Study to evaluate if eculizumab is efficient and safe enough to be used for treatment of children with atypical hemolytic-uremic syndrome

Phase 2

Active, not recruiting

• Conditions: Renal and Urogenital - Kidney disease; Atypical Hemolytic-Uremic Syndrome

• Registration Number: ACTRN12611000461998
• Lead Sponsor: Alexion Pharmaceuticals Australasia Pty Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2011-05-04
• Last Update Posted: 13 January 2020

Recruitment & Eligibility

• Status: Active, not recruiting
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

Signed informed consent.
Patients from 1 month to 18 years diagnosed with aHUS

Exclusion Criteria

Typical hemolytic-uremic syndrome (HUS), malignancy within 5 years of screening, HUS related to infection or bone marrow transplant or vitamin B12 deficiency, systemic lupus erythematosus (SLE), meningococcal/pneumococcal disease history.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Primary Safety Endpoint: safety assesments are based on routine physical examinations, vitals signs, AEs, lab data and ECGs.[Endpoints will be assessed after 26 weeks treatment for each patient.];Primary Efficacy Endpoint: proportion of patients with complete TMA response as evidenced by normalisation of hematological parameters and improvement in serum creatinine from baseline.[Endpoints will be assessed after 26 weeks treatment for each patient.]

Secondary Outcome Measures

Name	Time	Method
duration of complete TMA response[All endpoints will be assessed after 26 weeks treatment.];time to complete TMA response[All endpoints will be assessed after 26 weeks treatment.];assessment of hematologic response.[All endpoints will be assessed after 26 weeks treatment.]