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Search for Serum/Plasma Biomarkers in Pompe's Disease

NCT03045042 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 35

Last updated 2017-03-20

No results posted yet for this study

Summary

This study aims to analyze serum and plasma samples from patients with late onset Pompe disease treated and not treated with enzyme replacement therapy (ERT) to identify microRNA that could be specific of the disease. The investigators will correlate the concentration of these microRNA with several muscle function tests and quantitative muscle MRI to know whether they are good biomarkers of progression.

Conditions

  • Late Onset Pompe Disease
  • Pompe Disease

Interventions

DRUG

Enzyme Replacement Agent

Patients will be treated following the decision of their doctors. The investigators are going to analyze serum samples of these patients

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    collaborator INDUSTRY

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

    lead OTHER

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2017-04-30
Primary Completion
2018-01-31
Completion
2019-01-31

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 NCT03045042 on ClinicalTrials.gov