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Pompe Lactation Sub-Registry

This study is currently recruiting participants.
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Verified October 2016 by Sanofi ( Genzyme, a Sanofi Company )
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company ) Identifier:
First received: December 1, 2007
Last updated: October 20, 2016
Last verified: October 2016
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.

Condition Intervention Phase
Glycogen Storage Disease Pompe Disease Biological: alglucosidase alfa Phase 4

Study Type: Observational
Study Design: Time Perspective: Prospective
Official Title: A Sub-Registry to Determine the Presence of Alglucosidase Alfa in Breast Milk From Women With Pompe Disease Treated With Alglucosidase Alfa.

Resource links provided by NLM:

Further study details as provided by Sanofi ( Genzyme, a Sanofi Company ):

Primary Outcome Measures:
  • 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 ]

Estimated Enrollment: 5
Study Start Date: March 2012
Estimated Study Completion Date: December 2021
Estimated Primary Completion Date: November 2021 (Final data collection date for primary outcome measure)
Intervention Details:
    Biological: alglucosidase alfa
    At least one infusion of alglucosidase alfa post partum
    Other Names:
    • Myozyme
    • Lumizyme

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Females diagnosed with Pompe Disease and Lactating

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00566878

Contact: Medical Information 800-745-4447
Contact: Medical Information 617-252-7832

United States, Massachusetts
Registry participation is worldwide. Facilities not yet active may enroll upon identification of a patient Recruiting
Cambridge, Massachusetts, United States, 02142
United States, Michigan
Grand Rapids, Michigan, United States, 49503
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Responsible Party: Genzyme, a Sanofi Company Identifier: NCT00566878     History of Changes
Other Study ID Numbers: AGLU03406
LTS13972 ( Other Identifier: Sanofi )
Study First Received: December 1, 2007
Last Updated: October 20, 2016

Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
Glycogen Storage Disease
Pompe Disease
Pompe Disease (Late-Onset)
Acid Maltase Deficiency Disease
Glycogenosis II

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Glycogen Storage Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases processed this record on September 21, 2017