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A Trial of Tocilizumab in ALS Subjects (TCZALS-001)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02469896
Recruitment Status : Completed
First Posted : June 12, 2015
Results First Posted : December 18, 2019
Last Update Posted : December 18, 2019
Sponsor:
Collaborators:
ALS Association
Barrow Neurological Foundation
Massachusetts General Hospital
Genentech, Inc.
Information provided by (Responsible Party):
Shafeeq Ladha, MD, Barrow Neurological Institute

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions ALS
Amyotrophic Lateral Sclerosis
Lou Gehrig's Disease
Motor Neuron Disease
Interventions Drug: Tocilizumab
Other: Placebo
Enrollment 22
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Placebo Active Drug
Hide Arm/Group Description

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

16 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Period Title: Overall Study
Started 8 14
Completed 6 14
Not Completed 2 0
Reason Not Completed
Withdrawal by Subject             2             0
Arm/Group Title Placebo Active Drug Total
Hide Arm/Group Description

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

16 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Total of all reporting groups
Overall Number of Baseline Participants 8 14 22
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants 14 participants 22 participants
59.2  (8.6) 61.9  (8.7) 60.9  (8.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 14 participants 22 participants
Female
3
  37.5%
3
  21.4%
6
  27.3%
Male
5
  62.5%
11
  78.6%
16
  72.7%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 14 participants 22 participants
Hispanic or Latino
1
  12.5%
0
   0.0%
1
   4.5%
Not Hispanic or Latino
7
  87.5%
14
 100.0%
21
  95.5%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 14 participants 22 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
  12.5%
0
   0.0%
1
   4.5%
White
7
  87.5%
14
 100.0%
21
  95.5%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 8 participants 14 participants 22 participants
8
 100.0%
14
 100.0%
22
 100.0%
1.Primary Outcome
Title Number of Patients Tolerant to Study Drug
Hide Description Tolerability will be assessed by on the proportion of participants remaining on study drug through all 3 doses and remaining on study and free from possibly drug-related and dose-limiting SAEs to the end of follow-up. Safety will be assessed by the occurrence of severe adverse events (SAEs), overall rates of adverse events (AEs), clinically significant abnormal laboratory tests, and changes in vital signs.
Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Tolerability Active Drug Tolerability
Hide Arm/Group Description:

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

14 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Overall Number of Participants Analyzed 8 14
Measure Type: Count of Participants
Unit of Measure: Participants
6
  75.0%
12
  85.7%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo Tolerability, Active Drug Tolerability
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.602
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Method of Estimation Estimation Parameter Risk Ratio (RR)
Estimated Value 0.875
Confidence Interval (2-Sided) 95%
0.556 to 1.377
Estimation Comments [Not Specified]
2.Primary Outcome
Title Rates of All-cause Mortality
Hide Description Safety will be assessed by the occurrence of all-cause mortality.
Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Active Drug
Hide Arm/Group Description:

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

14 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Overall Number of Participants Analyzed 8 14
Measure Type: Count of Participants
Unit of Measure: Participants
Died
0
   0.0%
0
   0.0%
Alive
8
 100.0%
14
 100.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Other Statistical Analysis No statistical data was obtain because no patients died during the trial.
3.Secondary Outcome
Title Rate of Decline in Slow Vital Capacity (SVC)
Hide Description Efficacy will be assessed by the change in the rate of change of SVC as measured by change in percent predicted per month. The SVC is a measure of lung capacity that is reported as the percent of the predicted value expected based on gender and height. In ALS patients, this measure declines over time as a result of progressive respiratory muscle weakness.
Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Discrepancies in the number of subjects analyzed are a result of subjects with missed data collection during the specific time points.
Arm/Group Title Placebo Active Drug
Hide Arm/Group Description:

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

16 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Overall Number of Participants Analyzed 8 14
Least Squares Mean (Standard Error)
Unit of Measure: Percentage points change per month
-0.765  (0.829) -0.666  (0.560)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.922
Comments Unadjusted
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Slope
Estimated Value 0.099
Confidence Interval (2-Sided) 95%
-1.902 to 2.099
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Rate of Decline ALS Functional Rating Scale Revised (ALSFRS-R)
Hide Description Efficacy will be assessed by the mean change in ALSFRS-R total score.The ALSFRS-R scale measures the functional capabilities of an ALS patient in multiple domains such as swallowing, speech, fine motor, and breathing functions. It ranges from a maximum score of 48 for normal functioning to 0 for death or dependance on mechanical ventilation and declines by approximately 1 point per month on average for an ALS patient.
Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Discrepancies in the number of subjects analyzed are a result of subjects with missed data collection during the specific time points.
Arm/Group Title Placebo Active Drug
Hide Arm/Group Description:

