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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00838513
Other study ID # C08-003A
Secondary ID BB-IND 11075Eudr
Status Completed
Phase Phase 2
First received February 3, 2009
Last updated June 30, 2015
Start date July 2009
Est. completion date December 2013

Study information

Verified date June 2015
Source Alexion Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug AdministrationCanada: Health CanadaEuropean Union: European Medicines Agency
Study type Interventional

Clinical Trial 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).


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Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Drug:
eculizumab
All patients received open-label eculizumab administered intravenously on the following dose schedule: Induction dose - 900 mg per week for four weeks and a dose of 1200 mg one week later; Maintenance dose - 1200 mg every two weeks. Patients who received plasma exchange or infusion during the eculizumab treatment period received a supplemental dose of 600 mg within one hour before plasma infusion or within one hour after the completion of each plasma exchange.

Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Alexion Pharmaceuticals

Countries where clinical trial is conducted

Canada,  France,  Germany,  Italy,  Netherlands,  Sweden,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Percentage of Patients With TMA Event-free Status 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. Through 26 weeks No
Primary Percentage of Patients With Hematologic Normalization 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. Through 26 weeks No
Primary Percentage of Patients With Complete TMA Response 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. Through 26 weeks No
Secondary TMA Intervention Rate 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. Through 26 weeks No
Secondary Platelet Count Change From Baseline to 26 Weeks From Baseline to 26 Weeks No
Secondary Percentage of Patients With Platelet Count Normalization 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 Through 26 Weeks No
Secondary Percentage of Patients With TMA Event-free Status 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. Through End of Study, Median Exposure 156 Weeks No
Secondary Percentage of Patients With Hematologic Normalization 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. Through End of Study, Median Exposure 156 Weeks No
Secondary Percentage of Patients With Complete TMA Response 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. Through End of Study, Median Exposure 156 Weeks No
Secondary TMA Intervention Rate 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. Through End of Study, Median Exposure 156 Weeks No
Secondary Platelet Count Change From Baseline to 156 Weeks From Baseline to 156 Weeks No
Secondary Percentage of Patients With Platelet Count Normalization 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. Through End of Study, Median Exposure 156 Weeks No
Secondary Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration Induction Phase for 4 weeks followed by Maintenance Phase starting on Week 5 through 26 weeks or longer. No
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