Glycogenesis 2 Acid Maltase Deficiency, Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset)
Conditions
Keywords
Glycogen Storage Disease Type II (GSD-II), GSD-II, Pompe Disease, Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis II
Brief 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.
Detailed description
Study Design Time Perspective: Retrospective and Prospective
Interventions
None listed
Sponsors
Genzyme, a Sanofi Company
Study design
Observational model
OTHER
Time perspective
OTHER
Eligibility
Sex/Gender
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 criteria
There are no
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 | 10 Months |
| Follow-up of infants born to women with Pompe disease for 3 years post-partum | 3 years |
Countries
Australia, Belgium, Brazil, Croatia, Czechia, Germany, Italy, United Kingdom, United States
Contacts
CONTACTTrial Transparency email recommended (Toll free number for US & Canada)
CONTACTPompe Registry HelpLine
STUDY_DIRECTORStudy Director
Genzyme, a Sanofi Company
Outcome results
None listed