Trial Outcomes & Findings for A Non-Interventional Study in Rheumatoid Arthritis Patients Treated With Tocilizumab (RoActemra/Actemra) (NCT NCT01613378)

NCT ID: NCT01613378

Last Updated: 2016-10-04

Results Overview

• Recruitment status: COMPLETED
• Target enrollment: 200 participants
• Primary outcome timeframe: At 6 months
• Results posted on: 2016-10-04

Participant Flow

Participant milestones

Measure	Rheumatoid Arthritis Cohort The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Overall Study STARTED	200
Overall Study COMPLETED	161
Overall Study NOT COMPLETED	39

Reasons for withdrawal

Measure	Rheumatoid Arthritis Cohort The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Overall Study Lack of Efficacy	13
Overall Study Adverse Event	11
Overall Study Withdrawal by Subject	8
Overall Study Death	2
Overall Study Lost to Follow-up	1
Overall Study Other Reason	4

Baseline Characteristics

A Non-Interventional Study in Rheumatoid Arthritis Patients Treated With Tocilizumab (RoActemra/Actemra)

Baseline characteristics by cohort

Measure	Rheumatoid Arthritis Cohort n=200 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Age, Continuous	55.43 years STANDARD_DEVIATION 13.52 • n=5 Participants
Sex: Female, Male Female	160 Participants n=5 Participants
Sex: Female, Male Male	40 Participants n=5 Participants

PRIMARY outcome

Timeframe: At 6 months

Population: All participants enrolled in the study.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=200 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Percentage of Participants on Tocilizumab Treatment at 6 Months After Treatment Initiation	81.5 percentage of participants Interval 75.4 to 86.6

SECONDARY outcome

Timeframe: From baseline to Month 12

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the Tender Joint Count were included in this analysis.

Following an assessment of 68 joints for tenderness, joints were classified as tender or not tender by the investigator.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=146 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Change From Baseline in Tender Joint Count (TJC)	-12.71 tender joints Standard Deviation 12.84

SECONDARY outcome

Timeframe: From baseline to Month 12

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the Swollen Joint Count were included in this analysis.

Following an assessment of 66 joints for swelling, joints were classified as swollen or not swollen by the investigator.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=146 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Change From Baseline in Swollen Joint Count (SJC)	-8.66 swollen joints Standard Deviation 7.67

SECONDARY outcome

Timeframe: From baseline to Month 12

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the DAS28 assessment were included in this analysis.

The DAS28 scale is a combined index for measuring disease activity in rheumatoid arthritis. Scores range from 0 to 10, with higher scores representing more disease activity.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=117 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Change From Baseline in Disease Activity Score 28 (DAS28)	-3.00 units on a scale Standard Deviation 1.73

SECONDARY outcome

Timeframe: From baseline to Month 12

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the assessment of Duration of Morning Stiffness on the VAS were included in this analysis.

With VAS, participants specify their level of agreement to a statement by indicating a position along a continuous line between two endpoints, with 0 being the lowest level and 100 being the highest level of morning stiffness.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=143 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Change From Baseline in Duration of Morning Stiffness (Visual Analog Scale, VAS)	-24.31 units on a scale Standard Deviation 30.04

SECONDARY outcome

Timeframe: From baseline to Month 12

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the Assessment of Disease Activity on the VAS were included in this analysis.

With VAS, participants specify their level of agreement to a statement by indicating a position along a continuous line between two endpoints, with 0 being the lowest level and 100 being the highest level of disease activity.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=144 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Change From Baseline in Patient Global Assessment of Disease Activity (Visual Analog Scale, VAS)	-24.90 units on a scale Standard Deviation 29.30

SECONDARY outcome

Timeframe: From baseline to Month 12

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the Assessment of Disease Activity on the VAS were included in this analysis.

With VAS, physicians specify their level of agreement to a statement by indicating a position along a continuous line between two endpoints, with 0 being the lowest level and 100 being the highest level of disease activity.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=157 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Change From Baseline in Physician Global Assessment of Disease Activity (Visual Analog Scale, VAS)	-39.95 units on a scale Standard Deviation 28.28

SECONDARY outcome

Timeframe: From baseline to Month 12

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the Assessment of Pain on the VAS were included in this analysis.

With VAS, participants specify their level of agreement to a statement by indicating a position along a continuous line between two endpoints, with 0 being the lowest level and 100 being the highest level of pain.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=144 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Change From Baseline in Patient Assessment of Pain (Visual Analog Scale, VAS)	-27.99 units on a scale Standard Deviation 30.47

SECONDARY outcome

Timeframe: From baseline to Month 12

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the Patient Assessment of Fatigue on the VAS were included in this analysis.

