Hemolytic Uremic SyndromeSHUNCT02904863
Study of 'Vascular Competence' Profile and Endothelial Activation in the Hemolytic Uremic Syndrome in Children and Adults
A study testing Extra blood draw samples in people with Hemolytic Uremic Syndrome, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemolytic Uremic Syndrome
- What is being tested?
- Extra blood draw samples
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 1 siteFrance
- Study dates
- 11 Feb 2013to 27 Jul 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with a clinical diagnosis of typical or atypical HUS in acute phase
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- Conditions
- Hemolytic Uremic Syndrome
- Being tested
- Extra blood draw samples
- Medical subject headings
- Hemolytic-Uremic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Extra blood draw samplesBiological
| Study group | Role | What they receive |
|---|---|---|
| patients with HUS | Experimental | Not described Receives: Biological: Extra blood draw samples |
What is being measured
The primary outcome is the main question the study is designed to answer.
number of circulating endothelial cells (CECs) and endothelial progenitor cells (EPCs)
Measured: 36 months
number of endothelial progenitor cells (EPCs)
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2016 (estimated)
- Study start11 Feb 2013
- Primary completion7 Apr 2017
- Study completion27 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemolytic Uremic SyndromeStudies of the same condition, open and under way.
- More trials run by Assistance Publique Hopitaux De MarseilleThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Urielle Desalbres, Study director, Assistance Publique Hôpitaux de Marseille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02904863
- Sponsor's protocol ID
- 2012-03
- Other IDs
- 2012-A00217-36
- Acronym
- SHU
- Status last verified
- Jul 2023
- First submitted
- 8 Sep 2016
- Last updated
- 28 Jul 2023
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of 'Vascular Competence' Profile and Endothelial Activation in the Hemolytic Uremic Syndrome in Children and Adults
- ClinicalTrials.gov ID
- NCT02904863
- Acronym / sponsor ID
- SHU / 2012-03
- Conditions
- Hemolytic Uremic Syndrome
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Study start
- 11 Feb 2013 (actual)
- Primary completion
- 7 Apr 2017 (actual)
- Study completion
- 27 Jul 2023 (actual)
- First posted
- 19 Sep 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patients with HUS | Experimental | Biological: Extra blood draw samples |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Patients with a clinical diagnosis of typical or atypical HUS in acute phase
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Extra blood draw samples | Biological | patients with HUS |
Primary outcome
number of circulating endothelial cells (CECs) and endothelial progenitor cells (EPCs)
Time frame: 36 months
number of endothelial progenitor cells (EPCs)
Time frame: 36 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Urielle Desalbres study director
Assistance Publique Hôpitaux de Marseille
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Extra blood draw samples in people with Hemolytic Uremic Syndrome, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
number of circulating endothelial cells (CECs) and endothelial progenitor cells (EPCs) (time frame: 36 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jul 2023.