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Safety, Pharmacokinetics and Preliminary Efficacy Study of CFZ533 in Patients With Primary Sjögren's Syndrome

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: NCT02291029
Recruitment Status : Completed
First Posted : November 14, 2014
Results First Posted : August 14, 2019
Last Update Posted : August 14, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Primary Sjögren's Syndrome
Interventions Drug: CFZ533 active - Cohort 1
Drug: CFZ533 placebo- Cohort 1
Drug: CFZ533 active - Cohort 2
Drug: CFZ533 placebo - Cohort 2
Drug: CFZ533 active -Cohort 3
Drug: CFZ533 active - Cohort 3
Enrollment 69
Recruitment Details A total of 69 patients were enrolled in Germany (1 center), Hungary (1 center), Switzerland (1 center), United Kingdom (3 centers), United States (3 centers)
Pre-assignment Details  
Arm/Group Title Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo Cohort 3 CFZ533 Arm 1 Cohort 3 CFZ533 Arm 2
Hide Arm/Group Description CFZ533 3 mg/kg s.c. Placebo s.c./CFZ533 3 mg/kg s.c. CFZ533 10 mg/kg i.v. Placebo i.v./CFZ533 10 mg/kg i.v. CFZ533 600 mg s.c./CFZ533 300 mg s.c. CFZ533 10 mg/kg i.v./CFZ533 300 mg s.c.
Period Title: Overall Study
Started 8 4 21 11 13 12
Completed 8 3 20 11 13 12
Not Completed 0 1 1 0 0 0
Reason Not Completed
Withdrawal by Subject             0             0             1             0             0             0
Adverse Event             0             1             0             0             0             0
Arm/Group Title Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo Cohort 3 CFZ533 Arm 1 Cohort 3 CFZ533 Arm 2 Total
Hide Arm/Group Description CFZ533 3 mg/kg s.c. Placebo s.c./CFZ533 3 mg/kg s.c. CFZ533 10 mg/kg i.v. Placebo i.v./CFZ533 10 mg/kg i.v. CFZ533 600 mg s.c./CFZ533 300 mg s.c. CFZ533 10 mg/kg i.v./CFZ533 300 mg s.c. Total of all reporting groups
Overall Number of Baseline Participants 8 4 21 11 13 12 69
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 8 participants 4 participants 21 participants 11 participants 13 participants 12 participants 69 participants
56.4
(34 to 72)
48.8
(45 to 52)
51.7
(24 to 72)
50.6
(25 to 69)
52.3
(23 to 74)
54.8
(23 to 68)
52.6
(23 to 74)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 4 participants 21 participants 11 participants 13 participants 12 participants 69 participants
Female
8
 100.0%
4
 100.0%
19
  90.5%
11
 100.0%
12
  92.3%
10
  83.3%
64
  92.8%
Male
0
   0.0%
0
   0.0%
2
   9.5%
0
   0.0%
1
   7.7%
2
  16.7%
5
   7.2%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 4 participants 21 participants 11 participants 13 participants 12 participants 69 participants
Caucasian
7
  87.5%
4
 100.0%
18
  85.7%
10
  90.9%
12
  92.3%
10
  83.3%
61
  88.4%
Asian
1
  12.5%
0
   0.0%
2
   9.5%
1
   9.1%
1
   7.7%
2
  16.7%
7
  10.1%
Black
0
   0.0%
0
   0.0%
1
   4.8%
0
   0.0%
0
   0.0%
0
   0.0%
1
   1.4%
1.Primary Outcome
Title Change From Baseline in EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI)
Hide Description

The effect of CFZ533 on clinical disease activity was measured by the change in ESSDAI (EULAR Sjögren's syndrome disease activity index) between baseline and week 12. The instrument contains 12 organ-specific domains contributing to disease activity. For each domain, features of disease activity are scored in 3 or 4 levels according to their severity.

These scores are then summed across the 12 domains in a weighted manner to provide the total score (range 0-123). A reduction from baseline indicates improvement in patients.

Time Frame Baseline and Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
PD Population (all subjects with available Pharmacodynamics data and no protocol deviations with relevant impact on PD data)
Arm/Group Title Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo Cohort 3 CFZ533 Arm 1 Cohort 3 CFZ533 Arm 2
Hide Arm/Group Description:
CFZ533 3 mg/kg s.c.
Placebo s.c./CFZ533 3 mg/kg s.c.
CFZ533 10 mg/kg i.v.
