Trial Outcomes & Findings for A Long-Term Extension Study of RoActemra/Actemra (Tocilizumab) in Patients With Juvenile Idiopathic Arthritis Who Completed WA19977 Core Study (NCT NCT01667471)
NCT ID: NCT01667471
Last Updated: 2016-11-02
Results Overview
Adverse Events (AEs) and Serious Adverse Events (SAEs) were recorded from the first day of tocilizumab administration until 4 weeks after administration of the last dose of tocilizumab. AEs of special interest were Infections (including all opportunistic infections and non-serious infections as defined by those treated with IV anti-infectives), Myocardial infarction/Acute coronary syndrome, Gastrointestinal perforations and related events, Malignancies, Anaphylaxis/Hypersensitivity reactions, Demyelinating disorders, Stroke. Bleeding events, Hepatic events and Macrophage activation syndrome (MAS).
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
6 participants
Primary outcome timeframe
Baseline and every 4 weeks up to Week 76 and Final Follow-Up Visit (up to 82 weeks)
Results posted on
2016-11-02
Participant Flow
Participant milestones
| Measure |
Tocilizumab 8 mg/kg
Participants received tocilizumab 8 mg/kg intravenously (IV) every 4 weeks up to 104 weeks or until tocilizumab was commercially available for polyarticular-course Juvenile Idiopathic Arthritis (pcJIA).
|
|---|---|
|
Overall Study
STARTED
|
7
|
|
Overall Study
COMPLETED
|
7
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Long-Term Extension Study of RoActemra/Actemra (Tocilizumab) in Patients With Juvenile Idiopathic Arthritis Who Completed WA19977 Core Study
Baseline characteristics by cohort
| Measure |
Tocilizumab 8 mg/kg
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Age, Continuous
|
14.4 years
STANDARD_DEVIATION 3.2 • n=93 Participants
|
|
Gender
Female
|
6 Participants
n=93 Participants
|
|
Gender
Male
|
1 Participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Baseline and every 4 weeks up to Week 76 and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set
Adverse Events (AEs) and Serious Adverse Events (SAEs) were recorded from the first day of tocilizumab administration until 4 weeks after administration of the last dose of tocilizumab. AEs of special interest were Infections (including all opportunistic infections and non-serious infections as defined by those treated with IV anti-infectives), Myocardial infarction/Acute coronary syndrome, Gastrointestinal perforations and related events, Malignancies, Anaphylaxis/Hypersensitivity reactions, Demyelinating disorders, Stroke. Bleeding events, Hepatic events and Macrophage activation syndrome (MAS).
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
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|---|---|
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Number of Participants With Adverse Events of Special Interest and Study-Drug Related Adverse Events
Drug related AEs
|
6 participants
|
|
Number of Participants With Adverse Events of Special Interest and Study-Drug Related Adverse Events
Drug related SAEs
|
0 participants
|
|
Number of Participants With Adverse Events of Special Interest and Study-Drug Related Adverse Events
AEs of special interest
|
1 participants
|
|
Number of Participants With Adverse Events of Special Interest and Study-Drug Related Adverse Events
Drug-related AEs of special interest
|
1 participants
|
PRIMARY outcome
Timeframe: Baseline and every 4 weeks up to Week 76 and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set
AEs and SAEs were recorded from the first day of tocilizumab administration until 4 weeks after administration of the last dose of tocilizumab.
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Number of AEs of Special Interest and Study Drug Related AEs
Drug related AEs
|
22 adverse events
|
|
Number of AEs of Special Interest and Study Drug Related AEs
Drug related SAEs
|
0 adverse events
|
|
Number of AEs of Special Interest and Study Drug Related AEs
AEs of special interest
|
2 adverse events
|
|
Number of AEs of Special Interest and Study Drug Related AEs
Drug-related AEs of special interest
|
2 adverse events
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24, 36, 48, 60, 72 and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set; number (n) = number of participants analyzed for the given parameter at the specified visit.
The six JIA ACR components comprised of: 1) Physician's global assessment of disease activity, 2) Parent/Participant's global assessment of overall well-being, 3) Maximum number of joints with active arthritis, 4) Number of joints with limitation of movement, 5) Erythrocyte Sedimentation Rate (ESR) and/or C-reactive Protein (CRP), and 6) Childhood Health Assessment Questionnaire - Disease Index (CHAQ-DI). At an assessment visit, a JIA ACR30/50/70/90 response in comparison to Baseline was defined as: At least three of the six JIA ACR core components improving by at least 30 percent (%), 50%, 70%, or 90% respectively and no more than one of the remaining JIA ACR core components worsening by more than 30%.
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 12 JIA ACR30 (n=7)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 24 JIA ACR30 (n=7)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 36 JIA ACR30 (n=7)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 48 JIA ACR30 (n=6)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 60 JIA ACR30 (n=6)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Baseline JIA ACR50 (n=7)
|
85.7 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 12 JIA ACR50 (n=7)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 24 JIA ACR50 (n=7)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 36 JIA ACR50 (n=7)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 48 JIA ACR50 (n=6)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 60 JIA ACR50 (n=6)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 72 JIA ACR50 (n=2)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Follow-Up JIA ACR50 (n=7)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Baseline JIA ACR70 (n=6)
|
85.7 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 12 JIA ACR70 (n=7)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 24 JIA ACR70 (n=7)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 36 JIA ACR70 (n=7)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 48 JIA ACR70 (n=6)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 60 JIA ACR70 (n=6)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 72 JIA ACR70 (n=2)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Follow-Up JIA ACR70 (n=7)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Baseline JIA ACR90 (n=6)
|
85.7 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 12 JIA ACR90 (n=7)
|
85.7 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 24 JIA ACR90 (n=7)
|
57.1 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 36 JIA ACR90 (n=7)
|
71.4 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 48 JIA ACR90 (n=6)
|
83.3 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 60 JIA ACR90 (n=6)
|
66.7 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 72 JIA ACR90 (n=2)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Follow-Up JIA ACR90 (n=7)
|
85.7 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Baseline JIA ACR30 (n=7)
|
85.7 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Week 72 JIA ACR30 (n=2)
|
100.0 percentage of participants
|
|
Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30/50/70/90 by Visit
Follow-Up JIA ACR30 (n=7)
|
100.0 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24, 36, 48, 60, 72 and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set; n = number of participants analyzed for the given parameter at the specified visit.
A participant was defined to show inactive disease if all of the following criteria were applied: 1) No joints with active arthritis (no joints with swelling and no joints with lack of motion), 2) No fever, rash, serositis, splenomegaly, or generalized lymphadenopathy attributable to JIA, 3) No active uveitis, 4) ESR and/or CRP within normal range, and 5) Physician's global assessment of disease activity equals (=) 0 millimeters (mm) on a Visual analog scale (VAS).
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Percentage of Participants With Inactive Disease by Visit
Baseline(n=7)
|
57.1 percentage of participants
|
|
Percentage of Participants With Inactive Disease by Visit
Week 12 (n=7)
|
42.9 percentage of participants
|
|
Percentage of Participants With Inactive Disease by Visit
Week 24 (n=7)
|
14.3 percentage of participants
|
|
Percentage of Participants With Inactive Disease by Visit
Week 36 (n=7)
|
42.9 percentage of participants
|
|
Percentage of Participants With Inactive Disease by Visit
Week 48 (n=6)
|
16.7 percentage of participants
|
|
Percentage of Participants With Inactive Disease by Visit
Week 60 (n=6)
|
33.3 percentage of participants
|
|
Percentage of Participants With Inactive Disease by Visit
Week 72 (n=2)
|
50.0 percentage of participants
|
|
Percentage of Participants With Inactive Disease by Visit
Follow-Up (n=7)
|
57.1 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Screening, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 60, 64, 68, 72, 76 and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set; n = number of participants analyzed for the given parameter at the specified visit.
CR was defined as "clinical remission with medication (CRem)". A participant was in CR if inactive disease was observed for a minimum of 6 consecutive months.
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Baseline (n=7)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Screening (n=7)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 4 (n=7)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 8 (n=7)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 12 (n=7)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 16 (n=7)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 20 (n=7)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 24 (n=7)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 28 (n=7)
|
14.3 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 32 (n=7)
|
14.3 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 36 (n=7)
|
14.3 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 40 (n=6)
|
16.7 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 44 (n=6)
|
16.7 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 48 (n=6)
|
16.7 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 52 (n=6)
|
16.7 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 56 (n=6)
|
16.7 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 60 (n=6)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 64 (n=5)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 68 (n=3)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 72 (n=2)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Week 76 (n=1)
|
0.0 percentage of participants
|
|
Percentage of Participants Achieving Clinical Remission (CR) at Each Visit
Follow-Up (n=7)
|
0.0 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72 and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set; n = number of participants analyzed at the specified visit.
The participant's treating physician provided a rating of the participant's arthritis disease activity on a 0 to 100 mm horizontal scale. The extreme left end of the line, score 0 represented 'arthritis inactive' (ie, symptom-free and no arthritis symptoms) and the extreme right end score 100 represented 'arthritis very active'. A higher score indicated more disease activity.
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Physicians Assessment of Global Activity (VAS)
Week 48 (n=6)
|
5.7 mm
Standard Deviation 8.2
|
|
Physicians Assessment of Global Activity (VAS)
Baseline (n=7)
|
12.0 mm
Standard Deviation 30.4
|
|
Physicians Assessment of Global Activity (VAS)
Week 12 (n=7)
|
2.3 mm
Standard Deviation 2.9
|
|
Physicians Assessment of Global Activity (VAS)
Week 24 (n=7)
|
5.4 mm
Standard Deviation 6.1
|
|
Physicians Assessment of Global Activity (VAS)
Week 28 (n=1)
|
1.0 mm
Standard Deviation 0.0
|
|
Physicians Assessment of Global Activity (VAS)
Week 32 (n=2)
|
29.0 mm
Standard Deviation 8.5
|
|
Physicians Assessment of Global Activity (VAS)
Week 36 (n=7)
|
5.0 mm
Standard Deviation 7.9
|
|
Physicians Assessment of Global Activity (VAS)
Week 60 (n=6)
|
5.7 mm
Standard Deviation 8.7
|
|
Physicians Assessment of Global Activity (VAS)
Week 72 (n=2)
|
1.0 mm
Standard Deviation 1.4
|
|
Physicians Assessment of Global Activity (VAS)
Follow-Up (n=7)
|
5.0 mm
Standard Deviation 8.7
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72 and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set; n = number of participants analyzed at the specified visit.
The participant or parent/guardian, as appropriate, provided a rating of the participant's well-being on a 0 to 100 mm horizontal scale. The extreme left end of the line Score 0 represented 'very well' (ie, symptom-free and no arthritis disease activity) and the extreme right end score 100 represented 'very poor' (ie, maximum arthritis disease activity). A higher score indicated poorer well-being.
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Parent or Participant's Assessment of Global Activity (VAS)
Week 12 (n=7)
|
7.1 mm
Standard Deviation 8.1
|
|
Parent or Participant's Assessment of Global Activity (VAS)
Week 24 (n=7)
|
16.4 mm
Standard Deviation 19.9
|
|
Parent or Participant's Assessment of Global Activity (VAS)
Week 28 (n=1)
|
1.0 mm
Standard Deviation 0.0
|
|
Parent or Participant's Assessment of Global Activity (VAS)
Week 32 (n=2)
|
22.5 mm
Standard Deviation 24.7
|
|
Parent or Participant's Assessment of Global Activity (VAS)
Week 36 (n=7)
|
12.1 mm
Standard Deviation 18.4
|
|
Parent or Participant's Assessment of Global Activity (VAS)
Week 48 (n=6)
|
11.7 mm
Standard Deviation 13.9
|
|
Parent or Participant's Assessment of Global Activity (VAS)
Week 60 (n=6)
|
10.2 mm
Standard Deviation 13.8
|
|
Parent or Participant's Assessment of Global Activity (VAS)
Week 72 (n=2)
|
3.5 mm
Standard Deviation 4.9
|
|
Parent or Participant's Assessment of Global Activity (VAS)
Follow-Up (n=7)
|
9.7 mm
Standard Deviation 16.0
|
|
Parent or Participant's Assessment of Global Activity (VAS)
Baseline (n=7)
|
10.1 mm
Standard Deviation 20.0
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72 and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set; n = number of participants analyzed at the specified visit.
Joints with active arthritis were defined as joints with swelling or pain and limited of motion. The maximum number of joints with active arthritis was 71.The joint assessment was performed by an independent assessor who was not the treating physician and who was blinded to all other aspects of the participant's efficacy and safety data.
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Number of Joints With Active Arthritis
Baseline (n=7)
|
0.9 joints
Standard Deviation 2.3
|
|
Number of Joints With Active Arthritis
Week 12 (n=7)
|
0.3 joints
Standard Deviation 0.8
|
|
Number of Joints With Active Arthritis
Week 24 (n=7)
|
1.0 joints
Standard Deviation 2.6
|
|
Number of Joints With Active Arthritis
Week 28 (n=1)
|
1.0 joints
Standard Deviation 0.0
|
|
Number of Joints With Active Arthritis
Week 32 (n=2)
|
1.5 joints
Standard Deviation 0.7
|
|
Number of Joints With Active Arthritis
Week 36 (n=7)
|
1.1 joints
Standard Deviation 2.2
|
|
Number of Joints With Active Arthritis
Week 48 (n=6)
|
0.0 joints
Standard Deviation 0.0
|
|
Number of Joints With Active Arthritis
Week 60 (n=6)
|
0.3 joints
Standard Deviation 0.8
|
|
Number of Joints With Active Arthritis
Week 72 (n=2)
|
0.0 joints
Standard Deviation 0.0
|
|
Number of Joints With Active Arthritis
Follow-Up (n=7)
|
0.3 joints
Standard Deviation 0.8
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72 and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set; n = number of participants analyzed at the specified visit.
Joints with lack of movement were assessed. The maximum number of joints with lack of movement was 67. The joint assessment was performed by an independent assessor who was not the treating physician and who was blinded to all other aspects of the participant's efficacy and safety data.
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Number of Joints With Lack of Motion
Week 12 (n=7)
|
2.6 joints
Standard Deviation 5.1
|
|
Number of Joints With Lack of Motion
Week 24 (n=7)
|
2.4 joints
Standard Deviation 3.2
|
|
Number of Joints With Lack of Motion
Week 28 (n=1)
|
1.0 joints
Standard Deviation 0.0
|
|
Number of Joints With Lack of Motion
Week 32 (n=2)
|
5.5 joints
Standard Deviation 7.8
|
|
Number of Joints With Lack of Motion
Week 36 (n=7)
|
1.7 joints
Standard Deviation 2.4
|
|
Number of Joints With Lack of Motion
Week 48 (n=6)
|
2.7 joints
Standard Deviation 5.2
|
|
Number of Joints With Lack of Motion
Week 60 (n=6)
|
1.8 joints
Standard Deviation 3.0
|
|
Number of Joints With Lack of Motion
Week 72 (n=2)
|
0.0 joints
Standard Deviation 0.0
|
|
Number of Joints With Lack of Motion
Follow-Up (n=7)
|
2.9 joints
Standard Deviation 4.8
|
|
Number of Joints With Lack of Motion
Baseline (n=7)
|
1.4 joints
Standard Deviation 2.7
|
SECONDARY outcome
Timeframe: Baseline, Weeks 4, 8,12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 60, 64, 68, 72, 76 and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set; n = number of participants analyzed at the specified visit.
