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A Study of Tocilizumab as Monotherapy and in Combination With Methotrexate Versus Methotrexate in Patients With Early Moderate to Severe Rheumatoid Arthritis

Registration Number
NCT01007435
Lead Sponsor
Hoffmann-La Roche
Brief Summary

This randomized, double-blind, parallel group study will assess the safety, disease remission, and prevention of structural joint damage in patients with early moderate to severe rheumatoid arthritis treated with tocilizumab as monotherapy or in combination with methotrexate, versus methotrexate alone. Patients will be randomized to receive either (A) tocilizumab (8 mg/kg iv every 4 weeks) plus placebo, (B) tocilizumab (8 mg/kg iv every 4 weeks) plus methotrexate (7.5-20 mg po weekly), (C) tocilizumab (4 mg/kg iv every 4 weeks) plus methotrexate (7.5-20 mg po weekly), or (D) placebo plus methotrexate (7.5-20 mg po weekly). Patients in groups C and D who have not achieved low disease activity at week 52 can receive tocilizumab 8 mg/kg iv every 4 weeks. Anticipated time on study treatment is 104 weeks.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
1162
Inclusion Criteria
  • Adult patients ≥ 18 years of age.
  • Rheumatoid arthritis of ≤ 2 years duration.
  • Disease Activity Score 28 (DAS28) > 3.2.
  • Swollen joint count (SJC) ≥ 4 of 66 joints, tender joint count (TJC) ≥ 6 of 68 joints.
  • Rheumatoid factor (RF) and/or anti-cyclic citrullinated peptide (anti-CCP) positive (if RF and anti-CCP negative > 1 erosion required at screening).
  • Erythrocyte sedimentation rate (ESR) ≥ 28 mm/h or C-reactive protein (CRP) ≥ 10 mg/L at screening.
Exclusion Criteria
  • Previous treatment with tocilizumab.
  • Previous treatment with methotrexate or biologic agent.
  • Rheumatic autoimmune disease other than rheumatoid arthritis (RA).
  • History of or current inflammatory joint disease other than RA.
  • Functional class IV as defined by the American College of Rheumatology (ACR) Classification of Functional Status in RA.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
(A) Tocilizumab 8 mg/kg + placebo to methotrexateTocilizumabPatients received tocilizumab 8 mg/kg iv every 4 weeks + placebo to methotrexate orally once a week for 104 weeks.
(A) Tocilizumab 8 mg/kg + placebo to methotrexatePlacebo to methotrexatePatients received tocilizumab 8 mg/kg iv every 4 weeks + placebo to methotrexate orally once a week for 104 weeks.
(B) Tocilizumab 8 mg/kg + methotrexateTocilizumabPatients received tocilizumab 8 mg/kg iv every 4 weeks + methotrexate orally once a week for 104 weeks.
(C) Tocilizumab 4 mg/kg + methotrexateTocilizumabPatients received tocilizumab 4 mg/kg iv every 4 weeks + methotrexate orally once a week for 104 weeks.
(C) Tocilizumab 4 mg/kg + methotrexateMethotrexatePatients received tocilizumab 4 mg/kg iv every 4 weeks + methotrexate orally once a week for 104 weeks.
(D) Placebo to tocilizumab + methotrexatePlacebo to tocilizumabPatients received placebo tocilizumab intravenously (iv) every 4 weeks + methotrexate orally once a week for 104 weeks.
(B) Tocilizumab 8 mg/kg + methotrexateMethotrexatePatients received tocilizumab 8 mg/kg iv every 4 weeks + methotrexate orally once a week for 104 weeks.
(D) Placebo to tocilizumab + methotrexateMethotrexatePatients received placebo tocilizumab intravenously (iv) every 4 weeks + methotrexate orally once a week for 104 weeks.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants With a Disease Activity Score 28 (DAS28) Remission Response at Week 24Week 24

A participant has a DAS28 remission response if their DAS28 \< 2.6. The DAS28 is a combined index for measuring disease activity in rheumatic arthritis (RA) and includes swollen and tender joint counts, erythrocyte sedimentation rate (ESR), and general health (GH) status. The index is calculated with the following formula: DAS28 = (0.56 × √(TJC28)) + (0.28 × √(SJC28)) + (0.7 × log(ESR)) + (0.014 × GH), where TJC28 = tender joint count and SJC28 = swollen joint count, each on 28 joints. GH = a patient's global assessment of disease activity in the previous 24 hours on a 100 mm visual analog scale (left end = no disease activity \[symptom-free and no arthritis symptoms\], right end = maximum disease activity \[maximum arthritis disease activity\]). When ESR equaled 0 mm/hr, it was set to 1 mm/hr. The DAS28 scale ranges from 0 to 10, where higher scores represent higher disease activity.

