Hemolytic Uremic Syndrome of ChildhoodPhase 3ECULISHUNCT02205541
Eculizumab in Shiga-toxin Related Hemolytic and Uremic Syndrome Pediatric Patients - ECULISHU
A Phase 3 study testing Eculizumab in people with Hemolytic Uremic Syndrome of Childhood, run by University Hospital, Toulouse at 18 sites in France.
Trial summary
The investigators aim to perform the first controlled randomized prospective study using ECZ in pediatric STEC-HUS. This is of great interest as there is still no efficient specific therapy in that potentially devastating disease. Read more about this study
- Who is it for?
- Ages 1 month to 18 years
- Conditions the study is for
- Hemolytic Uremic Syndrome of Childhood
- What is being tested?
- Eculizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 100 participants
- Where
- 18 sitesFrance
- Study dates
- Jun 2015to Jun 2018
- Phase
- Phase 3
- 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 1 month to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Pediatric patient (1 month-18 years old)
- Affected by STEC-HUS defined by :
- Thrombocytopenia (<150 000/mm3)
- Mechanic hemolytic anemia (Hemoglobin < 10g/dL, haptoglobin <LLN, lactate dehydrogenase (LDH) >upper limit of normal (ULN) and/or bilirubin > ULN, presence of schizocytes)
- ARF defined by an estimated Schwartz 2009 creatinin clearance <75ml/min/1,73m²
- With prodromal diarrhea and/or presence of an enterohemorrhagic strain of Escherichia Coli and/or identification of the Stx 1 or 2 genes in the stool sample or rectal swab
- Written consent of the 2 parents
- Female patients of childbearing potential must be practicing an effective, reliable and medically acceptable contraceptive regimen during the entire duration of the study and 5 months after the end of the participation.
Who cannot join
- Neonatal HUS
- Malignancy
- Known HIV infection
- Pregnancy or lactation
- Identified drug exposure-related HUS
- Infection-related HUS
Show all 14Show fewer
- Known systemic lupus erythematosus or antiphospholipid antibody positivity or syndrome
- Patient already enrolled in a drug trial
- Patient with ongoing meningococcal infection
- Patient affected by aHUS or family history of aHUS
- STEC-HUS patient with severe multiorgan involvement at diagnostic:
- Neurological involvement (seizures, coma, focal deficit) with signs of microangiopathy on cerebral Magnetic Resonance Imaging.
- Cardiac involvement (cardiac failure, ischemic myocarditis, conduction or rhythm troubles)
- Digestive involvement (severe pancreatitis defined by lipasemia>500UI/L, severe hepatitis defined by transaminase >x10ULN and/or prothrombin time<60%, hemorrhagic colitis, bowel perforation, rectal prolapsus)
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
France18 sites
University Hospital
Amiens, 80054
University Hospital
Angers, 49100
University Hospital
Besançon, 25030
Pellegrin Hospital
Bordeaux, 33000
Morvan Hospital
Brest, 29609
University Hospital
Grenoble, 60107
Jeanne de Flandre Hospital
Lille, 59037
Mother and Child Hospital
Limoges, 87000
Women, Mother and Child Hospital
Lyon, 69500
La Timone Hospital
Marseille
University Hospital
Montpellier, 34295
Mother and Child Hospital
Nantes, 44000
Robert Debré Hospital
Paris, 75019
Trousseau Hospital
Paris, 75571
Necker Hospital
Paris, 75743
Anne de Bretagne University Hospital
Rennes, 35056
Purpan Children Hospital
Toulouse, 31059
Clocheville Hospital
Tours, 37044
About this study
Official title: Early Treatment With the Monoclonal C5 Antibody Eculizumab in Pediatric Patients Affected by Shiga-toxin Related Hemolytic and Uremic Syndrome: A Phase III Prospective Randomized Controlled Therapeutic Trial Versus Placebo
The investigators aim to perform the first controlled randomized prospective study using ECZ in pediatric STEC-HUS. This is of great interest as there is still no efficient specific therapy in that potentially devastating disease.