ESR is a marker of inflammation and was measured as millimeters per hour (mm/h). Healthy individuals have low ESR. Higher ESR indicate inflammation.
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Erythrocyte Sedimentation Rate
Baseline (n=7)
|
4.9 mm/h
Standard Deviation 5.1
|
|
Erythrocyte Sedimentation Rate
Week 4 (n=7)
|
3.0 mm/h
Standard Deviation 1.9
|
|
Erythrocyte Sedimentation Rate
Week 24 (n=7)
|
2.7 mm/h
Standard Deviation 1.4
|
|
Erythrocyte Sedimentation Rate
Week 28 (n=5)
|
3.4 mm/h
Standard Deviation 3.1
|
|
Erythrocyte Sedimentation Rate
Week 32 (n=7)
|
4.3 mm/h
Standard Deviation 4.2
|
|
Erythrocyte Sedimentation Rate
Week 36 (n=7)
|
4.3 mm/h
Standard Deviation 2.5
|
|
Erythrocyte Sedimentation Rate
Week 40 (n=6)
|
2.0 mm/h
Standard Deviation 1.8
|
|
Erythrocyte Sedimentation Rate
Week 44 (n=6)
|
2.7 mm/h
Standard Deviation 1.5
|
|
Erythrocyte Sedimentation Rate
Week 48 (n=6)
|
3.8 mm/h
Standard Deviation 1.5
|
|
Erythrocyte Sedimentation Rate
Week 52 (n=6)
|
3.2 mm/h
Standard Deviation 3.6
|
|
Erythrocyte Sedimentation Rate
Week 56 (n=6)
|
2.3 mm/h
Standard Deviation 1.6
|
|
Erythrocyte Sedimentation Rate
Week 60 (n=6)
|
10.2 mm/h
Standard Deviation 12.7
|
|
Erythrocyte Sedimentation Rate
Week 64 (n=5)
|
3.2 mm/h
Standard Deviation 2.4
|
|
Erythrocyte Sedimentation Rate
Week 68 (n=3)
|
10.0 mm/h
Standard Deviation 7.2
|
|
Erythrocyte Sedimentation Rate
Week 72 (n=2)
|
4.5 mm/h
Standard Deviation 0.7
|
|
Erythrocyte Sedimentation Rate
Week 76 (n=1)
|
2.0 mm/h
Standard Deviation 0.0
|
|
Erythrocyte Sedimentation Rate
Follow-Up (n=7)
|
4.3 mm/h
Standard Deviation 2.8
|
|
Erythrocyte Sedimentation Rate
Week 8 (n=7)
|
2.9 mm/h
Standard Deviation 2.3
|
|
Erythrocyte Sedimentation Rate
Week 12 (n=7)
|
3.0 mm/h
Standard Deviation 2.0
|
|
Erythrocyte Sedimentation Rate
Week 16 (n=7)
|
8.6 mm/h
Standard Deviation 16.1
|
|
Erythrocyte Sedimentation Rate
Week 20 (n=7)
|
2.6 mm/h
Standard Deviation 0.8
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72, and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set; n = number of participants analyzed at the specified visit.
The CHAQ-DI questionnaire consisted of 30 questions referring to eight domains: dressing/grooming, arising, eating, walking, hygiene, reach, grip and activities. Each domain had at least two component questions and if applicable to the participant there were four possible responses (0 = without any difficulty, 1 = with some difficulty, 2 = with much difficulty, 3 = unable to do). The CHAQ-DI score is the sum of the domain scores divided by the number of domains that have a non-missing score. This overall score ranges from 0 (best) to 3 (worst).
