Status:
UNKNOWN
A Natural History Study of Adult Onset Pompe Disease Using Muscle MRI
Lead Sponsor:
Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Collaborating Sponsors:
Genzyme, a Sanofi Company
Conditions:
Pompe Disease
Eligibility:
All Genders
8+ years
Brief Summary
This project is an observational prospective study in which patients affected by an adult onset Pompe disease will be followed-up during three years using different clinical, analytical and radiologic...
Detailed Description
Study aim: The principal objective of the study is to find biomarkers that quantify the natural progression of the disease and to know if they are useful to determine the improvement or lack of impai...
Eligibility Criteria
Inclusion
- Pompe disease confirmed using genetic study
- Onset of symptoms more than 2 years old
- To be able to come to the hospital and follow all the visits
- Patients with respiratory involvement are welcomed
Exclusion
- Patients unable to perform a MRI (respiratory problems can be solved using mechanical ventilation)
Key Trial Info
Start Date :
July 1 2013
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
July 1 2017
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT01914536
Start Date
July 1 2013
End Date
July 1 2017
Last Update
August 30 2013
Active Locations (1)
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1
Hospital de la Santa Creu iSant Pau
Barcelona, Barcelona, Spain, 08025