Furthermore, published data concerning the use of ECZ in STEC-HUS are controversial, reflecting statistical bias in retrospective or uncontrolled studies, thus emphasizing the need for prospective studies.
Read the detailed description
Hemolytic and uremic syndrome (HUS), characterized by thrombocytopenia, hemolytic anemia and acute renal failure (ARF), mainly affects children younger than 5 years old. Shiga-toxin (Stx) related HUS (STEC-HUS) is due to Stx secreting bacteria (mainly enterohemorrhagic Escherichia Coli strains). Acute phase of STEC-HUS is severe with at least 50% of affected children requiring dialysis, 20% presenting neurological involvement and 5% cardiac involvement. Mortality rates can reach 5% in pediatric series and long-term renal sequels have been reported in at least 30% of surviving patients. Apart from supportive care, no specific treatment (such as plasma exchange) has proven its efficacy in this life-threatening disease.
Recently, activation of the complement alternative pathway (CAP) has been demonstrated in STEC-HUS patients and experimental studies have highlighted that Stx induce CAP activation on human endothelial cells and platelet-leucocytes complexes, in addition to its direct cell toxicity inducing apoptosis, both processes ending up in microvasculature thrombosis. CAP activation has been demonstrated as the cause of atypical HUS (aHUS) and ECZ, a monoclonal C5 antibody, which inhibits the terminal complement complex (TCC) formation, can efficiently prevent evolution to end stage renal disease in aHUS patients. In 2011, Lapeyraque et al. reported its possible efficacy in 3 severe STEC-HUS pediatric patients. Nevertheless, in STEC-HUS, ECZ has only been used in uncontrolled studies, mostly during the 2011 German outbreak, with conflicting results. Considering the lack of therapy to prevent life-threatening complications and renal sequels in STEC-HUS and the logically expected efficacy of ECZ, controlled studies are mandatory. In recruiting centers, STEC-HUS patients with ARF will be proposed to enroll the trial with the exception of patients with multiorgan. After parental consent, patients will be randomized to receive either ECZ or a dextrose-based placebo in a single blinded fashion. According to the patient body weight, there will be 3 to 5 injections at day (D) 0, D7, D14, D21 and D28. According to the length of initial hospital stay, patients may have the remaining injections in the day ward of the recruiting center. Dosage of ECZ will be based on previous trials using ECZ in pediatric aHUS patients. We designed a single blinded study because patients treated with placebo who will develop severe multiorgan involvement will be switched to the ECZ arm. ECZ or placebo will be administrated intravenously as a 30-minute injection. In patients receiving hemodialysis, ECZ injection will be performed after a dialysis session. Before first injection, patients will receive a tetravalent meningococcal vaccine and an oral antibiotic prophylaxis to be continued up to 14 days after last injection.
This is a single blinded, phase III, randomized, multi-center controlled versus placebo clinical trial of eculizumab in STEC-HUS patients.
- Conditions
- Hemolytic Uremic Syndrome of Childhood
- Being tested
- Eculizumab, Placebo
- Keywords
- Eculizumab, Shiga-toxin, hemolytic and uremic syndrome, pediatric
- 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.
EculizumabDrug
Also called Soloris®
According to the patient body weight, there will be 3 to 5 injections at D0, D7, D14, D21 and D28. According to the length of initial hospital stay, patients may have the remaining injections in the day ward of the recruiting center. Dosage of ECZ will be based on previous trials using ECZ in pediatric aHUS patients.
ECZ or placebo will be administrated intravenously as a 30-minute injection
PlaceboDrug
Also called Solution with 5% glucose
According to the patient body weight, there will be 3 to 5 injections at D0, D7, D14, D21 and D28.
ECZ or placebo will be administrated intravenously as a 30-minute injection
| Study group | Role | What they receive |
|---|---|---|
| Eculizumab | Experimental | 300mg concentrate for solution for infusion. According to the patient body weight, there will be 3 to 5 injections administered in IV infusion at D0, D7, D14, D21 and D28.
