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Phase 3 N=112 Randomized Quadruple-blind Treatment

A Study of RoActemra/Actemra (Tocilizumab) in Patients With Active Systemic Juvenile Idiopathic Arthritis (JIA)

Juvenile Idiopathic Arthritis

Enrolled (actual)
112
Serious AEs
42.9%
Results posted
Oct 2011
Primary outcome: Primary: Part I: Percentage of Participants With ≥30% Improvement in Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) Core Set and Absence of Fever — 85.3; 24.3 Percentage of participants

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
tocilizumab [RoActemra/Actemra] (Drug); Placebo (Drug); Non-steroidal anti-inflammatory drugs (NSAIDs) (Drug); methotrexate (Drug); corticosteroids (Drug)
Age
Pediatric · 2+ yrs
Sex
All
Sponsor
Hoffmann-La Roche
Primary completion
Sep 2009

Outcome Measures

OutcomeResultp-value
PRIMARY
Part I: Percentage of Participants With ≥30% Improvement in Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) Core Set and Absence of Fever
85.3; 24.3
PRIMARY
Part II: Percentage of Participants With Decreases in Oral Corticosteroid Dose at Week 104
76; 73; 62; 47
SECONDARY
Part I: Percentage of Participants With JIA Core Set ACR 30/50/70/90 Response at Week 12
90.7; 24.3; 85.3; 10.8; 70.7; 8.1
SECONDARY
Part I: Percentage Change From Baseline in JIA Core Set ACR Score Component: Physician's Global Assessment of Disease Activity
-69.6; -41.1
SECONDARY
Part I: Percentage Change From Baseline in JIA Core Set ACR Score Component: Parent/Patient Global Assessment of Overall Well-being
-65.8; -1.4
SECONDARY
Part I: Percentage Change From Baseline in JIA Core Set ACR Score Component: Maximum Number of Joints With Active Arthritis
-70.6; -37.2
SECONDARY
Part I: Percentage Change From Baseline in JIA Core Set ACR Score Component: Number of Joints With Limitation of Movement
-51.6; -22.5
SECONDARY
Part I: Percentage Change From Baseline in JIA Core Set ACR Score Component: Erythrocyte Sedimentation Rate
-88.2; 33.6
SECONDARY
Part I: Percentage Change From Baseline in JIA Core Set ACR Score Component: Childhood Health Assessment Questionnaire Disability Index (CHAQ-DI)
-45.6; -10.3
SECONDARY
Part I: Percentage of Participants With Fever Due to Systemic Juvenile Idiopathic Arthritis (sJIA) at Baseline Who Are Free of Fever at Week 12
85.4; 20.8
SECONDARY
Part I: Percentage of Participants With Changes in Laboratory Indicators: High-sensitivity C-Reactive Protein(hsCRP), Hemoglobin (Hb), Platelets and Leukocytes From Abnormal at Baseline to Normal at Week 12
98.6; 5.9; 80.0; 6.9; 90.4; 3.8
SECONDARY
Part I: Percentage of Participants With Concomitant Corticosteroid Reduction
24.3; 3.2
SECONDARY
Part I: Change From Baseline in the Pain Visual Analog Scale (VAS) at Week 12
-41.0; -1.1
SECONDARY
Part I: Percentage of Patients With Minimally Important Improvement in CHAQ-DI Score at Week 12
77.3; 18.9
SECONDARY
Part I: Percentage of Patients With Rash at Baseline Who Are Free From Rash at Week 12
63.6; 11.1
SECONDARY
Part I: Percentage of Patients With Anemia at Baseline With a ≥10 g/L Increase in Hemoglobin at Week 6 and Week 12
88.0; 3.4; 88.0; 3.4
SECONDARY
Part II: Percentage of Participants With JIA ACR70 and JIA ACR90 Responses Week 104
76.0; 61.3
SECONDARY
Part II: Number of Active Joints at Week 104
1.9
SECONDARY
Part II: Percentage of Participants With no Active Joints at Week 104
47.4
SECONDARY
Part II: Percentage of Participants With Inactive Disease at Week 104
26.7
SECONDARY
Part II: Childhood Health Assessment Questionnaire-Disability Index (CHAQ-DI) Score at Week 104
1.68; 0.55
SECONDARY
Part II: Percentage of Participants With Oral Corticosteroid Cessation at Week 104
60
SECONDARY
Part II: Rate of Serious Adverse Events (SAEs), Serious Infection Adverse Events (AEs), Related SAEs, Macrophage Activation Syndrome, AEs Leading to Withdrawal and Deaths Per 100 Patient Years to Week 104
23.3; 10.9; 7.4; 1.5; 3.0; 1.5
SECONDARY
Part III: Percentage of Participants With at Least 30%, 50%, 70%, and 90% Improvement in JIA Core Set According to ACR
100.0; 100.0; 100.0; 100.0; 92.9; 95.7
SECONDARY
Part III: Percentage of Participants Who Maintain JIA ACR30, JIA ACR50, JIA ACR70, JIA ACR90 Response for 6 Months Previous to the Specified Week
NA; NA; NA; NA; NA; NA
SECONDARY
Part III: Doses of Oral Corticosteroids
0.203; 0.356; 0.020; 0.047; 0.022; 0.046
SECONDARY
Part III: Percentage of Participants on Corticosteroids at Baseline Able to Discontinue Corticosteroids by Weeks 104,116, 128, 140, 152, 164, 176, 188, 200, 212, 224, 236, 248, and 260
66.0; 67.0; 67.0; 68.0; 69.0; 70.0
SECONDARY
Part III: Percentage of Participants With a >=20/50/75/90% Decrease From Baseline in Oral Corticosteroid Dose at Visits
100.0; 97.8; 94.7; 91.3; 84.2; 82.6
SECONDARY
Part III: Percentage of Participants With Inactive Disease
45.2; 46.8; 48.8; 43.5; 55.0; 48.8
SECONDARY
Part III: Percentage of Participants in Clinical Remission
14.6; 17.4; 32.5; 23.3; 34.2; 25.0
SECONDARY
Part III: Percentage of Participants on Corticosteroids at Baseline in Clinical Remission Off All Oral Corticosteroids for 6 Months Prior to Specified Visits
12.2; 17.4; 32.5; 20.9; 28.9; 20.0
SECONDARY
Part III: Percentage of Participants on Methotrexate At Baseline in Clinical Remission Off Corticosteroids and Methotrexate for 6 Months Prior to Specified Visits
7.3; 8.7; 20.0; 11.6; 18.4; 5.0
SECONDARY
Part III: Percentage of Participants in Clinical Remission Off All Arthritis Medications Except Tocilizumab for 6 Months Prior to Specified Visits
4.9; 8.7; 15.0; 9.3; 15.8; 5.0
SECONDARY
Part III: Percentage of Participants Who Developed Antibodies To Tocilizumab During Weeks 104 to 260
0.0; 0.0
SECONDARY
Part III: Percentage of Participants Who Developed Anti-TCZ Antibodies Associated With The Occurrence of Drug Hypersensitivity Reactions.
0.0; 0.0

