Traitement précoce de patients pédiatriques présentant un syndrome hémolytique et urémique typique par l’anticorps monoclonal anti-C5 eculizumab : essai thérapeutique prospectif randomisé contrôlé contre placebo de phase III

Phase 1

• Conditions: Hemolytic and uremic syndrome; MedDRA version: 17.0 Level: LLT Classification code 10042814 Term: Syndrome hemolytic uremic System Organ Class: 100000004851; Therapeutic area: Diseases [C] - Symptoms and general pathology [C23]

• Registration Number: EUCTR2014-001169-28-FR
• Lead Sponsor: Centre Hospitalier de Toulouse

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-07-31
• Last Update Posted: 1 February 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 100

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 ULN and/or bilirubine >ULN, presence of schizocytes)
- ARF defined by an estimated Schwartz 2009 creatinin clairance <75ml/min/1,73m²
- With prodromal diarrhea and/or presence of an enterohemorragic strain of Escherichia Coli and/or identification of the Stx 1 or 2 genes in the stool sample or rectal swab

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

. Neonatal HUS
. Malignancy
. Known HIV infection
. Pregnancy or lactation
. Identified drug exposure-related HUS
. Infection-related HUS
. Known systemic lupus erythematosus or antiphospholid 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)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified