Clinical Trial: Eval. of Potential Predictors of Disease Progression in Patients With aHUS Including Genetics, Biomarkers and Treatment

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

Official Title: Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment

Brief Summary: This is a prospective, open-label study with no patient randomization. Treatment for atypical hemolytic uremic syndrome (aHUS) will remain observational and at the discretion of the treating physician. The purpose of this study is to assess disease manifestations of complement mediated thrombotic microangiopathy (TMA) and potential clinical predictors of disease manifestations and progression in patients with aHUS with or without eculizumab treatment in the clinical setting.

Detailed Summary:
Sponsor: Alexion Pharmaceuticals

Current Primary Outcome: Rate of TMA manifestations during eculizumab treatment compared to off- treatment [ Time Frame: Up to 47 Months ]

TMA is defined as one of following:

  1. Hematologic or renal events due to aHUS
  2. Extra-renal clinical signs and symptoms of aHUS
  3. Tissue (eg, kidney transplant) biopsy demonstrating TMA


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in estimated Glomerular Filtration Rate (eGFR) over time [ Time Frame: Up to 47 Months ]
    Change in estimated Glomerular Filtration Rate (eGFR) over time using the chronic kidney disease-epidemiology (CKD-EPI) formula
  • Rate of TMA manifestations in subsets of patients [ Time Frame: Up to 47 Months ]
    1. While patient is on a labeled eculizumab dosing regimen
    2. While patient is on a non-labeled eculizumab dosing regimen (e.g. reduced dose and or reduced frequency)
  • Incidence of Plasma Exchange and Plasma Infusion (PE/PI) [ Time Frame: Up to 47 Months ]


Original Secondary Outcome: Same as current

Information By: Alexion Pharmaceuticals

Dates:
Date Received: November 24, 2015
Date Started: November 2015
Date Completion: April 2020
Last Updated: January 11, 2017
Last Verified: January 2017