Alglucosidase Alfa Pompe Safety Sub-Registry

Genzyme

Status: Completed

Conditions: Pompe Disease

Treatments: Biological: alglucosidase alfa

Study type: Observational

Funder types: Industry

Identifiers

NCT01710813

LTS13930 (Other Identifier)

AGLU06909

Details and patient eligibility

About

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.

Enrollment: 110 patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

110 participants in 1 patient group

pompe safety sub-registry

Description:

patients are selected from those who are enrolled in the Pompe Registry, and will be followed for safety evaluation in this sub-registry

Treatment:

Biological: alglucosidase alfa