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A Study to Compare Subcutaneous Versus Intravenous Administration of RoActemra/Actemra (Tocilizumab) in Participants With Moderate to Severe Active Rheumatoid Arthritis

Phase 3
Completed
Conditions
Rheumatoid Arthritis
Interventions
Drug: tocilizumab SC
Drug: tocilizumab IV
Drug: placebo to tocilizumab IV
Drug: placebo to tocilizumab SC
Drug: Disease-modifying antirheumatic drugs (DMARDs)
Registration Number
NCT01194414
Lead Sponsor
Hoffmann-La Roche
Brief Summary

This randomized, double-blind, parallel group study compares the efficacy and safety of subcutaneous (sc) versus intravenous (iv) administration of tocilizumab in participants with moderate to severe active rheumatoid arthritis. Participants were randomized to receive either tocilizumab 162 mg sc weekly plus iv placebo every 4 weeks, or tocilizumab 8 mg/kg iv every 4 weeks plus sc placebo weekly during the double-blind period from baseline to Week 24. The double-blind period was followed by a 72-week open-label treatment with some switching of sc and iv administration. No placebo was administered in the open-label phase. Participants continued on their stable dose of disease-modifying antirheumatic drugs (DMARDs) throughout the study. Anticipated time on study treatment was 2 years.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
1262
Inclusion Criteria
  • Adult participants, ≥ 18 years of age
  • Rheumatoid arthritis of ≥ 6 months duration, according to American College of Rheumatology (ACR) criteria
  • Swollen joint count (SJC) ≥ 4 (66 joint count), tender joint count (TJC) ≥ 4 (68 joint count) at screening and baseline
  • Inadequate response to current DMARD therapy
  • Permitted DMARDs must be at stable dose for ≥ 8 weeks prior to baseline
  • Oral corticosteroids (≤ 10 mg/day prednisone or equivalent) and NSAIDs (up to maximum recommended dose) must be at stable dose for ≥ 4 weeks prior to baseline
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Exclusion Criteria
  • Major surgery (including joint surgery) within 8 weeks prior to screening or planned major surgery within 6 months following randomization
  • Rheumatic autoimmune disease other than RA
  • Functional class IV (ACR classification)
  • Diagnosis of juvenile idiopathic arthritis (JIA) or juvenile rheumatoid arthritis (JRA) and/or RA before the age of 16
  • Prior history of or current inflammatory joint disease other than RA
  • Intra-articular or parenteral corticosteroids within 4 weeks prior to baseline
  • Previous treatment with tocilizumab
  • Active current or history of recurrent infection
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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Tocilizumab SC Then Tocilizumab IVtocilizumab SCParticipants who received tocilizumab 162 mg subcutaneous (SC) injection weekly plus placebo to tocilizumab IV infusion every 4 weeks for 24 weeks in double blind treatment period switched to tocilizumab 8 mg/kg IV infusion every 4 weeks for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose continued throughout the study.
Tocilizumab IV Then Tocilizumab SCtocilizumab SCParticipants who received tocilizumab 8 mg/kg infusion (IV) every 4 weeks plus placebo to tocilizumab SC injection weekly in double blind treatment period switched to tocilizumab 162 mg SC injection weekly for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose will be continued throughout the study.
Tocilizumab SCtocilizumab SCParticipants received tocilizumab 162 mg subcutaneous (SC) injection weekly plus placebo to tocilizumab intravenous (IV) infusion every 4 weeks for a total of 24 weeks in the double-blind period. Participants continued to receive tocilizumab 162 mg SC injection weekly for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose continued throughout the study.
Tocilizumab SC Then Tocilizumab IVtocilizumab IVParticipants who received tocilizumab 162 mg subcutaneous (SC) injection weekly plus placebo to tocilizumab IV infusion every 4 weeks for 24 weeks in double blind treatment period switched to tocilizumab 8 mg/kg IV infusion every 4 weeks for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose continued throughout the study.
Tocilizumab SCplacebo to tocilizumab IVParticipants received tocilizumab 162 mg subcutaneous (SC) injection weekly plus placebo to tocilizumab intravenous (IV) infusion every 4 weeks for a total of 24 weeks in the double-blind period. Participants continued to receive tocilizumab 162 mg SC injection weekly for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose continued throughout the study.
Tocilizumab SCDisease-modifying antirheumatic drugs (DMARDs)Participants received tocilizumab 162 mg subcutaneous (SC) injection weekly plus placebo to tocilizumab intravenous (IV) infusion every 4 weeks for a total of 24 weeks in the double-blind period. Participants continued to receive tocilizumab 162 mg SC injection weekly for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose continued throughout the study.
Tocilizumab IVtocilizumab IVParticipants received tocilizumab 8 mg/kg infusion (IV) every 4 weeks plus placebo to tocilizumab SC injection weekly for a total of 24 weeks in the double-blind period. Participants continued to receive tocilizumab 8 mg/kg IV infusion every 4 weeks for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose continued throughout the study.
Tocilizumab IVplacebo to tocilizumab SCParticipants received tocilizumab 8 mg/kg infusion (IV) every 4 weeks plus placebo to tocilizumab SC injection weekly for a total of 24 weeks in the double-blind period. Participants continued to receive tocilizumab 8 mg/kg IV infusion every 4 weeks for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose continued throughout the study.
Tocilizumab IVDisease-modifying antirheumatic drugs (DMARDs)Participants received tocilizumab 8 mg/kg infusion (IV) every 4 weeks plus placebo to tocilizumab SC injection weekly for a total of 24 weeks in the double-blind period. Participants continued to receive tocilizumab 8 mg/kg IV infusion every 4 weeks for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose continued throughout the study.
Tocilizumab SC Then Tocilizumab IVplacebo to tocilizumab IVParticipants who received tocilizumab 162 mg subcutaneous (SC) injection weekly plus placebo to tocilizumab IV infusion every 4 weeks for 24 weeks in double blind treatment period switched to tocilizumab 8 mg/kg IV infusion every 4 weeks for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose continued throughout the study.
Tocilizumab SC Then Tocilizumab IVDisease-modifying antirheumatic drugs (DMARDs)Participants who received tocilizumab 162 mg subcutaneous (SC) injection weekly plus placebo to tocilizumab IV infusion every 4 weeks for 24 weeks in double blind treatment period switched to tocilizumab 8 mg/kg IV infusion every 4 weeks for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose continued throughout the study.
Tocilizumab IV Then Tocilizumab SCtocilizumab IVParticipants who received tocilizumab 8 mg/kg infusion (IV) every 4 weeks plus placebo to tocilizumab SC injection weekly in double blind treatment period switched to tocilizumab 162 mg SC injection weekly for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose will be continued throughout the study.
Tocilizumab IV Then Tocilizumab SCplacebo to tocilizumab SCParticipants who received tocilizumab 8 mg/kg infusion (IV) every 4 weeks plus placebo to tocilizumab SC injection weekly in double blind treatment period switched to tocilizumab 162 mg SC injection weekly for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose will be continued throughout the study.
Tocilizumab IV Then Tocilizumab SCDisease-modifying antirheumatic drugs (DMARDs)Participants who received tocilizumab 8 mg/kg infusion (IV) every 4 weeks plus placebo to tocilizumab SC injection weekly in double blind treatment period switched to tocilizumab 162 mg SC injection weekly for a total of 72 weeks in open label extension period. Non-biologic disease-modifying anti-rheumatic drugs (DMARDs) at a stable dose will be continued throughout the study.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants With Adverse Events, Serious Adverse Events and Clinically Significant Laboratory AssessmentsBaseline to up to 3 months after last dose of study drug (approximately up to 2 years)
Percentage of Participants Achieving an American College of Rheumatology Criteria (ACR20) Response at Week 24Baseline, 24 weeks

