Pompe Lactation Sub-Registry

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2014 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT00566878
First received: December 1, 2007
Last updated: October 21, 2014
Last verified: October 2014
  Purpose

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:

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 ] [ Designated as safety issue: No ]
  • breast milk production and composition in women with Pompe disease who receive alglucosidase alfa [ Time Frame: 6 Months ] [ Designated as safety issue: No ]

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
  Eligibility

Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Females diagnosed with Pompe Disease and Lactating

Criteria

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 ClinicalTrials.gov identifier: NCT00566878

Contacts
Contact: Medical Information 800-745-4447 MedInfo@genzyme.com
Contact: Medical Information 617-252-7832 MedInfo@genzyme.com

Locations
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
Recruiting
Grand Rapids, Michigan, United States, 49503
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00566878     History of Changes
Other Study ID Numbers: AGLU03406, LTS13972
Study First Received: December 1, 2007
Last Updated: October 21, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Sanofi:
Glycogen Storage Disease
GSD-II
Pompe Disease
Pompe Disease (Late-Onset)
Acid Maltase Deficiency Disease
Glycogenosis II

Additional relevant MeSH terms:
Glycogen Storage Disease
Glycogen Storage Disease Type II
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Carbohydrate Metabolism, Inborn Errors
Central Nervous System Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases

ClinicalTrials.gov processed this record on October 22, 2014