Pompe Pregnancy Sub-Registry

Study Overview
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Brief Summary
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This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Pompe Registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglucosidase alfa) and irrespective of the commercial product with which she may be treated. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician. The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Pompe disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with alglucosidase alfa.

Detailed Description
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Study Design Time Perspective: Retrospective and Prospective

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

Conditions
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Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset), Glycogenesis 2 Acid Maltase Deficiency

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Eligible women must: be enrolled in the pompe registry (NCT00231400) be pregnant, or have been pregnant with appropriate medical documentation available. provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed. Note: It is recommended that pregnancy data be collected on eligible women regardless of infant enrollment. In the event of patients having multiple pregnancies, participation in this Sub-Registry is encouraged for each individual pregnancy. Exclusion Criteria: There are no exclusion criteria for this Sub-Registry
Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Pregnant women with confirmed diagnosis of pompe Disease<br>No experimental intervention is given. Pregnant women with confirmed diagnosis of Pompe disease that are participating in the Pompe Registry (NCT00231400) and consented to participate in the Pompe Pregnancy Sub-registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglusidase alfa) and irrespective of the commercial product with which she may be treated. | |
| Pregnant women receiving no treatment for pompe disease<br>Pregnant women with pompe disease enrolled in the pompe disease registry (NCT00231400) who are not receiving treatment | |
| Infants born to mothers receiving treatment for pompe disease<br>The infants of mothers with pompe disease enrolled in the pompe disease registry (NCT00231400) where the mothers are receiving treatment of alglucosidase alpha (Myozyme) | |
| Infants born to mothers receiving no treatment for pompe disease<br>The infants of mothers with pompe disease enrolled in the Pompe Disease Registry (NCT00231400) where the mothers are not receiving Treatment | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pregnancy outcomes, including complications and infant growth, in all women with pompe disease during pregnancy, regardless of whether they receive disease-specific therapy such as ERT with alglucosidase alfa |  | 10 Months | 
| Follow-up of infants born to women with Pompe disease for 3 years post-partum |  | 3 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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GSD-II, Glycogen Storage Disease Type II (GSD-II), Pompe Disease, Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis II