Clinical Trial: Single Arm Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title:
Brief Summary: The purpose of the study is to assess the efficacy of ALXN1210 to control disease activity in adolescent and adult patients with aHUS who have not previously used a complement inhibitor.
Detailed Summary:
Sponsor: Alexion Pharmaceuticals
Current Primary Outcome: Complete TMA response [ Time Frame: 26 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Dialysis requirement status [ Time Frame: 26 weeks ]
- Time to Complete TMA Response [ Time Frame: 26 weeks ]
- Complete TMA Response status over time [ Time Frame: 26 weeks ]
- Observed value and change from baseline in estimated glomerular filtration rate (eGFR) [ Time Frame: 26 weeks ]
- Change from baseline in chronic kidney disease (CKD) stage [ Time Frame: 26 weeks ]
- Change from baseline in hematologic parameters (platelets, LDH, hemoglobin) [ Time Frame: 26 weeks ]
- Increase in hemoglobin ≥ 20g/L from baseline [ Time Frame: 26 weeks ]
- Change from baseline in quality of life, as measured by the EQ-5D-3L (all patients) [ Time Frame: 26 weeks ]
- Change from baseline in quality of life, as measured by the FACIT Fatigue Version 4 questionnaire (patients ≥ 18 years of age) [ Time Frame: 26 weeks ]
- Change from baseline in quality of life, as measured by the Pediatric FACIT Fatigue questionnaire (patients 12 to < 18 years of age) [ Time Frame: 26 weeks ]
Original Secondary Outcome: Same as current
Information By: Alexion Pharmaceuticals
Dates:
Date Received: October 25, 2016
Date Started: November 2016
Date Completion: December 2019
Last Updated: May 19, 2017
Last Verified: May 2017