Trial Outcomes & Findings for A Study of Subcutaneous (SC) Tocilizumab (RoActemra/Actemra) in Participants With Active Rheumatoid Arthritis (RA) and Inadequate Response to Disease-Modifying Anti-Rheumatic Drugs (DMARDs) (NCT NCT02046616)

NCT ID: NCT02046616

Last Updated: 2018-04-13

Results Overview

CDAI was derived as the sum of the following: tender joint count (TJC), swollen joint count (SJC), participant global assessment (PGA) of disease activity, and physician assessment of disease activity. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA and physician assessment of disease activity were scored 0-100 millimeters (mm) and rounded to the nearest centimeter (cm) on a visual analog scale (VAS), where higher scores indicate greater perceived disease activity. The total CDAI score range was 0-76, where higher scores indicate increased disease activity. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA disease activity.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

133 participants

Primary outcome timeframe

Baseline, Week 12

Results posted on

2018-04-13

Participant Flow

One hundred thirty-three participants entered the 24-week Treatment Period. Those who completed treatment entered the Follow-Up (FU) Period for an additional 8 weeks.

Participant milestones

Participant milestones
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 milligrams (mg), irrespective of body weight, administered subcutaneously weekly (QW) for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
24-Week Treatment Period
STARTED
133
24-Week Treatment Period
COMPLETED
114
24-Week Treatment Period
NOT COMPLETED
19
8-Week FU Period
STARTED
114
8-Week FU Period
COMPLETED
113
8-Week FU Period
NOT COMPLETED
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 milligrams (mg), irrespective of body weight, administered subcutaneously weekly (QW) for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
24-Week Treatment Period
Anaphylaxis/Serious Hypersensitivity
1
24-Week Treatment Period
Any Other Adverse Event
13
24-Week Treatment Period
Insufficient Therapeutic Response
1
24-Week Treatment Period
Physician Decision
3
24-Week Treatment Period
Other
1
8-Week FU Period
Lost to Follow-up
1

Baseline Characteristics

A Study of Subcutaneous (SC) Tocilizumab (RoActemra/Actemra) in Participants With Active Rheumatoid Arthritis (RA) and Inadequate Response to Disease-Modifying Anti-Rheumatic Drugs (DMARDs)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Age, Continuous
55.9 years
STANDARD_DEVIATION 12.0 • n=5 Participants
Sex: Female, Male
Female
108 Participants
n=5 Participants
Sex: Female, Male
Male
25 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 12

Population: ITT Set; only those patients who provided data at baseline and at least one post-baseline assessment were analyzed.

CDAI was derived as the sum of the following: tender joint count (TJC), swollen joint count (SJC), participant global assessment (PGA) of disease activity, and physician assessment of disease activity. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA and physician assessment of disease activity were scored 0-100 millimeters (mm) and rounded to the nearest centimeter (cm) on a visual analog scale (VAS), where higher scores indicate greater perceived disease activity. The total CDAI score range was 0-76, where higher scores indicate increased disease activity. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA disease activity.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Change From Baseline in Clinical Disease Activity Index (CDAI) at Week 12
Baseline
24.9 units on a scale
Standard Deviation 10.5
Change From Baseline in Clinical Disease Activity Index (CDAI) at Week 12
Change at Week 12
-16.6 units on a scale
Standard Deviation 12.1

SECONDARY outcome

Timeframe: Baseline and Weeks 2, 4, 8, 12, 16, 20, 24

Population: ITT Set; only those patients who provided data at baseline and at least one post-baseline assessment were analyzed.

DAS28-ESR was based on TJC, SJC, PGA of disease activity, and laboratory-derived ESR. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA of disease activity was scored 0-100 mm on a VAS, where higher scores indicate greater perceived disease activity. The total DAS28-ESR score was transformed to a single score range of 0-10, where higher scores indicate increased disease activity. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA disease activity.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Change From Baseline in Disease Activity Score 28 (DAS28)-Erythrocyte Sedimentation Rate (ESR) Score
Baseline
5.0 units on a scale
Standard Deviation 1.1
Change From Baseline in Disease Activity Score 28 (DAS28)-Erythrocyte Sedimentation Rate (ESR) Score
Change at Week 2
-1.4 units on a scale
Standard Deviation 1.0
Change From Baseline in Disease Activity Score 28 (DAS28)-Erythrocyte Sedimentation Rate (ESR) Score
Change at Week 4
-2.1 units on a scale
Standard Deviation 1.1
Change From Baseline in Disease Activity Score 28 (DAS28)-Erythrocyte Sedimentation Rate (ESR) Score
Change at Week 8
-2.8 units on a scale
Standard Deviation 1.3
Change From Baseline in Disease Activity Score 28 (DAS28)-Erythrocyte Sedimentation Rate (ESR) Score
Change at Week 12
-2.9 units on a scale
Standard Deviation 1.5
Change From Baseline in Disease Activity Score 28 (DAS28)-Erythrocyte Sedimentation Rate (ESR) Score
Change at Week 16
-3.2 units on a scale
Standard Deviation 1.4
Change From Baseline in Disease Activity Score 28 (DAS28)-Erythrocyte Sedimentation Rate (ESR) Score
Change at Week 20
-3.3 units on a scale
Standard Deviation 1.4
Change From Baseline in Disease Activity Score 28 (DAS28)-Erythrocyte Sedimentation Rate (ESR) Score
Change at Week 24
-3.3 units on a scale
Standard Deviation 1.4

SECONDARY outcome

Timeframe: Weeks 2, 4, 8, 12, 16, 20, 24

Population: ITT Set

ACR response was assessed on the basis of percent improvement (20% for ACR20, 50% for ACR50, 70% for ACR70) in both TJC and SJC as well as at least three of the following: physician assessment of disease activity, PGA of disease activity, PGA of pain, Health Assessment Questionnaire-Disability Index (HAQ-DI), and either ESR or C-reactive protein level. TJC and SJC were taken as the number of tender and swollen joints, out of 68 and 66 assessed joints, respectively. PGA and physician assessments were scored 0-100 mm on a VAS, where higher scores indicate greater perceived disease activity or pain. HAQ-DI was scored using participant responses to 20 questions assessing activities of daily living (ADLs), with total score scale of 0-3, where higher scores indicate increased functional disability. The percentage of participants meeting criteria for each level of ACR response was reported.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR50, Week 20
64.9 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR20, Week 2
25.2 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR20, Week 4
51.5 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR20, Week 8
70.4 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR20, Week 12
70.8 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR20, Week 16
82.5 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR20, Week 20
77.2 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR20, Week 24
78.1 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR50, Week 2
8.7 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR50, Week 4
22.3 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR50, Week 8
46.4 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR50, Week 12 (n=120)
51.7 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR50, Week 16
65.8 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR50, Week 24
63.2 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR70, Week 2
0.8 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR70, Week 4
8.5 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR70, Week 8
24.0 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR70, Week 12
30.0 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR70, Week 16
44.2 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR70, Week 20
42.1 percentage of participants
Percentage of Participants With American College of Rheumatology (ACR) Response
ACR70, Week 24
47.4 percentage of participants

SECONDARY outcome

Timeframe: Weeks 2, 4, 8, 12, 16, 20, 24

Population: ITT Set

EULAR response was assessed by change from baseline and absolute DAS28-ESR score. EULAR response classification was as follows: Good (change \>1.2 with absolute score \</=3.2), Moderate (change \>1.2 with absolute score \>3.2 or change \>0.6 with absolute score \</=5.1), None (change \</=0.6 or absolute score \>5.1). DAS28-ESR was based on TJC, SJC, and PGA of disease activity, and laboratory-derived ESR. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA of disease activity was scored 0-100 mm on a VAS, where higher scores indicate greater perceived disease activity. The total DAS28-ESR score was transformed to a single score range of 0-10, where higher scores indicate increased disease activity. The percentage of participants meeting criteria for each level of EULAR response was reported.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 24, Moderate
9.6 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 24, None
2.6 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 2, Good
29.1 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 2, Moderate
47.2 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 2, None
23.6 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 4, Good
51.2 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 4, Moderate
39.5 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 4, None
9.3 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 8, Good
78.4 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 8, Moderate (n=125)
16.8 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 8, None
4.8 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 12, Good (n=119)
78.2 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 12, Moderate
16.0 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 12, None
5.9 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 16, Good
87.5 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 16, Moderate
9.2 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 16, None
3.3 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 20, Good
86.7 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 20, Moderate
8.8 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 20, None
4.4 percentage of participants
Percentage of Participants With European League Against Rheumatism (EULAR) Response
Week 24, Good
87.7 percentage of participants

SECONDARY outcome

Timeframe: Baseline and Weeks 2, 4, 8, 16, 20, 24

Population: ITT Set; only those patients who provided data at baseline and at least one post-baseline assessment were analyzed.

CDAI was derived as the sum of the following: TJC, SJC, PGA of disease activity, and physician assessment of disease activity. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA and physician assessment of disease activity were scored 0-100 mm and rounded to the nearest cm on a VAS, where higher scores indicate greater perceived disease activity. The total CDAI score range was 0-76, where higher scores indicate increased disease activity. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA disease activity.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Change From Baseline in CDAI at Weeks 2, 4, 8, 16, 20, and 24
Change at Week 2
-6.2 units on a scale
Standard Deviation 8.4
Change From Baseline in CDAI at Weeks 2, 4, 8, 16, 20, and 24
Change at Week 4
-10.5 units on a scale
Standard Deviation 9.8
Change From Baseline in CDAI at Weeks 2, 4, 8, 16, 20, and 24
Change at Week 8
-15.7 units on a scale
Standard Deviation 11.0
Change From Baseline in CDAI at Weeks 2, 4, 8, 16, 20, and 24
Change at Week 16
-18.8 units on a scale
Standard Deviation 11.5
Change From Baseline in CDAI at Weeks 2, 4, 8, 16, 20, and 24
Change at Week 20
-18.9 units on a scale
Standard Deviation 11.7
Change From Baseline in CDAI at Weeks 2, 4, 8, 16, 20, and 24
Change at Week 24
-19.0 units on a scale
Standard Deviation 11.7

SECONDARY outcome

Timeframe: Baseline and Weeks 2, 4, 8, 12, 16, 20, 24

Population: ITT Set; only those patients who provided data at baseline and at least one post-baseline assessment were analyzed.

SDAI was derived as the sum of the following: TJC, SJC, PGA of disease activity, physician assessment of disease activity, and laboratory-derived C-reactive protein level. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA and physician assessment of disease activity were scored 0-100 mm and rounded to the nearest cm on a VAS, where higher scores indicate greater perceived disease activity. The total SDAI score range was 0-86, where higher scores indicate increased disease activity. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA disease activity.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=132 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Change From Baseline in Simplified Disease Activity Index (SDAI)
Baseline
35.76 units on a scale
Standard Deviation 20.71
Change From Baseline in Simplified Disease Activity Index (SDAI)
Change at Week 2
-15.37 units on a scale
Standard Deviation 16.71
Change From Baseline in Simplified Disease Activity Index (SDAI)
Change at Week 4
-20.32 units on a scale
Standard Deviation 19.24
Change From Baseline in Simplified Disease Activity Index (SDAI)
Change at Week 8
-25.89 units on a scale
Standard Deviation 20.88
Change From Baseline in Simplified Disease Activity Index (SDAI)
Change at Week 12
-26.19 units on a scale
Standard Deviation 23.21
Change From Baseline in Simplified Disease Activity Index (SDAI)
Change at Week 16
-29.03 units on a scale
Standard Deviation 21.15
Change From Baseline in Simplified Disease Activity Index (SDAI)
Change at Week 20
-28.47 units on a scale
Standard Deviation 21.26
Change From Baseline in Simplified Disease Activity Index (SDAI)
Change at Week 24
-28.93 units on a scale
Standard Deviation 20.93

SECONDARY outcome

Timeframe: Baseline and Weeks 2, 4, 8, 12, 16, 20, 24

Population: ITT Set; only those patients who provided data at baseline and at least one post-baseline assessment were analyzed.

TJC was taken as the number of tender joints out of 28 assessed joints.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=28 Joints
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Change From Baseline in TJC
Baseline
8.8 tender joints
Standard Deviation 5.2
Change From Baseline in TJC
Change at Week 2
-2.1 tender joints
Standard Deviation 5.8
Change From Baseline in TJC
Change at Week 4
-3.5 tender joints
Standard Deviation 6.1
Change From Baseline in TJC
Change at Week 8
-5.8 tender joints
Standard Deviation 6.3
Change From Baseline in TJC
Change at Week 12
-5.5 tender joints
Standard Deviation 7.1
Change From Baseline in TJC
Change at Week 16
-6.7 tender joints
Standard Deviation 6.9
Change From Baseline in TJC
Change at Week 20
-6.7 tender joints
Standard Deviation 6.5
Change From Baseline in TJC
Change at Week 24
-7.1 tender joints
Standard Deviation 5.8

SECONDARY outcome

Timeframe: Baseline and Weeks 2, 4, 8, 12, 16, 20, 24

Population: ITT Set; only those patients who provided data at baseline and at least one post-baseline assessment were analyzed.

SJC was taken as the number of swollen joints out of 28 assessed joints.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=28 Joints
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Change From Baseline in SJC
Baseline
6.8 swollen joints
Standard Deviation 5.5
Change From Baseline in SJC
Change at Week 2
-2.3 swollen joints
Standard Deviation 4.3
Change From Baseline in SJC
Change at Week 4
-3.1 swollen joints
Standard Deviation 4.7
Change From Baseline in SJC
Change at Week 8
-4.8 swollen joints
Standard Deviation 5.1
Change From Baseline in SJC
Change at Week 12
-5.3 swollen joints
Standard Deviation 5.3
Change From Baseline in SJC
Change at Week 16
-5.9 swollen joints
Standard Deviation 5.2
Change From Baseline in SJC
Change at Week 20
-6.0 swollen joints
Standard Deviation 5.4
Change From Baseline in SJC
Change at Week 24
-5.4 swollen joints
Standard Deviation 5.3

SECONDARY outcome

Timeframe: Baseline up to Week 24

Population: ITT Set

The percentage of participants with at least one adverse event leading to dose/frequency reduction or temporary dose hold was reported.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Percentage of Participants With At Least One Adverse Event Leading to Dosage Modification
Dose/Frequency Reduced Due to Adverse Event
18.80 percentage of participants
Percentage of Participants With At Least One Adverse Event Leading to Dosage Modification
Dose Held Due to Adverse Event
27.07 percentage of participants

SECONDARY outcome

Timeframe: Baseline to FU Week 8 (up to 32 weeks overall)

Population: ITT Set

Participants were evaluated for the presence of anti-tocilizumab antibodies. Confirmatory assays were performed in the case of a positive screen assay result.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=132 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Number of Participants With Neutralizing Anti-Tocilizumab Antibodies
1 participants

SECONDARY outcome

Timeframe: Predose (30 minutes) at baseline; Weeks 12, 24; and FU Week 8 (up to 32 weeks overall)

Population: ITT Set. The Analysis was conducted on those participants with quantifiable tocilizumab concentration at the specified assessment.

Tocilizumab concentration was determined, averaged among all participants, and expressed in micrograms per milliliter (mcg/mL).

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Tocilizumab Concentration
Baseline
0.6 mcg/mL
Standard Deviation 0.3
Tocilizumab Concentration
Week 12
47.4 mcg/mL
Standard Deviation 28.1
Tocilizumab Concentration
Week 24
48.0 mcg/mL
Standard Deviation 27.2
Tocilizumab Concentration
FU Week 8
32.8 mcg/mL
Standard Deviation 19.3

SECONDARY outcome

Timeframe: Predose (30 minutes) at baseline; Weeks 12, 24; and FU Week 8 (up to 32 weeks overall)

Population: ITT Set

sIL-6R concentration was determined, averaged among all participants, and expressed in nanograms per milliliter (ng/mL).

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Soluble Interleukin-6 Receptor (sIL-6R) Concentration
Baseline
37.0 ng/mL
Standard Deviation 12.7
Soluble Interleukin-6 Receptor (sIL-6R) Concentration
Week 12
509.4 ng/mL
Standard Deviation 138.7
Soluble Interleukin-6 Receptor (sIL-6R) Concentration
Week 24
520.2 ng/mL
Standard Deviation 156.4
Soluble Interleukin-6 Receptor (sIL-6R) Concentration
FU Week 8
166.0 ng/mL
Standard Deviation 203.5

SECONDARY outcome

Timeframe: Baseline and Weeks 2, 4, 8, 12, 16, 20, 24

Population: ITT Set; only those patients who provided data at baseline and at least one post-baseline assessment were analyzed.

PGA of disease activity was scored 0-100 mm on a VAS, where higher scores indicate greater perceived disease activity. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA disease activity.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Change From Baseline in Patient Global Assessment of Disease Activity According to VAS
Baseline
53.2 mm
Standard Deviation 21.1
Change From Baseline in Patient Global Assessment of Disease Activity According to VAS
Change at Week 2
-6.8 mm
Standard Deviation 18.6
Change From Baseline in Patient Global Assessment of Disease Activity According to VAS
Change at Week 4
-20.3 mm
Standard Deviation 21.3
Change From Baseline in Patient Global Assessment of Disease Activity According to VAS
Change at Week 8
-24.6 mm
Standard Deviation 25.8
Change From Baseline in Patient Global Assessment of Disease Activity According to VAS
Change at Week 12
-30.3 mm
Standard Deviation 25.5
Change From Baseline in Patient Global Assessment of Disease Activity According to VAS
Change at Week 16
-32.3 mm
Standard Deviation 23.9
Change From Baseline in Patient Global Assessment of Disease Activity According to VAS
Change at Week 20
-32.2 mm
Standard Deviation 27.1
Change From Baseline in Patient Global Assessment of Disease Activity According to VAS
Change at Week 24
-34.3 mm
Standard Deviation 24.8

SECONDARY outcome

Timeframe: Baseline and Weeks 2, 4, 8, 12, 16, 20, 24

Population: ITT Set; only those patients who provided data at baseline and at least one post-baseline assessment were analyzed.

PGA of RA-related pain was scored 0-100 mm on a VAS, where higher scores indicate greater perceived pain. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA-related pain.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=132 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Change From Baseline in Patient Global Assessment of RA-Related Pain According to VAS
Baseline
54.8 mm
Standard Deviation 22.1
Change From Baseline in Patient Global Assessment of RA-Related Pain According to VAS
Change at Week 2
-9.1 mm
Standard Deviation 20.8
Change From Baseline in Patient Global Assessment of RA-Related Pain According to VAS
Change at Week 4 (n=128)
-22.5 mm
Standard Deviation 24.4
Change From Baseline in Patient Global Assessment of RA-Related Pain According to VAS
Change at Week 8
-28.9 mm
Standard Deviation 26.6
Change From Baseline in Patient Global Assessment of RA-Related Pain According to VAS
Change at Week 12
-32.8 mm
Standard Deviation 27.5
Change From Baseline in Patient Global Assessment of RA-Related Pain According to VAS
Change at Week 16
-35.6 mm
Standard Deviation 25.9
Change From Baseline in Patient Global Assessment of RA-Related Pain According to VAS
Change at Week 20
-35.0 mm
Standard Deviation 26.7
Change From Baseline in Patient Global Assessment of RA-Related Pain According to VAS
Change at Week 24
-37.8 mm
Standard Deviation 26.3

SECONDARY outcome

Timeframe: Baseline and Weeks 2, 4, 8, 12, 16, 20, 24

Population: ITT Set; only those patients who provided data at baseline and at least one post-baseline assessment were analyzed.

HAQ-DI consisted of 20 questions assessing ADLs in 8 domains (dress/groom, arise, eat, walk, reach, grip, hygiene) with each item rated 0 (no difficulty) to 3 (unable to do). The highest score recorded for any question in a domain determined the score for that domain, unless assistance was required. The total HAQ-DI score was the sum of domain scores divided by the number of domains answered/scored, for a single score range of 0-3, where higher scores indicate increased functional disability. Change from baseline was averaged among all participants. Negative values indicate improvement in ability to perform ADLs.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Change From Baseline in HAQ-DI Score
Baseline (n=133)
1.2 units on a scale
Standard Deviation 0.6
Change From Baseline in HAQ-DI Score
Change at Week 2
-0.1 units on a scale
Standard Deviation 0.4
Change From Baseline in HAQ-DI Score
Change at Week 4
-0.3 units on a scale
Standard Deviation 0.5
Change From Baseline in HAQ-DI Score
Change at Week 8
-0.5 units on a scale
Standard Deviation 0.5
Change From Baseline in HAQ-DI Score
Change at Week 12
-0.5 units on a scale
Standard Deviation 0.6
Change From Baseline in HAQ-DI Score
Change at Week 16
-0.6 units on a scale
Standard Deviation 0.6
Change From Baseline in HAQ-DI Score
Change at Week 20
-0.6 units on a scale
Standard Deviation 0.6
Change From Baseline in HAQ-DI Score
Change at Week 24
-0.6 units on a scale
Standard Deviation 0.6

SECONDARY outcome

Timeframe: Baseline up to Week 24

Population: ITT Set

Participants were provided with diary cards to record home injections. Compliance with treatment was calculated individually for each participant as the actual number of injections as a percentage of the planned number of injections (up to the point of discontinuation for those who discontinued study treatment prematurely) and then averaged among all participants.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Compliance With Treatment According to Percentage of Injections Administered
86.78 percentage of injections
Standard Deviation 23.04 • Interval 4.2 to 108.3

SECONDARY outcome

Timeframe: Baseline and Weeks 2, 4, 8, 12, 16, 20, 24

Population: ITT Set; only those patients who provided data at baseline and at least one post-baseline assessment were analyzed.

FACIT-F consisted of 40 questions/statements assessing chronic illness therapy with special emphasis on fatigue over the past 7 days, with each item rated 0 (not at all) to 4 (very much). During score calculations, negatively-worded item scales (e.g., "I have a lack of energy") were reversed so that higher scores indicated more favorable conditions. The total FACIT-F score was the sum of all item scores and ranged 0-160, and the brief FACIT-F score was the sum of 13 item scores and ranged 0-52, where higher scores indicate greater well-being. Change from baseline was averaged among all participants. Positive values indicate improvement in well-being.

Outcome measures

Outcome measures
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 Participants
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Brief Score, Baseline (n=133)
32.9 units on a scale
Standard Deviation 11.1
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Brief Score, Change at Week 2
3.4 units on a scale
Standard Deviation 6.9
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Brief Score, Change at Week 4
5.7 units on a scale
Standard Deviation 8.3
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Brief Score, Change at Week 8
6.6 units on a scale
Standard Deviation 8.0
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Brief Score, Change at Week 12
7.6 units on a scale
Standard Deviation 7.8
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Brief Score, Change at Week 16
7.9 units on a scale
Standard Deviation 9.7
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Brief Score, Change at Week 20
8.0 units on a scale
Standard Deviation 9.1
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Total Score, Change at Week 2
6.9 units on a scale
Standard Deviation 14.0
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Total Score, Change at Week 4
13.0 units on a scale
Standard Deviation 16.6
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Total Score, Change at Week 16
18.0 units on a scale
Standard Deviation 20.1
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Total Score, Change at Week 20
18.6 units on a scale
Standard Deviation 19.8
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Total Score, Change at Week 24
20.1 units on a scale
Standard Deviation 19.3
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Brief Score, Change at Week 24
8.4 units on a scale
Standard Deviation 8.5
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Total Score, Baseline
106.8 units on a scale
Standard Deviation 23.6
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Total Score, Change at Week 8
15.1 units on a scale
Standard Deviation 16.2
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Score
Total Score, Change at Week 12
17.1 units on a scale
Standard Deviation 16.2

Adverse Events

Tocilizumab Alone or Combined With Methotrexate or Other DMARD

Serious events: 12 serious events
Other events: 120 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 participants at risk
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Psychiatric disorders
Acute psychosis
0.75%
1/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Immune system disorders
Anaphylactic reaction
0.75%
1/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Musculoskeletal and connective tissue disorders
Back pain
0.75%
1/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Gastrointestinal disorders
Constipation
0.75%
1/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Gastrointestinal disorders
Gastrooesophageal reflux disease
0.75%
1/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Nervous system disorders
Headache
0.75%
1/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Vascular disorders
Hypertension
1.5%
2/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Infections and infestations
Infectious pleural effusion
0.75%
1/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lung adenocarcinoma
0.75%
1/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Infections and infestations
Pneumonia
1.5%
2/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Rectal cancer
0.75%
1/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Nervous system disorders
Transient ischaemic attack
0.75%
1/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set

Other adverse events

Other adverse events
Measure
Tocilizumab Alone or Combined With Methotrexate or Other DMARD
n=133 participants at risk
All participants received tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs at a dose of 162 mg, irrespective of body weight, administered subcutaneously QW for 24 weeks. An additional 8 weeks were allotted for post-treatment evaluation of safety/immunogenicity.
Gastrointestinal disorders
Nausea
8.3%
11/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
General disorders
Injection site erythema
5.3%
7/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Infections and infestations
Nasopharyngitis
15.0%
20/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Infections and infestations
Upper respiratory tract infection
7.5%
10/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Investigations
Alanine aminotransferase increased
6.8%
9/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Investigations
Neutrophil count decreased
5.3%
7/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Musculoskeletal and connective tissue disorders
Back pain
5.3%
7/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Nervous system disorders
Dizziness
5.3%
7/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Nervous system disorders
Headache
6.8%
9/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Skin and subcutaneous tissue disorders
Pruritus
6.0%
8/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Skin and subcutaneous tissue disorders
Rash
7.5%
10/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set
Vascular disorders
Hypertension
12.8%
17/133 • Baseline up to FU Week 8 (up to 32 weeks overall)
ITT Set

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 800-821-8590

Results disclosure agreements

  • Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/ or the publication be postponed in order to protect the Sponsor's intellectual property rights.
  • Publication restrictions are in place

Restriction type: OTHER