Clinical Trial: Study of ALXN1210 in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional
Official Title: A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
Brief Summary: The purpose of the study is to assess the efficacy of ALXN1210 to control disease activity in children and adolescents 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 ]The proportion of patients who do not require dialysis, among those who had received dialysis within 56 days prior to study drug treatment initiation
- Time to Complete TMA response [ Time Frame: 26 weeks ]Time from start of treatment to achievement of complete TMA response, defined as normalization of hematological parameters and ≥ 25% improvement in serum creatinine from Baseline
- Complete TMA Response status over time [ Time Frame: 26 weeks ]Proportion of patients achieving complete TMA response at each time point.
- Observed value and change from baseline in estimated glomerular filtration rate (eGFR) [ Time Frame: 26 weeks ]Observed value and change from baseline in eGFR
- Change from baseline in chronic kidney disease (CKD) stage [ Time Frame: 26 weeks ]The number and proportion of patients with improvement, worsening, and no change in CKD stage compared to baseline.
- Change from baseline in hematologic parameters (platelets, lactate dehydrogenase (LDH), hemoglobin) [ Time Frame: 26 weeks ]Observed value and change from baseline in platelets, LDH, and hemoglobin.
- Increase in hemoglobin of ≥ 20 g/L from baseline [ Time Frame: 26 weeks ]A proportion of patients with an increase in hemoglobin ≥ 20 g/L from baseline to Day 183
- Change from baseline in quality of life, as measured by Pediatric Functional Assessment of Chronic Therapy (FACIT) Fatigue questionnaire (patients ≥ 5 years of age) [ Time Frame: 26 weeks ]Change in pediatric FACIT-Fatigue scores from baseline to Day 183
Original Secondary Outcome: Same as current
Information By: Alexion Pharmaceuticals
Dates:
Date Received: April 24, 2017
Date Started: May 2017
Date Completion: October 2020
Last Updated: April 24, 2017
Last Verified: April 2017