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Trial record 10 of 54 for:    Devic syndrome

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

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ClinicalTrials.gov Identifier: NCT00904826
Recruitment Status : Completed
First Posted : May 20, 2009
Results First Posted : November 4, 2013
Last Update Posted : November 4, 2013
Sponsor:
Collaborator:
Alexion Pharmaceuticals
Information provided by:
Mayo Clinic

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Neuromyelitis Optica
Devic's Disease
Intervention Drug: Eculizumab
Enrollment 14
Recruitment Details 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  
Arm/Group Title Eculizumab
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 14
Completed 14
Not Completed 0
Arm/Group Title Eculizumab
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 14
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 14 participants
41.1
(18.4 to 67.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants
Female
14
 100.0%
Male
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 14 participants
14
Ethnic origin  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 14 participants
African American 2
Hispanic 3
White 9
Diagnosis  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 14 participants
Neuromyelitis optica 8
Relapsing optic neuritis 2
Relapsing transverse myelitis 4
Coexisting autoimmune diseases  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 14 participants
Myasthenia gravis 2
Idiopathic thrombocytopenic purpura 2
Mixed connective tissue disease 2
No coexisting autoimmune disease 10
Type of previous attacks at enrollment  
Measure Type: Number
Unit of measure:  Attacks
Number Analyzed 14 participants
Optic neuritis 28
Transverse myelitis 52
Brainstem 1
Multifocal optic neuritis and transverse myelitis 3
Other multifocal 2
Number of previous attacks per subject  
Mean (Full Range)
Unit of measure:  Attacks per subject
Number Analyzed 14 participants
5.5
(2.0 to 10.0)
Expanded Disability Status Scale (EDSS) Score   [1] 
Mean (Full Range)
Unit of measure:  Units on a scale
Number Analyzed 14 participants
4.8
(2.0 to 8.0)
[1]
Measure Description: The EDSS is an ordinal clinical rating scale ranging from 0 (normal neurologic examination) to 10 (death) in half-point increments.
1.Primary Outcome
Title Median Number of Neuromyelitis Optica (NMO) Attacks Per Year
Hide Description [Not Specified]
Time Frame baseline, after 12 months of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 14
Median (Full Range)
Unit of Measure: attacks per year
Baseline
3
(2 to 4)
After 12 months of treatment
0
(0 to 1)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Eculizumab
Comments Comparison between before treatment and one year after treatment.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
2.Secondary Outcome
Title Number Subjects Experiencing an NMO Attack in 12 Months of Eculizumab Treatment
Hide Description [Not Specified]
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: participants
2
3.Secondary Outcome
Title Change in Expanded Disability Status Scale (EDDS) Score
Hide Description The EDSS is an ordinal clinical rating scale ranging from 0 (normal neurologic examination) to 10 (death) in half-point increments.
Time Frame baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 14
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
-0.7
(-1.2 to -0.2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Eculizumab
Comments Comparison was made between baseline and after 12 months of treatment.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0078
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
4.Secondary Outcome
Title Number of Subjects With Change in Visual Acuity in at Least One Eye by at Least One Point
Hide Description Visual acuity was measured using the the Visual Acuity subscale of the Opticospinal Impairment Score (OSIS) for Exacerbations. This subscale ranges from 0 (normal) to 8 (no light perception).
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: participants
5
5.Secondary Outcome
Title Number of Subjects With Change in Ambulation by at Least 1 Point
Hide Description Ambulation was measured by the Hauser Ambulation Index, which ranges from 0 (asymptomatic; fully active) to 9 (restricted to wheelchair; unable to transfer self independently.)
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: participants
3
6.Secondary Outcome
Title Mean Serum Concentration of Eculizumab