Summary

This study will evaluate the efficacy and safety of RoActemra/Actemra (tocilizumab) in patients with active systemic juvenile idiopathic arthritis (sJIA) who have an inadequate clinical response to NSAIDs and corticosteroids. In Part I of the study patients will be randomized 2:1 to receive iv infusions of RoActemra/Actemra (8mg/kg iv for patients >=30kg, or 12mg/kg for patients <30kg) or placebo, every 2 weeks. Stable NSAIDs and methotrexate will be continued throughout. After 12 weeks of double-blind treatment, all patients will have the option to enter Part II of the study to receive open-label treatment with RoActemra/Actemra for a further 92 weeks, followed by a 3-year continuation of the study in Part III in which, for patients who meet specific criteria, an optional alternative dosing schedule decreasing the study drug administration frequency will be introduced. Anticipated time on study treatment is up to 5 years.

Eligibility Criteria

Inclusion Criteria

  • Patients aged 2-17 years of age
  • Systemic juvenile idiopathic arthritis with >= 6 months persistent activity
  • Presence of active disease (>=5 active joints, or >=2 active joints + fever + steroids)
  • Inadequate clinical response to nonsteroidal anti-inflammatory drugs (NSAIDs) and corticosteroids due to toxicity or lack of efficacy

Exclusion Criteria

  • Wheelchair-bound or bed-ridden
  • Any other autoimmune, rheumatic disease or overlap syndrome other than systemic juvenile idiopathic arthritis
  • Intravenous long-acting corticosteroids or intra-articular corticosteroids within 4 weeks of baseline, or throughout study
  • Disease-modifying antirheumatic drugs (DMARDs), other than methotrexate
  • Previous treatment with tocilizumab
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00642460). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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