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
CHAQ-DI Score
Baseline (n=7)
|
0.0 score on a scale
Standard Deviation 0.0
|
|
CHAQ-DI Score
Week 32 (n=2)
|
0.19 score on a scale
Standard Deviation 0.27
|
|
CHAQ-DI Score
Week 36 (n=7)
|
0.13 score on a scale
Standard Deviation 0.33
|
|
CHAQ-DI Score
Week 48 (n=6)
|
0.00 score on a scale
Standard Deviation 0.00
|
|
CHAQ-DI Score
Follow-Up (n=7)
|
0.00 score on a scale
Standard Deviation 0.00
|
|
CHAQ-DI Score
Week 12 (n=7)
|
0.04 score on a scale
Standard Deviation 0.09
|
|
CHAQ-DI Score
Week 24 (n=7)
|
0.14 score on a scale
Standard Deviation 0.20
|
|
CHAQ-DI Score
Week 28 (n=1)
|
0.00 score on a scale
Standard Deviation 0.0
|
|
CHAQ-DI Score
Week 60 (n=6)
|
0.21 score on a scale
Standard Deviation 0.51
|
|
CHAQ-DI Score
Week 72 (n=2)
|
0.00 score on a scale
Standard Deviation 0.00
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24, 28, 32, 36, 48, 60, 72, and Final Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set; n = number of participants analyzed at the specified visit.
Parents or participants rated participant's pain by placing a horizontal line on a VAS of 0 (no pain)- 100 mm (severe pain).
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Parent or Participant's Assessment of Pain (VAS)
Week 12 (n=7)
|
7.0 mm
Standard Deviation 10.0
|
|
Parent or Participant's Assessment of Pain (VAS)
Week 24 (n=7)
|
13.7 mm
Standard Deviation 16.7
|
|
Parent or Participant's Assessment of Pain (VAS)
Week 28 (n=1)
|
1.0 mm
Standard Deviation 0.0
|
|
Parent or Participant's Assessment of Pain (VAS)
Week 32 (n=2)
|
28.5 mm
Standard Deviation 17.7
|
|
Parent or Participant's Assessment of Pain (VAS)
Week 36 (n=7)
|
10.6 mm
Standard Deviation 15.9
|
|
Parent or Participant's Assessment of Pain (VAS)
Week 48 (n=6)
|
12.3 mm
Standard Deviation 13.6
|
|
Parent or Participant's Assessment of Pain (VAS)
Week 60 (n=6)
|
10.2 mm
Standard Deviation 13.8
|
|
Parent or Participant's Assessment of Pain (VAS)
Week 72 (n=2)
|
4.0 mm
Standard Deviation 5.7
|
|
Parent or Participant's Assessment of Pain (VAS)
Follow-Up (n=7)
|
9.9 mm
Standard Deviation 15.9
|
|
Parent or Participant's Assessment of Pain (VAS)
Baseline (n=7)
|
10.9 mm
Standard Deviation 21.8
|
SECONDARY outcome
Timeframe: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 60, 64, 68, 72, 76 and Final Follow-Up Follow-Up Visit (up to 82 weeks)Population: Safety Analysis Set; n = number of participants analyzed at the specified visit.
CRP an acute phase protein, is a marker of inflammation. CRP was measured as milligrams per deciliter (mg/dL).