Eculizumab (ECZ) will be administrated intravenously as a 30-minute injection. Receives: Drug: Eculizumab |
| Placebo | Placebo comparator | Infusion of a solution with 5% glucose. The administration scheme will be the same as the Eculizumab arm : there will be 3 to 5 injections at D0, D7, D14, D21 and D28.
Placebo will be administrated intravenously as a 30-minute injection. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
the duration in days of extrarenal epuration
Extrarenal epuration means peritoneal dialysis or hemodialysis, and is assessed at each visit.
Measured: From the inclusion date and assessed up to 13 months
Secondary outcomes (9)
Number of adverse events as a measure of Safety and tolerance of treatment injections (ECZ or placebo)
Measured: At each injection (treatment visits 2, 3, 4, 5, 6 at respectively day 0, 7, 14, 21, 28) and at each follow-up visit (7, 8, 9 respectively at month 2, 7, 13)
Adverse reactions related to the treatment (ECZ or placebo)
Measured: At each injection (treatment visits 2, 3, 4, 5, 6 at respectively day 0, 7, 14, 21, 28) and at each follow-up visit (7, 8, 9 respectively at month 2, 7, 13)
Duration of Acute Renal Failure (ARF)
Duration of ARF will be evaluated as follow : urine output measurement, creatinin clearance estimated with the Schwartz 2009 assay (based on creatinin plasma levels), ionogram, proteinuria.
Measured: Inclusion Visit (1 at day -3 to -1 ), treatment period (visits 2, 3, 4, 5, 6 respectively at day 0, 7, 14, 21, 28), follow-up period (visits 7, 8, 9 respectively at month 2, 7, 13)
Renal sequels
Renal sequels will be evaluated as follow : blood pressure, creatinin clearance, ionogram, proteinuria and microalbuminuria.
Measured: At 1, 6 and 12 months after last injection of ECZ (follow-up visits 7, 8, 9)
Hematological abnormalities
- Duration (in days) of the thrombocytopenia
- Duration (in days) of the hemolytic anemia
Measured: Inclusion Visit (1 at day -3 to -1), treatment period (visits 2, 3, 4, 5, 6 respectively at day 0, 7, 14, 21, 28), follow-up period (visits 7, 8, 9 respectively at month 2, 7, 13)
Blood parameters of Complement Alternative Pathway (CAP)
Blood parameters of CAP will be evaluated with plasmatic dosages of the complement components C3 and CD46.
Measured: Inclusion visit (1 at day -3 to -1), Treatment visits (3, 4, 5, 6 at day 7, 14, 21, 28), follow-up visit (7 at month 2)
Inhibition of the Terminal Complement Complex (TCC)
Inhibition of the TCC will be evaluated trough the CH50 assay and plasmatic free ECZ levels.
Measured: Inclusion visit (1 at day -3 to -1), Treatment visits (3, 4, 5, 6 at day 7, 14, 21, 28), follow-up visit (7 at month 2)
Incidence of extrarenal manifestations
- Neurological involvement (seizures, coma, focal deficit)
- Cardiac involvement (cardiac failure, ischemic myocarditis, conduction or rhythm troubles)
- Digestive involvement (pancreatitis, hepatitis, hemorrhagic colitis, bowel perforation, rectal prolapsus)
Measured: Inclusion Visit (1 at day -3 to -1), treatment period (visits 2, 3, 4, 5, 6 respectively at day 0, 7, 14, 21, 28)
Mortality
Measured: Inclusion Visit (1 at day -3 to -1), treatment period (visits 2, 3, 4, 5, 6 respectively at day 0, 7, 14, 21, 28), follow-up period (visits 7, 8, 9 respectively at month 2, 7, 13)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2014 (estimated)
- Study startJun 2015
- Primary completionJun 2018
- Study completionJun 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Hemolytic Uremic Syndrome of ChildhoodStudies of the same condition, open and under way.
