Pompe Lactation Sub-Registry

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.

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Key information

Conditions

Glycogen Storage Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glycogen Storage Disease Type II Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Pompe Disease

Sex eligibility

Female

Study type

Observational

Primary location

Registry participation is worldwide. Facilities not yet active may enroll upon identification of a patient, Cambridge, Massachusetts, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

alglucosidase alfa

Biological

At least one infusion of alglucosidase alfa post partum

Other names: Myozyme, Lumizyme

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

Sponsors and collaborators

Lead sponsor

Genzyme, a Sanofi Company

Industry

Registry information

Official study title

A Sub-Registry to Determine the Presence of Alglucosidase Alfa in Breast Milk From Women With Pompe Disease Treated With Alglucosidase Alfa.

Important dates

Study start: 2012
Primary completion: 2024
Study completion: 2024
First posted: Dec 4, 2007
Registry last updated: Mar 19, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed