Trial Outcomes & Findings for A Long-Term Extension Study of RoActemra/Actemra (Tocilizumab) in Patients With Juvenile Idiopathic Arthritis From France Who Completed WA19977 Core Study (NCT NCT01673919)

NCT ID: NCT01673919

Last Updated: 2025-09-12

Results Overview

An adverse event (AE) is defined as any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered to be related to the medicinal product. An serious adverse event (SAE) is any untoward medical occurrence that, at any dose, results in death, is life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, or results in a congenital anomaly/birth defect.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 7 participants
• Primary outcome timeframe: Approximately 2 years
• Results posted on: 2025-09-12

Participant Flow

A total of seven participants who completed the core study WA19977 were enrolled in this extension study conducted from 13 February 2012 to 15 January 2014 at four centers in France.

Participant milestones

Measure	Tocilizumab (8 mg/kg) Eligible participants received tocilizumab (TCZ) 8 milligram/kilogram (mg/kg) intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for polyarticular-course Juvenile Idiopathic Arthritis (pcJIA) in France, whichever came first.
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 From France Who Completed WA19977 Core Study

Baseline characteristics by cohort

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Age, Customized 2 to 11 years	3 participants n=93 Participants
Age, Customized 12 to 17 years	2 participants n=93 Participants
Age, Customized 18 to 64 years	2 participants n=93 Participants
Sex: Female, Male Female	7 Participants n=93 Participants
Sex: Female, Male Male	0 Participants n=93 Participants

PRIMARY outcome

Timeframe: Approximately 2 years

Population: Safety population included all participants who received at least a single dose of study drug.

An adverse event (AE) is defined as any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered to be related to the medicinal product. An serious adverse event (SAE) is any untoward medical occurrence that, at any dose, results in death, is life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, or results in a congenital anomaly/birth defect.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Participants With Any Adverse Events and Any Serious Adverse Events Any AEs	7 participants
Number of Participants With Any Adverse Events and Any Serious Adverse Events Any SAEs	0 participants

PRIMARY outcome

Timeframe: Approximately 2 years

Population: Safety population included all participants who received at least a single dose of study drug.

An AE is defined as any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered to be related to the medicinal product. The AEs of special interests included gingival bleeding, tooth abscess, acarodermatitis, ear infection, gastroenteritis, herpes zoster ophthalmic, lice infestation, nasopharyngitis, oral fungal infection, oral herpes, pharyngitis, rhinitis, sinusitis, tonsillitis, tracheitis, tracheobronchitis, urinary tract infection, menorrhagia, asthma, epistaxis, and hematoma.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Participants With Adverse Events of Special Interest Gingival bleeding	2 participants
Number of Participants With Adverse Events of Special Interest Tooth abscess	2 participants
Number of Participants With Adverse Events of Special Interest Acarodermatitis	1 participants
Number of Participants With Adverse Events of Special Interest Ear infection	1 participants
Number of Participants With Adverse Events of Special Interest Gastroenteritis	1 participants
Number of Participants With Adverse Events of Special Interest Herpes zoster ophthalmic	1 participants
Number of Participants With Adverse Events of Special Interest Lice infestation	1 participants
Number of Participants With Adverse Events of Special Interest Nasopharyngitis	1 participants
Number of Participants With Adverse Events of Special Interest Oral fungal infection	1 participants
Number of Participants With Adverse Events of Special Interest Oral herpes	1 participants
Number of Participants With Adverse Events of Special Interest Pharyngitis	1 participants
Number of Participants With Adverse Events of Special Interest Rhinitis	1 participants
Number of Participants With Adverse Events of Special Interest Sinusitis	1 participants
Number of Participants With Adverse Events of Special Interest Tonsillitis	1 participants
Number of Participants With Adverse Events of Special Interest Tracheitis	1 participants
Number of Participants With Adverse Events of Special Interest Tracheobronchitis	1 participants
Number of Participants With Adverse Events of Special Interest Urinary tract infection	1 participants
Number of Participants With Adverse Events of Special Interest Menorrhagia	1 participants
Number of Participants With Adverse Events of Special Interest Asthma	1 participants
Number of Participants With Adverse Events of Special Interest Epistaxis	1 participants
Number of Participants With Adverse Events of Special Interest Hematoma	1 participants

PRIMARY outcome

Timeframe: Approximately 2 years

Population: Safety population included all participants who received at least a single dose of study drug.

An AE is defined as any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered to be related to the medicinal product. Relatedness of any AEs was reported as possibly related, probably related, or remotely related to TCZ.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Participants With Adverse Events Related to Tocilizumab AEs possibly related to TCZ	5 participants
Number of Participants With Adverse Events Related to Tocilizumab AEs probably related to TCZ	2 participants
Number of Participants With Adverse Events Related to Tocilizumab AEs remotely related to TCZ	1 participants

SECONDARY outcome

Timeframe: Approximately 2 years

Population: Safety population included all participants who received at least a single dose of study drug.

Participants received TCZ for Week 104 or when TCZ was commercially available for pcJIA participants, whichever comes first in France. The mean TCZ exposure (time from first to last administration) was reported.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Mean Exposure to Study Treatment	17.08 Months Standard Deviation 1.55

SECONDARY outcome

Timeframe: Approximately 2 years

Population: Safety population included all participants who received at least a single dose of study drug.

The participants were followed-up from Day 1 to last visit date (approximately 2 years). Mean time for which participants were followed up in the study was reported.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Mean Duration of Study Follow-Up	21.08 Months Standard Deviation 1.64

SECONDARY outcome

Timeframe: Approximately 2 years

Population: Safety population included all participants who received at least a single dose of study drug.

Number of participants with AEs leading to TCZ modification, AEs leading to death, anaphylaxis or serious hypersensitivity, and deaths were reported.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Participants With AEs Leading to TCZ Modification, AEs Leading to Death, Anaphylaxis or Serious Hypersensitivity and Deaths AEs leading to TCZ modification	2 participants
Number of Participants With AEs Leading to TCZ Modification, AEs Leading to Death, Anaphylaxis or Serious Hypersensitivity and Deaths AEs leading to death	0 participants
Number of Participants With AEs Leading to TCZ Modification, AEs Leading to Death, Anaphylaxis or Serious Hypersensitivity and Deaths Anaphylaxis or serious hypersensitivity	0 participants
Number of Participants With AEs Leading to TCZ Modification, AEs Leading to Death, Anaphylaxis or Serious Hypersensitivity and Deaths Death	0 participants

SECONDARY outcome

Timeframe: Approximately 2 years

Population: Safety population included all participants who received at least a single dose of study drug.

Clinically significant abnormal parameters included eosinophil count, alanine aminotransferase, total bilirubin, and protein and blood in urine. Number of participants with these abnormal lab parameters was reported.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Participants With Clinically Significant Abnormal Laboratory Parameters Total bilirubin	1 participants
Number of Participants With Clinically Significant Abnormal Laboratory Parameters Protein and blood in urine	2 participants
Number of Participants With Clinically Significant Abnormal Laboratory Parameters Eosinophil count	1 participants
Number of Participants With Clinically Significant Abnormal Laboratory Parameters Alanine aminotransferase	1 participants

SECONDARY outcome

Timeframe: Approximately 2 years

Population: Safety population included all participants who received at least a single dose of study drug.

Participants with abnormal physical examinations of ear, nose and throat (asthma); extremities (synovitis, sequelae with flexion of the 5th right proximal interphalangeal joint, hallux valgus, deviations of metatarsophalangeal joints, and callus under metatarsal head); lung (mild bronchospasm); skin (vitiligo and hematoma, fatty subcutaneous infiltration on the neck, cutaneous eruption, and scalp pediculosis); and musculoskeletal system (discomfort in right hip) were reported.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Participants With Abnormality in Physical Examinations Ear, nose and throat	1 participants
Number of Participants With Abnormality in Physical Examinations Extremities	1 participants
Number of Participants With Abnormality in Physical Examinations Lung	1 participants
Number of Participants With Abnormality in Physical Examinations Musculoskeletal system	1 participants
Number of Participants With Abnormality in Physical Examinations Skin	3 participants

SECONDARY outcome

Timeframe: Weeks 12, 24, 36, 48, 60, 72, 84, and 108

Population: The ITT population included all participants who had at least one efficacy assessment. Number (n) = number of participants analyzed for the given parameter at the specified visit.

The Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) is comprised of six components: Maximum number of joints with active arthritis; Number of joints with limitation of movement; Erythrocyte Sedimentation Rate (ESR) and/or C-reactive Protein (CRP); Childhood Health Assessment Questionnaire-Disease Index (CHAQ-DI) graded on 4-point scales [0 = without any difficulty and 3 = unable to do] of 30 items grouped into 8 domains of physical function; Physician's global assessment of disease activity and Participant's global assessment of overall well-being (both assessed on a 0 to 100 mm Visual Analogue Scale [VAS], where score 0 = inactive arthritis and 100 = very active arthritis). A JIA ACR50/70 response is defined as improvement in at least three of the six core components by at least 50 percent (%), or 70%, respectively and no more than one of the remaining core components worsening by more than 30%.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=6 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR50, Week 12 (n = 5)	1 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR50, Week 24 (n = 5)	2 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR50, Week 36 (n = 6)	1 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR50, Week 48 (n = 5)	1 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR50, Week 60 (n = 5)	0 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR50, Week 72 (n = 2)	0 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR50, Week 84 (n = 1)	1 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR50, Week 108 (n = 6)	2 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR70, Week 12 (n = 5)	4 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR70, Week 24 (n = 5)	3 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR70, Week 36 (n = 6)	5 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR70, Week 48 (n = 5)	4 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR70, Week 60 (n = 5)	5 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR70, Week 72 (n = 2)	2 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR70, Week 84 (n = 1)	0 participants
Number of Participants With Juvenile Idiopathic Arthritis American College of Rheumatology Response 50/70 JIA ACR70, Week 108 (n = 6)	4 participants

SECONDARY outcome

Timeframe: Weeks 24, 36, 48, 72, and 108

Population: The ITT population included all participants who had at least one efficacy assessment. n = number of participants analyzed for the given parameter at the specified visit.

Inactive disease was defined as: 1) No joints with active arthritis (no swollen, painful and lack of motion joints), 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) No disease activity according to Physician's global assessment of disease activity (<= 10 millimeters [mm] on a VAS). The participant's treating physician provided a rating of the participant's arthritis disease activity on a 0 to 100 mm horizontal scale where score 0 represented 'arthritis inactive' (i.e., symptom-free and no arthritis symptoms) and score 100 represented 'arthritis very active'.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Participants With Inactive Disease Week 24 (n = 6)	1 participants
Number of Participants With Inactive Disease Week 36 (n = 7)	1 participants
Number of Participants With Inactive Disease Week 48 (n = 6)	1 participants
Number of Participants With Inactive Disease Week 72 (n = 4)	1 participants
Number of Participants With Inactive Disease Week 108 (n = 7)	1 participants

SECONDARY outcome

Timeframe: Weeks 24, 36, 48, 72, and 108

Population: The ITT population included all participants who had at least one efficacy assessment. n = number of participants analyzed for the given parameter at the specified visit.

Clinical remission was defined as inactive disease observed for at least 6 continuous months. Clinical remission was defined as per medication uptake as: Level 1 (clinical remission on medication), Level 2 (clinical remission off oral corticosteroid medication [still on TCZ]), Level 3 (clinical remission off both oral corticosteroid and methotrexate medication [still on TCZ]), and Level 4 (clinical remission off all anti-inflammatory medications [still on TCZ]). Number of participants at each clinical remission level was reported.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Participants Achieving Clinical Remission Week 24, Remission level 1 (n = 7)	1 participants
Number of Participants Achieving Clinical Remission Week 36, Remission level 1 (n = 6)	5 participants
Number of Participants Achieving Clinical Remission Week 48, Remission level 1 (n = 6)	1 participants
Number of Participants Achieving Clinical Remission Week 72, Remission level 1 (n = 4)	1 participants
Number of Participants Achieving Clinical Remission Week 108, Remission level 1 (n = 7)	1 participants

SECONDARY outcome

Timeframe: Weeks 12, 24, 36, 48, 60, 72, 84, and 108

Population: The ITT population included all the participants who had at least one efficacy assessment. n = number of participants analyzed for the given parameter at the specified visit.

The CHAQ-DI included questions on dressing and grooming, arising, eating, walking, hygiene, reach, grip, and activities. The disability index (DI) of the original CHAQ was graded on 4-point categorical scales of 30 items grouped into 8 domains of physical function. The highest scoring item in each domain determined the score for that domain. The score for the disability index was the mean of domain scores ranging from 0 to 3 (0 = without any difficulty and 3 = unable to do) with higher scores meaning higher disability. Minimally important improvement in CHAQ-DI was defined as a change from baseline of WA19977 core study (Day 1/Visit 1) >=0.13 at each visit.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Participants With a Minimally Important Improvement in the Childhood Health Assessment Questionnaire-Disability Index Week 12 (n=7)	5 participants
Number of Participants With a Minimally Important Improvement in the Childhood Health Assessment Questionnaire-Disability Index Week 24 (n=7)	5 participants
Number of Participants With a Minimally Important Improvement in the Childhood Health Assessment Questionnaire-Disability Index Week 36 (n=7)	6 participants
Number of Participants With a Minimally Important Improvement in the Childhood Health Assessment Questionnaire-Disability Index Week 48 (n=7)	5 participants
Number of Participants With a Minimally Important Improvement in the Childhood Health Assessment Questionnaire-Disability Index Week 60 (n=7)	6 participants
Number of Participants With a Minimally Important Improvement in the Childhood Health Assessment Questionnaire-Disability Index Week 72 (n=3)	2 participants
Number of Participants With a Minimally Important Improvement in the Childhood Health Assessment Questionnaire-Disability Index Week 84 (n=1)	0 participants
Number of Participants With a Minimally Important Improvement in the Childhood Health Assessment Questionnaire-Disability Index Week 108 (n=7)	6 participants

SECONDARY outcome

Timeframe: Baseline (Day 1), Weeks 12, 24, 36, 48, 60, 72, 84, and 108

Population: The ITT population included all the participants who had at least one efficacy assessment. n = number of participants analyzed for the given parameter at the specified visit.

The most frequent symptom reported by most participants was a limitation of motion (LOM) of joints and it was determined by physical examination. The mean joints with limitation of motion were reported.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Joints With Limitation of Motion Day 1 (n=7)	11.4 joints Standard Deviation 14.3
Number of Joints With Limitation of Motion Week 12 (n=7)	9.4 joints Standard Deviation 11.9
Number of Joints With Limitation of Motion Week 24 (n=6)	6.7 joints Standard Deviation 8.4
Number of Joints With Limitation of Motion Week 36 (n=7)	8.1 joints Standard Deviation 9.5
Number of Joints With Limitation of Motion Week 48 (n=7)	8.6 joints Standard Deviation 10.5
Number of Joints With Limitation of Motion Week 60 (n=7)	10.3 joints Standard Deviation 14.8
Number of Joints With Limitation of Motion Week 72 (n=4)	8.5 joints Standard Deviation 16.3
Number of Joints With Limitation of Motion Week 84 (n=1)	31.0 joints Standard Deviation NA Data was available for only one participant; therefore, standard deviation cannot be calculated.
Number of Joints With Limitation of Motion Week 108 (n=7)	10.6 joints Standard Deviation 13.4

SECONDARY outcome

Timeframe: Baseline (Day 1), Weeks 12, 24, 36, 48, 60, 72, 84, and 108

Population: The ITT population included all the participants who had at least one efficacy assessment. n = number of participants analyzed for the given parameter at the specified visit.

The active range of motion joints was counted by physical examination and mean joints was reported.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Joints With Active Range of Motion Day 1 (n=7)	2.3 joints Standard Deviation 2.5
Number of Joints With Active Range of Motion Week 12 (n=7)	1.4 joints Standard Deviation 2.3
Number of Joints With Active Range of Motion Week 24 (n=6)	2.2 joints Standard Deviation 2.2
Number of Joints With Active Range of Motion Week 36 (n=7)	1.3 joints Standard Deviation 1.8
Number of Joints With Active Range of Motion Week 48 (n=7)	2.0 joints Standard Deviation 1.9
Number of Joints With Active Range of Motion Week 60 (n=7)	2.0 joints Standard Deviation 2.2
Number of Joints With Active Range of Motion Week 72 (n=4)	0.3 joints Standard Deviation 0.5
Number of Joints With Active Range of Motion Week 84 (n=1)	0.0 joints Standard Deviation NA Data was available for only one participant; therefore, standard deviation cannot be calculated.
Number of Joints With Active Range of Motion Week 108 (n=7)	1.1 joints Standard Deviation 1.2

SECONDARY outcome

Timeframe: Baseline (Day 1), Weeks 12, 24, 36, 48, 60, 72, 84, and 108

Population: The ITT population included all the participants who had at least one efficacy assessment. n = number of participants analyzed for the given parameter at the specified visit.

The swollen joints was counted by physical examination and mean swollen joints were reported.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Swollen Joints Day 1 (n=7)	1.6 joints Standard Deviation 2.1
Number of Swollen Joints Week 12 (n=7)	1.0 joints Standard Deviation 2.2
Number of Swollen Joints Week 24 (n=6)	1.0 joints Standard Deviation 1.7
Number of Swollen Joints Week 36 (n=7)	0.9 joints Standard Deviation 1.5
Number of Swollen Joints Week 48 (n=7)	1.9 joints Standard Deviation 1.8
Number of Swollen Joints Week 60 (n=7)	1.7 joints Standard Deviation 2.2
Number of Swollen Joints Week 72 (n=4)	0.3 joints Standard Deviation 0.5
Number of Swollen Joints Week 84 (n=1)	0.0 joints Standard Deviation NA Data was available for only one participant; therefore, standard deviation cannot be calculated.
Number of Swollen Joints Week 108 (n=7)	0.7 joints Standard Deviation 1.3

SECONDARY outcome

Timeframe: Baseline (Day 1), Weeks 12, 24, 36, 48, 60, 72, 84, and 108

Population: The ITT population included all the participants who had at least one efficacy assessment. n = number of participants analyzed for the given parameter at the specified visit.

The painful joints were counted by physical examination and mean painful joints was reported.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Number of Painful Joints Day 1 (n=7)	1.9 joints Standard Deviation 2.3
Number of Painful Joints Week 12 (n=7)	0.6 joints Standard Deviation 0.8
Number of Painful Joints Week 24 (n=6)	2.0 joints Standard Deviation 2.8
Number of Painful Joints Week 36 (n=7)	0.7 joints Standard Deviation 1.0
Number of Painful Joints Week 48 (n=7)	0.9 joints Standard Deviation 1.1
Number of Painful Joints Week 60 (n=7)	0.7 joints Standard Deviation 1.0
Number of Painful Joints Week 72 (n=4)	0.5 joints Standard Deviation 1.0
Number of Painful Joints Week 84 (n=1)	0.0 joints Standard Deviation NA Data was available for only one participant; therefore, standard deviation cannot be calculated.
Number of Painful Joints Week 108 (n=7)	2.0 joints Standard Deviation 4.0

SECONDARY outcome

Timeframe: Baseline (Day 1), Weeks 12, 24, 36, 48, 60, 72, 84, and 108

Population: The ITT population included all the participants who had at least one efficacy assessment. n = number of participants analyzed for the given parameter at the specified visit.

