Pompe Pregnancy Sub-Registry

Tracking Pregnancy Outcomes in Women with Pompe Disease

Recruiting
Female
Phase N/A
20 participants needed
9 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 20 Nov 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, leading to muscle weakness and other health issues. The study does not involve any experimental treatments. Instead, it focuses on observing and collecting information about the pregnancies of women who are part of the Pompe Registry, which is a large database of individuals with this condition.

Participants will undergo clinical assessments as part of their regular medical care, as determined by their doctors. The study will gather data on pregnancy complications and the growth of infants born to these women. This information will help researchers understand more about the effects of Pompe disease on pregnancy and infant development.

  • Who can participate: Women who are enrolled in the Pompe Registry and are pregnant, or have been pregnant with appropriate medical documentation, can participate. They must provide signed informed consent before any data collection.
  • Study details: Participants will continue with their standard medical care as determined by their physician. The study will collect data on their pregnancies, including any complications and infant growth. No experimental treatments will be administered.

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