- 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-ES
- Lead Sponsor
- ALEXION PHARMACEUTICALS, INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 22
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: 15
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: -
- Secondary Outcome Measures
Name Time Method Secondary end point(s): -;Timepoint(s) of evaluation of this end point: -