Hide Description [Not Specified]
Time Frame 6 weeks, 3 months, 6 months, 9 months, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Patient 13 was excluded from the 3 months measurement because she had temporarily discontinued treatment.
Arm/Group Title Eculizumab
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: micrograms/mL
6 weeks 206  (77)
3 months 187  (91.2)
9 months 230  (85.3)
12 months 246  (102)
7.Secondary Outcome
Title Percentage Hemolysis
Hide Description Percentage of hemolysis is a measure of complement activity. Less than 20% lysis is deemed to be complete complement inhibition.
Time Frame baseline, 6 weeks, 3 months, 6 months, 9 months, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Subject 13 was excluded at 3 months visit because she had temporarily discontinued treatment.
Arm/Group Title Eculizumab
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: percentage of hemolysis
Baseline 88.5  (13.2)
6 weeks 0.4  (0.8)
3 months 0  (0)
6 months 0.2  (0.6)
9 months 0.4  (0.9)
12 months 0.9  (1.5)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Eculizumab
Comments Comparison is between 6 weeks and baseline.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Eculizumab
Comments Comparison is between 3 months and baseline.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Eculizumab
Comments Comparison is between 6 months and baseline
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Eculizumab
Comments Comparison is between 9 months and baseline.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 5 Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Eculizumab
Comments Comparison is between 12 months and baseline.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
8.Secondary Outcome
Title Mean Eculizumab Concentration in Cerebrospinal Fluid (CSF)
Hide Description [Not Specified]
Time Frame 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
12 subjects including subject 13 agreed to have CSF draw at the 3 month visit, but subject 13 was excluded because she had temporarily discontinued treatment.
Arm/Group Title Eculizumab
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: ng/mL
34.7  (18.7)
9.Secondary Outcome
Title Mean Complement Protein 5 (C5) Concentration in CSF
Hide Description [Not Specified]
Time Frame baseline, 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
At 3 months, C5 was undetectable in 6 subjects; patient 13 was excluded because she had temporarily discontinued treatment.
Arm/Group Title Eculizumab
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: ng/mL
Baseline 144  (75.5)
3 months 60.8  (23.3)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Eculizumab
Comments Comparison was between 3 months and baseline.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0019
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Eculizumab
Hide Arm/Group Description 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.
All-Cause Mortality
Eculizumab
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Eculizumab
Affected / at Risk (%) # Events
Total   1/14 (7.14%)    
Infections and infestations   
Meningococcal infection   1/14 (7.14%)  1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Eculizumab
Affected / at Risk (%) # Events
Total   9/14 (64.29%)    
Blood and lymphatic system disorders   
Swelling or ankle swelling   2/14 (14.29%)  2
Gastrointestinal disorders   
Nausea   6/14 (42.86%)  18
Diarrhea   6/14 (42.86%)  7
General disorders   
Headache   9/14 (64.29%)  23
Dizziness   6/14 (42.86%)  13
Abdominal Pain   4/14 (28.57%)  5
Flu like symptoms   2/14 (14.29%)  3
Fatigue   2/14 (14.29%)  3
Nasal congestion or watery eyes   2/14 (14.29%)  3
Infections and infestations   
Urinary tract infection   2/14 (14.29%)  6
Musculoskeletal and connective tissue disorders   
Muscle cramps   2/14 (14.29%)  9
Respiratory, thoracic and mediastinal disorders   
Coughing   5/14 (35.71%)  6
Skin and subcutaneous tissue disorders   
Rash   3/14 (21.43%)  3
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Sean J. Pittock
Organization: Mayo Clinic
Phone: 507-284-4741
EMail: pittock.sean@mayo.edu
Other Publications:
Layout table for additonal information
Responsible Party: Sean J. Pittock, M.D., Mayo Clinic
ClinicalTrials.gov Identifier: NCT00904826     History of Changes
Other Study ID Numbers: 09-001240
First Submitted: May 18, 2009
First Posted: May 20, 2009
Results First Submitted: August 30, 2013
Results First Posted: November 4, 2013
Last Update Posted: November 4, 2013