Alglucosidase Alfa Pompe Safety Sub-Registry
Information source: Sanofi
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Pompe Disease
Intervention: alglucosidase alfa (Biological)
Phase: N/A
Status: Recruiting
Sponsored by: Genzyme, a Sanofi Company Official(s) and/or principal investigator(s): Medical Monitor, Study Director, Affiliation: Genzyme, a Sanofi Company
Overall contact: For site information, send an email with site number to, Email: Contact-Us@sanofi.com
Summary
To collect uniform and meaningful data on patients with Pompe disease who experience
anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic
immune complex-mediated reactions following treatment with alglucosidase alfa.
Clinical Details
Official title: 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
Study design: Observational Model: Cohort, Time Perspective: Prospective
Primary outcome: number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions
Eligibility
Minimum age: N/A.
Maximum age: N/A.
Gender(s): Both.
Criteria:
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.
Locations and Contacts
For site information, send an email with site number to, Email: Contact-Us@sanofi.com
Investigational Site Number 056001, Gent 9000, Belgium; Recruiting
Investigational Site Number 151747, Fairfax, Virginia 22030, United States; Recruiting
Additional Information
Starting date: March 2015
Last updated: June 3, 2015
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