- Conditions
- Pediatric patients with Atypical Hemolytic-Uremic Syndrome (aHUS)MedDRA version: 14.0Level: LLTClassification code 10019515Term: Hemolytic uremic syndromeSystem Organ Class: 10005329 - Blood and lymphatic system disordersTherapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2010-020310-28-BE
- Lead Sponsor
- ALEXION PHARMACEUTICALS, INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 15
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
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.;Timepoint(s) of evaluation of this end point: Study endpoints will be assessed at every dosing visit on an ongoing basis during and at the end of the Treatment Period. An evaluation of these parameters will also occur at every dosing visit during and at the end of the extension treatment period
- Secondary Outcome Measures
Name Time Method Secondary end point(s): -;Timepoint(s) of evaluation of this end point: -