The Physician's global assessment of disease activity was recorded on a 0 to 100 mm horizontal VAS where score 0 represented 'arthritis inactive' (i.e., symptom-free and no arthritis symptoms) and score 100 represented 'arthritis very active' (higher score indicate worsening of disease).

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Physician's Global Assessment of Disease Activity Day 1 (n=7)	8.6 millimeter (mm) Standard Deviation 7.7
Physician's Global Assessment of Disease Activity Week 12 (n=6)	4.2 millimeter (mm) Standard Deviation 6.4
Physician's Global Assessment of Disease Activity Week 24 (n=7)	9.3 millimeter (mm) Standard Deviation 12.2
Physician's Global Assessment of Disease Activity Week 36 (n=7)	6.7 millimeter (mm) Standard Deviation 6.9
Physician's Global Assessment of Disease Activity Week 48 (n=7)	3.7 millimeter (mm) Standard Deviation 3.6
Physician's Global Assessment of Disease Activity Week 60 (n=7)	4.0 millimeter (mm) Standard Deviation 3.4
Physician's Global Assessment of Disease Activity Week 72 (n=4)	2.8 millimeter (mm) Standard Deviation 4.3
Physician's Global Assessment of Disease Activity Week 84 (n=1)	4.0 millimeter (mm) Standard Deviation NA Data was available for only one participant; therefore, standard deviation cannot be calculated.
Physician's Global Assessment of Disease Activity Week 108 (n=7)	6.6 millimeter (mm) Standard Deviation 7.1

SECONDARY outcome

Timeframe: Baseline (Day 1), Weeks 12, 24, 36, 48, 60, 72, 84, and 108

Population: The ITT population included all the participants who had at least one efficacy assessment. n = number of participants analyzed for the given parameter at the specified visit.

Parent/patient global assessment of disease activity was performed using 0 to 100 mm VAS, and higher the score of VAS, worse the disease status (0= absence of activity or sign or symptom; 100= maximal activity or signs or symptoms). No disease activity was defined as VAS <=10 mm.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Parent/Patient's Global Assessment of Disease Activity Day 1 (n=7)	14.9 mm Standard Deviation 27.5
Parent/Patient's Global Assessment of Disease Activity Week 12 (n=7)	8.0 mm Standard Deviation 12.2
Parent/Patient's Global Assessment of Disease Activity Week 24 (n=7)	12.7 mm Standard Deviation 23.3
Parent/Patient's Global Assessment of Disease Activity Week 36 (n=7)	3.9 mm Standard Deviation 6.8
Parent/Patient's Global Assessment of Disease Activity Week 48 (n=7)	10.1 mm Standard Deviation 17.4
Parent/Patient's Global Assessment of Disease Activity Week 60 (n=7)	7.4 mm Standard Deviation 8.3
Parent/Patient's Global Assessment of Disease Activity Week 72 (n=3)	0.3 mm Standard Deviation 0.6
Parent/Patient's Global Assessment of Disease Activity Week 84 (n=1)	73.0 mm Standard Deviation NA Data was available for only one participant; therefore, standard deviation cannot be calculated.
Parent/Patient's Global Assessment of Disease Activity Week 108 (n=7)	12.1 mm Standard Deviation 17.8

SECONDARY outcome

Timeframe: Baseline (Day 1), Weeks 12, 24, 36, 48, 60, 72, 84, and 108

Population: The ITT population included all the participants who had at least one efficacy assessment. n = number of participants analyzed for the given parameter at the specified visit.

Participants or parents rated participant's pain by placing a horizontal line on a VAS of 0 (no pain) - 100 mm (unbearable pain). To describe the pain, a cut-off at 10 mm was used, and VAS <10 mm was defined as no pain.

