Glycogen Storage Disease, Pompe Disease
Conditions
Keywords
Glycogen Storage Disease, GSD-II, Pompe Disease, Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis II
Brief summary
The objective is to determine if alglucosidase alfa is present in breast milk from mothers with Pompe Disease being treated with alglucosidase alfa and to measure breast milk production and composition in women with Pompe Disease who receive alglucosidase alfa.
Interventions
BIOLOGICALalglucosidase alfa
At least one infusion of alglucosidase alfa post partum
Sponsors
Genzyme, a Sanofi Company
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
FEMALE
Healthy volunteers
No
Inclusion criteria
* Must be enrolled in Pompe Registry (NCT00231400) * Must be pregnant and intend to breast-feed or be currently lactating and receive at least one infusion of alglucosidase alfa while lactating * Provide a signed Patient Information and Authorization form to participate in the sub-registry prior to any sub-registry-related assessments are performed * Agree to adhere to the sub-registry guidelines for antibody testing and recommended schedule of assessments.
Exclusion criteria
* Patients will be excluded from this sub-registry if they have received an investigational drug (excluding alglucosidase alfa in regions where alglucosidase alfa is not commercially available) within 30 days prior to Visit 1 breast milk collection
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| alglucosidase alfa accumulation in immature (1-3 months post partum) or mature (4-6 months post partum) breast milk sample from mothers with Pompe disease who are being treated with alglucosidase alfa. | 6 Months |
| breast milk production and composition in women with Pompe disease who receive alglucosidase alfa | 6 Months |
Countries
United States
Outcome results
None listed