Pompe Lactation Sub-Registry

NCT00566878 | Completed

• 2 other identifiers: AGLU03406LTS13972
• Study Type: observational
• Target: 2
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Glycogen Storage Disease; Pompe Disease

Keywords

Glycogen Storage Disease; GSD-II; Pompe Disease; Pompe Disease (Late-Onset); Acid Maltase Deficiency Disease; Glycogenosis II

Outcome Measures

Primary Outcomes (2)

• 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

Interventions

alglucosidase alfa BIOLOGICAL

At least one infusion of alglucosidase alfa post partum

Also known as: Myozyme, Lumizyme

Eligibility Criteria

• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Females diagnosed with Pompe Disease and Lactating

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (2)

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

Cambridge, Massachusetts, 02142, United States

Location

Unknown Facility

Grand Rapids, Michigan, 49503, United States

Location

MeSH Terms

Conditions

Glycogen Storage Disease; Glycogen Storage Disease Type II

Interventions

GAA protein, human

Condition Hierarchy (Ancestors)

Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Lysosomal Storage Diseases

Study Officials

• Medical Monitor

  Genzyme, a Sanofi Company

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 1, 2007
• First Posted: December 4, 2007
• Study Start: March 31, 2012
• Primary Completion: February 9, 2024
• Study Completion: February 9, 2024
• Last Updated: March 19, 2024

Record last verified: 2024-03

Locations

US (2)