Pompe Pregnancy Sub-Registry

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2013 by Genzyme, a Sanofi Company
Sponsor:
Information provided by (Responsible Party):
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00567073
First received: December 1, 2007
Last updated: September 10, 2013
Last verified: September 2013
  Purpose

The objective is to track pregnancy outcomes in women with Pompe Disease and to follow infants born to women with Pompe Disease.


Condition Intervention Phase
Glycogen Storage Disease Type II (GSD-II)
Pompe Disease (Late-onset)
Glycogenesis 2 Acid Maltase Deficiency
Biological: alglucosidase alpha
Other: No Treatment
Phase 4

Study Type: Observational
Official Title: A Sub-Registry to Observe the Effect of Myozyme (Alglucosidase Alfa) Treatment on Pregnancy and Infant Growth in Women With Pompe Disease

Resource links provided by NLM:


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

Primary Outcome Measures:
  • Pregnancy outcomes, including complications, in women with Pompe disease who receive Myozyme during pregnancy and in women with Pompe disease that do not receive Myozyme [ Time Frame: 10 Months ] [ Designated as safety issue: No ]
  • Follow-up of infants born to women with Pompe disease for 3 years post-partum [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples Without DNA

Blood


Estimated Enrollment: 20
Study Start Date: November 2011
Estimated Study Completion Date: December 2021
Estimated Primary Completion Date: November 2021 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Pregnant Women Receiving Treatment for Pompe Disease
Pregnant Women with Pompe Disease Enrolled in the Pompe Disease Registry (NCT00231400)That Are Receiving Treatment of alglucosidase alpha (Myozyme)
Biological: alglucosidase alpha
Other Name: Myozyme
Pregnant Women Receiving No Treatment for Pompe Disease
Pregnant Women with Pompe Disease Enrolled in the Pompe Disease Registry (NCT00231400)That Are Not Receiving Treatment
Other: No Treatment
Infants Born to Mothers Receiving Treatment for Pompe
The Infants of Mothers with Pompe Disease Enrolled in the Pompe Disease Registry (NCT00231400)Where the Mothers Are Receiving Treatment of alglucosidase alpha (Myozyme)
Other: No Treatment
Infants Born to Mothers Receiving No Treatment for Pompe
The Infants of Mothers with Pompe Disease Enrolled in the Pompe Disease Registry (NCT00231400)Where the Mothers Are Not Receiving Treatment
Other: No Treatment

Detailed Description:

Study Design Time Perspective: Retrospective and Prospective

  Eligibility

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

Pregnant Females with Pompe disease and/or infants born to females with Pompe disease. Participants may or may not be receiving therapy.

Criteria

Inclusion Criteria:

  • Enroll in or agree to enroll in Pompe Registry
  • Be pregnant or have been pregnant with appropriate medical documentation
  • Provide a signed Patient Information and Authorization Form to participate in the sub-registry prior to any sub-registry related data collection is performed

Exclusion Criteria:

  • No Exclusion Criteria
  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: NCT00567073

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 and not limited to this facility; facilities not yet active may enroll upon identification of a patient Recruiting
Cambridge, Massachusetts, United States, 02142
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00567073     History of Changes
Other Study ID Numbers: AGLU03506
Study First Received: December 1, 2007
Last Updated: September 10, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Genzyme, a Sanofi Company:
Glycogen Storage Disease Type II (GSD-II)
GSD-II
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
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on August 28, 2014