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A Natural History Study of Adult Onset Pompe Disease Using Muscle MRI

NCT01914536 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 30

Last updated 2013-08-30

No results posted yet for this study

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 radiological tests in order to know which is the natural history of the disease and which is the impact that treatment with recombinant enzyme has in the progression of the disease.

Conditions

  • Pompe Disease

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    collaborator INDUSTRY

  • Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau

    lead OTHER

Principal Investigators

  • Jordi Díaz Manera, MD PhD · Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau

  • Isabel Illa, MD pHD · Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau

Eligibility

Min Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-07-31
Primary Completion
2014-07-31
Completion
2017-07-31

Countries

  • Spain

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01914536 on ClinicalTrials.gov