Pompe Lactation Sub-Registry

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

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 Overview

• Status: Completed
• Conditions: Glycogen Storage Disease; Pompe Disease
• Intervention / Treatment: Biological: alglucosidase alfa

• Study Type: Observational
• Enrollment (Actual): 2

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Cambridge, Massachusetts, United States, 02142
      • Registry participation is worldwide. Facilities not yet active may enroll upon identification of a patient
  • Michigan
    • Grand Rapids, Michigan, United States, 49503

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Females diagnosed with Pompe Disease and Lactating

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome 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

Collaborators and Investigators

• Sponsor: Genzyme, a Sanofi Company
• Investigators: Study Director: Medical Monitor, Genzyme, a Sanofi Company

Study record dates

Study Major Dates

• Study Start (Actual): March 31, 2012
• Primary Completion (Actual): February 9, 2024
• Study Completion (Actual): February 9, 2024

Study Registration Dates

• First Submitted: December 1, 2007
• First Submitted That Met QC Criteria: December 1, 2007
• First Posted (Estimated): December 4, 2007

Study Record Updates

• Last Update Posted (Actual): March 19, 2024
• Last Update Submitted That Met QC Criteria: March 15, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: GSD-II; Pompe Disease; Acid Maltase Deficiency Disease; Glycogen Storage Disease; Glycogenosis II; Pompe Disease (Late-Onset)
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Lysosomal Storage Diseases, Nervous System; Glycogen Storage Disease Type II; Glycogen Storage Disease
• Other Study ID Numbers: AGLU03406; LTS13972 (Other Identifier: Sanofi)