A Sub-registry to Observe the Effect of Alglucosidase Alfa or Avalglucosidase Alfa Treatment on Pregnancy and Infant Growth in Women With Pompe Disease
Genzyme, a Sanofi Company
Summary
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.
Description
Study Design Time Perspective: Retrospective and Prospective
Eligibility
- Age range
- Not specified
- Sex
- Female
- Healthy volunteers
- No
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 Cr…
Locations (35)
- Barrow Neurol Group- Site Number : 840087Phoenix, Arizona
- University of Florida Pediatrics Genetics- Site Number : 157138Jacksonville, Florida
- Emory University School Of Medicine- Site Number : 840060Atlanta, Georgia
- Indianapolis University School of Medicine- Site Number : 840027Indianapolis, Indiana
- University of Iowa- Site Number : 150294Iowa City, Iowa
- Spectrum for Health- Site Number : 840019Grand Rapids, Michigan