With VAS, participants specify their level of agreement to a statement by indicating a position along a continuous line between two endpoints, with 0 being the lowest level and 100 being the highest level of fatigue.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=143 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Change From Baseline in Patient Assessment of Fatigue (Visual Analog Scale, VAS)	-23.94 units on a scale Standard Deviation 30.55

SECONDARY outcome

Timeframe: From baseline to Month 12

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the CRP assessment were included in this analysis.

The serum concentration of C-reactive protein (CRP) was measured. A reduction in the level of CRP was considered an improvement.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=120 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Change From Baseline in C-Reactive Protein (CRP)	-16.35 mg/L Standard Deviation 31.68

SECONDARY outcome

Timeframe: From baseline to Month 12

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the ESR assessment were included in this analysis.

The erythrocyte sedimentation rate (ESR) was analyzed at the site using the kit provided by the central laboratory. A reduction in the level of ESR was considered an improvement.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=104 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Change From Baseline in Erythrocyte Sedimentation Rate (ESR)	-16.79 mm/hr Standard Deviation 22.71

SECONDARY outcome

Timeframe: At 12 months

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the assessment of Extra-Articular Manifestations were included in this analysis.

The extra-articular RA manifestations ascertained were the nodules, Raynaud's phenomenon, secondary Sjogren's syndrome, pulmonary fibrosis, pericarditis, polyneuropathy, scleritis, severe cutaneous vasculitis, weight loss and anemia. Percentages are based on the total number of participants with available data.

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=161 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Percentage of Participants With Changes in Extra-Articular Manifestations at 12 Months Nodules	3.1 percentage of participants
Percentage of Participants With Changes in Extra-Articular Manifestations at 12 Months Raynaud"s Phenomenon	0 percentage of participants
Percentage of Participants With Changes in Extra-Articular Manifestations at 12 Months Secondary Sjogren"s Syndrome	2.5 percentage of participants
Percentage of Participants With Changes in Extra-Articular Manifestations at 12 Months Pulmonary Fibrosis	0 percentage of participants
Percentage of Participants With Changes in Extra-Articular Manifestations at 12 Months Pericarditis	0 percentage of participants
Percentage of Participants With Changes in Extra-Articular Manifestations at 12 Months Polyneuropathy	0 percentage of participants
Percentage of Participants With Changes in Extra-Articular Manifestations at 12 Months Scleritis	0 percentage of participants
Percentage of Participants With Changes in Extra-Articular Manifestations at 12 Months Severe Cutaneous Vasculitis	0 percentage of participants
Percentage of Participants With Changes in Extra-Articular Manifestations at 12 Months Weight Loss	0.6 percentage of participants
Percentage of Participants With Changes in Extra-Articular Manifestations at 12 Months Anemia	1.2 percentage of participants

SECONDARY outcome

Timeframe: At 12 months

Population: Full analysis set (FAS) population consisted of all participants included in the study who received at least one dose of Tocilizumab (n=198). Only participants who completed the assessment of Extra-Articular Manifestations were included in this analysis.

The severity of extra-articular RA manifestations ascertained were the nodules, Raynaud's phenomenon, secondary Sjogren's syndrome, pulmonary fibrosis, pericarditis, polyneuropathy, scleritis, severe cutaneous vasculitis, weight loss and anemia. Percentages are based on the total number of participants who responded "YES" to changes in extra- articular RA manifestations (new presence or change in severity) since the last visit. NA=Not applicable

Outcome measures

Measure	Rheumatoid Arthritis Cohort n=161 Participants The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Nodules: Mild	5 participants
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Nodules: Moderate	0 participants
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Nodules: Severe	0 participants
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Raynaud"s Phenomenon: Mild	NA participants No change was evident.
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Secondary Sjogren"s Syndrome: Mild	3 participants
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Secondary Sjogren"s Syndrome: Moderate	1 participants
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Secondary Sjogren"s Syndrome: Severe	0 participants
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Pulmonary Fibrosis: Moderate	NA participants No change was evident.
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Pulmonary Fibrosis: Severe	NA participants No change was evident.
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Pericarditis: Mild	NA participants No change was evident.
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Severe Cutaneous Vasculitis: Mild	NA participants No change was evident.
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Severe Cutaneous Vasculitis: Moderate	NA participants No change was evident.
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Weight Loss: Mild	1 participants
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Weight Loss: Moderate	0 participants
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Weight Loss: Severe	0 participants
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Anemia: Mild	2 participants
Number of Participants With Changes in Severity of Extra-Articular Manifestations at 12 Months Anemia: Moderate	0 participants

