AN OPEN-LABEL, MULTI-CENTER CLINICAL TRIAL OF ECULIZUMAB IN PEDIATRIC PATIENTS WITH ATYPICAL HEMOLYTIC-UREMIC SYNDROME (aHUS)

Conditions: Pediatric patients with Atypical Hemolytic-Uremic Syndrome (aHUS)
MedDRA version: 14.1Level: LLTClassification code 10019515Term: Hemolytic uremic syndromeSystem Organ Class: 10005329 - Blood and lymphatic system disorders

Registration Number: EUCTR2010-020310-28-DE

Lead Sponsor: ALEXION PHARMACEUTICALS, INC.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 20

Inclusion Criteria

1. Patient’s parent/legal guardian must be willing and able to give written informed
consent and the patient must be willing to give written informed assent [if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees (IRB/IEC)].
2. Pediatric patients with aHUS. Patients may be newly diagnosed, or with previously
diagnosed disease, or post-kidney transplant with the disease.
3. Patients from 1 month up to 18 years of age and body weight = 5 kg.
4. Patient exhibit Thrombocytopenia, hemolysis and elevated Serum Creatinine
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

1. Plasma Therapy for > 5 weeks prior to enrollment.
2. Chronic dialysis
3. Prior eculizumab use or hypersensitivity to eculizumab, to murine proteins or to one of the excipients.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.;Secondary Objective: Efficacy;Primary end point(s): Primary Efficacy Endpoint:<br>Proportion of patients with complete TMA response.<br><br>Primary Safety Endpoint:<br>Safety assessments.

Secondary Outcome Measures