Eculizumab in Shiga-toxin Related Hemolytic and Uremic Syndrome Pediatric Patients - ECULISHU (ECULISHU)
Hemolytic Uremic Syndrome of Childhood
About this trial
This is an interventional treatment trial for Hemolytic Uremic Syndrome of Childhood focused on measuring Eculizumab, Shiga-toxin, hemolytic and uremic syndrome, pediatric
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- Neonatal HUS
- Malignancy
- Known HIV infection
- Pregnancy or lactation
- Identified drug exposure-related HUS
- Infection-related HUS
- 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)
Sites / Locations
- University Hospital
- University Hospital
- University Hospital
- Pellegrin Hospital
- Morvan Hospital
- University Hospital
- Jeanne de Flandre Hospital
- Mother and Child Hospital
- Women, Mother and Child Hospital
- La Timone Hospital
- University Hospital
- Mother and Child Hospital
- Robert Debré Hospital
- Trousseau Hospital
- Necker Hospital
- Anne de Bretagne University Hospital
- Purpan Children Hospital
- Clocheville Hospital
Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Eculizumab
Placebo
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.
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.