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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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26 July 2021 |
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Main ID: |
NCT01710813 |
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Date of registration:
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17/10/2012 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Alglucosidase Alfa Pompe Safety Sub-Registry
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Scientific title:
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A Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune Complex Mediated Reactions With Alglucosidase Alfa Treatment |
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Date of first enrolment:
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March 20, 2015 |
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Target sample size:
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110 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01710813 |
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Study type:
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Observational [Patient Registry] |
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Study design:
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Phase:
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Countries of recruitment
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Belgium
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Czech Republic
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Czechia
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Germany
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Italy
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Taiwan
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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 must be enrolled in the Pompe Registry;
- Provide a signed patient information and authorization form;
- Have a confirmed diagnosis of Pompe disease (confirmation of diagnosis is defined as
documented GAA enzyme deficiency from any tissue source and/or documentation of 2 GAA
gene mutations);
- Be naïve to and plan to be treated with alglucosidase alfa at or prior to enrollment,
or are being treated with alglucosidase alfa.
Exclusion Criteria:
- Patients will be excluded if they have received an investigational drug (excluding
alglucosidase alfa) within 30 days prior to signing a Safety Sub-Registry Patient
Information and Authorization form, or if they are taking or plan to take any
investigational product while enrolled in the Safety Sub-Registry.
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Pompe Disease
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Intervention(s)
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Biological: alglucosidase alfa
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Primary Outcome(s)
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number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions
[Time Frame: 4 Years]
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Secondary ID(s)
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LTS13930
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AGLU06909
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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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