Trials / Unknown
UnknownNCT03045042
Search for Serum/Plasma Biomarkers in Pompe's Disease
- Status
- Unknown
- Phase
- —
- Study type
- Observational
- Enrollment
- 35 (estimated)
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau · Academic / Other
- Sex
- All
- Age
- 2 Years
- Healthy volunteers
- Accepted
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
Interventions
| Type | Name | Description |
|---|---|---|
| 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 |
Timeline
- Start date
- 2017-04-01
- Primary completion
- 2018-01-01
- Completion
- 2019-01-01
- First posted
- 2017-02-07
- Last updated
- 2017-03-20
Locations
1 site across 1 country: Spain
Source: ClinicalTrials.gov record NCT03045042. Inclusion in this directory is not an endorsement.