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A Study of Subcutaneous (SC) Tocilizumab (RoActemra/Actemra) in Participants With Active Rheumatoid Arthritis (RA) and Inadequate Response to Disease-Modifying Anti-Rheumatic Drugs (DMARDs)

Phase 3
Completed
Conditions
Rheumatoid Arthritis
Interventions
Drug: Non-Biologic DMARDs
Registration Number
NCT02046616
Lead Sponsor
Hoffmann-La Roche
Brief Summary

This Phase IIIb, open-label, single-arm study will evaluate the safety, efficacy, and tolerability of SC tocilizumab (RoActemra/Actemra) in monotherapy or in combination with methotrexate or other non-biologic DMARDs in participants with active RA who are naive to tocilizumab. Participants will receive tocilizumab 162 milligrams (mg) subcutaneously weekly (QW) for 24 weeks.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
133
Inclusion Criteria
  • Active RA according to the revised ACR (1987) criteria or EULAR/ACR (2010) criteria
  • Moderate to severe RA with a DAS28-ESR score >3.2 points
  • Inadequate response and/or intolerance to MTX or other non-biologic DMARDs and/or where MTX or other non-biologic DMARDs are inappropriate
  • Oral corticosteroids (less than or equal to [</=] 10 mg per day prednisolone or equivalent) and nonsteroidal anti-inflammatory drugs (NSAIDs) permitted if on stable dose regimen for greater than or equal to [>/=] 4 weeks prior to baseline
  • Permitted non-biologic DMARDs allowed if at stable dose for >/=4 weeks prior to baseline
  • Receiving treatment on an outpatient basis, not including tocilizumab
  • Agreement to use reliable means of contraception as defined by protocol, among females of childbearing potential and males with female partners of childbearing potential
Exclusion Criteria
  • Major surgery (including joint surgery) within 8 weeks prior to screening or planned major surgery within 6 months following baseline
  • Rheumatic autoimmune disease other than RA
  • Functional Class IV as defined by the ACR Classification of Functional Status in Rheumatoid Arthritis
  • Diagnosis of juvenile idiopathic arthritis or juvenile RA and/or RA before the age of 16
  • Prior history of or current inflammatory joint disease other than RA
  • Exposure to tocilizumab or any other biologic DMARDs at any time prior to baseline
  • Treatment with any investigational agent within 4 weeks (or 5 half-lives of the investigational drug, whichever is longer) of screening
  • Intra-articular or parenteral corticosteroids within 4 weeks prior to baseline
  • History of severe allergic or anaphylactic reactions to human, humanized, or murine monoclonal antibodies
  • Evidence of serious concomitant disease or disorder
  • Known active current or history of recurrent infection
  • Any major episode of infection requiring hospitalization or treatment with intravenous (IV) antibiotics within 4 weeks of screening or oral antibiotics within 2 weeks of screening
  • Active tuberculosis requiring treatment within the previous 3 years
  • Positive for hepatitis B or hepatitis C
  • History of or current active primary or secondary immunodeficiency
  • Pregnant or lactating women
  • Neuropathies or other conditions that might interfere with pain evaluation
  • Inadequate hematologic, renal, or liver function

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Tocilizumab Alone or Combined with Methotrexate or Other DMARDNon-Biologic DMARDsAll participants will receive tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs, irrespective of body weight, for 24 weeks.
Tocilizumab Alone or Combined with Methotrexate or Other DMARDTocilizumabAll participants will receive tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs, irrespective of body weight, for 24 weeks.
Tocilizumab Alone or Combined with Methotrexate or Other DMARDMethotrexateAll participants will receive tocilizumab as a single fixed dose (monotherapy) or in combination with methotrexate or other non-biologic DMARDs, irrespective of body weight, for 24 weeks.
Primary Outcome Measures
NameTimeMethod
Change From Baseline in Clinical Disease Activity Index (CDAI) at Week 12Baseline, Week 12

CDAI was derived as the sum of the following: tender joint count (TJC), swollen joint count (SJC), participant global assessment (PGA) of disease activity, and physician assessment of disease activity. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA and physician assessment of disease activity were scored 0-100 millimeters (mm) and rounded to the nearest centimeter (cm) on a visual analog scale (VAS), where higher scores indicate greater perceived disease activity. The total CDAI score range was 0-76, where higher scores indicate increased disease activity. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA disease activity.

Secondary Outcome Measures
NameTimeMethod
Change From Baseline in SJCBaseline and Weeks 2, 4, 8, 12, 16, 20, 24

SJC was taken as the number of swollen joints out of 28 assessed joints.

Number of Participants With Neutralizing Anti-Tocilizumab AntibodiesBaseline to FU Week 8 (up to 32 weeks overall)

Participants were evaluated for the presence of anti-tocilizumab antibodies. Confirmatory assays were performed in the case of a positive screen assay result.

