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Pompe Pregnancy Sub-Registry

This study is currently recruiting participants.
Verified August 2017 by Sanofi ( Genzyme, a Sanofi Company )
Sponsor:
ClinicalTrials.gov Identifier:
NCT00567073
First Posted: December 4, 2007
Last Update Posted: August 22, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
  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
Glycogen Storage Disease Type II (GSD-II) Pompe Disease (Late-onset) Glycogenesis 2 Acid Maltase Deficiency Biological: alglucosidase alpha Other: No Treatment

Study Type: Observational
Study Design: Observational Model: Other
Time Perspective: Other
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 Sanofi ( 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 ]
  • Follow-up of infants born to women with Pompe disease for 3 years post-partum [ Time Frame: 3 years ]

Biospecimen Retention:   Samples Without DNA
Blood

Estimated Enrollment: 20
Actual Study Start Date: November 2011
Estimated Study Completion Date: September 2022
Estimated Primary Completion Date: September 2022 (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

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes 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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00567073     History of Changes
Other Study ID Numbers: AGLU03506
First Submitted: December 1, 2007
First Posted: December 4, 2007
Last Update Posted: August 22, 2017
Last Verified: August 2017

Keywords provided by Sanofi ( 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
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases