Pompe Disease
Conditions
Keywords
pompe disease, alglucosidase alfa, anaphylaxis, severe cutaneous, systemic immune complex-mediated reactions
Brief 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.
Interventions
BIOLOGICALalglucosidase alfa
Alglucosidase alfa IV infusion of 20 mg/kg; qow
Sponsors
Genzyme, a Sanofi Company
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions | 4 Years | collect meaningful data on patients with these outcomes following treatment with alglucosidase alfa |
Countries
Belgium, Czechia, Germany, Italy, Taiwan, United States
Outcome results
None listed