Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00844844
First received: February 13, 2009
Last updated: November 26, 2014
Last verified: June 2014
Results First Received: October 21, 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
C08-002A/B combined 2 studies: one for adults (C08-002A, N=16) and one for adolescents (C08-002B, N=1) Please refer to NCT00844545 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 had to exhibit a decrease in platelet count despite at least 4 Plasma Therapy (PT) treatments in the 1 week immediately prior to screening. Patients who met the eligibility criteria during screening 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 4 periods

Period 1:   Screening Period
    Eculizumab  
STARTED     17  
COMPLETED     17  
NOT COMPLETED     0  

Period 2:   Treatment Period (26 Weeks)
    Eculizumab  
STARTED     17  
COMPLETED     15  
NOT COMPLETED     2  
Adverse Event                 1  
Protocol Violation                 1  

Period 3:   Extension Treatment Period
    Eculizumab  
STARTED     13  
COMPLETED     11  
NOT COMPLETED     2  
Adverse Event                 2  

Period 4:   Post Treatment Period
    Eculizumab  
STARTED     6  
COMPLETED     5  
NOT COMPLETED     1  
Withdrawal by Subject                 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 17 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]
  17  
Age  
[units: Years]
Mean ± Standard Deviation
  31.8  ± 13.32  
Gender  
[units: participants]
 
Female     12  
Male     5  
Race (NIH/OMB)  
[units: participants]
 
American Indian or Alaska Native     0  
Asian     1  
Native Hawaiian or Other Pacific Islander     0  
Black or African American     1  
White     15  
More than one race     0  
Unknown or Not Reported     0  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Assess the Effect of Eculizumab to Reduce Thrombotic Microangiopathy (TMA) as Assessed by Platelet Count Change From Baseline   [ Time Frame: From Baseline through 26 weeks ]

2.  Primary:   Assess the Effect of Eculizumab to Reduce Thrombotic Microangiopathy (TMA) as Assessed by Platelet Count Normalization   [ Time Frame: Through 26 weeks ]

3.  Primary:   Assess the Effect of Eculizumab to Reduce Thrombotic Microangiopathy (TMA) as Assessed by Hematologic Normalization   [ Time Frame: Through 26 weeks ]

4.  Secondary:   Additional Efficacy Endpoints Related to Manifestations of TMA as Assessed by Complete TMA Response   [ Time Frame: Through 26 weeks ]

5.  Secondary:   Additional Efficacy Endpoints Related to Manifestations of TMA as Assessed by TMA Intervention Rate   [ Time Frame: Through 26 weeks ]

6.  Secondary:   Assess the Effect of Eculizumab to Reduce Thrombotic Microangiopathy (TMA) as Assessed by Platelet Count Change From Baseline   [ Time Frame: Through End of Study, Median Exposure 100.29 Weeks ]

7.  Secondary:   Assess the Effect of Eculizumab to Reduce Thrombotic Microangiopathy (TMA) as Assessed by Platelet Count Normalization   [ Time Frame: Through End of Study ]

8.  Secondary:   Assess the Effect of Eculizumab to Reduce Thrombotic Microangiopathy (TMA) as Assessed by Hematologic Normalization   [ Time Frame: Through End of Study ]

9.  Secondary:   Additional Efficacy Endpoints Related to Manifestations of TMA as Assessed by Complete TMA Response   [ Time Frame: Through End of Study ]

10.  Secondary:   Additional Efficacy Endpoints Related to Manifestations of TMA as Assessed by TMA Intervention Rate   [ Time Frame: Through End of Study ]

11.  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: NCT00844844     History of Changes
Other Study ID Numbers: C08-002B, BB-IND-11075, EudraCT Number 2008-006953-41
Study First Received: February 13, 2009
Results First Received: October 21, 2014
Last Updated: November 26, 2014
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
European Medicines Agency: EMA