Thrombotic MicroangiopathiesCOMPETENCT04745195
Complement Prospective Evaluation of Thrombotic Microangiopathy on Endothelium
An observational study of Thrombotic Microangiopathies and Hemolytic Uremic Syndrome, Atypical, run by Maastricht University Medical Center at 1 site in Netherlands.
Trial summary
Thrombotic microangiopathy (TMA) is a severe and life-threatening condition, often affecting the kidneys and brain. It can occur on the background of various clinical conditions. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombotic Microangiopathies, Hemolytic Uremic Syndrome, Atypical, Hemolytic-Uremic Syndrome
- How many people
- 42 participants
- Where
- 1 siteNetherlands
- Study dates
- 11 Aug 2021to 31 Dec 2026
- How long will I take part?
- 12 Months
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients of at least 18 years of age presenting with TMA, either on peripheral blood or kidney biopsy, will be recruited at the emergency departments of (regional) hospitals affiliated to the Limburg Renal Registry, Maastricht University Medical Center, Maastricht, NLD. Patients who meet the eligibility criteria will be included.
Who may be able to join
- Males or females at least 18 years of age;
- Have acute kidney injury, defined as estimated GFR <45 mL/min/1.73m2;
- Have documented TMA either on peripheral blood, defined as Coombs negative microangiopathic hemolytic anemia (hematocrit <30%, hemoglobin <6.5 mmol/L [<10 g/dL], lactate dehydrogenase >500 U/L, and either schistocytes on peripheral blood smear or undetectable haptoglobin), and platelets <150,000 per µL, or kidney biopsy;
- Have primary atypical HUS or a coexisting condition linked to complement dysregulation:
- Hypertensive emergency, defined as SBP/DBP of >180/120 mmHg and impending organ damage secondary to hypertension (at least one of the following: neurologic disease, hypertensive retinopathy grade III and/or IV, left ventricular hypertrophy); OR
- Pregnancy, including 12 weeks postpartum; OR
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- Kidney donor recipient; OR
- Systemic auto-immune disease associated with TMA, including systemic sclerosis, systemic lupus erythematosus, anti-phospholipid syndrome;
- Have the ability to understand the requirements of the study, provide written informed consent, and comply with the study protocol procedures.
Who cannot join
- Have secondary causes of hypertensive emergency, including renovascular hypertension, Cushing syndrome, aldosteronism, pheochromocytoma, thyroid disease;
- Have a nephropathy not related to thrombosis on kidney biopsy;
- Have ADAMTS13 deficiency, defined as ADAMTS13 activity <10%;
- Have a positive stool culture for Shiga toxin producing bacteria;
- Have positive serologic test for viral infections, including HIV and CMV;
- Have a history of malignant disease, excluding non-melanoma skin cancer;
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- Have a history of bone marrow or solid organ transplantation, excluding kidney transplantation;
- Received at least one of the following agents: chemotherapeutics, sirolimus, anti-VEGF agents;
- Have a history of recent past exposure to illicit drug(s).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Netherlands1 site, 1 recruiting
- Recruiting
Maastricht University Medical Center
Maastricht, Limburg, 6229HX
Sjoerd A.M.E.G. Timmermans, MD, PhD, +31(0)433871198, sjoerd.timmermans@mumc.nl
Daan P.C. van Doorn, MD, +31(0)433871198, daan.vandoorn@maastrichtuniversity.nl
About this study
Official title: Diagnostic and Risk Criteria for Complement Defects in Thrombotic Microangiopathy and Amplifying Conditions, Such as Severe Hypertension: The COMPETE Study.
Thrombotic microangiopathy (TMA) is a severe and life-threatening condition, often affecting the kidneys and brain. It can occur on the background of various clinical conditions. Dysregulation of the alternative pathway of complement may be the etiological factor and this type of TMA is classified, according to the current nomenclature, as primary atypical hemolytic uremic syndrome (HUS). Half the patients with primary atypical HUS present with rare variants in complement genes, although coexisting conditions are often needed for the TMA to become manifest. In patients with secondary atypical HUS, certain coexisting conditions appear to drive the disease and treatment should target the underlying condition to remit the TMA.
Recently, the investigators demonstrated, by using a novel in-house developed functional endothelial cell-based test, that complement dysregulation and overactivation is the dominant cause of disease and its sequelae in a subset of patients with secondary atypical HUS, having impact on treatment and prognosis. The investigators did first prove this concept in patients presenting with TMA and hypertensive emergency. A prospective study is needed to further corroborate these findings along the spectrum of TMA. The investigators hypothesize that their functional endothelial cell-based test, the so-called "HMEC" test, can better categorizes the TMA into different groups with potential therapeutic and prognostic implications. Thus, paving the road to the ultimate goal of precision medicine.
