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Recruiting NCT00567073

Pompe Pregnancy Sub-Registry

Conditions: Glycogen Storage Disease Type II (GSD-II) Pompe Disease (Late-onset) Glycogenesis 2 Acid Maltase Deficiency

Sponsor: Genzyme, a Sanofi Company

trial.available_in: БГ
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.
Description
Study Design Time Perspective: Retrospective and Prospective
Who can participate
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
Locations 9
Australia (1)
Investigational Site Number : 151069
Westmead , New South Wales
Belgium (1)
Investigational Site Number : 056001
Ghent
Brazil (1)
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 150978
São Paulo
Croatia (1)
Investigational Site Number : 1910001
Zagreb
Czech Republic (1)
Investigational Site Number : 2030001
Prague
Germany (1)
Investigational Site Number : 152200
Bonn
Italy (1)
Investigational Site Number : 380005
Florence , Firenze
United Kingdom (1)
Investigational Site Number : 87147
Cardiff , Cardiff [Caerdydd Gb-crd]
United States (1)
Barrow Neurol Group- Site Number : 840087
Phoenix , Arizona
Technical details
Status
Recruiting
Study type
OBSERVATIONAL
Sex
Female only
Healthy volunteers
No
Start date
18.06.2007
Completion date
31.01.2034
Registry ID
NCT00567073
Source
anzctr
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