Pompe Lactation Sub-Registry - Full Text View

Sponsored by:	Genzyme
Information provided by:	Genzyme
ClinicalTrials.gov Identifier:	NCT00566878

Purpose

The objective is to determine if alglucosidase alfa is present in breast milk from mothers with Pompe Disease being treated with Myozyme® and to measure breast milk production and composition in women with Pompe Disease who receive Myozyme®.

Condition
Glycogen Storage Diseae Type II Pompe Disease

Genetics Home Reference related topics: Pompe disease

Drug Information available for: Alglucosidase Alfa Glucan 1,4-alpha-Glucosidase

U.S. FDA Resources

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

Further study details as provided by Genzyme:

Primary Outcome Measures:

To track and evaluate pregnancy outcomes in women with Pompe disease who receive Myozyme during pregnancy and in women with Pompe disease who do not receive Myozyme. [ Time Frame: 46 weeks ] [ Designated as safety issue: No ]
To follow infants born to women with Pompe disease. [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Biospecimen Retention: Samples Without DNA

Biospecimen Description:

Blood, Breast milk

Study Start Date:

December 2007

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
Must be pregnant and intend to breast-feed or be currently lactating and receive at least one infusion of Myozyme 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:

No exclusion criteria

Contacts and Locations

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

Sponsors and Collaborators

Genzyme

Investigators

Study Director:

Deborah Marsden, MD

Genzyme

More Information

Study ID Numbers:	AGLU03406
Study First Received:	December 1, 2007
Last Updated:	October 21, 2008
ClinicalTrials.gov Identifier:	NCT00566878
Health Authority:	United States: Food and Drug Administration

Keywords provided by Genzyme:

Glycogen Storage Disease Type II (GSD-II)
GSD-II
Pompe Disease

Pompe Disease (Late-Onset)
Acid Maltase Deficiency Disease
Glycogenosis II

Study placed in the following topic categories:

Metabolic Diseases
Glycogen Storage Disease
Lysosomal Storage Diseases
Central Nervous System Diseases
Glycogen Storage Disease Type II
Brain Diseases
Glycogen storage disease type 2

Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Metabolic disorder
Deficiency Diseases
Brain Diseases, Metabolic

Additional relevant MeSH terms:

Lysosomal Storage Diseases, Nervous System
Nervous System Diseases
Carbohydrate Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on January 16, 2009