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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00838513
First received: February 3, 2009
Last updated: June 30, 2015
Last verified: June 2015
Results First Received: December 15, 2014  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Atypical Hemolytic Uremic Syndrome
Intervention: Drug: eculizumab

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
C08-003A/B combined 2 studies: one for adults (C08-003A, N=15) and one for adolescents (C08-003B, N=5) Please refer to NCT00844428 for combined studies with enrollment number corresponding to each individual study

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Patients receiving PT for aHUS and observed to receive ≥ 1 PT every two weeks, and no more than 3 PT treatments/week for at least 8 weeks before the first dose of eculizumab. Patients who met the eligibility criteria during the Observation Period were enrolled into the Treatment Period which commenced with the first eculizumab dose.

Reporting Groups
  Description
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.

Participant Flow for 5 periods

Period 1:   Screening Period
    Eculizumab  
STARTED     23  
COMPLETED     20  
NOT COMPLETED     3  
Screen Failure                 3  

Period 2:   Observation Period (8 Weeks)
    Eculizumab  
STARTED     20  
COMPLETED     20  
NOT COMPLETED     0  

Period 3:   Treatment Period (26 Weeks)
    Eculizumab  
STARTED     20  
COMPLETED     20  
NOT COMPLETED     0  

Period 4:   Extension Treatment Period
    Eculizumab  
STARTED     19  
COMPLETED     18  
NOT COMPLETED     1  
Death                 1  

Period 5:   Post Treatment Period (Patients Who Disc
    Eculizumab  
STARTED     5  
COMPLETED     4  
NOT COMPLETED     1  
Death                 1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The intent-to-treat (ITT) population was defined as all patients who received any amount of eculizumab, and were considered evaluable for safety and efficacy analyses. For both the efficacy and safety analyses, all 20 patients who were treated with study drug were included in the ITT population.

Reporting Groups
  Description
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.

Baseline Measures
    Eculizumab  
Number of Participants  
[units: participants]
  20  
Age  
[units: Years]
Mean (Standard Deviation)
  32.3  (14.92)  
Gender  
[units: participants]
 
Female     12  
Male     8  
Race (NIH/OMB)  
[units: participants]
 
American Indian or Alaska Native     0  
Asian     0  
Native Hawaiian or Other Pacific Islander     0  
Black or African American     2  
White     18  
More than one race     0  
Unknown or Not Reported     0  



  Outcome Measures
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1.  Primary:   Percentage of Patients With TMA Event-free Status   [ Time Frame: Through 26 weeks ]

2.  Primary:   Percentage of Patients With Hematologic Normalization   [ Time Frame: Through 26 weeks ]

3.  Primary:   Percentage of Patients With Complete TMA Response   [ Time Frame: Through 26 weeks ]

4.  Secondary:   TMA Intervention Rate   [ Time Frame: Through 26 weeks ]

5.  Secondary:   Platelet Count Change From Baseline to 26 Weeks   [ Time Frame: From Baseline to 26 Weeks ]

6.  Secondary:   Percentage of Patients With Platelet Count Normalization   [ Time Frame: Through 26 Weeks ]

7.  Secondary:   Percentage of Patients With TMA Event-free Status   [ Time Frame: Through End of Study, Median Exposure 156 Weeks ]

8.  Secondary:   Percentage of Patients With Hematologic Normalization   [ Time Frame: Through End of Study, Median Exposure 156 Weeks ]

9.  Secondary:   Percentage of Patients With Complete TMA Response   [ Time Frame: Through End of Study, Median Exposure 156 Weeks ]

10.  Secondary:   TMA Intervention Rate   [ Time Frame: Through End of Study, Median Exposure 156 Weeks ]

11.  Secondary:   Platelet Count Change From Baseline to 156 Weeks   [ Time Frame: From Baseline to 156 Weeks ]

12.  Secondary:   Percentage of Patients With Platelet Count Normalization   [ Time Frame: Through End of Study, Median Exposure 156 Weeks ]

13.  Secondary:   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. ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Alexion Pharmaceuticals Inc
Organization: Alexion Pharmaceuticals, Inc.
e-mail: clinicaltrials@alxn.com


No publications provided by Alexion Pharmaceuticals

Publications automatically indexed to this study:

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00838513     History of Changes
Other Study ID Numbers: C08-003A, BB-IND 11075, EudraCT Number 2008-006954-17
Study First Received: February 3, 2009
Results First Received: December 15, 2014
Last Updated: June 30, 2015
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
European Union: European Medicines Agency