ACR20 response is defined as a ≥ 20% improvement (reduction) compared with baseline for both total joint count-68 joints (TJC68) and swollen joint count-66 joints (SJC66), as well as for three of the five additional ACR core set variables: Patient's Assessment of Pain over the previous 24 hours using a Visual Analog Scale (VAS) where left end of the line 0=no pain to right end of the line 100=unbearable pain; Patient's Global Assessment of Disease Activity and Physician's Global Assessment of Disease Activity over the previous 24 hours using a VAS where left end of the line 0=no disease activity to right end of the line 100=maximum disease activity; Health Assessment Questionnaire: 20 questions in 8 areas (dressing/grooming, arising, eating, walking, hygiene, reach, grip and activities) answered on a scale of 0=without difficulty to 3=unable to do; and acute-phase reactant (either C-reactive protein \[CRP\] or Erythrocyte Sedimentation Rate \[ESR\]).

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants Achieving an American College of Rheumatology Criteria (ACR70) Response at Week 24Baseline, 24 weeks

ACR70 response is defined as a ≥ 70% improvement (reduction) compared with baseline for both total joint count-68 joints (TJC68) and swollen joint count-66 joints (SJC66), as well as for three of the five additional ACR core set variables: Patient's Assessment of Pain over the previous 24 hours using a Visual Analog Scale (VAS) where left end of the line 0=no pain to right end of the line 100=unbearable pain; Patient's Global Assessment of Disease Activity and Physician's Global Assessment of Disease Activity over the previous 24 hours using a VAS where left end of the line 0=no disease activity to right end of the line 100=maximum disease activity; Health Assessment Questionnaire: 20 questions in 8 areas (dressing/grooming, arising, eating, walking, hygiene, reach, grip and activities) answered on a scale of 0=without difficulty to 3=unable to do; and acute-phase reactant (either C-reactive protein or Erythrocyte Sedimentation Rate).

Percentage of Participants Achieving an American College of Rheumatology Criteria (ACR50) Response at Week 24Baseline, 24 weeks

ACR50 response is defined as a ≥ 50% improvement (reduction) compared with baseline for both total joint count-68 joints (TJC68) and swollen joint count-66 joints (SJC66), as well as for three of the five additional ACR core set variables: Patient's Assessment of Pain over the previous 24 hours using a Visual Analog Scale (VAS) where left end of the line 0=no pain to right end of the line 100=unbearable pain; Patient's Global Assessment of Disease Activity and Physician's Global Assessment of Disease Activity over the previous 24 hours using a VAS where left end of the line 0=no disease activity to right end of the line 100=maximum disease activity; Health Assessment Questionnaire: 20 questions in 8 areas (dressing/grooming, arising, eating, walking, hygiene, reach, grip and activities) answered on a scale of 0=without difficulty to 3=unable to do; and acute-phase reactant (either C-reactive protein or Erythrocyte Sedimentation Rate).

Percentage of Participants With Disease Activity Score 28 (DAS28) Remission at Week 24Week 24

The DAS28 (ESR) score is a measure of the subject's disease activity. It is calculated using the tender joint count (28 joints), swollen joint count (28 joints), patient's global assessment of disease activity VAS where left side of the line 0=no disease activity to right side of the line 100=extreme disease activity and ESR. DAS28-(ESR) total scores range from 0 - 10. Remission is defined as achieving a DAS28-ESR score of less than 2.6.

Percentage of Participants Achieving a Decrease of ≥ 0.3 in the Health Assessment Questionnaire-Disability Index (HAQ-DI) From Baseline to Week 24Baseline, 24 Weeks

The Stanford Health Assessment Questionnaire disability index (HAQ-DI) is a participant completed questionnaire specific for rheumatoid arthritis. It consists of 20 questions referring to 8 domains: dressing/grooming, arising, eating, walking, hygiene, reach, grip and common daily activities. Each domain has at least two component questions. There are four possible responses for each component ranging from 0(without any difficulty) to 4 (unable to do).HAQ-DI=sum of worst scores in each domain divided by the number of domains answered. A decrease indicates improvement.

Percentage of Participants Who Withdrew Because of Lack of Therapeutic Response at Week 2424 Weeks

The percentage of participants who withdrew from the study because they were not responding to treatment with the study drug.