Outcome measures
| Measure |
Tocilizumab
n=7 Participants
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
CRP Levels
Baseline (n=7)
|
0.03 mg/dL
Standard Deviation 0.01
|
|
CRP Levels
Week 4 (n=7)
|
0.05 mg/dL
Standard Deviation 0.07
|
|
CRP Levels
Week 8 (n=7)
|
0.02 mg/dL
Standard Deviation 0.02
|
|
CRP Levels
Week 12 (n=7)
|
0.03 mg/dL
Standard Deviation 0.01
|
|
CRP Levels
Week 28 (n=6)
|
0.02 mg/dL
Standard Deviation 0.02
|
|
CRP Levels
Week 32 (n=7)
|
0.03 mg/dL
Standard Deviation 0.02
|
|
CRP Levels
Week 36 (n=7)
|
0.02 mg/dL
Standard Deviation 0.02
|
|
CRP Levels
Week 40 (n=6)
|
0.03 mg/dL
Standard Deviation 0.02
|
|
CRP Levels
Week 44 (n=6)
|
0.03 mg/dL
Standard Deviation 0.02
|
|
CRP Levels
Week 48 (n=6)
|
0.03 mg/dL
Standard Deviation 0.02
|
|
CRP Levels
Week 52 (n=6)
|
0.02 mg/dL
Standard Deviation 0.02
|
|
CRP Levels
Week 56 (n=6)
|
0.03 mg/dL
Standard Deviation 0.02
|
|
CRP Levels
Week 60 (n=6)
|
0.38 mg/dL
Standard Deviation 0.90
|
|
CRP Levels
Week 64 (n=5)
|
0.03 mg/dL
Standard Deviation 0.03
|
|
CRP Levels
Week 68 (n=3)
|
0.09 mg/dL
Standard Deviation 0.10
|
|
CRP Levels
Week 72 (n=2)
|
0.04 mg/dL
Standard Deviation 0.04
|
|
CRP Levels
Week 76 (n=1)
|
0.05 mg/dL
Standard Deviation 0.0
|
|
CRP Levels
Follow-Up (n=7)
|
0.05 mg/dL
Standard Deviation 0.06
|
|
CRP Levels
Week 16 (n=7)
|
0.23 mg/dL
Standard Deviation 0.54
|
|
CRP Levels
Week 20 (n=7)
|
0.05 mg/dL
Standard Deviation 0.07
|
|
CRP Levels
Week 24 (n=7)
|
0.03 mg/dL
Standard Deviation 0.02
|
Adverse Events
Tocilizumab 8 mg/kg
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Tocilizumab 8 mg/kg
n=7 participants at risk
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Infections and infestations
Appendicitis
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
Other adverse events
| Measure |
Tocilizumab 8 mg/kg
n=7 participants at risk
Participants received tocilizumab 8 mg/kg IV every 4 weeks up to 104 weeks or until tocilizumab was commercially available for pcJIA.
|
|---|---|
|
Blood and lymphatic system disorders
Neutropenia
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Cardiac disorders
Aortic valve incompetence
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Cardiac disorders
Mitral valve incompetence
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Gastrointestinal disorders
Abdominal pain
|
28.6%
2/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Gastrointestinal disorders
Abdominal pain upper
|
28.6%
2/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Gastrointestinal disorders
Constipation
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Gastrointestinal disorders
Epigastric discomfort
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Gastrointestinal disorders
Gastritis
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Gastrointestinal disorders
Nausea
|
28.6%
2/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Gastrointestinal disorders
Vomiting
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
General disorders
Injection site swelling
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
General disorders
Local swelling
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
General disorders
Vaccination site reaction
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Acute tonsillitis
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Bronchitis
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Cystitis
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Gastroenteritis
|
57.1%
4/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Herpes simplex
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Lice infestation
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Nasopharyngitis
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Otitis externa
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Rhinitis
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Scarlet fever
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Sinusitis
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Upper respiratory tract infection
|
42.9%
3/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Infections and infestations
Vulvovaginal mycotic infection
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Injury, poisoning and procedural complications
Excoriation
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Injury, poisoning and procedural complications
Contusion
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Injury, poisoning and procedural complications
Fall
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Investigations
Biopsy kidney
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Investigations
Hepatic enzyme increased
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Investigations
White blood cell count decreased
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
57.1%
4/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Musculoskeletal and connective tissue disorders
Foot deformity
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Musculoskeletal and connective tissue disorders
Joint effusion
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Musculoskeletal and connective tissue disorders
Juvenile idiopathic arthritis
|
42.9%
3/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar disorder
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Skin and subcutaneous tissue disorders
Psoriasis
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
14.3%
1/7 • Adverse events were collected from the date of screening until the final Follow-Up Visit (up to 82 weeks).
|
Additional Information
Medical Communications
Hoffmann-LaRoche or Genentech, Inc
Phone: 800-821-8590
Email: [email protected]
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 only after the first publication or presentation that involves the Overall Study. The 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