Compliance With Treatment According to Percentage of Injections AdministeredBaseline up to Week 24

Participants were provided with diary cards to record home injections. Compliance with treatment was calculated individually for each participant as the actual number of injections as a percentage of the planned number of injections (up to the point of discontinuation for those who discontinued study treatment prematurely) and then averaged among all participants.

Change From Baseline in TJCBaseline and Weeks 2, 4, 8, 12, 16, 20, 24

TJC was taken as the number of tender joints out of 28 assessed joints.

Soluble Interleukin-6 Receptor (sIL-6R) ConcentrationPredose (30 minutes) at baseline; Weeks 12, 24; and FU Week 8 (up to 32 weeks overall)

sIL-6R concentration was determined, averaged among all participants, and expressed in nanograms per milliliter (ng/mL).

Change From Baseline in Patient Global Assessment of RA-Related Pain According to VASBaseline and Weeks 2, 4, 8, 12, 16, 20, 24

PGA of RA-related pain was scored 0-100 mm on a VAS, where higher scores indicate greater perceived pain. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA-related pain.

Change From Baseline in Disease Activity Score 28 (DAS28)-Erythrocyte Sedimentation Rate (ESR) ScoreBaseline and Weeks 2, 4, 8, 12, 16, 20, 24

DAS28-ESR was based on TJC, SJC, PGA of disease activity, and laboratory-derived ESR. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA of disease activity was scored 0-100 mm on a VAS, where higher scores indicate greater perceived disease activity. The total DAS28-ESR score was transformed to a single score range of 0-10, where higher scores indicate increased disease activity. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA disease activity.

Change From Baseline in Simplified Disease Activity Index (SDAI)Baseline and Weeks 2, 4, 8, 12, 16, 20, 24

SDAI was derived as the sum of the following: TJC, SJC, PGA of disease activity, physician assessment of disease activity, and laboratory-derived C-reactive protein level. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA and physician assessment of disease activity were scored 0-100 mm and rounded to the nearest cm on a VAS, where higher scores indicate greater perceived disease activity. The total SDAI score range was 0-86, where higher scores indicate increased disease activity. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA disease activity.

Percentage of Participants With At Least One Adverse Event Leading to Dosage ModificationBaseline up to Week 24

The percentage of participants with at least one adverse event leading to dose/frequency reduction or temporary dose hold was reported.

Tocilizumab ConcentrationPredose (30 minutes) at baseline; Weeks 12, 24; and FU Week 8 (up to 32 weeks overall)

Tocilizumab concentration was determined, averaged among all participants, and expressed in micrograms per milliliter (mcg/mL).

Change From Baseline in Patient Global Assessment of Disease Activity According to VASBaseline and Weeks 2, 4, 8, 12, 16, 20, 24

PGA of disease activity was scored 0-100 mm on a VAS, where higher scores indicate greater perceived disease activity. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA disease activity.

Percentage of Participants With American College of Rheumatology (ACR) ResponseWeeks 2, 4, 8, 12, 16, 20, 24

ACR response was assessed on the basis of percent improvement (20% for ACR20, 50% for ACR50, 70% for ACR70) in both TJC and SJC as well as at least three of the following: physician assessment of disease activity, PGA of disease activity, PGA of pain, Health Assessment Questionnaire-Disability Index (HAQ-DI), and either ESR or C-reactive protein level. TJC and SJC were taken as the number of tender and swollen joints, out of 68 and 66 assessed joints, respectively. PGA and physician assessments were scored 0-100 mm on a VAS, where higher scores indicate greater perceived disease activity or pain. HAQ-DI was scored using participant responses to 20 questions assessing activities of daily living (ADLs), with total score scale of 0-3, where higher scores indicate increased functional disability. The percentage of participants meeting criteria for each level of ACR response was reported.

Percentage of Participants With European League Against Rheumatism (EULAR) ResponseWeeks 2, 4, 8, 12, 16, 20, 24

EULAR response was assessed by change from baseline and absolute DAS28-ESR score. EULAR response classification was as follows: Good (change \>1.2 with absolute score \</=3.2), Moderate (change \>1.2 with absolute score \>3.2 or change \>0.6 with absolute score \</=5.1), None (change \</=0.6 or absolute score \>5.1). DAS28-ESR was based on TJC, SJC, and PGA of disease activity, and laboratory-derived ESR. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA of disease activity was scored 0-100 mm on a VAS, where higher scores indicate greater perceived disease activity. The total DAS28-ESR score was transformed to a single score range of 0-10, where higher scores indicate increased disease activity. The percentage of participants meeting criteria for each level of EULAR response was reported.