Placebo i.v./CFZ533 10 mg/kg i.v.
CFZ533 600 mg s.c./CFZ533 300 mg s.c.
CFZ533 10 mg/kg i.v./CFZ533 300 mg s.c.
Overall Number of Participants Analyzed 8 4 21 11 13 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 8 participants 4 participants 21 participants 11 participants 13 participants 12 participants
12.0  (3.78) 11.8  (3.86) 10.6  (4.44) 11.0  (5.16) 12.7  (6.06) 10.4  (5.87)
Week 12 Number Analyzed 8 participants 4 participants 20 participants 11 participants 13 participants 12 participants
9.6  (5.45) 9.8  (3.30) 4.2  (4.25) 9.7  (9.05) 7.2  (6.69) 2.8  (2.48)
Change from Baseline to Week 12 Number Analyzed 8 participants 4 participants 20 participants 11 participants 13 participants 12 participants
-2.4  (2.77) -2.0  (2.45) -6.4  (4.00) -1.3  (8.06) -5.5  (5.49) -7.6  (7.14)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1 CFZ533, Cohort 1 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.397
Comments One-sided p-value
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.41
Confidence Interval (2-Sided) 95%
-3.70 to 2.89
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 2 CFZ533, Cohort 2 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.009
Comments One-sided p-value
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -5.21
Confidence Interval (2-Sided) 95%
-9.46 to -0.96
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Cohort 3 CFZ533 Arm 1, Cohort 3 CFZ533 Arm 2
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.344
Comments two-sided p-value
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 2.34
Confidence Interval (2-Sided) 95%
-2.78 to 7.45
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change From Baseline in EULAR Sjögren's Syndrome Patient Reported Intensity (ESSPRI)
Hide Description The ESSPRI is a patient self-reported outcome measure to assess dryness, limb pain, fatigue and mental fatigue, where each of the domains normally reported as 0 (not at all) to 10 (extremely severe). The final ESSPRI score is the average of three: dryness, pain and fatigue. A reduction from baseline indicates the improvement of symptoms.
Time Frame Baseline and Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
PD Population
Arm/Group Title Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo Cohort 3 CFZ533 Arm 1 Cohort 3 CFZ533 Arm 2
Hide Arm/Group Description:
CFZ533 3 mg/kg s.c.
Placebo s.c./CFZ533 3 mg/kg s.c.
CFZ533 10 mg/kg i.v.
Placebo i.v./CFZ533 10 mg/kg i.v.
CFZ533 600 mg s.c./CFZ533 300 mg s.c.
CFZ533 10 mg/kg i.v./CFZ533 300 mg s.c.
Overall Number of Participants Analyzed 8 4 21 11 13 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 8 participants 4 participants 21 participants 11 participants 13 participants 12 participants
6.75  (1.909) 7.00  (1.826) 6.71  (1.678) 7.18  (1.486) 7.00  (1.604) 6.00  (2.344)
Week 12 Number Analyzed 8 participants 4 participants 20 participants 11 participants 13 participants 12 participants
5.71  (1.240) 7.08  (2.251) 5.03  (2.413) 6.24  (2.039) 5.33  (2.269) 4.83  (2.552)
Change from Baseline to Week 12 Number Analyzed 8 participants 4 participants 20 participants 11 participants 13 participants 12 participants
-1.04  (1.201) 0.08  (0.631) -1.68  (1.954) -0.94  (1.246) -1.67  (1.841) -1.17  (2.333)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1 CFZ533, Cohort 1 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.205
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.09
Confidence Interval (2-Sided) 95%
-2.97 to 0.80
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 2 CFZ533, Cohort 2 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.188
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.95
Confidence Interval (2-Sided) 95%
-2.41 to 0.50
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Cohort 3 CFZ533 Arm 1, Cohort 3 CFZ533 Arm 2
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.663
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.37
Confidence Interval (2-Sided) 95%
-2.08 to 1.35
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change From Baseline in Physician Global Assessment of the Patient's Overall Disease Activity (VAS)
Hide Description The visual analogue scale used is a 100 mm VAS ranging from "no disease" (0 mm) to "maximal disease activity" (100 mm).
Time Frame Baseline and Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
PD Population (Statistical Analysis only for Cohort 1 and 2)
Arm/Group Title Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo Cohort 3 CFZ533 Arm 1 Cohort 3 CFZ533 Arm 2
Hide Arm/Group Description:
CFZ533 3 mg/kg s.c.
Placebo s.c./CFZ533 3 mg/kg s.c.
