Status:

COMPLETED

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

Lead Sponsor:

University Hospital, Toulouse

Conditions:

Hemolytic Uremic Syndrome of Childhood

Eligibility:

All Genders

1-18 years

Phase:

PHASE3

Brief 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 po...

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 ...

Eligibility Criteria

Inclusion

  • 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

  • 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)

Key Trial Info

Start Date :

June 1 2015

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

June 1 2018

Estimated Enrollment :

100 Patients enrolled

Trial Details

Trial ID

NCT02205541

Start Date

June 1 2015

End Date

June 1 2018

Last Update

July 12 2019

Active Locations (18)

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Page 1 of 5 (18 locations)

1

University Hospital

Amiens, France, 80054

2

University Hospital

Angers, France, 49100

3

University Hospital

Besançon, France, 25030

4

Pellegrin Hospital

Bordeaux, France, 33000