Role of the Endothelium in Stroke-like Episode Among CDG Patients (PECDG)

March 30, 2026 updated by: Assistance Publique - Hôpitaux de Paris
The aim of the study is to evaluate the role of the endothelial barrier in the occurrence of stroke-like episode. And to study the protein C system on the surface of the endothelium.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

The study consists in isolating endothelial progenitor cells in peripheral blood. After blood puncture, PBMC (peripheral blood mononuclear cell) are isolated by Ficoll gradient and then culturing in appropriated medium.

When cells are confluent, investigators can study the endothelial barrier by using permeability assay and transendothelial resistance test (iCELLigence technology).

Then, protein C system will be studied. Indeed, EPCR (Endothelial protein C receptor) and TM (thrombomodulin), are the two receptors responsible for the activation of protein C. The expression of these receptors will be assessed in flow cytometry. The activated protein C is able to interact with the third receptor PAR1 (Protease activated receptor 1) which is responsible for the protective effects of the on the endothelial barrier. The PAR1 expression will be studied in flow cytometry also.

In order to measure the capacity of the protein C system to generate activated protein C investigators will assay an activated protein C generation on the cell surface.

All these assays will be realized in basal condition and in heat stress (42°C), to mimic the stroke-like episode environment. Indeed, stroke-like episodes occur always during febrile illness.

Study Type

Interventional

Enrollment (Actual)

6

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
    • Paris
      • Paris, Paris, France, 75015
        • Hopital Necker Enfants Malades

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • patients with CDG molecularly diagnosed
  • with stroke-like history or not

Exclusion Criteria:

  • rejection to participate in research

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Basic Science
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: CDG with stroke-like history
Biological: Peripheral blood puncture

Endothelial progenitor cells will be isolated from peripheral blood by density gradient and cultured in 10%FBS EGM2 medium

On the EPCs we will study:

  • the endothelial permeability
  • the protein C system
Other: CDG without stroke-like history
Biological: Peripheral blood puncture

Endothelial progenitor cells will be isolated from peripheral blood by density gradient and cultured in 10%FBS EGM2 medium

On the EPCs we will study:

  • the endothelial permeability
  • the protein C system

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Endothelial permeability assay in basal condition
Time Frame: at inclusion
at inclusion
Endothelial permeability assay after an heat shock
Time Frame: at inclusion
at inclusion

Secondary Outcome Measures

Outcome Measure
Time Frame
Activated protein C generation assay on the endothelial cell surface
Time Frame: at inclusion
at inclusion
Quantification of the endothelial receptors (EPCR, TM, PAR1)
Time Frame: at inclusion
at inclusion
Endothelial permeability assay after an activated protein C incubation
Time Frame: at inclusion
at inclusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Collaborators

URC-CIC Paris Descartes Necker Cochin

Investigators

  • Principal Investigator: Pascale De Lonlay, MD, PhD, Assistance Publique - Hôpitaux de Paris
  • Study Director: Delphine Borgel, MD, PhD, Assistance Publique - Hôpitaux de Paris

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 23, 2017

Primary Completion (Actual)

April 26, 2019

Study Completion (Actual)

April 26, 2019

Study Registration Dates

First Submitted

August 11, 2017

First Submitted That Met QC Criteria

August 11, 2017

First Posted (Actual)

August 16, 2017

Study Record Updates

Last Update Posted (Actual)

April 3, 2026

Last Update Submitted That Met QC Criteria

March 30, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • K170703J
  • 2017-A02141-52 (Registry Identifier: ANSM-IDRCB)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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