Eculizumab in Shiga-toxin Related Hemolytic and Uremic Syndrome Pediatric Patients - ECULISHU

• Status: Completed
• Trial end date: 2018-06-01
• Target enrollment: 0
• Participant gender: All

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.

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University Hospital, Toulouse
• Treatments: Eculizumab; Pharmaceutical Solutions; Shiga Toxins

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, haptoglobindehydrogenase (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)