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Pompe Pregnancy Sub-Registry

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ClinicalTrials.gov Identifier: NCT00567073
Recruitment Status : Recruiting
First Posted : December 4, 2007
Last Update Posted : June 10, 2019
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

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

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

Detailed Description:
Study Design Time Perspective: Retrospective and Prospective

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Study Type : Observational
Estimated Enrollment : 20 participants
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
Actual Study Start Date : November 2011
Estimated Primary Completion Date : January 31, 2034
Estimated Study Completion Date : January 31, 2034


Group/Cohort Intervention/treatment
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



Primary Outcome Measures :
  1. 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 ]
  2. 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


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Ages Eligible for Study:   Child, Adult, Older Adult
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

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

Locations
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United States, New York
Investigational Site Number 840040 Recruiting
New York, New York, United States, 10016
United States, Virginia
Investigational Site Number 840025 Recruiting
Fairfax, Virginia, United States, 22030
Italy
Investigational Site Number 380008 Recruiting
Brescia, Italy, 25123
Investigational Site Number 380006 Recruiting
Cagliari, Italy, 09126
Investigational Site Number 380005 Recruiting
Firenze, Italy, 50141
Investigational Site Number 380004 Recruiting
Genova, Italy, 16147
Investigational Site Number 380013 Recruiting
Messina, Italy, 98125
Investigational Site Number 380007 Recruiting
Milano, Italy, 20133
Investigational Site Number 380009 Recruiting
Monza, Italy
Investigational Site Number 380002 Recruiting
Padova, Italy, 35128
Investigational Site Number 380011 Recruiting
Padova, Italy
Investigational Site Number 380003 Recruiting
Pavia, Italy, 27100
Investigational Site Number 380015 Recruiting
Roma, Italy, 00165
Investigational Site Number 380012 Recruiting
Roma, Italy
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
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Study Director: Medical Monitor Genzyme, a Sanofi Company

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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00567073     History of Changes
Other Study ID Numbers: AGLU03506
First Posted: December 4, 2007    Key Record Dates
Last Update Posted: June 10, 2019
Last Verified: June 2019
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:
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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