Hemolytic Uremic Syndrome of Childhood Clinical Trial
— ECULISHUOfficial 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
Verified date | July 2019 |
Source | University Hospital, Toulouse |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 100 |
Est. completion date | June 2018 |
Est. primary completion date | June 2018 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Month to 18 Years |
Eligibility |
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) |
Country | Name | City | State |
---|---|---|---|
France | University Hospital | Amiens | |
France | University Hospital | Angers | |
France | University Hospital | Besançon | |
France | Pellegrin Hospital | Bordeaux | |
France | Morvan Hospital | Brest | |
France | University Hospital | Grenoble | |
France | Jeanne de Flandre Hospital | Lille | |
France | Mother and Child Hospital | Limoges | |
France | Women, Mother and Child Hospital | Lyon | |
France | La Timone Hospital | Marseille | |
France | University Hospital | Montpellier | |
France | Mother and Child Hospital | Nantes | |
France | Necker Hospital | Paris | |
France | Robert Debré Hospital | Paris | |
France | Trousseau Hospital | Paris | |
France | Anne de Bretagne University Hospital | Rennes | |
France | Purpan Children Hospital | Toulouse | |
France | Clocheville Hospital | Tours |
Lead Sponsor | Collaborator |
---|---|
University Hospital, Toulouse |
France,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | the duration in days of extrarenal epuration | Extrarenal epuration means peritoneal dialysis or hemodialysis, and is assessed at each visit. | From the inclusion date and assessed up to 13 months | |
Secondary | Number of adverse events as a measure of Safety and tolerance of treatment injections (ECZ or placebo) | 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) | ||
Secondary | Adverse reactions related to the treatment (ECZ or placebo) | 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) | ||
Secondary | 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. | 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) | |
Secondary | Renal sequels | Renal sequels will be evaluated as follow : blood pressure, creatinin clearance, ionogram, proteinuria and microalbuminuria. | At 1, 6 and 12 months after last injection of ECZ (follow-up visits 7, 8, 9) | |
Secondary | Hematological abnormalities | Duration (in days) of the thrombocytopenia Duration (in days) of the hemolytic anemia |
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) | |
Secondary | Blood parameters of Complement Alternative Pathway (CAP) | Blood parameters of CAP will be evaluated with plasmatic dosages of the complement components C3 and CD46. | 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) | |
Secondary | Inhibition of the Terminal Complement Complex (TCC) | Inhibition of the TCC will be evaluated trough the CH50 assay and plasmatic free ECZ levels. | 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) | |
Secondary | 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) |
Inclusion Visit (1 at day -3 to -1), treatment period (visits 2, 3, 4, 5, 6 respectively at day 0, 7, 14, 21, 28) | |
Secondary | Mortality | 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) |
Status | Clinical Trial | Phase | |
---|---|---|---|
Not yet recruiting |
NCT03690024 -
Outcame of Cases With Hemolytic Uremic Syndrome Attending Assiut University Child Hospital
|