- More trials run by University Hospital, ToulouseThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Arnaud Garnier, MD, Principal investigator, University Hospital, ToulouseKarine Brochard, Principal investigator, University Hospital, Toulouse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02205541
- Sponsor's protocol ID
- RC31/13/7052
- Other IDs
- PHRC 13-0060, 2014-001169-28, 2014
- Acronym
- ECULISHU
- Status last verified
- Apr 2026
- First submitted
- 28 Jul 2014
- Last updated
- 4 May 2026
- Results posted
- No
- Data monitoring committee
- No
Publications
Garnier A, Brochard K, Kwon T, Sellier-Leclerc AL, Lahoche A, Launay EA, Nobili F, Caillez M, Taque S, Harambat J, Michel-Bourdat G, Guigonis V, Fila M, Cloarec S, Djamal-Dine D, de Parscaux L, Allard L, Salomon R, Ulinski T, Fremeaux-Bacchi V, Morin C, Olivier-Abbal P, Colineaux H, Auriol F, Arnaud C, Kieffer I, Brusq C. Efficacy and Safety of Eculizumab in Pediatric Patients Affected by Shiga Toxin-Related Hemolytic and Uremic Syndrome: A Randomized, Placebo-Controlled Trial. J Am Soc Nephrol. 2023 Sep 1;34(9):1561-1573. doi: 10.1681/ASN.0000000000000182. Epub 2023 Jun 12.
PubMed 37303085Imdad A, Nelson JR, Tanner-Smith EE, Huang D, Gomez-Duarte OG. Interventions for preventing diarrhoea-associated haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2025 Apr 25;4(4):CD012997. doi: 10.1002/14651858.CD012997.pub3.
PubMed 40277027Groussolles M, Winer N, Sentilhes L, Biquart F, Massoud M, Vivanti AJ, Bouchghoul H, Rozenberg P, Olivier P, Desbriere R, Chauleur C, Perrotin F, Coatleven F, Fuchs F, Bretelle F, Tsatsaris V, Salomon LJ, Sananes N, Kayem G, Houflin-Debarge V, Schmitz T, Benoist G, Arnaud C, Ehlinger V, Vayssiere C; Groupe de Recherche en Gynecologie Obstetrique. Arabin pessary to prevent adverse perinatal outcomes in twin pregnancies with a short cervix: a multicenter randomized controlled trial (PESSARONE). Am J Obstet Gynecol. 2022 Aug;227(2):271.e1-271.e13. doi: 10.1016/j.ajog.2022.01.038. Epub 2022 Feb 3.
PubMed 35123930Imdad A, Mackoff SP, Urciuoli DM, Syed T, Tanner-Smith EE, Huang D, Gomez-Duarte OG. Interventions for preventing diarrhoea-associated haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2021 Jul 5;7(7):CD012997. doi: 10.1002/14651858.CD012997.pub2.
PubMed 34219224Matrat L, Bacchetta J, Ranchin B, Tanne C, Sellier-Leclerc AL. Pediatric atypical hemolytic-uremic syndrome due to auto-antibodies against factor H: is there an interest to combine eculizumab and mycophenolate mofetil? Pediatr Nephrol. 2021 Jun;36(6):1647-1650. doi: 10.1007/s00467-021-05025-8. Epub 2021 Mar 28.
PubMed 33774745Muff-Luett M, Sanderson KR, Engen RM, Zahr RS, Wenderfer SE, Tran CL, Sharma S, Cai Y, Ingraham S, Winnicki E, Weaver DJ, Hunley TE, Kiessling SG, Seamon M, Woroniecki R, Miyashita Y, Xiao N, Omoloja AA, Kizilbash SJ, Mansuri A, Kallash M, Yu Y, Sherman AK, Srivastava T, Nester CM. Eculizumab exposure in children and young adults: indications, practice patterns, and outcomes-a Pediatric Nephrology Research Consortium study. Pediatr Nephrol. 2021 Aug;36(8):2349-2360. doi: 10.1007/s00467-021-04965-5. Epub 2021 Mar 10.
