Study of 'Vascular Competence' Profile and Endothelial Activation in the Hemolytic Uremic Syndrome in Children and Adults

Hemolytic Uremic Syndrome Clinical Trial

— SHU  

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02904863
• Other study ID #: 2012-03
• Secondary ID: 2012-A00217-36
• Status: Completed
• Phase: N/A
• Start date: February 11, 2013
• Est. completion date: July 27, 2023

Study information

• Verified date: July 2023
• Source: Assistance Publique Hopitaux De Marseille
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The Hemolytic Uremic Syndrome (HUS) is a rare thrombotic microangiopathy (TMA), affecting both children and adults. HUS is characterized by the abnormal occurrence of diffuse thrombosis in the microcirculation resulting in the occurrence of ischemic events affecting especially the kidneys and is associated with hemolytic anemia. One of the major problems encountered in the management of HUS is the absence of reliable marker of treatment response or relapse; conventional hematological markers being too insensitive to judge therapeutic efficacy or identify early relapse. Data from the literature suggest that the endothelial cell is a major target of this syndrome. Our hypothesis is that an initial micro-endothelial activation plays a critical role in the initiation and / or relapse of the disease.The main objective of this study is to define a "vascular competence" profile in a population of patients with typical or atypical HUS; both in the acute phase and in remission of the disease.

Description:

The Hemolytic Uremic Syndrome (HUS) is a rare thrombotic microangiopathy (TMA), affecting both children and adults. HUS is characterized by the abnormal occurrence of diffuse thrombosis in the microcirculation resulting in the occurrence of ischemic events affecting especially the kidneys and is associated with hemolytic anemia.Its prognosis is severe, with a mortality of 1% in children, 10% in adults and the occurrence of renal failure in 50% of cases.In its typical form, HUS occurs in the aftermath of a diarrheic intestinal infection by bacteria which produce a Shiga toxin. In its unusual shape, which affects both children and adults, there are genetic abnormalities alternate way of regulating complement proteins explaining frequent relapses.One of the major problems encountered in the management of HUS is the absence of reliable marker of treatment response or relapse; conventional hematological markers being too insensitive to judge therapeutic efficacy or identify early relapse. Data from the literature suggest that the endothelial cell is a major target of this syndrome. Our hypothesis is that an initial micro-endothelial activation plays a critical role in the initiation and / or relapse of the disease through the sudden release of high molecular weight ultralarge von Willebrand factor (UL-vWHf) and procoagulant endothelial microparticles.The main objective of this study is to define a "vascular competence" profile in a population of patients with typical or atypical HUS; both in the acute phase and in remission of the disease.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 19
• Est. completion date: July 27, 2023
• Est. primary completion date: April 7, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Patients with a clinical diagnosis of typical or atypical HUS in acute phase

Exclusion Criteria:

• Patient with positive serological screening test for infection with the HIV.

• Patients with a history of cancer.

• Patients who have undergone organ transplantation or bone marrow.

• Patient with a vivid picture of autoimmune thrombotic thrombocytopenic purpura

Related Conditions & MeSH terms

• Hemolysis
• Hemolytic Uremic Syndrome
• Hemolytic-Uremic Syndrome
• Syndrome

Intervention

Biological:
• Extra blood draw samples

Locations

Country	Name	City	State
France	Assistance Publique Hôpitaux de Marseille	Marseille

Sponsors (1)

Lead Sponsor	Collaborator
Assistance Publique Hopitaux De Marseille

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	number of circulating endothelial cells (CECs) and endothelial progenitor cells (EPCs)		36 months
Primary	number of endothelial progenitor cells (EPCs)		36 months