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 ]

The proportion of patients who achieved complete thrombotic microangiopathy (TMA) response as assessed by normalization of hematological parameters between Baseline and Day 183


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