PubMed 33693990
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Treatment With the Monoclonal C5 Antibody Eculizumab in Pediatric Patients Affected by Shiga-toxin Related Hemolytic and Uremic Syndrome: A Phase III Prospective Randomized Controlled Therapeutic Trial Versus Placebo
- ClinicalTrials.gov ID
- NCT02205541
- Acronym / sponsor ID
- ECULISHU / RC31/13/7052
- Conditions
- Hemolytic Uremic Syndrome of Childhood
- Keywords
- Eculizumab, Shiga-toxin, hemolytic and uremic syndrome, pediatric
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Study start
- Jun 2015 (actual)
- Primary completion
- Jun 2018 (actual)
- Study completion
- Jun 2018 (actual)
- First posted
- 31 Jul 2014 (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 investigators aim to perform the first controlled randomized prospective study using ECZ in pediatric STEC-HUS. This is of great interest as there is still no efficient specific therapy in that potentially devastating disease.
Furthermore, published data concerning the use of ECZ in STEC-HUS are controversial, reflecting statistical bias in retrospective or uncontrolled studies, thus emphasizing the need for prospective studies.
Detailed description
Hemolytic and uremic syndrome (HUS), characterized by thrombocytopenia, hemolytic anemia and acute renal failure (ARF), mainly affects children younger than 5 years old. Shiga-toxin (Stx) related HUS (STEC-HUS) is due to Stx secreting bacteria (mainly enterohemorrhagic Escherichia Coli strains). Acute phase of STEC-HUS is severe with at least 50% of affected children requiring dialysis, 20% presenting neurological involvement and 5% cardiac involvement. Mortality rates can reach 5% in pediatric series and long-term renal sequels have been reported in at least 30% of surviving patients. Apart from supportive care, no specific treatment (such as plasma exchange) has proven its efficacy in this life-threatening disease.
Recently, activation of the complement alternative pathway (CAP) has been demonstrated in STEC-HUS patients and experimental studies have highlighted that Stx induce CAP activation on human endothelial cells and platelet-leucocytes complexes, in addition to its direct cell toxicity inducing apoptosis, both processes ending up in microvasculature thrombosis. CAP activation has been demonstrated as the cause of atypical HUS (aHUS) and ECZ, a monoclonal C5 antibody, which inhibits the terminal complement complex (TCC) formation, can efficiently prevent evolution to end stage renal disease in aHUS patients. In 2011, Lapeyraque et al. reported its possible efficacy in 3 severe STEC-HUS pediatric patients. Nevertheless, in STEC-HUS, ECZ has only been used in uncontrolled studies, mostly during the 2011 German outbreak, with conflicting results. Considering the lack of therapy to prevent life-threatening complications and renal sequels in STEC-HUS and the logically expected efficacy of ECZ, controlled studies are mandatory. In recruiting centers, STEC-HUS patients with ARF will be proposed to enroll the trial with the exception of patients with multiorgan. After parental consent, patients will be randomized to receive either ECZ or a dextrose-based placebo in a single blinded fashion. According to the patient body weight, there will be 3 to 5 injections at day (D) 0, D7, D14, D21 and D28. According to the length of initial hospital stay, patients may have the remaining injections in the day ward of the recruiting center. Dosage of ECZ will be based on previous trials using ECZ in pediatric aHUS patients. We designed a single blinded study because patients treated with placebo who will develop severe multiorgan involvement will be switched to the ECZ arm. ECZ or placebo will be administrated intravenously as a 30-minute injection. In patients receiving hemodialysis, ECZ injection will be performed after a dialysis session. Before first injection, patients will receive a tetravalent meningococcal vaccine and an oral antibiotic prophylaxis to be continued up to 14 days after last injection.
