease |
Date of first enrolment:
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May 2001 |
Target sample size:
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8 |
Recruitment status: |
Completed |
URL:
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http://clinicaltrials.gov/show/NCT00025896 |
Study type:
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Interventional |
Study design:
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Allocation: Non-Randomized, 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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United States
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Clinical diagnosis of Classical Infantile Pompe Disease
- endogenous GAA activity < 1.0%
- cardiomegaly
- cardiomyopathy
- CRIM (+)
- ability to comply with the clinical protocol which will require extensive clinical
evaluations
Exclusion Criteria:
- respiratory insufficiency
- cardiac failure
- major congenital abnormality
- any other medical condition that could potentially decrease survival
- CRIM (-)
Age minimum:
N/A
Age maximum:
N/A
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Acid Maltase Deficiency Disease
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Pompe Disease
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Glycogen Storage Disease Type II
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Glycogenosis 2
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Intervention(s)
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Drug: recombinant human acid alpha-glucosidase (rhGAA)
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Secondary ID(s)
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AGLU-001-00
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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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