Outcome measures

Measure	Tocilizumab (8 mg/kg) n=7 Participants Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Parent/Patient's Discomfort Index (Pain) Week 36 (n=7)	3.0 mm Standard Deviation 3.1
Parent/Patient's Discomfort Index (Pain) Day 1 (n=7)	15.0 mm Standard Deviation 27.2
Parent/Patient's Discomfort Index (Pain) Week 12 (n=7)	5.6 mm Standard Deviation 7.7
Parent/Patient's Discomfort Index (Pain) Week 24 (n=7)	7.7 mm Standard Deviation 11.1
Parent/Patient's Discomfort Index (Pain) Week 48 (n=7)	12.9 mm Standard Deviation 18.5
Parent/Patient's Discomfort Index (Pain) Week 60 (n=7)	6.4 mm Standard Deviation 7.3
Parent/Patient's Discomfort Index (Pain) Week 72 (n=3)	0.7 mm Standard Deviation 0.6
Parent/Patient's Discomfort Index (Pain) Week 84 (n=1)	51.0 mm Standard Deviation NA Data was available for only one participant; therefore, standard deviation cannot be calculated.
Parent/Patient's Discomfort Index (Pain) Week 108 (n=7)	5.1 mm Standard Deviation 9.0

Adverse Events

Tocilizumab (8 mg/kg)

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Tocilizumab (8 mg/kg) n=7 participants at risk Eligible participants received TCZ 8 mg/kg intravenously every 4 weeks up to 104 weeks or until TCZ was commercially available for pcJIA in France, whichever came first.
Blood and lymphatic system disorders Neutropenia	28.6% 2/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Congenital, familial and genetic disorders Von Willebrand's disease	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Eye disorders Chalazion	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Eye disorders Conjunctivitis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Gastrointestinal disorders Abdominal pain	28.6% 2/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Gastrointestinal disorders Gingival bleeding	28.6% 2/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Gastrointestinal disorders Dental caries	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Gastrointestinal disorders Gastrointestinal disorder	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Gastrointestinal disorders Odynophagia	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
General disorders Vaccination site pain	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Immune system disorders Allergy to arthropod bite	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Tooth abscess	28.6% 2/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Acarodermatitis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Ear infection	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Gastroenteritis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Herpes zoster ophthalmic	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Lice infestation	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Nasopharyngitis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Oral fungal infection	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Oral herpes	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Pharyngitis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Rhinitis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Sinusitis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Tonsillitis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Tracheitis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Tracheobronchitis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Infections and infestations Urinary tract infection	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Injury, poisoning and procedural complications Accidental overdose	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Injury, poisoning and procedural complications Bite	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Injury, poisoning and procedural complications Joint injury	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Injury, poisoning and procedural complications Limb injury	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Injury, poisoning and procedural complications Multiple injuries	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Injury, poisoning and procedural complications Skin wound	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Musculoskeletal and connective tissue disorders Arthralgia	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Musculoskeletal and connective tissue disorders Neck pain	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Musculoskeletal and connective tissue disorders Osteoarthritis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Musculoskeletal and connective tissue disorders Osteonecrosis of jaw	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Musculoskeletal and connective tissue disorders Pain in extremity	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Nervous system disorders Headache	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Psychiatric disorders Depression	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Psychiatric disorders Sleep disorder	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Reproductive system and breast disorders Dysmenorrhoea	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Reproductive system and breast disorders Menorrhagia	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Respiratory, thoracic and mediastinal disorders Asthma	28.6% 2/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Respiratory, thoracic and mediastinal disorders Allergic cough	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Respiratory, thoracic and mediastinal disorders Asthmatic crisis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Respiratory, thoracic and mediastinal disorders Cough	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Respiratory, thoracic and mediastinal disorders Epistaxis	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Skin and subcutaneous tissue disorders Alopecia	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Skin and subcutaneous tissue disorders Eczema	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Skin and subcutaneous tissue disorders Rash	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Surgical and medical procedures Curetting of chalazion	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Surgical and medical procedures Tooth extraction	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.
Vascular disorders Hematoma	14.3% 1/7 • Approximately 2 years Safety population included all participants who received at least a single dose of study drug.

Additional Information

Roche Trial Information Hotline

F. Hoffmann-La Roche AG

Phone: +41 61 6878333

Email: global.trial_information@roche.com

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