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An Open Label Study of the Effects of Eculizumab in Neuromyelitis Optica

This study has been completed.
Sponsor:
Collaborator:
Alexion Pharmaceuticals
Information provided by:
Mayo Clinic
ClinicalTrials.gov Identifier:
NCT00904826
First received: May 18, 2009
Last updated: August 30, 2013
Last verified: August 2013
Results First Received: August 30, 2013  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Neuromyelitis Optica
Devic's Disease
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
Between October 2009 and November 2010, subjects were directly recruited at the Mayo Clinics in Rochester, Minnesota and Scottsdale, Arizona, or identified through the Mayo Clinic study-specific repository or clinicoserological database.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Eculizumab The patient will receive eculizumab at a dose of 600mg intravenously (an infusion given into the vein) each week for 4 weeks, then 900mg intravenously at the fifth week, then 900mg every 2 weeks for 48 weeks. Subjects will receive therapy for a total of 12 months.

Participant Flow:   Overall Study
    Eculizumab  
STARTED     14  
COMPLETED     14  
NOT COMPLETED     0  



  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.
No text entered.

Reporting Groups
  Description
Eculizumab The patient will receive eculizumab at a dose of 600mg intravenously (an infusion given into the vein) each week for 4 weeks, then 900mg intravenously at the fifth week, then 900mg every 2 weeks for 48 weeks. Subjects will receive therapy for a total of 12 months.

Baseline Measures
    Eculizumab  
Number of Participants  
[units: participants]
  14  
Age  
[units: years]
Mean ( Full Range )
  41.1  
  ( 18.4 to 67.6 )  
Gender  
[units: participants]
 
Female     14  
Male     0  
Region of Enrollment  
[units: participants]
 
United States     14  
Ethnic origin  
[units: participants]
 
African American     2  
Hispanic     3  
White     9  
Diagnosis  
[units: participants]
 
Neuromyelitis optica     8  
Relapsing optic neuritis     2  
Relapsing transverse myelitis     4  
Coexisting autoimmune diseases  
[units: participants]
 
Myasthenia gravis     2  
Idiopathic thrombocytopenic purpura     2  
Mixed connective tissue disease     2  
No coexisting autoimmune disease     10  
Type of previous attacks at enrollment  
[units: attacks]
 
Optic neuritis     28  
Transverse myelitis     52  
Brainstem     1  
Multifocal optic neuritis and transverse myelitis     3  
Other multifocal     2  
Number of previous attacks per subject  
[units: attacks per subject]
Mean ( Full Range )
  5.5  
  ( 2.0 to 10.0 )  
Expanded Disability Status Scale (EDSS) Score [1]
[units: units on a scale]
Mean ( Full Range )
  4.8  
  ( 2.0 to 8.0 )  
[1] The EDSS is an ordinal clinical rating scale ranging from 0 (normal neurologic examination) to 10 (death) in half-point increments.



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Median Number of Neuromyelitis Optica (NMO) Attacks Per Year   [ Time Frame: baseline, after 12 months of treatment ]

2.  Secondary:   Number Subjects Experiencing an NMO Attack in 12 Months of Eculizumab Treatment   [ Time Frame: 12 months ]

3.  Secondary:   Change in Expanded Disability Status Scale (EDDS) Score   [ Time Frame: baseline, 12 months ]

4.  Secondary:   Number of Subjects With Change in Visual Acuity in at Least One Eye by at Least One Point   [ Time Frame: 12 months ]

5.  Secondary:   Number of Subjects With Change in Ambulation by at Least 1 Point   [ Time Frame: 12 months ]

6.  Secondary:   Mean Serum Concentration of Eculizumab   [ Time Frame: 6 weeks, 3 months, 6 months, 9 months, 12 months ]

7.  Secondary:   Percentage Hemolysis   [ Time Frame: baseline, 6 weeks, 3 months, 6 months, 9 months, 12 months ]

8.  Secondary:   Mean Eculizumab Concentration in Cerebrospinal Fluid (CSF)   [ Time Frame: 3 months ]

9.  Secondary:   Mean Complement Protein 5 (C5) Concentration in CSF   [ Time Frame: baseline, 3 months ]


  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:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. Sean J. Pittock
Organization: Mayo Clinic
phone: 507-284-4741
e-mail: pittock.sean@mayo.edu


Publications of Results:
Other Publications:

Responsible Party: Sean J. Pittock, M.D., Mayo Clinic
ClinicalTrials.gov Identifier: NCT00904826     History of Changes
Other Study ID Numbers: 09-001240
Study First Received: May 18, 2009
Results First Received: August 30, 2013
Last Updated: August 30, 2013
Health Authority: United States: Institutional Review Board