An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome
- Registration Number
- NCT01194973
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Brief Summary
The record Primary purpose is to assess the efficacy of eculizumab in adult patients with Atypical Hemolytic- Uremic Syndrome (aHUS) to control Thrombotic Microangiopathy (TMA) as characterized by thrombocytopenia, hemolysis and renal impairment.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 44
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Eculizumab Eculizumab -
- Primary Outcome Measures
Name Time Method Percentage of Patients With Modified Complete TMA Response 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.
Percentage of Patients With Complete TMA Response 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.
- Secondary Outcome Measures
Name Time Method Percentage of Patients With Complete Hematologic Response 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.
Percentage of Patients With Platelet Count Normalization 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
Percentage of Patients With Modified Complete TMA Response 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.
Platelet Count Change From Baseline to 52 Weeks Through 52 Weeks Percentage of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement 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/1.73m\^2 from baseline sustained for at least two consecutive measurements obtained at least four weeks apart
Platelet Count Change From Baseline to 26 Weeks Through 26 weeks Percentage of Patients With Complete TMA Response 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.