Adverse Events

Rheumatoid Arthritis Cohort

Serious events: 31 serious events

Other events: 68 other events

Deaths: 0 deaths

Serious adverse events

Measure	Rheumatoid Arthritis Cohort n=198 participants at risk The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Blood and lymphatic system disorders Neutropenia	0.51% 1/198 • Number of events 1 • 12 months
Cardiac disorders Acute myocardial infarction	0.51% 1/198 • Number of events 1 • 12 months
Cardiac disorders Chest discomfort	0.51% 1/198 • Number of events 1 • 12 months
Cardiac disorders Myocardial infarction	0.51% 1/198 • Number of events 1 • 12 months
Cardiac disorders Stress cardiomyopathy	0.51% 1/198 • Number of events 1 • 12 months
Gastrointestinal disorders Small intestinal obstruction	0.51% 1/198 • Number of events 1 • 12 months
Gastrointestinal disorders Vomiting	0.51% 1/198 • Number of events 1 • 12 months
General disorders Mass	0.51% 1/198 • Number of events 1 • 12 months
Hepatobiliary disorders Drug-induced liver injury	0.51% 1/198 • Number of events 1 • 12 months
Immune system disorders Anaphylactic reaction	1.0% 2/198 • Number of events 2 • 12 months
Infections and infestations Cellulitis	0.51% 1/198 • Number of events 1 • 12 months
Infections and infestations Cystitis	0.51% 1/198 • Number of events 1 • 12 months
Infections and infestations Pneumonia	1.0% 2/198 • Number of events 2 • 12 months
Infections and infestations Postoperative wound infection	0.51% 1/198 • Number of events 1 • 12 months
Infections and infestations Pyelonephritis	0.51% 1/198 • Number of events 1 • 12 months
Infections and infestations Subcutaneous abscess	0.51% 1/198 • Number of events 1 • 12 months
Infections and infestations Urosepsis	0.51% 1/198 • Number of events 1 • 12 months
Psychiatric disorders Delirium	0.51% 1/198 • Number of events 1 • 12 months
Renal and urinary disorders Nephrolithiasis	0.51% 1/198 • Number of events 1 • 12 months
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	0.51% 1/198 • Number of events 1 • 12 months
Skin and subcutaneous tissue disorders Petechiae	0.51% 1/198 • Number of events 1 • 12 months
Vascular disorders Hypersensitivity vasculitis	0.51% 1/198 • Number of events 1 • 12 months
Injury, poisoning and procedural complications Fall	1.5% 3/198 • Number of events 3 • 12 months
Injury, poisoning and procedural complications Fat embolism	0.51% 1/198 • Number of events 1 • 12 months
Injury, poisoning and procedural complications Hip fracture	1.0% 2/198 • Number of events 2 • 12 months
Injury, poisoning and procedural complications Lumbar vertebral fracture	0.51% 1/198 • Number of events 1 • 12 months
Musculoskeletal and connective tissue disorders Arthralgia	1.0% 2/198 • Number of events 2 • 12 months
Musculoskeletal and connective tissue disorders Back pain	0.51% 1/198 • Number of events 1 • 12 months
Musculoskeletal and connective tissue disorders Joint instability	0.51% 1/198 • Number of events 1 • 12 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Lung adenocarcinoma	1.0% 2/198 • Number of events 2 • 12 months
Pregnancy, puerperium and perinatal conditions Pregnancy	0.51% 1/198 • Number of events 1 • 12 months
Psychiatric disorders Depression	0.51% 1/198 • Number of events 1 • 12 months
Renal and urinary disorders Bladder prolapse	0.51% 1/198 • Number of events 1 • 12 months
Reproductive system and breast disorders Menorrhagia	0.51% 1/198 • Number of events 1 • 12 months
Surgical and medical procedures Knee arthroplasty	0.51% 1/198 • Number of events 1 • 12 months
Vascular disorders Obstructive shock	0.51% 1/198 • Number of events 1 • 12 months

Other adverse events

Measure	Rheumatoid Arthritis Cohort n=198 participants at risk The cohort included participants with moderate to severe rheumatoid arthritis (RA) in whom the treating physician made the decision to initiate tocilizumab treatment. No intervention: No intervention administered in this study
Gastrointestinal disorders Nausea	5.1% 10/198 • Number of events 11 • 12 months
General disorders Fatigue	5.1% 10/198 • Number of events 11 • 12 months
Infections and infestations Bronchitis	5.1% 10/198 • Number of events 10 • 12 months
Infections and infestations Upper respiratory tract infection	11.6% 23/198 • Number of events 30 • 12 months
Infections and infestations Urinary tract infection	5.1% 10/198 • Number of events 17 • 12 months
Musculoskeletal and connective tissue disorders Rheumatoid arthritis	5.1% 10/198 • Number of events 13 • 12 months
Nervous system disorders Headache	5.6% 11/198 • Number of events 11 • 12 months

Additional Information

Medical Communications

Hoffmann-La Roche

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 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