Trial Outcomes & Findings for A Study of RoActemra/Actemra (Tocilizumab) in Combination With DMARDs Versus Current Best Practice DMARD Therapy in Patients With Rheumatoid Arthritis (NCT NCT01251120)

NCT ID: NCT01251120

Last Updated: 2014-11-11

Results Overview

The DAS28 is a combined index for measuring disease activity in Rheumatoid Arthritis (RA). The index includes swollen and tender joint counts (SJC and TJC), acute phase response, and general health status. For this study Erythrocyte sedimentation Rate (ESR) was to be used to calculate DAS28 score. The DAS28, which uses a 28 joint count, is derived from the original DAS which includes a 44 swollen joint count. The DAS28 has been validated in RA. The index is calculated using the following formula: DAS28 equals (=) 0.56 times (×) square root of √(TJC) plus (+) 0.28 × √(SJC) + 0.70 × ln(ESR) + 0.014 × GH Where, TJC28 = tender joint count on 28 joints, SJC28 = swollen joint count on 28 joints, ln = natural log, ESR measured as millimeters per hour (mm/hr), and GH = general health, which is participant's global assessment of disease activity (100-mm Visual Analog Scale \[VAS\]). DAS28 less than/equal to (≤) 2.6 defined remission.

• Recruitment status: TERMINATED
• Study phase: PHASE4
• Target enrollment: 2 participants
• Primary outcome timeframe: Month 12
• Results posted on: 2014-11-11

Participant Flow

Participant milestones

Measure	Tocilizumab Participants received tocilizumab 8 milligrams per kilogram (mg/kg) intravenously every 4 weeks along with background disease-modifying antirheumatic drugs (DMARDs) including methotrexate. All participants who received methotrexate also received at least 5 mg oral folic acid weekly.	Non-biologic DMARDs Participants received non-biologic DMARDs (including methotrexate) according to current best practice. All participants who received methotrexate also received at least 5 mg oral folic acid weekly.
Overall Study STARTED	1	1
Overall Study COMPLETED	1	0
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Measure	Tocilizumab Participants received tocilizumab 8 milligrams per kilogram (mg/kg) intravenously every 4 weeks along with background disease-modifying antirheumatic drugs (DMARDs) including methotrexate. All participants who received methotrexate also received at least 5 mg oral folic acid weekly.	Non-biologic DMARDs Participants received non-biologic DMARDs (including methotrexate) according to current best practice. All participants who received methotrexate also received at least 5 mg oral folic acid weekly.
Overall Study Lack of Efficacy	0	1

Baseline Characteristics

A Study of RoActemra/Actemra (Tocilizumab) in Combination With DMARDs Versus Current Best Practice DMARD Therapy in Patients With Rheumatoid Arthritis

Baseline characteristics by cohort

Measure	Tocilizumab n=1 Participants Participants received tocilizumab 8 mg/kg intravenously every 4 weeks plus background DMARDs (including methotrexate)	Placebo n=1 Participants Participants received Non-biologic DMARDs (including methotrexate) according to current best practice	Total n=2 Participants Total of all reporting groups
Age, Continuous	63.2 years STANDARD_DEVIATION 0.0 • n=93 Participants	48.2 years STANDARD_DEVIATION 0.0 • n=4 Participants	55.7 years STANDARD_DEVIATION 7.5 • n=27 Participants
Sex: Female, Male Female	1 Participants n=93 Participants	1 Participants n=4 Participants	2 Participants n=27 Participants
Sex: Female, Male Male	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants

PRIMARY outcome

Timeframe: Month 12

Population: Because of the limited number (n) of participants (n=2), the study was terminated prematurely. No statistical analysis could be performed.

The DAS28 is a combined index for measuring disease activity in Rheumatoid Arthritis (RA). The index includes swollen and tender joint counts (SJC and TJC), acute phase response, and general health status. For this study Erythrocyte sedimentation Rate (ESR) was to be used to calculate DAS28 score. The DAS28, which uses a 28 joint count, is derived from the original DAS which includes a 44 swollen joint count. The DAS28 has been validated in RA. The index is calculated using the following formula:

DAS28 equals (=) 0.56 times (×) square root of √(TJC) plus (+) 0.28 × √(SJC) + 0.70 × ln(ESR) + 0.014 × GH Where, TJC28 = tender joint count on 28 joints, SJC28 = swollen joint count on 28 joints, ln = natural log, ESR measured as millimeters per hour (mm/hr), and GH = general health, which is participant's global assessment of disease activity (100-mm Visual Analog Scale [VAS]). DAS28 less than/equal to (≤) 2.6 defined remission.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline and 6 and 12 months

Population: Because of the limited number of participants (n=2), the study was terminated prematurely. No statistical analysis could be performed.

