Pompe Pregnancy Sub-Registry

Brief description of study

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.

Study Design Time Perspective: Retrospective and Prospective

Clinical Study Identifier: NCT00567073

You may be eligible for this study if you meet the following criteria:

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

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

Last updated on 06 Jan 2021

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  • You can expect the study team to contact you via email or phone in the next few days.
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