Pompe Lactation Sub-Registry

Genzyme, a Sanofi Company2 enrolled

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Glycogen Storage Disease Pompe Disease

Interventions

alglucosidase alfaBIOLOGICAL

At least one infusion of alglucosidase alfa post partum

Eligibility

Sex: FEMALE

Medical Language ↔ Plain English

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

Locations (2)

Registry participation is worldwide. Facilities not yet active may enroll upon identification of a patient

Cambridge, Massachusetts, United States

Unnamed facility

Grand Rapids, Michigan, United States

Outcomes

Primary Outcomes

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.

Time frame: 6 Months

breast milk production and composition in women with Pompe disease who receive alglucosidase alfa

Time frame: 6 Months

Data from ClinicalTrials.gov

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