Clinical Trial: Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (aHUS)

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

Official Title: An Open-label, Multi-center Controlled Clinical Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (AHUS)

Brief Summary: The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of adult patients with plasma therapy-sensitive Atypical Hemolytic-Uremic Syndrome (aHUS).

Detailed Summary:
Sponsor: Alexion Pharmaceuticals

Current Primary Outcome:

• Percentage of Patients With TMA Event-free Status [ Time Frame: Through 26 weeks ]
  TMA Event-free status is defined as the absence for at least 12 weeks of [1] decrease in platelet count of > 25% from the Platelet Count Pre-PT Baseline Set Point; [2] PT while the patient is receiving eculizumab, and [3] new dialysis.
• Percentage of Patients With Hematologic Normalization [ Time Frame: Through 26 weeks ]
  Hematologic Normalization was defined as normalization of both platelet count and lactic dehydrogenase (LDH) sustained for at least two consecutive measurements which spanned a period of at least four weeks.
• Percentage of Patients With Complete TMA Response [ Time Frame: Through 26 weeks ]
  The proportion of patients who achieved a Complete TMA Response from baseline through 26 weeks of treatment with eculizumab was determined. Complete TMA Response was defined as Hematologic Normalization plus improvement in renal function (defined as (≥25% reduction from baseline in serum creatinine), which was sustained for two consecutive measurements over a period of at least four weeks.

Original Primary Outcome: Assess the effect of eculizumab on thrombotic microangiopathy (TMA). [ Time Frame: Through 26 weeks ]

Current Secondary Outcome:

• TMA Intervention Rate [ Time Frame: Through 26 weeks ]
  TMA Intervention Rate (# PE/PI and # Dialysis Events/Patient/Day) in the eculizumab treatment period (from baseline through 26 weeks) for PE/PI and (from the fifteenth day following the first eculizumab dose through 26 weeks) for new dialysis events was compared with the TMA Intervention Rate during the pre-eculizumab treatment period.
• Platelet Count Change From Baseline to 26 Weeks [ Time Frame: From Baseline to 26 Weeks ]
• Percentage of Patients With Platelet Count Normalization [ Time Frame: Through 26 Weeks ]
  Platelet count normalization was defined as the platelet count observed to be ≥150 x 10^9/L on at least two consecutive measurements which span a period of at least four weeks
• Percentage of Patients With TMA Event-free Status [ Time Frame: Through End of Study, Median Exposure 156 Weeks ]
  TMA Event-free status is defined as the absence for at least 12 weeks of [1] decrease in platelet count of > 25% from the Platelet Count Pre-PT Baseline Set Point; [2] PT while the patient is receiving eculizumab, and [3] new dialysis.
• Percentage of Patients With Hematologic Normalization [ Time Frame: Through End of Study, Median Exposure 156 Weeks ]
  Hematologic Normalization was defined as normalization of both platelet count and lactic dehydrogenase (LDH) sustained for at least two consecutive measurements which spanned a period of at least four weeks.
• Percentage of Patients With Complete TMA Response [ Time Frame: Through End of Study, Median Exposure 156 Weeks ]
  The proportion of patients who achieved a Complete TMA Response from baseline through end of study with eculizumab was determined. Complete TMA Response was defined as Hematologic Normalization plus improvement in renal function (defined as (≥25% reduction from baseline in serum creatinine), which was sustained for two consecutive measurements over a period of at least four weeks.
• TMA Intervention Rate [ Time Frame: Through End of Study, Median Exposure 156 Weeks ]
  TMA Intervention Rate (# PE/PI and # Dialysis Events/Patient/Day) in the eculizumab treatment period (from baseline through end of study) for PE/PI and (from the fifteenth day following the first eculizumab dose through end of study) for new dialysis events was compared with the TMA Intervention Rate during the pre-eculizumab treatment period.
• Platelet Count Change From Baseline to 156 Weeks [ Time Frame: From Baseline to 156 Weeks ]
• Percentage of Patients With Platelet Count Normalization [ Time Frame: Through End of Study, Median Exposure 156 Weeks ]
  Platelet count normalization was defined as the platelet count observed to be ≥150 x 10^9/L on at least two consecutive measurements which span a period of at least four weeks Not specified.
• Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration [ Time Frame: Induction Phase for 4 weeks followed by Maintenance Phase starting on Week 5 through 26 weeks or longer. ]

Original Secondary Outcome:

• Additional efficacy endpoints related to manifestations of TMA. [ Time Frame: Through 26 weeks ]
• Overall safety and tolerability of eculizumab [ Time Frame: Through 26 weeks ]
• Pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in patients with aHUS. [ Time Frame: Through 26 weeks ]

Dates:
Date Received: February 3, 2009
Date Started: July 2009
Date Completion:
Last Updated: June 30, 2015
Last Verified: June 2015