- Conditions
- Thrombotic Microangiopathies, Hemolytic Uremic Syndrome, Atypical, Hemolytic-Uremic Syndrome
- Medical subject headings
- Thrombotic Microangiopathies, Atypical Hemolytic Uremic Syndrome, Hemolytic-Uremic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Complement-mediated thrombotic microangiopathy | Not described | |
| Thrombotic microangiopathty with normal complement regulation | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
The prevalence of complement-mediated TMA along the spectrum of TMA
Measured: 1 year
Secondary outcomes (5)
The diagnostic performance of the "HMEC" test for the diagnosis of complement-mediated TMA
Measured: 1 year
The dynamics of ex vivo C5b9 formation on the endothelium during the course of TMA
Measured: every month for up to 1 year
The dynamics of ex vivo C5b9 formation on the endothelium as compared to routine complement measures during the course of TMA
Measured: every month for up to 1 year
Composite kidney endpoint: 50% eGFR decline, chronic kidney disease stage G5, end-stage kidney disease
The diagnostic and prognostic value of pathologic features and chronicity indices on kidney biopsy at the time of presentation
Measured: 1 year
composite TMA endopoint: TMA recurrence, end-stage kidney disease
The prognostic value of genetic variants in complement genes
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Feb 2021
- Study start11 Aug 2021
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thrombotic MicroangiopathiesStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 42 participants (estimated)
- Target duration
- 12 Months
Sponsor and investigators
- Lead sponsor
- Maastricht University Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sjoerd Timmermans, Maastricht University Medical Center
- Investigators
- Pieter van Paassen, MD, PhD, Principal investigator, Maastricht University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04745195
- Sponsor's protocol ID
- NL74928.068.20
- Other IDs
- 23OK1056
- Acronym
- COMPETE
- Status last verified
- Sep 2025
- First submitted
- 6 Jan 2021
- Last updated
- 1 Oct 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Diagnostic and Risk Criteria for Complement Defects in Thrombotic Microangiopathy and Amplifying Conditions, Such as Severe Hypertension: The COMPETE Study.
- ClinicalTrials.gov ID
- NCT04745195
- Acronym / sponsor ID
- COMPETE / NL74928.068.20
- Conditions
- Thrombotic Microangiopathies, Hemolytic Uremic Syndrome, Atypical, Hemolytic-Uremic Syndrome
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Maastricht University Medical CenterUniversity, hospital or other
- Study start
- 11 Aug 2021 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 9 Feb 2021 (actual)
- 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.
Thrombotic microangiopathy (TMA) is a severe and life-threatening condition, often affecting the kidneys and brain. It can occur on the background of various clinical conditions. Dysregulation of the alternative pathway of complement may be the etiological factor and this type of TMA is classified, according to the current nomenclature, as primary atypical hemolytic uremic syndrome (HUS). Half the patients with primary atypical HUS present with rare variants in complement genes, although coexisting conditions are often needed for the TMA to become manifest. In patients with secondary atypical HUS, certain coexisting conditions appear to drive the disease and treatment should target the underlying condition to remit the TMA.
Recently, the investigators demonstrated, by using a novel in-house developed functional endothelial cell-based test, that complement dysregulation and overactivation is the dominant cause of disease and its sequelae in a subset of patients with secondary atypical HUS, having impact on treatment and prognosis. The investigators did first prove this concept in patients presenting with TMA and hypertensive emergency. A prospective study is needed to further corroborate these findings along the spectrum of TMA. The investigators hypothesize that their functional endothelial cell-based test, the so-called "HMEC" test, can better categorizes the TMA into different groups with potential therapeutic and prognostic implications. Thus, paving the road to the ultimate goal of precision medicine.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 12 Months
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Complement-mediated thrombotic microangiopathy | – | – |
| Thrombotic microangiopathty with normal complement regulation | – | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients of at least 18 years of age presenting with TMA, either on peripheral blood or kidney biopsy, will be recruited at the emergency departments of (regional) hospitals affiliated to the Limburg Renal Registry, Maastricht University Medical Center, Maastricht, NLD. Patients who meet the eligibility criteria will be included.
Key inclusion criteria (9 listed)
- Males or females at least 18 years of age;
- Have acute kidney injury, defined as estimated GFR <45 mL/min/1.73m2;
- Have documented TMA either on peripheral blood, defined as Coombs negative microangiopathic hemolytic anemia (hematocrit <30%, hemoglobin <6.5 mmol/L [<10 g/dL], lactate dehydrogenase >500 U/L, and either schistocytes on peripheral blood smear or undetectable haptoglobin), and platelets <150,000 per µL, or kidney biopsy;
- Have primary atypical HUS or a coexisting condition linked to complement dysregulation:
- Have the ability to understand the requirements of the study, provide written informed consent, and comply with the study protocol procedures.
Key exclusion criteria (9 listed)
- Have secondary causes of hypertensive emergency, including renovascular hypertension, Cushing syndrome, aldosteronism, pheochromocytoma, thyroid disease;
- Have a nephropathy not related to thrombosis on kidney biopsy;
- Have ADAMTS13 deficiency, defined as ADAMTS13 activity <10%;
- Have a positive stool culture for Shiga toxin producing bacteria;
- Have positive serologic test for viral infections, including HIV and CMV;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
The prevalence of complement-mediated TMA along the spectrum of TMA
Time frame: 1 year
5 secondary outcomes
The diagnostic performance of the "HMEC" test for the diagnosis of complement-mediated TMA
Time frame: 1 year
The dynamics of ex vivo C5b9 formation on the endothelium during the course of TMA
Time frame: every month for up to 1 year
The dynamics of ex vivo C5b9 formation on the endothelium as compared to routine complement measures during the course of TMA
Time frame: every month for up to 1 year
Composite kidney endpoint: 50% eGFR decline, chronic kidney disease stage G5, end-stage kidney disease
Time frame: 1 year
composite TMA endopoint: TMA recurrence, end-stage kidney disease
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2025.
- Individual participant dataSharing planned: Undecided
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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.
Pieter van Paassen, MD, PhD principal investigator
Maastricht University Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thrombotic Microangiopathies and Hemolytic Uremic Syndrome, Atypical, run by Maastricht University Medical Center at 1 site in Netherlands.
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 9 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 1 Oct 2025. 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 Netherlands. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sjoerd A.M.E.G. Timmermans, MD, PhD (+31(0)433871198, sjoerd.timmermans@mumc.nl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The prevalence of complement-mediated TMA along the spectrum of TMA (time frame: 1 year). 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 1 Oct 2025.