Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02904863
Recruitment Status : Unknown
Verified September 2016 by Assistance Publique Hopitaux De Marseille.
Recruitment status was:  Recruiting
First Posted : September 19, 2016
Last Update Posted : September 19, 2016
Sponsor:
Information provided by (Responsible Party):
Assistance Publique Hopitaux De Marseille

Brief 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.

Condition or disease Intervention/treatment Phase
Hemolytic Uremic Syndrome Biological: Extra blood draw samples Not Applicable

Detailed 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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 48 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Study Start Date : February 2013
Estimated Primary Completion Date : February 2017
Estimated Study Completion Date : February 2018


Arm Intervention/treatment
Experimental: patients with HUS Biological: Extra blood draw samples



Primary Outcome Measures :
  1. number of circulating endothelial cells (CECs) and endothelial progenitor cells (EPCs) [ Time Frame: 36 months ]
  2. number of endothelial progenitor cells (EPCs) [ Time Frame: 36 months ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02904863


Contacts
Layout table for location contacts
Contact: Gilles Kaplanski, Professor gilles.kaplanski@ap-hm.fr

Locations
Layout table for location information
France
Assistance Publique Hôpitaux de Marseille Recruiting
Marseille, France
Contact: Gilles Kaplanski       gilles.kaplanski@ap-hm.fr   
Sponsors and Collaborators
Assistance Publique Hopitaux De Marseille
Investigators
Layout table for investigator information
Study Director: Urielle Desalbres Assistance Publique Hôpitaux de Marseille

Layout table for additonal information
Responsible Party: Assistance Publique Hopitaux De Marseille
ClinicalTrials.gov Identifier: NCT02904863     History of Changes
Other Study ID Numbers: 2012-03
2012-A00217-36 ( Other Identifier: ANSM )
First Posted: September 19, 2016    Key Record Dates
Last Update Posted: September 19, 2016
Last Verified: September 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Layout table for MeSH terms
Syndrome
Hemolysis
Azotemia
Hemolytic-Uremic Syndrome
Disease
Pathologic Processes
Uremia
Kidney Diseases
Urologic Diseases
Anemia, Hemolytic
Anemia
Hematologic Diseases
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders