Trial Outcomes & Findings for An Observational Study of Tocilizumab (Actemra) in Participants With Moderate to Severe Rheumatoid Arthritis (NCT NCT01741688)

NCT ID: NCT01741688

Last Updated: 2017-08-01

Results Overview

• Recruitment status: COMPLETED
• Target enrollment: 16 participants
• Primary outcome timeframe: At 6 months
• Results posted on: 2017-08-01

Participant Flow

Participant milestones

Measure	Tocilizumab Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Overall Study STARTED	16
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	Tocilizumab Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Overall Study Lack of insurance coverage	2
Overall Study Withdrawal by Subject	1

Baseline Characteristics

An Observational Study of Tocilizumab (Actemra) in Participants With Moderate to Severe Rheumatoid Arthritis

Baseline characteristics by cohort

Measure	Tocilizumab n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Age, Continuous	55 years n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment Peru	16 participants n=5 Participants

PRIMARY outcome

Timeframe: At 6 months

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Number of Participants on Tocilizumab at 6 Months After Treatment Initiation	14 participants

SECONDARY outcome

Timeframe: At baseline

Systemic manifestation measured by C-reactive protein levels > 3

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Percentage of Participants With Systemic Manifestations of Rheumatoid Arthritis	93.8 percentage of participants

SECONDARY outcome

Timeframe: At baseline

DMARDs exposure was evaluated for all participants. "Prior DMARDs treatment" includes participants, who were treated with DMARDs 6 months before being included in the study. "DMARDs treatment at baseline" includes participants, who were receiving DMARDs when they were included in the study and continued with this concomitant medication to tocilizumab.

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Percentage of Participants Starting Tocilizumab After Prior and Baseline Disease-Modifying Anti-rheumatic Drugs (DMARDs) Exposure Prior DMARDs treatment	18.8 percentage of participants
Percentage of Participants Starting Tocilizumab After Prior and Baseline Disease-Modifying Anti-rheumatic Drugs (DMARDs) Exposure DMARDs treatment at baseline	62.5 percentage of participants

SECONDARY outcome

Timeframe: At baseline

Other biologic agents include anti-Tumor Necrosis Factor (TNF) antibody.

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Number of Participants Starting Tocilizumab After Failing Other Biologic Agents	1 participants

SECONDARY outcome

Timeframe: At 6 months

Population: Number of participants remaining in the study at 6 months

Outcome measures

Measure	Cohort n=14 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Median Dose at 6 Months	8 mg/kg of body weight Interval 6.3 to 9.2

SECONDARY outcome

Timeframe: At 6 months

Outcome measures

Measure	Cohort n=14 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Number of Participants With Dose Modifications at 6 Months	4 participants

SECONDARY outcome

Timeframe: Approximately 16 months

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Median Duration of Treatment	6 months Interval 3.4 to 6.8

SECONDARY outcome

Timeframe: Approximately 16 months

Safety variable measuring number of patients that discontinued tocilizumab due to adverse reactions to tocilizumab.

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Percentage of Participants Discontinued From Tocilizumab for Safety	0 percentage of participants

SECONDARY outcome

Timeframe: Approximately 16 months

Efficacy variable that measures the rate of participants discontinued from tocilizumab due to lack of efficacy according to criteria of treating physician.

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Percentage of Participants Discontinued From Tocilizumab for Lack of Efficacy	0 percentage of participants

SECONDARY outcome

Timeframe: Approximately 16 months

This variable measures the number of events not related to safety or efficacy leading to discontinuation of tocilizumab treatment.

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Number of Participants Discontinued From Tocilizumab for Other Reasons	3 participants

SECONDARY outcome

Timeframe: Approximately 16 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Approximately 16 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: At baseline

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Percentage of Participants on Tocilizumab Monotherapy at Study Entry	12.5 percentage of participants

SECONDARY outcome

Timeframe: At Visit 1 (Baseline), Observation 1: up to 4 weeks, Observation 2: up to 8 weeks; Observation 3: up to 12 weeks; Observation 4: up to 16 weeks and Observation 5: up to 20 weeks

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Total Tender Joint Count (TJC) Visit 1	18 Number of tender joints/participant Interval 12.0 to 22.0
Total Tender Joint Count (TJC) Observation 1	0 Number of tender joints/participant Interval 0.0 to 5.0
Total Tender Joint Count (TJC) Observation 2	0 Number of tender joints/participant Interval 0.0 to 6.0
Total Tender Joint Count (TJC) Observation 3	0 Number of tender joints/participant Interval 0.0 to 0.0
Total Tender Joint Count (TJC) Observation 4	0 Number of tender joints/participant Interval 0.0 to 0.0
Total Tender Joint Count (TJC) Observation 5	0 Number of tender joints/participant Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: At Visit 1 (Baseline), Observation 1: up to 4 weeks, Observation 2: up to 8 weeks; Observation 3: up to 12 weeks; Observation 4: up to 16 weeks and Observation 5: up to 20 weeks

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Total Swollen Joint Count (SJC) Visit 1	18 Number of swollen joints/participant Interval 5.0 to 18.0
Total Swollen Joint Count (SJC) Observation 1	0.5 Number of swollen joints/participant Interval 0.0 to 8.0
Total Swollen Joint Count (SJC) Observation 2	0 Number of swollen joints/participant Interval 0.0 to 6.0
Total Swollen Joint Count (SJC) Observation 3	0 Number of swollen joints/participant Interval 0.0 to 10.0
Total Swollen Joint Count (SJC) Observation 4	0 Number of swollen joints/participant Interval 0.0 to 0.0
Total Swollen Joint Count (SJC) Observation 5	0 Number of swollen joints/participant Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: At Visit 1 (Baseline), Observation 1: up to 4 weeks, Observation 2: up to 8 weeks; Observation 3: up to 12 weeks; Observation 4: up to 16 weeks and Observation 5: up to 20 weeks

The DAS28 is a measure of disease activity in rheumatoid arthritis (RA) and the number 28 refers to the 28 joints that are examined in this assessment. To calculate the DAS28 the following assessments are done: 1) count the number of swollen joints (out of the 28 [sw28]), 2) count the number of tender joints (out of the 28 [t28]), 3) measure Erythrocyte Sedimentation Rate (ESR), and 4) ask the participant to make a 'global assessment of health' (GH) indicated by marking a 10 cm line between very good and very bad.

The Score is developed under the follow formula:

DAS28(4) = 0.56*sqrt(t28) + 0.28*sqrt(sw28) + 0.70*Ln(ESR) + 0.014*GH Where, t=tender joints; sw=swollen joints; ESR= Erythrocyte Sedimentation Rate; GH=Global assessment of health score.

This score may range from 0 to 9.3, where a DAS28 of greater than 5.1 implies active disease, less than 3.2 low disease activity, and less than 2.6 remission.

Outcome measures

Measure	Cohort n=16 Participants Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Disease Activity Score Based on 28 Joints (DAS28) Visit 1	6.29 DAS28 score Interval 4.82 to 6.72
Disease Activity Score Based on 28 Joints (DAS28) Observation 1	2.87 DAS28 score Interval 1.89 to 3.67
Disease Activity Score Based on 28 Joints (DAS28) Observation 2	2.23 DAS28 score Interval 2.02 to 3.38
Disease Activity Score Based on 28 Joints (DAS28) Observation 3	2.16 DAS28 score Interval 1.82 to 2.8
Disease Activity Score Based on 28 Joints (DAS28) Observation 4	2.45 DAS28 score Interval 2.45 to 2.45
Disease Activity Score Based on 28 Joints (DAS28) Observation 5	2.41 DAS28 score Interval 2.41 to 2.41

Adverse Events

Tocilizumab

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Measure

Tocilizumab n=16 participants at risk Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.

Hepatobiliary disorders Cholelithiasis

6.2% 1/16 • Number of events 1 • From baseline to end of study, approximately 16 months.

Other adverse events

Measure	Tocilizumab n=16 participants at risk Participants with moderate to severe rheumatoid arthritis (RA), according to the American College of Rheumatology (ACR) criteria and the Disease Activity Score Based on 28 Joints (DAS28), in whom the attending physician has decided to start treatment with tocilizumab (according to the local label). Tocilizumab: Tocilizumab was administered according to the local label.
Eye disorders Keratitis	6.2% 1/16 • Number of events 1 • From baseline to end of study, approximately 16 months.
Eye disorders Corneal ulcer	6.2% 1/16 • Number of events 1 • From baseline to end of study, approximately 16 months.
Respiratory, thoracic and mediastinal disorders Flu	6.2% 1/16 • Number of events 1 • From baseline to end of study, approximately 16 months.
Nervous system disorders Migraine	6.2% 1/16 • Number of events 1 • From baseline to end of study, approximately 16 months.
Nervous system disorders Headache	6.2% 1/16 • Number of events 1 • From baseline to end of study, approximately 16 months.
Renal and urinary disorders Urinary tract infection	6.2% 1/16 • Number of events 1 • From baseline to end of study, approximately 16 months.

Additional Information

Medical Communications

Hoffmann-LaRoche

Phone: 800-821-8590

Email: genentech@druginfo.com

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 after the first publication or presentation that involves the overall study. 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