Pompe Lactation Sub-Registry
Study on Breast Milk and Treatment for a Rare Genetic Disorder
Study Overview
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.
Eligibility Criteria
You may be eligible for this study if you meet the following criteria:
- Conditions: Glycogen Storage Disease, Pompe Disease
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Gender: Female
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
This study investigates the presence of an investigational medication in the breast milk of mothers with Pompe Disease. The purpose of this study is to understand if the investigational medication, which is used to treat Pompe Disease, is found in the breast milk of mothers who are being treated with it.
Participants in this study will have their breast milk production and composition measured. The study involves collecting samples of breast milk to analyze the presence of the investigational medication. This will help researchers understand how the treatment may affect lactating women with Pompe Disease.
- Who can participate: Women enrolled in the Pompe Registry who are pregnant or lactating and receiving the investigational medication while lactating are eligible. Participants must consent and adhere to study guidelines.
- Study details: Participants will provide breast milk samples for analysis and follow guidelines for antibody testing according to a recommended schedule.
