NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

Search for Serum/Plasma Biomarkers in Pompe's Disease

UnknownN/A

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

Conditions: Late Onset Pompe Disease, Pompe Disease

Interventions: Enzyme Replacement Agent

Countries: Spain

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.

Eligibility overview

Sex: ALL

Age: 2 Years to —

Healthy volunteers: Yes

Study type: OBSERVATIONAL

Eligibility criteria

Inclusion Criteria:

* Genetic confirmation of Pompe disease

Exclusion Criteria:

* Pregnancy

Locations (1)

Barcelona, Barcelona, Spain