An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Enrollment
- 44
- Primary Endpoint
- Percentage of Patients With Modified Complete TMA Response
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Eculizumab
Intervention: Eculizumab (Drug)
Outcomes
Primary Outcomes
Percentage 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.
Percentage 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.
Secondary Outcomes
- Percentage of Patients With Complete Hematologic Response(Through End of Study, Median Exposure 52 Weeks)
- Percentage of Patients With Platelet Count Normalization(Through End of Study, Median Exposure 52 Weeks)
- Percentage of Patients With Modified Complete TMA Response(Through End of Study, Median Exposure 52 Weeks)
- 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)
- 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)