Clinical Trial: An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric 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 Pediatric Patients With Atypical Hemolytic-Uremic Syndrome

Brief Summary: The primary purpose is to assess the efficacy and safety of eculizumab in pediatric 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 ]

Original Primary Outcome: Study endpoints will be assessed at every dosing visit on an ongoing basis during and at the end of the Treatment Period. An evaluation of these parameters will also occur at every dosing visit during and at the end of the extension treatment period. [ Time Frame: 6 month enrollment, 6 to 24 (or more) months treatment and extension period respectively. ]

Current Secondary Outcome:

• Proportion of Patients With Complete Hematologic Response [ Time Frame: Through 26 weeks ]
  Proportion of Patients with Complete Hematologic response through 26 weeks 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 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 55 Weeks ]
  Proportion of Patients with Complete TMA response was determined and defined by normalization of hematological parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline which was 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 55 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 End of Study, Median Exposure 55 Weeks ]
  Proportion of Patients with Platelet Count Normalization through end of study 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 55 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 ]
• Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3 [ Time Frame: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort ]
• Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7 [ Time Frame: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort ]
• Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg) [ Time Frame: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort ]
• Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1 [ Time Frame: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort ]
• Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5 [ Time Frame: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort ]

Original Secondary Outcome:

Information By: Alexion Pharmaceuticals

Dates:
Date Received: August 31, 2010
Date Started: September 2010
Date Completion:
Last Updated: April 13, 2015
Last Verified: April 2015