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

16 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Overall Number of Participants Analyzed 8 14
Least Squares Mean (Standard Error)
Unit of Measure: units on a scale
-0.583  (0.313) -0.591  (0.211)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.983
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Difference of slopes
Estimated Value -0.008
Confidence Interval (2-Sided) 95%
-0.761 to 0.745
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Rate of Decline Handheld Dynamometry (HHD)
Hide Description Efficacy will be assessed by the change in the rate of change of HHD upper and lower extremity mega-scores. HHD utilizes an electronic pressure sensor to measure strength of individual muscles in kilograms. To calculate megascores, the mean and standard deviation of each muscle or muscle group, without regard to laterality, will be calculated from the baseline assessment of all participants. Strength estimates of each bilateral muscle or muscle group will be converted to Z scores by subtracting the relevant mean and dividing by the relevant standard deviation. Z scores for all upper extremity measurements (shoulder flexion, elbow flexion, elbow extension, wrist extension, and first dorsal interosseous contraction) and all lower extremity measurements (hip flexion, knee flexion, knee extension, and ankle dorsiflexion) will be averaged to yield upper and lower extremity megascores. Larger values indicate greater strength.
Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Discrepancies in the number of subjects analyzed are a result of subjects with missed data collection during the specific time points.
Arm/Group Title Placebo Active Drug
Hide Arm/Group Description:

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

16 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Overall Number of Participants Analyzed 8 14
Mean (Standard Error)
Unit of Measure: Z-score
HHD Upper extremity mega-score Number Analyzed 8 participants 14 participants
-0.028  (0.035) -0.070  (0.024)
HHD Lower extremity mega-score Number Analyzed 7 participants 14 participants
-0.018  (0.039) -0.032  (0.026)
6.Secondary Outcome
Title Change in Peripheral Blood Mononuclear Cell (PBMC) Gene Expression
Hide Description Target engagement will be assessed by comparing the PBMC fold change in cytokine gene expression from baseline to week 4-16 average of ALS patients receiving drug versus placebo.
Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Discrepancies in the number of subjects analyzed are a result of subjects with missed data collection during the specific time points.
Arm/Group Title Placebo Active Drug
Hide Arm/Group Description:

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

16 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Overall Number of Participants Analyzed 8 14
Geometric Mean (95% Confidence Interval)
Unit of Measure: Fold change in gene expression
PBMC IL-6 fold-change Number Analyzed 8 participants 14 participants
1.21
(0.60 to 2.54)
1.39
(0.52 to 5.00)
PBMC IL-8 fold-change Number Analyzed 8 participants 14 participants
1.33
(0.08 to 39.4)
1.79
(0.38 to 14.8)
PBMC Matrix Metalloproteinase 1 (MMP1)fold-change Number Analyzed 8 participants 13 participants
1.01
(0.37 to 2.13)
1.21
(0.42 to 3.28)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments PBMC IL-6 fold-change
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.684
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of geometric means
Estimated Value 1.150
Confidence Interval (2-Sided) 95%
0.566 to 2.337
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.663
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of geometric means
Estimated Value 1.344
Confidence Interval (2-Sided) 95%
0.331 to 2.080
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.500
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of geometric means
Estimated Value 1.198
Confidence Interval (2-Sided) 95%
0.691 to 2.080
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Changes in Cytokine Levels in the Plasma
Hide Description Target engagement will be assessed by mean change in plasma cytokine concentration between weeks 4 and 16 in ALS subjects receiving placebo or active drug.
Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Discrepancies in the number of subjects analyzed are a result of subjects with missed data collection during the specific time points.
Arm/Group Title Placebo Active Drug
Hide Arm/Group Description:

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

16 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Overall Number of Participants Analyzed 8 14
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Fold change in concentration
CRP (ug/mL) Number Analyzed 8 participants 13 participants
1.051
(0.279 to 3.952)
0.049
(0.019 to 0.126)
IL-1beta (pg/mL) Number Analyzed 8 participants 14 participants
0.969
(0.608 to 1.545)
1.266
(0.871 to 1.840)
IL-6 (pg/mL) Number Analyzed 8 participants 14 participants
0.897
(0.532 to 1.515)
17.974
(12.900 to 25.043)
IL-8 (pg/mL) Number Analyzed 8 participants 14 participants
1.241
(0.862 to 1.787)
1.569
(1.169 to 2.105)
IL-17 (pg/mL) Number Analyzed 6 participants 14 participants
0.962
(0.470 to 1.967)
1.342
(0.809 to 2.226)
Tumor necrosis factor (TNF)-alpha (pg/mL) Number Analyzed 8 participants 14 participants
1.180
(0.981 to 1.419)
1.054
(0.923 to 1.204)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 0.047
Confidence Interval (2-Sided) 95%
0.010 to 0.217
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.247
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 1.306
Confidence Interval (2-Sided) 95%
0.828 to 2.059
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 19.591
Confidence Interval (2-Sided) 95%
11.143 to 34.446
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.185
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 1.264
Confidence Interval (2-Sided) 95%
0.892 to 1.791
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.427
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 1.396
Confidence Interval (2-Sided) 95%
0.608 to 3.206
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.285
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 0.893
Confidence Interval (2-Sided) 95%
0.725 to 1.100
Estimation Comments [Not Specified]
8.Secondary Outcome
Title Change in Mean Concentration Cytokines in the Cerebrospinal Fluid (CSF)
Hide Description Target engagement will be assessed by the mean change in CSF cytokine concentration between baseline and week 8 in ALS subjects receiving placebo or active drug.
Time Frame 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Active Drug
Hide Arm/Group Description:

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

14subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Overall Number of Participants Analyzed 8 14
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Fold change in concentration
CRP (ng/mL)
0.641
(0.216 to 1.903)
0.107
(0.044 to 0.260)
IL-1beta (pg/mL)
1.498
(0.542 to 4.138)
1.110
(0.459 to 2.679)
IL-6 (pg/mL)
1.242
(0.781 to 1.974)
4.115
(2.813 to 6.018)
IL-8 (pg/mL)
0.946
(0.758 to 1.181)
1.020
(0.854 to 1.218)
TNF-alpha (pg/mL)
0.989
(0.727 to 1.346)
1.066
(0.833 to 1.365)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.011
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 0.167
Confidence Interval (2-Sided) 95%
0.043 to 0.643
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.604
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 0.741
Confidence Interval (2-Sided) 95%
0.228 to 2.405
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 2.940
Confidence Interval (2-Sided) 95%
1.823 to 4.740
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.587
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 1.078
Confidence Interval (2-Sided) 95%
0.813 to 1.431
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.697
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 1.078
Confidence Interval (2-Sided) 95%
0.728 to 1.595
Estimation Comments [Not Specified]
9.Secondary Outcome
Title Change in CSF Soluble Interleukin-6 (sIL-6) Receptor Concentrations
Hide Description Target engagement will be assessed by comparing the mean change in CSF sIL-6 receptor concentrations (ng/mL) between baseline and week 8 of the placebo and active drug groups.
Time Frame 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Discrepancies in the number of subjects analyzed are a result of subjects with missed data collection during the specific time points.
Arm/Group Title Placebo Active Drug
Hide Arm/Group Description:

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

16 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Overall Number of Participants Analyzed 8 14
Least Squares Mean (95% Confidence Interval)
Unit of Measure: ng/mL
1.029
(0.886 to 1.195)
1.836
(1.621 to 2.080)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Active Drug
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of fold change
Estimated Value 1.785
Confidence Interval (2-Sided) 95%
1.502 to 2.121
Estimation Comments [Not Specified]
10.Secondary Outcome
Title Peripheral Benzodiazepine Receptor 28 (PBR28) Positron Emission Tomography (PET)
Hide Description Measure the effects of tocilizumab on reducing glial activation measured by PBR28 PET in a subset of trial participants.
Time Frame 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Only 2 patients met inclusion criteria for the PET portion of the study and thus, the data could not be statistically analyzed.
Arm/Group Title Placebo Active Drug
Hide Arm/Group Description:

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

16 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

Overall Number of Participants Analyzed 1 1
Measure Type: Number
Unit of Measure: Standardized Uptake Variable Ratio(SUVR)
Baseline 1.225282 1.059685
Week 8 1.361542 1.056888
Time Frame Adverse events were collected over the study lasting up to 16 weeks in each subject.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Active Drug
Hide Arm/Group Description

8 subjects will receive matching IV placebo every 4 weeks for 3 months.

Placebo: IV Infusion

16 subjects will receive 8mg/kg of IV tocilizumab every 4 weeks for 3 months.