Percentage of Participants With American College of Rheumatology Criteria (ACR20, ACR50, ACR70) at Week 97Week 97

ACR20, ACR50 and ACR70: ≥20%, ≥50% and ≥70% reduction from baseline for both TJC68 and SJC66, as well as for 3 of 5 additional ACR variables: Patient's Assessment of Pain in last 24 hours using a Visual Analog Scale (VAS) (0=no pain and 100=unbearable pain); Patient's and Physician's Global Assessment of Disease Activity in last 24 hours using a VAS (0=no disease activity and100=maximum disease activity); Health Assessment Questionnaire: 20 questions in 8 areas (dressing/grooming, arising, eating, walking, hygiene, reach, grip and activities) answered on a scale of 0=without difficulty to 3=unable to do; and acute-phase reactant (either CRP or ESR). CRP was used for calculation of ACR. If missing, ESR was used. LOCF was used for missing joint counts, no imputation for other ACR components.

Percentage of Participants With Disease Activity Score 28 (DAS28) Remission at Week 97Week 97

The DAS28 (ESR) score is a measure of the subject's disease activity. It is calculated using the tender joint count (28 joints), swollen joint count (28 joints), patient's global assessment of disease activity VAS where left side of the line 0=no disease activity to right side of the line 100=extreme disease activity and ESR. DAS28-(ESR) total scores range from 0 - 10. Remission is defined as achieving a DAS28-ESR score of less than 2.6. LOCF used for tender and swollen joint counts, no imputation used for ESR and Patient's Global Assessment of Disease Activity VAS.

Percentage of Participants Achieving a Decrease of ≥0.3 in the Health Assessment Questionnaire-Disability Index (HAQ-DI) From Baseline to Week 97Baseline, Week 97

The Stanford Health Assessment Questionnaire disability index (HAQ-DI) is a patient completed questionnaire specific for rheumatoid arthritis. It consists of 20 questions referring to 8 domains: dressing/grooming, arising, eating, walking, hygiene, reach, grip and common daily activities. Each domain has at least two component questions. There are four possible responses for each component ranging from 0(without any difficulty) to 4 (unable to do).HAQ-DI=sum of worst scores in each domain divided by the number of domains answered. A decrease indicates improvement. No imputation of missing scores was made other than for missing baseline scores, for which last score prior to baseline will be carried forward. For participants who prematurely withdrew, data collected at withdrawal visit was used and data thereafter is missing.

Percentage of Participants Who Withdrew Because of Lack of Therapeutic Response at Week 97Week 97

The percentage of participants who withdrew from the study because they were not responding to treatment with the study drug.

Area Under the Serum Concentration Curve of Tocilizumab After First SC Injection or IV InfusionWeek 0: at 6 hours (hr), 24 hr, 48 hr, 96 hr, 120 hr and 168 hr after first dose
Area Under the Serum Concentration Curve of Tocilizumab at Steady State for SC and IV TreatmentWeek 20: at 6 hours (hr), 24 hr, 48 hr, 96 hr, 120 hr and 168 hr after dose.
Minimum Serum Concentration (Cmin) of TocilizumabWeek 0, Week 20: at 6 hours (hr), 24 hr, 48 hr, 96 hr, 120 hr and 168 hr after dose
Maximum Serum Concentration (Cmax) of TocilizumabWeek 0, Week 20: at 6 hours (hr), 24 hr, 48 hr, 96 hr, 120 hr and 168 hr after dose
Time to Maximum Serum Concentration (Tmax) of TocilizumabWeek 0, Week 20: at 6 hours (hr), 24 hr, 48 hr, 96 hr, 120 hr and 168 hr after dose
Change From Baseline in Serum Interleukin-6 (IL-6) Concentration at Week 25Baseline, Week 25
Change From Baseline in Serum Soluble Interleukin-6 Receptor (sIL-6R) Concentration at Week 97Baseline, Week 97
Percentage of Participants Who Developed Antibodies To Tocilizumab at Week 97Week 97
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