CFZ533 10 mg/kg i.v.
Placebo i.v./CFZ533 10 mg/kg i.v.
CFZ533 600 mg s.c./CFZ533 300 mg s.c.
CFZ533 10 mg/kg i.v./CFZ533 300 mg s.c.
Overall Number of Participants Analyzed 8 4 21 11 13 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 7 participants 4 participants 21 participants 11 participants 13 participants 12 participants
57.9  (15.72) 57.8  (17.19) 51.9  (12.62) 47.9  (19.18) 50.4  (12.39) 47.1  (18.34)
Week 12 Number Analyzed 8 participants 4 participants 20 participants 11 participants 13 participants 12 participants
40.5  (16.42) 55.5  (12.01) 22.8  (11.78) 34.2  (13.90) 25.4  (16.65) 27.3  (16.74)
Change from Baseline to Week 12 Number Analyzed 7 participants 4 participants 20 participants 11 participants 13 participants 12 participants
-17.6  (24.60) -2.3  (10.90) -28.7  (16.02) -13.7  (22.97) -25.0  (15.30) -19.8  (21.96)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1 CFZ533, Cohort 1 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.161
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -15.26
Confidence Interval (2-Sided) 95%
-37.90 to 7.38
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 2 CFZ533, Cohort 2 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.017
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -12.16
Confidence Interval (2-Sided) 95%
-21.94 to -2.38
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Change From Baseline in Patient's Global Assessment of Their Disease Activity (VAS)
Hide Description The visual analogue scale used is a 100 mm VAS ranging from "no disease" (0 mm) to "maximal disease activity" (100 mm).
Time Frame Baseline and Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
PD Population (Statistical Analysis only for Cohort 1 and 2)
Arm/Group Title Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo Cohort 3 CFZ533 Arm 1 Cohort 3 CFZ533 Arm 2
Hide Arm/Group Description:
CFZ533 3 mg/kg s.c.
Placebo s.c./CFZ533 3 mg/kg s.c.
CFZ533 10 mg/kg i.v.
Placebo i.v./CFZ533 10 mg/kg i.v.
CFZ533 600 mg s.c./CFZ533 300 mg s.c.
CFZ533 10 mg/kg i.v./CFZ533 300 mg s.c.
Overall Number of Participants Analyzed 8 4 21 11 13 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 8 participants 4 participants 21 participants 11 participants 13 participants 12 participants
47.13  (32.406) 73.00  (12.623) 58.43  (19.881) 54.91  (21.002) 63.69  (25.799) 52.08  (22.138)
Week 12 Number Analyzed 8 participants 4 participants 20 participants 11 participants 13 participants 12 participants
49.06  (24.519) 75.50  (24.393) 34.85  (24.564) 42.27  (24.483) 38.46  (26.965) 53.17  (25.305)
Change from Baseline to Week 12 Number Analyzed 8 participants 4 participants 20 participants 11 participants 13 participants 12 participants
1.94  (26.023) 2.50  (17.861) -23.05  (26.920) -12.64  (26.871) -25.23  (29.833) 1.08  (23.283)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1 CFZ533, Cohort 1 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.456
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -9.45
Confidence Interval (2-Sided) 95%
-36.20 to 17.30
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 2 CFZ533, Cohort 2 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.376
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -8.14
Confidence Interval (2-Sided) 95%
-26.67 to 10.39
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Change From Baseline in Short Form (36) Health Survey (SF-36) Physical Component Score
Hide Description The SF-36 is a 36-item, patient self-reported outcome measure (questionnaires) of patient health. The outcome of the questionnaires in eight scales results in two summary scores, physical component and mental component, both ranging from 0 - 100. An increase from baseline in either component summary score indicates reduced disease burden.
Time Frame Baseline and Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
PD Population for Cohort 1 and Cohort 2. Data were not collected from participants in Cohort 3 CFZ533 Arm 1 and Cohort 3 CFZ533 Arm 2.
Arm/Group Title Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo
Hide Arm/Group Description:
CFZ533 3 mg/kg s.c.
Placebo s.c./CFZ533 3 mg/kg s.c.
CFZ533 10 mg/kg i.v.
Placebo i.v./CFZ533 10 mg/kg i.v.
Overall Number of Participants Analyzed 8 4 21 11
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 6 participants 4 participants 20 participants 11 participants
42.218  (6.9437) 31.215  (12.5562) 38.163  (8.5905) 38.819  (5.9689)
Week 12 Number Analyzed 8 participants 4 participants 18 participants 11 participants
40.374  (9.2230) 36.123  (13.0002) 44.001  (9.3943) 40.298  (8.9392)
Change from Baseline to Week 12 Number Analyzed 6 participants 4 participants 17 participants 11 participants
-1.005  (4.5380) 4.908  (4.2349) 5.546  (7.1760) 1.479  (8.2497)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1 CFZ533, Cohort 1 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.172
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -5.50
Confidence Interval (2-Sided) 95%
-13.91 to 2.91
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 2 CFZ533, Cohort 2 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.175
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 3.83
Confidence Interval (2-Sided) 95%
-1.81 to 9.48
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Change From Baseline in Short Form (36) Health Survey (SF-36) Mental Component Score
Hide Description The SF-36 is a 36-item, patient self-reported outcome measure (questionnaires) of patient health. The outcome of the questionnaires in eight scales results in two summary scores, physical component and mental component, both ranging from 0 - 100. An increase from baseline in either component summary score indicates reduced disease burden.
Time Frame Baseline and Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
PD Population for Cohort 1 and Cohort 2. Data were not collected from participants in Cohort 3 CFZ533 Arm 1 and Cohort 3 CFZ533 Arm 2.
Arm/Group Title Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo
Hide Arm/Group Description:
CFZ533 3 mg/kg s.c.
Placebo s.c./CFZ533 3 mg/kg s.c.
CFZ533 10 mg/kg i.v.
Placebo i.v./CFZ533 10 mg/kg i.v.
Overall Number of Participants Analyzed 8 4 21 11
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 6 participants 4 participants 20 participants 11 participants
46.838  (7.8986) 43.118  (16.3701) 37.071  (12.2914) 39.512  (15.4212)
Week 12 Number Analyzed 8 participants 4 participants 18 participants 11 participants
48.076  (12.5197) 43.660  (13.9997) 44.688  (10.2469) 43.785  (13.2982)
Change from Baseline to Week 12 Number Analyzed 6 participants 4 participants 17 participants 11 participants
0.373  (6.3174) 0.543  (4.0309) 8.212  (11.1378) 4.273  (10.7671)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1 CFZ533, Cohort 1 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.986
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.07
Confidence Interval (2-Sided) 95%
-8.49 to 8.35
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 2 CFZ533, Cohort 2 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.175
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 3.83
Confidence Interval (2-Sided) 95%
-1.81 to 9.48
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Change From Baseline in Multidimensional Fatigue Inventory (MFI)
Hide Description The MFI is a patient self-reported outcome measure (questionnaires) to assess fatigue covering the following dimensions: General Fatigue, Physical Fatigue, Mental Fatigue, Reduced Motivation and Reduced Activity. Each dimension has a possible range from 4-20. The reported total score has a range from 20-100. A reduction from baseline in MFI indicates improvement.
Time Frame Baseline and Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
PD Population for Cohort 1 and Cohort 2. Data were not collected from participants in Cohort 3 CFZ533 Arm 1 and Cohort 3 CFZ533 Arm 2.
Arm/Group Title Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo
Hide Arm/Group Description:
CFZ533 3 mg/kg s.c.
Placebo s.c./CFZ533 3 mg/kg s.c.
CFZ533 10 mg/kg i.v.
Placebo i.v./CFZ533 10 mg/kg i.v.
Overall Number of Participants Analyzed 8 4 21 11
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 6 participants 4 participants 20 participants 11 participants
54.1  (16.23) 78.0  (17.80) 70.0  (17.51) 66.2  (17.59)
Week 12 Number Analyzed 8 participants 4 participants 18 participants 11 participants
53.5  (13.96) 69.8  (17.75) 55.2  (16.65) 63.3  (16.99)
Change from Baseline to Week 12 Number Analyzed 6 participants 4 participants 17 participants 11 participants
-0.6  (8.12) -8.3  (8.18) -14.5  (18.09) -2.9  (12.37)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1 CFZ533, Cohort 1 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.807
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.34
Confidence Interval (2-Sided) 95%
-10.48 to 13.15
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 2 CFZ533, Cohort 2 Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.074
Comments [Not Specified]
Method Repeated measures model
Comments with time (as nominal study week), the interaction between time and treatment (all as fixed effects) and with baseline as a covariate
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -9.83
Confidence Interval (2-Sided) 95%
-20.66 to 1.01
Estimation Comments [Not Specified]
Time Frame Cohort 1 and 2: 255 days (30 days post study completion) Cohort 3: 171 days (30 days post study completion)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo Cohort 3 CFZ533 Arm 1 Cohort 3 CFZ533 Arm 2
Hide Arm/Group Description CFZ533 3 mg/kg s.c. Placebo s.c./CFZ533 3 mg/kg s.c. CFZ533 10 mg/kg i.v. Placebo i.v./CFZ533 10 mg/kg i.v. CFZ533 600 mg s.c./CFZ533 300 mg s.c. CFZ533 10 mg/kg i.v./CFZ533 300 mg s.c.
All-Cause Mortality
Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo Cohort 3 CFZ533 Arm 1 Cohort 3 CFZ533 Arm 2
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/8 (0.00%)   0/4 (0.00%)   0/21 (0.00%)   0/11 (0.00%)   0/13 (0.00%)   0/12 (0.00%) 
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Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo Cohort 3 CFZ533 Arm 1 Cohort 3 CFZ533 Arm 2
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/8 (12.50%)   0/4 (0.00%)   1/21 (4.76%)   0/11 (0.00%)   0/13 (0.00%)   1/12 (8.33%) 
Cardiac disorders             
Atrial fibrillation  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Infections and infestations             
Conjunctivitis bacterial  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Injury, poisoning and procedural complications             
Post procedural swelling  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Procedural pain  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Musculoskeletal and connective tissue disorders             
Haemarthrosis  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
1
Term from vocabulary, MedDRA (21.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Cohort 1 CFZ533 Cohort 1 Placebo Cohort 2 CFZ533 Cohort 2 Placebo Cohort 3 CFZ533 Arm 1 Cohort 3 CFZ533 Arm 2
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   8/8 (100.00%)   4/4 (100.00%)   11/21 (52.38%)   7/11 (63.64%)   12/13 (92.31%)   12/12 (100.00%) 
Blood and lymphatic system disorders             
Anaemia  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Increased tendency to bruise  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Iron deficiency anaemia  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Lymphadenopathy  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Lymphopenia  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Cardiac disorders             
Palpitations  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Ear and labyrinth disorders             
Cerumen impaction  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Deafness  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Hypoacusis  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Tinnitus  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Eye disorders             
Blepharitis  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Cataract  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Diplopia  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Dry eye  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Eye pain  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Ocular hyperaemia  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Vitreous detachment  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Vitreous floaters  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Gastrointestinal disorders             
Abdominal discomfort  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Abdominal distension  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Abdominal pain upper  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Constipation  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  2/12 (16.67%) 
Diarrhoea  1  1/8 (12.50%)  1/4 (25.00%)  2/21 (9.52%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Dyspepsia  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  2/12 (16.67%) 
Dysphagia  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Gastrooesophageal reflux disease  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Nausea  1  1/8 (12.50%)  1/4 (25.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Parotid gland enlargement  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Salivary gland enlargement  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Tongue ulceration  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Toothache  1  1/8 (12.50%)  1/4 (25.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Vomiting  1  2/8 (25.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
General disorders             
Chills  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Cyst  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Fatigue  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Inflammation  1  1/8 (12.50%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Injection site bruising  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Injection site erythema  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Injection site haematoma  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  2/13 (15.38%)  0/12 (0.00%) 
Injection site reaction  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Nodule  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Non-cardiac chest pain  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Peripheral swelling  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  1/12 (8.33%) 
Vessel puncture site bruise  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Immune system disorders             
Drug hypersensitivity  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Seasonal allergy  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Infections and infestations             
Angular cheilitis  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Bacterial infection  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Body tinea  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Bronchitis  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  2/12 (16.67%) 
Candida infection  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Cellulitis  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Conjunctivitis  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Conjunctivitis viral  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Cystitis  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Ear infection  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Gastroenteritis viral  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Gingivitis  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Herpes zoster  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Influenza  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Localised infection  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Lower respiratory tract infection  1  2/8 (25.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Lymph gland infection  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Nail bed infection  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Nasopharyngitis  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  2/11 (18.18%)  1/13 (7.69%)  4/12 (33.33%) 
Oral herpes  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  2/12 (16.67%) 
Otitis media  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Paronychia  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Pharyngitis  1  0/8 (0.00%)  1/4 (25.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Rhinitis  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Sinusitis  1  0/8 (0.00%)  1/4 (25.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Skin infection  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Tonsillitis  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Tooth infection  1  0/8 (0.00%)  1/4 (25.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Upper respiratory tract infection  1  2/8 (25.00%)  2/4 (50.00%)  2/21 (9.52%)  2/11 (18.18%)  4/13 (30.77%)  2/12 (16.67%) 
Urinary tract infection  1  2/8 (25.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  2/12 (16.67%) 
