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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. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT00520143 |
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Date of registration:
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21/08/2007 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Alglucosidase Alfa Temporary Access Program
ATAP |
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Scientific title:
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Alglucosidase Alfa Temporary Access Program |
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Date of first enrolment:
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July 2007 |
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Target sample size:
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Recruitment status: |
Approved for marketing |
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URL:
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http://clinicaltrials.gov/show/NCT00520143 |
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Study type:
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Expanded Access |
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Study design:
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N/A
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Phase:
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N/A
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Countries of recruitment
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United States
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Contacts
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Name:
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Medical Monitor |
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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 or patient's legal guardian must provide signed, informed consent prior
to performing any study-related procedures.
- The patient must reside in the US.
- The patient must have a confirmed diagnosis of Pompe disease defined as documented
acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or GAA gene
mutations.
- The patient must have/had documented clinical signs and symptoms of Pompe disease.
- The patient must have/had prior treatment with alglucosidase alfa produced at
commercial scale OR be naive to enzyme replacement therapy (ERT) for the treatment of
Pompe disease and meet at least 1 of the following criteria: require a wheelchair OR
require some respiratory assistance for any number of hours (including night time)
through non-invasive ventilation.
- The patient must be capable of complying with the required program schedule of
assessments.
Exclusion Criteria:
- Females who are pregnant or lactating
- The patient has a clinical condition unrelated to Pompe disease that would interfere
with program assessments.
- The patient is currently enrolled in any clinical studies.
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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Glycogenosis 2
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Pompe Disease (Late-Onset)
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Acid Maltase Deficiency Disease
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Glycogen Storage Disease Type II (GSD-II)
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Intervention(s)
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Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
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Secondary ID(s)
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AGLU03907
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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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