Pompe Lactation Sub-Registry

Research on Breast Milk Composition in Mothers with Pompe Disease

Not Recruiting
Female
Phase N/A
2 participants needed

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
  • 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

Updated on 19 Mar 2024. Study ID: NCT00566878

This study investigates the presence of an investigational medication in the breast milk of mothers with Pompe Disease. Pompe Disease is a rare genetic disorder that affects the body's ability to break down a complex sugar called glycogen due to a deficiency of an enzyme. The purpose of this study is to determine if the investigational medication, which is used to treat Pompe Disease, can be found in breast milk and to measure the production and composition of breast milk in these women.

Participants in this study will have their breast milk collected and analyzed. The study will involve regular assessments and adherence to specific guidelines, including antibody testing. Participants will be required to provide their breast milk samples while receiving the investigational medication.

  • Who can participate: Women with Pompe Disease who are pregnant or currently lactating and are part of the Pompe Registry can participate. They must also agree to follow the study guidelines, provide a signed Patient Information and Authorization form, and have received at least one infusion of the investigational medication while lactating.
  • Study details: Participants must be part of the Pompe Registry and will provide breast milk samples for analysis. They will follow specific guidelines for antibody testing and other assessments. A placebo is not used in this study. Participants need to provide a signed Patient Information and Authorization form.