Urogenital infection bacterial  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Viral upper respiratory tract infection  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Injury, poisoning and procedural complications             
Arthropod bite  1  1/8 (12.50%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Contusion  1  0/8 (0.00%)  0/4 (0.00%)  2/21 (9.52%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Corneal abrasion  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Epicondylitis  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Fall  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Incision site hypoaesthesia  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Limb injury  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Post procedural swelling  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Procedural dizziness  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Procedural nausea  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Procedural pain  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Skin abrasion  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Spinal compression fracture  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Tendon injury  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Tooth fracture  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Investigations             
Blood alkaline phosphatase increased  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Blood pressure increased  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Body temperature increased  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
C-reactive protein increased  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Electrocardiogram abnormal  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Gamma-glutamyltransferase increased  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Intraocular pressure increased  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Lipase increased  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  2/11 (18.18%)  0/13 (0.00%)  0/12 (0.00%) 
Lymphocyte count decreased  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Red blood cells urine positive  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
White blood cell count decreased  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
White blood cells urine positive  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Metabolism and nutrition disorders             
Appetite disorder  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Dehydration  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  1/12 (8.33%) 
Musculoskeletal and connective tissue disorders             
Arthralgia  1  2/8 (25.00%)  1/4 (25.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  2/12 (16.67%) 
Arthritis  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Back pain  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Joint range of motion decreased  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Joint stiffness  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Myalgia  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Neck pain  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Osteoarthritis  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  1/13 (7.69%)  0/12 (0.00%) 
Plantar fasciitis  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Sjogren's syndrome  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)             
Abdominal wall neoplasm  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Nervous system disorders             
Amnesia  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Carpal tunnel syndrome  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Dizziness  1  2/8 (25.00%)  1/4 (25.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Headache  1  0/8 (0.00%)  1/4 (25.00%)  2/21 (9.52%)  1/11 (9.09%)  2/13 (15.38%)  4/12 (33.33%) 
Hemianopia homonymous  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Paraesthesia  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Syncope  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Tremor  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Psychiatric disorders             
Abnormal dreams  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Anxiety  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Depressed mood  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Depression  1  0/8 (0.00%)  1/4 (25.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Insomnia  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Reproductive system and breast disorders             
Amenorrhoea  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Breast cyst  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Endometrial disorder  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Menstruation irregular  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Polymenorrhoea  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Uterine pain  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Uterine prolapse  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Respiratory, thoracic and mediastinal disorders             
Allergic sinusitis  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Asthma  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Cough  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Dry throat  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Dysphonia  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Hyperventilation  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Nasal congestion  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Nasal dryness  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Oropharyngeal pain  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  1/11 (9.09%)  1/13 (7.69%)  0/12 (0.00%) 
Rhinalgia  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
Skin and subcutaneous tissue disorders             
Alopecia  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Cutaneous vasculitis  1  0/8 (0.00%)  1/4 (25.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Dermatitis allergic  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Erythema  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Hyperhidrosis  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  1/12 (8.33%) 
Onychoclasis  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Photosensitivity reaction  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Pruritus  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  1/13 (7.69%)  0/12 (0.00%) 
Rash  1  2/8 (25.00%)  1/4 (25.00%)  1/21 (4.76%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Rash macular  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  1/11 (9.09%)  0/13 (0.00%)  0/12 (0.00%) 
Rosacea  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Swelling face  1  1/8 (12.50%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Urticaria  1  0/8 (0.00%)  0/4 (0.00%)  1/21 (4.76%)  0/11 (0.00%)  0/13 (0.00%)  0/12 (0.00%) 
Vascular disorders             
Hypotension  1  0/8 (0.00%)  0/4 (0.00%)  0/21 (0.00%)  0/11 (0.00%)  0/13 (0.00%)  1/12 (8.33%) 
1
Term from vocabulary, MedDRA (21.0)
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.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
EMail: Novartis.email@novartis.com
Layout table for additonal information
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT02291029    
Other Study ID Numbers: CCFZ533X2203
First Submitted: November 4, 2014
First Posted: November 14, 2014
Results First Submitted: June 28, 2019
Results First Posted: August 14, 2019
Last Update Posted: August 14, 2019