Work productivity measures include absence from work (participant reported and registries), permanent work disability (pension, participant reported and registries), presenteeism (Quantity and Quality instrument, QQ).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Month 6

Population: Because of the limited number of participants (n=2), the study was terminated prematurely. No statistical analysis could be performed.

The DAS28 is a combined index for measuring disease activity in RA. The index includes SJC and TJC, acute phase response, and general health status. For this study ESR was to be used to calculate DAS28 score. The DAS28, which uses a 28 joint count, is derived from the original DAS which includes a 44 swollen joint count. The DAS28 has been validated in RA. The index is calculated using the following formula:

DAS28 = 0.56 × √(TJC) + 0.28 × √(SJC) + 0.70 × ln(ESR) + 0.014 × GH Where, TJC28 = tender joint count on 28 joints, SJC28 = swollen joint count on 28 joints, ln = natural log, ESR measured as millimeters per hour (mm/hr), and GH = general health, which is participant's global assessment of disease activity (100-mm VAS). DAS28 ≤ 2.6 defined remission.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Months 6 and 12

Population: Because of the limited number of participants (n=2), the study was terminated prematurely. No statistical analysis could be performed.

The ACR definition of response includes tender and swollen joint counts, VAS scales for pain, participant and investigator global assessment of disease activity, participant-assessed disability using Health Assessment Questionnaire (HAQ) and acute phase response.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Months 6 and 12

Population: Because of the limited number of participants (n=2), the study was terminated prematurely. No statistical analysis could be performed.

The CDAI is a purely clinical index to measure disease activity. The index includes swollen and tender joint counts, participant global assessment of disease activity, and evaluator global assessment of disease activity (EGA).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline and Months 6 and 12

Population: Because of the limited number of participants (n=2), the study was terminated prematurely. No statistical analysis could be performed.

The FACIT-Fatigue score was calculated according to a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function. FACIT-F is a 13-item questionnaire. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). The larger the participant's response to the questions (with the exception of 2 negatively stated), the greater the participants fatigue. For all questions, except for the 2 negatively stated ones, the code was reversed and a new score was calculated as (4 minus the participant's response). The sum of all responses resulted in the FACIT-Fatigue score for a total possible score of 0 (worse score) to 52 (better score). Clinically relevant improvement is defined as a ≥5-point change from Baseline.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline and Months 6 and 12

Population: Because of the limited number of participants (n=2), the study was terminated prematurely. No statistical analysis could be performed.

EQ-5D is a participant rated questionnaire to assess health-related quality of life in terms of a single utility score. Health State Profile component assesses level of current health for 5 domains: mobility, self-care, usual activities, pain and discomfort, and anxiety and depression; 1 indicates better health state (no problems); 3 indicates worst health state (confined to bed). Scoring formula developed by EuroQoL Group assigns a utility value for each domain in the profile. Score is transformed and results in a total score range -0.594 to 1.000; higher score indicates a better health state.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline and Months 6 and 12

Population: Because of the limited number of participants (n=2), the study was terminated prematurely. No statistical analysis could be performed.

HAQ-DI includes 20 questions concerning participant's activities of daily life, grouped in 8 scales of 2 to 3 questions for each activity. To respond to each question, a four-level response (score of 0 to 3 points), with higher scores showing larger functional limitations, was chosen. Overall score was computed as the sum of the domain scores and divided by the number of domains answered. Total possible score range was 0-3 where 0=without difficulties; 1= with some difficulties; 2=with great difficulties; and 3=unable to perform these actions at all.

Outcome measures

Outcome data not reported

Adverse Events

Tocilizumab

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Non-biologic DMARDs

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Tocilizumab n=1 participants at risk Participants received tocilizumab 8 mg/kg intravenously every 4 weeks along with background DMARDs including methotrexate. All participants who received methotrexate also received at least 5 mg oral folic acid weekly.	Non-biologic DMARDs n=1 participants at risk Participants received non-biologic DMARDs (including methotrexate) according to current best practice. All participants who received methotrexate also received at least 5 mg oral folic acid weekly.
Gastrointestinal disorders Gastointestinal Disorder	100.0% 1/1 • The study was terminated prematurely due to lack of enrollment. Adverse events were recorded from the date of screening until study termination.	0.00% 0/1 • The study was terminated prematurely due to lack of enrollment. Adverse events were recorded from the date of screening until study termination.

Additional Information

Medical Communications

Hoffmann- LaRoche

Phone: 1-800-821-8590

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