Clinical Trial: An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome

Brief Summary: The record Primary purpose is to assess the efficacy of eculizumab in adult patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.

Detailed Summary:
Sponsor: Alexion Pharmaceuticals

Current Primary Outcome:

  • Proportion of Patients With Complete TMA Response [ Time Frame: Through 26 weeks ]
    Proportion of Patients with Complete TMA response was determined and defined by normalization of hematological parameters (platelet count and LDH) and preservation of renal function (defined as < 25% increase in serum creatinine from baseline) which were sustained for at least two consecutive measurements obtained at least four weeks apart.
  • Proportion of Patients With Modified Complete TMA Response [ Time Frame: Through 26 weeks ]
    Proportion of Patients with Modified Complete TMA response through 26 weeks of treatment was determined and defined by normalization of hematological parameters (platelet count and LDH) and improvement in renal function (defined as ≥ 25% reduction from the baseline value in serum creatinine, which were sustained for at least two consecutive measurements obtained at least four weeks apart.


Original Primary Outcome: Proportion of patients with complete TMA response. [ Time Frame: includes 26 weeks for the primary endpoint analysis with a possibility of continued treatment if patient continues to benefit from treatment for up to 2 years or until marketing approval for this indication. ]

Current Secondary Outcome:

  • Proportion of Patients With Complete Hematologic Response [ Time Frame: Through 26 weeks ]
    Proportion of Patients with Complete Hematologic response through end of study was determined and defined by normalization of platelet count and LDH sustained for at least two consecutive measurements obtained at least four weeks apart.
  • Proportion of Patients With Platelet Count Normalization [ Time Frame: Through 26 weeks ]
    Proportion of Patients with Platelet Count Normalization through 26 weeks of treatment was determined and defined as the platelet count observed to be ≥ 150 x 109/L on at least two consecutive measurements which span a period of at least four weeks
  • Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement [ Time Frame: Through 26 weeks ]
    Proportion of Patients with Estimated Glomerular Filtration Rate (eGFR) Improvement was determined and defined as an increase in eGFR by ≥ 15 mL/min/1.73m2 from baseline, sustained for at least two consecutive measurements obtained at least four weeks apart.
  • Platelet Count Change From Baseline to 26 Weeks [ Time Frame: Through 26 weeks ]
  • Proportion of Patients With Complete TMA Response [ Time Frame: Through End of Study, Median Exposure 52 Weeks ]
    Proportion of Patients with Complete TMA response through end of study was determined and defined by normalization of hematological parameters (platelet count and LDH) and preservation of renal function (defined as < 25% increase in serum creatinine from baseline) which were sustained for at least two consecutive measurements obtained at least four weeks apart.
  • Proportion of Patients With Modified Complete TMA Response [ Time Frame: Through End of Study, Median Exposure 52 Weeks ]
    Proportion of Patients with Modified Complete TMA response through end of study was determined and defined by normalization of hematological parameters (platelet count and LDH) and improvement in renal function (defined as ≥ 25% reduction from the baseline value in serum creatinine, which were sustained for at least two consecutive measurements obtained at least four weeks apart.
  • Proportion of Patients With Complete Hematologic Response [ Time Frame: Through End of Study, Median Exposure 52 Weeks ]
    Proportion of Patients with Complete Hematologic response through end of study of treatment was determined and defined by normalization of platelet count and LDH sustained for at least two consecutive measurements obtained at least four weeks apart.
  • Proportion of Patients With Platelet Count Normalization [ Time Frame: Through End of Study, Median Exposure 52 Weeks ]
    Proportion of Patients with Platelet Count Normalization through end of study of treatment was determined and defined as the platelet count observed to be ≥ 150 x 109/L on at least two consecutive measurements which span a period of at least four weeks
  • Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement [ Time Frame: Through End of Study, Median Exposure 52 Weeks ]
    Proportion of Patients with Estimated Glomerular Filtration Rate (eGFR) Improvement was determined and defined as an increase in eGFR by ≥ 15 mL/min/1.73m2 from baseline sustained for at least two consecutive measurements obtained at least four weeks apart
  • Platelet Count Change From Baseline to 52 Weeks [ Time Frame: Through 52 Weeks ]


Original Secondary Outcome:

Information By: Alexion Pharmaceuticals

Dates:
Date Received: August 31, 2010
Date Started: July 2010
Date Completion:
Last Updated: April 7, 2015
Last Verified: April 2015