Search for Serum/Plasma Biomarkers in Pompe's Disease

Pompe Disease Clinical Trial

— BIOPOMPE  

Official title:

Search for Serum/Plasma Biomarkers in Pompe's Disease

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT03045042
• Other study ID #: IIBSP-BIO-2017-02
• Secondary ID: GZ-2015-11342
• Status: Not yet recruiting
• Phase: N/A
• First received: January 27, 2017
• Last updated: March 17, 2017
• Start date: April 2017
• Est. completion date: January 2019

Study information

• Verified date: January 2017
• Source: Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
• Contact: Eduard Gallardo, PhD
• Phone: 9355657682
• Email: egallardo[@]santpau.cat
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 35
• Est. completion date: January 2019
• Est. primary completion date: January 2018
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 2 Years and older

Eligibility

Inclusion Criteria:

• Genetic confirmation of Pompe disease

Exclusion Criteria:

• Pregnancy

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Late Onset Pompe Disease
• Pompe Disease

Intervention

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

Locations

Country	Name	City	State
Spain	Hospital de la Santa Creu i Sant Pau	Barcelona

Sponsors (2)

Lead Sponsor	Collaborator
Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau	Genzyme, a Sanofi Company

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	MicroRNA in serum samples	Investigators are going to study microRNA following a microarray analysis of serum samples from patients treated and non treated	Baseline
Secondary	Muscle function test	6 minutes walking test (6MWT), time to climb 4 steps, time to descend 4 steps, Activlim, time to walk 10 meter, Medical Research Council scale (MRC), myometry	Baseline and one year
Secondary	Whole body muscle MRI	2 point dixon studies of thigh muscles	Baseline a and one year
Secondary	Changes in microRNA concentration	Investigators will analyze concentration of microRNA after one year follow up in every single patients	One year period