This is a single blinded, phase III, randomized, multi-center controlled versus placebo clinical trial of eculizumab in STEC-HUS patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Eculizumab | Experimental | Drug: Eculizumab300mg concentrate for solution for infusion. According to the patient body weight, there will be 3 to 5 injections administered in IV infusion at D0, D7, D14, D21 and D28. Eculizumab (ECZ) will be administrated intravenously as a 30-minute injection. |
| Placebo | Placebo comparator | Drug: PlaceboInfusion of a solution with 5% glucose. The administration scheme will be the same as the Eculizumab arm : there will be 3 to 5 injections at D0, D7, D14, D21 and D28. Placebo will be administrated intravenously as a 30-minute injection. |
Who may qualify
- Ages
- 1 month to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Pediatric patient (1 month-18 years old)
- Affected by STEC-HUS defined by :
- Thrombocytopenia (<150 000/mm3)
- Mechanic hemolytic anemia (Hemoglobin < 10g/dL, haptoglobin <LLN, lactate dehydrogenase (LDH) >upper limit of normal (ULN) and/or bilirubin > ULN, presence of schizocytes)
- ARF defined by an estimated Schwartz 2009 creatinin clearance <75ml/min/1,73m²
Key exclusion criteria (14 listed)
- Neonatal HUS
- Malignancy
- Known HIV infection
- Pregnancy or lactation
- Identified drug exposure-related HUS
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 |
|---|---|---|
| EculizumabAlso: Soloris®According to the patient body weight, there will be 3 to 5 injections at D0, D7, D14, D21 and D28. According to the length of initial hospital stay, patients may have the remaining injections in the day ward of the recruiting center. Dosage of ECZ will be based on previous trials using ECZ in pediatric aHUS patients. ECZ or placebo will be administrated intravenously as a 30-minute injection | Drug | Eculizumab |
| PlaceboAlso: Solution with 5% glucoseAccording to the patient body weight, there will be 3 to 5 injections at D0, D7, D14, D21 and D28. ECZ or placebo will be administrated intravenously as a 30-minute injection | Drug | Placebo |
Primary outcome
the duration in days of extrarenal epuration
Time frame: From the inclusion date and assessed up to 13 months
Extrarenal epuration means peritoneal dialysis or hemodialysis, and is assessed at each visit.
9 secondary outcomes
Number of adverse events as a measure of Safety and tolerance of treatment injections (ECZ or placebo)
Time frame: At each injection (treatment visits 2, 3, 4, 5, 6 at respectively day 0, 7, 14, 21, 28) and at each follow-up visit (7, 8, 9 respectively at month 2, 7, 13)
Adverse reactions related to the treatment (ECZ or placebo)
Time frame: At each injection (treatment visits 2, 3, 4, 5, 6 at respectively day 0, 7, 14, 21, 28) and at each follow-up visit (7, 8, 9 respectively at month 2, 7, 13)
Duration of Acute Renal Failure (ARF)
Time frame: Inclusion Visit (1 at day -3 to -1 ), treatment period (visits 2, 3, 4, 5, 6 respectively at day 0, 7, 14, 21, 28), follow-up period (visits 7, 8, 9 respectively at month 2, 7, 13)
Renal sequels
Time frame: At 1, 6 and 12 months after last injection of ECZ (follow-up visits 7, 8, 9)
Hematological abnormalities
Time frame: Inclusion Visit (1 at day -3 to -1), treatment period (visits 2, 3, 4, 5, 6 respectively at day 0, 7, 14, 21, 28), follow-up period (visits 7, 8, 9 respectively at month 2, 7, 13)
Blood parameters of Complement Alternative Pathway (CAP)
Time frame: Inclusion visit (1 at day -3 to -1), Treatment visits (3, 4, 5, 6 at day 7, 14, 21, 28), follow-up visit (7 at month 2)
Inhibition of the Terminal Complement Complex (TCC)
Time frame: Inclusion visit (1 at day -3 to -1), Treatment visits (3, 4, 5, 6 at day 7, 14, 21, 28), follow-up visit (7 at month 2)
Incidence of extrarenal manifestations
Time frame: Inclusion Visit (1 at day -3 to -1), treatment period (visits 2, 3, 4, 5, 6 respectively at day 0, 7, 14, 21, 28)
Mortality
Time frame: Inclusion Visit (1 at day -3 to -1), treatment period (visits 2, 3, 4, 5, 6 respectively at day 0, 7, 14, 21, 28), follow-up period (visits 7, 8, 9 respectively at month 2, 7, 13)
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 4 May 2026.
