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Alglucosidase Alfa Pompe Safety Sub-Registry

CompletedN/A

Sponsor: Genzyme, a Sanofi Company

Conditions: Pompe Disease

Interventions: alglucosidase alfa

Countries: United States, Belgium, Czechia, Germany, Italy, Taiwan

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.

Eligibility overview

Sex: ALL

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility 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 (18)

Phoenix, Arizona, United States
Durham, North Carolina, United States
Hershey, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
Salt Lake City, Utah, United States
Fairfax, Virginia, United States
Ghent, Belgium
Leuven, Belgium
Leuven, Belgium
Prague, Czechia
Halle, Germany
Brescia, Italy
Cagliari, Italy
Messina, Italy
Milan, Italy
Padova, Italy
Roma, Italy
Taipei, Taiwan