Pompe Pregnancy Sub-Registry
Study on Pregnancy Outcomes in Women with Pompe Disease
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
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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
This study investigates the outcomes of pregnancies 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. The study includes women who are part of the Pompe Registry, whether or not they receive specific treatments for the disease, such as enzyme replacement therapy (ERT).
Participants will undergo clinical assessments and receive standard care as determined by their doctors. The study aims to track various pregnancy outcomes, including any complications and the growth of infants born to these women. No experimental treatments are involved.
- Who can participate: Eligible participants are women enrolled in the Pompe Registry who are currently pregnant or have been pregnant, with proper medical documentation. They must provide informed consent to join the Sub-Registry.
- Study details: Participants will receive standard care as directed by their physicians and undergo clinical assessments to track pregnancy outcomes. The study does not involve any experimental treatments.
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