- Results publication: PubMed 37303085Garnier A, Brochard K, Kwon T, Sellier-Leclerc AL, Lahoche A, Launay EA, Nobili F, Caillez M, Taque S, Harambat J, Michel-Bourdat G, Guigonis V, Fila M, Cloarec S, Djamal-Dine D, de Parscaux L, Allard L, Salomon R, Ulinski T, Fremeaux-Bacchi V, Morin C, Olivier-Abbal P, Colineaux H, Auriol F, Arnaud C, Kieffer I, Brusq C. Efficacy and Safety of Eculizumab in Pediatric Patients Affected by Shiga Toxin-Related Hemolytic and Uremic Syndrome: A Randomized, Placebo-Controlled Trial. J Am Soc Nephrol. 2023 Sep 1;34(9):1561-1573. doi: 10.1681/ASN.0000000000000182. Epub 2023 Jun 12.
- Derived from this study: PubMed 40277027Imdad A, Nelson JR, Tanner-Smith EE, Huang D, Gomez-Duarte OG. Interventions for preventing diarrhoea-associated haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2025 Apr 25;4(4):CD012997. doi: 10.1002/14651858.CD012997.pub3.
- Derived from this study: PubMed 35123930Groussolles M, Winer N, Sentilhes L, Biquart F, Massoud M, Vivanti AJ, Bouchghoul H, Rozenberg P, Olivier P, Desbriere R, Chauleur C, Perrotin F, Coatleven F, Fuchs F, Bretelle F, Tsatsaris V, Salomon LJ, Sananes N, Kayem G, Houflin-Debarge V, Schmitz T, Benoist G, Arnaud C, Ehlinger V, Vayssiere C; Groupe de Recherche en Gynecologie Obstetrique. Arabin pessary to prevent adverse perinatal outcomes in twin pregnancies with a short cervix: a multicenter randomized controlled trial (PESSARONE). Am J Obstet Gynecol. 2022 Aug;227(2):271.e1-271.e13. doi: 10.1016/j.ajog.2022.01.038. Epub 2022 Feb 3.
- Derived from this study: PubMed 34219224Imdad A, Mackoff SP, Urciuoli DM, Syed T, Tanner-Smith EE, Huang D, Gomez-Duarte OG. Interventions for preventing diarrhoea-associated haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2021 Jul 5;7(7):CD012997. doi: 10.1002/14651858.CD012997.pub2.
- Derived from this study: PubMed 33774745Matrat L, Bacchetta J, Ranchin B, Tanne C, Sellier-Leclerc AL. Pediatric atypical hemolytic-uremic syndrome due to auto-antibodies against factor H: is there an interest to combine eculizumab and mycophenolate mofetil? Pediatr Nephrol. 2021 Jun;36(6):1647-1650. doi: 10.1007/s00467-021-05025-8. Epub 2021 Mar 28.
- Derived from this study: PubMed 33693990Muff-Luett M, Sanderson KR, Engen RM, Zahr RS, Wenderfer SE, Tran CL, Sharma S, Cai Y, Ingraham S, Winnicki E, Weaver DJ, Hunley TE, Kiessling SG, Seamon M, Woroniecki R, Miyashita Y, Xiao N, Omoloja AA, Kizilbash SJ, Mansuri A, Kallash M, Yu Y, Sherman AK, Srivastava T, Nester CM. Eculizumab exposure in children and young adults: indications, practice patterns, and outcomes-a Pediatric Nephrology Research Consortium study. Pediatr Nephrol. 2021 Aug;36(8):2349-2360. doi: 10.1007/s00467-021-04965-5. Epub 2021 Mar 10.
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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.
Arnaud Garnier, MD principal investigator
University Hospital, Toulouse
Karine Brochard principal investigator
University Hospital, Toulouse
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Eculizumab in people with Hemolytic Uremic Syndrome of Childhood, run by University Hospital, Toulouse at 18 sites 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 8 inclusion and 14 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 4 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
the duration in days of extrarenal epuration (time frame: From the inclusion date and assessed up to 13 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02205541, which also lists 6 linked publications.
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 4 May 2026.