Change From Baseline in CDAI at Weeks 2, 4, 8, 16, 20, and 24Baseline and Weeks 2, 4, 8, 16, 20, 24

CDAI was derived as the sum of the following: TJC, SJC, PGA of disease activity, and physician assessment of disease activity. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA and physician assessment of disease activity were scored 0-100 mm and rounded to the nearest cm on a VAS, where higher scores indicate greater perceived disease activity. The total CDAI score range was 0-76, where higher scores indicate increased disease activity. Change from baseline was averaged among all participants. Negative values indicate improvement/reduction in RA disease activity.

Change From Baseline in HAQ-DI ScoreBaseline and Weeks 2, 4, 8, 12, 16, 20, 24

HAQ-DI consisted of 20 questions assessing ADLs in 8 domains (dress/groom, arise, eat, walk, reach, grip, hygiene) with each item rated 0 (no difficulty) to 3 (unable to do). The highest score recorded for any question in a domain determined the score for that domain, unless assistance was required. The total HAQ-DI score was the sum of domain scores divided by the number of domains answered/scored, for a single score range of 0-3, where higher scores indicate increased functional disability. Change from baseline was averaged among all participants. Negative values indicate improvement in ability to perform ADLs.

Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) ScoreBaseline and Weeks 2, 4, 8, 12, 16, 20, 24

FACIT-F consisted of 40 questions/statements assessing chronic illness therapy with special emphasis on fatigue over the past 7 days, with each item rated 0 (not at all) to 4 (very much). During score calculations, negatively-worded item scales (e.g., "I have a lack of energy") were reversed so that higher scores indicated more favorable conditions. The total FACIT-F score was the sum of all item scores and ranged 0-160, and the brief FACIT-F score was the sum of 13 item scores and ranged 0-52, where higher scores indicate greater well-being. Change from baseline was averaged among all participants. Positive values indicate improvement in well-being.

Trial Locations

Locations (27)

Aalborg Universitetshospital Nord, Reumatologisk Afdeling

🇩🇰

Alborg, Denmark

Gentofte Hospital, Medicinsk Afd. C, Klinik for Gigt- og Rygsygdomme

🇩🇰

Hellerup, Denmark

Glostrup Hospital, Reumatologisk Afdeling, Ambulatoriet

🇩🇰

Glostrup, Denmark

Sjællands Universitetshospital, Køge; Reumatologisk Afdeling

🇩🇰

Køge, Denmark

Holbæk Sygehus, Medicinsk Afd., Reumatologisk Amb.15-2

🇩🇰

Holbæk, Denmark

Svendborg Sygehus, Reumatologisk Ambulatorium

🇩🇰

Svendborg, Denmark

Helsinki University Central Hospital; Rheumatology Clinic

🇫🇮

Helsinki, Finland

Kiljavan Lääketutkimus Oy

🇫🇮

Hyvinkää, Finland

Lappeenranta Central Hospital; Outpatient Clinic of Internal Medicine

🇫🇮

Lappeenranta, Finland

Haukeland Universitetssykehus; Revmatologisk Avdeling

🇳🇴

Bergen, Norway

Drammen sykehus Vestre Viken HF, Revmatologisk avd.

🇳🇴

Drammen, Norway

Länssjukhuset Ryhov; Ortoped- och Reumatolog kliniken

🇸🇪

Jönköping, Sweden

Uni Hospital Linkoeping; Dept. of Rheumatology

🇸🇪

Linkoeping, Sweden

Skånes Universitetssjukhus Lund; Reumatologkliniken

🇸🇪

Lund, Sweden

Skånes Universitetssjukhus Malmö; Reumatologkliniken

🇸🇪

Malmo, Sweden

Örebro Uni Hospital; Rheumatology

🇸🇪

Oerebro, Sweden

Akademiska sjukhuset, Reumatologkliniken

🇸🇪

Uppsala, Sweden

Simrishamns Sjukhus

🇸🇪

Simrishamn, Sweden

Karolinska Sjukhuset; Reumatologkliniken D2-1

🇸🇪

Stockholm, Sweden

Västmanlands sjukhus Västerås, Reumatologkliniken

🇸🇪

Västerås, Sweden

Central Hospital of Pohjois-Karjala; Outpatient Clinic of Rheumatology

🇫🇮

Joensuu, Finland

Keski-Suomen Keskussairaala; Sisätautien Klinikka

🇫🇮

Jyväskylä, Finland

Oulu University Hospital; Rheumatology

🇫🇮

Oulu, Finland

Diakonhjemmet; Reumatolgisk Avdeling

🇳🇴

Oslo, Norway

St. Olavs Hospital; Revmatologisk avdeling

🇳🇴

Trondheim, Norway

Ã…lesund Sykehus; Revmatologisk Avdeling

🇳🇴

Ã…lesund, Norway

Odense Universitetshospital, Reumatologisk Afdeling C, Ambulatoriet

🇩🇰

Odense, Denmark

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