Pompe Pregnancy Sub-Registry

Pregnancy Outcomes in Women with Pompe Disease

Recruiting
Female
Phase N/A
20 participants needed
11 Locations

Study Overview

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 or avalglucosidase 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 or avalglucosidase alfa.

Study Details

Study Design Time Perspective: Retrospective and Prospective

Eligibility Criteria

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

Updated on 08 Apr 2024. Study ID: NCT00567073

This study investigates pregnancy outcomes in women with Pompe disease. Pompe disease is a rare genetic disorder that affects the body's ability to break down a complex sugar called glycogen due to a deficiency of the enzyme acid alpha-glucosidase. This study aims to track pregnancy complications and infant growth in women enrolled in the Pompe Registry, regardless of whether they receive enzyme replacement therapy (ERT) or other disease-specific treatments.

Participants in this observational study will undergo clinical assessments and receive standard care as determined by their physician. An observational study is a type of research study where data is collected by observing participants receiving routine care, without changing their treatment, which may focus on people using specific medications or having certain conditions to better understand how treatments work.

  • Who can participate: Eligible women must be enrolled in the Pompe Registry and be pregnant or have been pregnant. They must also provide a signed informed consent form.
  • Study details: Participants will be part of a sub-registry that tracks pregnancy outcomes and will receive standard care as determined by their physician. No experimental interventions will be given.

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