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants With a Disease Activity Score 28 (DAS28) Remission Response at Week 52Week 52
Percentage of Patients With an Improvement ≥ 20%, 50%, or 70% in American College of Rheumatology (ACR) Score (ACR20/50/70) From Baseline to Weeks 24 and 52Baseline to Weeks 24 and 52

Improvement must be seen in tender (68) and swollen (66) joint counts. Joints were assessed and classified as swollen/not swollen and tender/not tender by pressure and joint manipulation. Improvement must also be seen in at least 3 of the following 5 parameters: Separate patient and physician assessments of patient disease activity in the previous 24 hours on a visual analog scale (VAS, the extreme left end of the line "no disease activity" \[symptom-free and no arthritis symptoms\] and the extreme right end "maximum disease activity"; patient assessment of pain in previous the 24 hours on a VAS (extreme left end of the line "no pain" and the extreme right end "unbearable pain"); Health Assessment Questionnaire-Disability Index (20 questions, 8 components: dressing/grooming, arising, eating, walking, hygiene, reach, grip, and activities, 0=without difficulty to 3=unable to do); and C-reactive protein (CRP), or erythrocyte sedimentation rate if CRP was missing.

Change From Baseline in Modified Total Sharp Score (mTSS) at Week 52Baseline to Week 52

The mTSS is a measure of joint damage and includes measures of joint erosion (JE) and joint space narrowing (JSN). The JE score, using the van der Heijde modification, measures erosion severity in 32 hand joints and 12 foot joints. Each hand joint is scored from 0 to 5 and each foot joint is scored from 0 to 10; the total score ranges from 0 to 280. Each joint is scored according to the surface area involved. A score of 10 indicates extensive loss of bone from more than one-half of the articulating bone; a score of 0 indicates no erosion. The JSN score measures the severity of JSN in 30 hand joints (15 per hand) and 12 foot joints (6 per foot). Each joint, including subluxation, is scored from 0 to 4; the total score ranges from 0 to 168. A higher score indicates more joint space narrowing. The mTSS ranges from 0 to 448 (280+168). A higher mTSS score indicates greater damage. A negative change score indicates improvement.

Change From Baseline in Modified Sharp Erosion Score at Week 52Baseline to Week 52
Change From Baseline in Sharp Joint Space Narrowing Score at Week 52Baseline to Week 52
Percentage of Participants With a Major Clinical Response at Week 52Baseline to Week 52

A major clinical response is defined as an ACR70 response that is maintained for 6 consecutive months (24 weeks) for any 24-week period between Week 2 and Week 52.

Change From Baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) Score at Weeks 24 and 52Baseline to Weeks 24 and 52

The Stanford HAQ-DI is a patient completed questionnaire specific for rheumatoid arthritis. The HAQ-DI assesses how well the patient is able to perform 8 activities: Dressing/grooming, arising, eating, walking, hygiene, reach, grip, and activities. The patient answers 20 questions with 1 of 4 responses with the past week as the time frame: 0=without difficulty, 1=with some difficulty, 2=with much difficulty, and 3=unable to do. The highest score for any question in a category determines the category score. The total score ranges from 0 (no disability) to 3 (completely disabled). A negative change score indicates improvement.

Change From Baseline in Short Form 36 (SF-36) Physical Component Summary (PCS) Scores at Weeks 24 and 52Baseline to Weeks 24 and 52

The SF-36 Health Survey (Version 2) is a standardized questionnaire consisting of 36 questions that measures patient-reported symptoms on 8 dimensions; it is used to assess health-related quality of life (HRQoL). The Physical Component Summary (PCS) score summarizes the subscales Physical Functioning, Role-Physical, Bodily Pain, and General Health. The Mental Component Summary (MCS) score summarizes the subscales Vitality, Social Functioning, Role-Emotional, and Mental Health. Each score was scaled from 0 to 100. A positive change score indicates better HRQoL.

Trial Locations

Locations (236)

Pinnacle Research Group; Llc, Central

🇺🇸

Anniston, Alabama, United States

Rheumatology Associates of North Alabama

🇺🇸

Huntsville, Alabama, United States

Clnical & Translational Reseach Center for Alabama, PC

🇺🇸

Tuscaloosa, Alabama, United States

Arthrocare, Arthritis Care and Research, P.C.

🇺🇸

Mesa, Arizona, United States

Sun Valley Arthritis Center

🇺🇸

Peoria, Arizona, United States

Talbert Medical Group

🇺🇸

Huntington Beach, California, United States

Uni of California - San Diego; Division of Rheumatology

🇺🇸

La Jolla, California, United States

Valerius Medical Group & Research Ctr of Greater Long Beach

🇺🇸

Los Alamitos, California, United States

Sharp Rees-Stealy Medical Group

🇺🇸

San Diego, California, United States

San Diego Arthritis Med Clnc

🇺🇸

San Diego, California, United States

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Pinnacle Research Group; Llc, Central
🇺🇸Anniston, Alabama, United States

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