Tocilizumab: IV Infusion

All-Cause Mortality
Placebo Active Drug
Affected / at Risk (%) Affected / at Risk (%)
Total   0/8 (0.00%)      0/14 (0.00%)    
Hide Serious Adverse Events
Placebo Active Drug
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/8 (12.50%)      0/14 (0.00%)    
Respiratory, thoracic and mediastinal disorders     
Pneumonia Aspiration  1  1/8 (12.50%)  1 0/14 (0.00%)  0
1
Term from vocabulary, MedDRA (17.1)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Active Drug
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   6/8 (75.00%)      11/14 (78.57%)    
Blood and lymphatic system disorders     
Neutropenia  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Ear and labyrinth disorders     
Ear Pruritus  1  1/8 (12.50%)  1 0/14 (0.00%)  0
Gastrointestinal disorders     
Abdominal Distension  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Abdominal Pain  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Aphthous Stomatitis  1  1/8 (12.50%)  1 0/14 (0.00%)  0
Constipation  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Faecal Incontinence  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Nausea  1  2/8 (25.00%)  4 1/14 (7.14%)  1
Salivary Hypersecretion  1  0/8 (0.00%)  0 2/14 (14.29%)  2
General disorders     
Catheter Site Related Reaction  1  1/8 (12.50%)  1 0/14 (0.00%)  0
Fatigue  1  1/8 (12.50%)  2 2/14 (14.29%)  2
Infusion Site Pruritus  1  1/8 (12.50%)  1 0/14 (0.00%)  0
Injection Site Pain  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Puncture Site Pain  1  4/8 (50.00%)  4 2/14 (14.29%)  2
Infections and infestations     
Catheter Site Infection  1  1/8 (12.50%)  1 0/14 (0.00%)  0
Clostridium Difficile Colitis  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Gingival Abscess  1  1/8 (12.50%)  1 0/14 (0.00%)  0
Nasopharyngitis  1  0/8 (0.00%)  0 2/14 (14.29%)  2
Pneumonia  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Upper Respiratory Tract Infection  1  1/8 (12.50%)  1 1/14 (7.14%)  1
Urinary Tract Infection  1  1/8 (12.50%)  1 0/14 (0.00%)  0
Injury, poisoning and procedural complications     
Contusion  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Fall  1  2/8 (25.00%)  3 3/14 (21.43%)  4
Foot Fracture  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Post Lumbar Puncture Syndrome  1  0/8 (0.00%)  0 2/14 (14.29%)  2
Procedural Pain  1  1/8 (12.50%)  2 3/14 (21.43%)  4
Investigations     
Alanine Aminotransferase Increased  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Aspartate Aminotransferase Increased  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Blood Pressure Increased  1  0/8 (0.00%)  0 2/14 (14.29%)  2
Platelet Count Increased  1  1/8 (12.50%)  1 0/14 (0.00%)  0
White Blood Cell Count Increased  1  1/8 (12.50%)  1 0/14 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Muscular Weakness  1  1/8 (12.50%)  1 3/14 (21.43%)  4
Musculoskeletal Pain  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Nervous system disorders     
Dizziness  1  2/8 (25.00%)  3 1/14 (7.14%)  1
Headache  1  0/8 (0.00%)  0 2/14 (14.29%)  3
Presyncope  1  0/8 (0.00%)  0 1/14 (7.14%)  1
Renal and urinary disorders     
Urinary Tract Pain  1  1/8 (12.50%)  1 0/14 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Cough  1  2/8 (25.00%)  2 0/14 (0.00%)  0
Dysphonia  1  0/8 (0.00%)  0 2/14 (14.29%)  2
Dyspnoea  1  1/8 (12.50%)  1 3/14 (21.43%)  3
Oropharyngeal Pain  1  1/8 (12.50%)  1 1/14 (7.14%)  1
Skin and subcutaneous tissue disorders     
Pruritus  1  1/8 (12.50%)  1 1/14 (7.14%)  3
1
Term from vocabulary, MedDRA 17.1
Indicates events were collected by systematic assessment
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Shafeeq Ladha, MD
Organization: Barrow Neurological Institute
Phone: 602-406-8989
EMail: Shafeeq.Ladha@Barrowneuro.org
Layout table for additonal information
Responsible Party: Shafeeq Ladha, MD, Barrow Neurological Institute
ClinicalTrials.gov Identifier: NCT02469896    
Other Study ID Numbers: 2015TCZALS-001
First Submitted: June 3, 2015
First Posted: June 12, 2015
Results First Submitted: August 6, 2019
Results First Posted: December 18, 2019
Last Update Posted: December 18, 2019