Trial Outcomes & Findings for Pediatric Arthritis Study of Certolizumab Pegol (NCT NCT01550003)

NCT ID: NCT01550003

Last Updated: 2024-10-23

Results Overview

Certolizumab Pegol (CZP) plasma concentration level was measured in micrograms per milliliter (ug/ml).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

193 participants

Primary outcome timeframe

Week 16

Results posted on

2024-10-23

Participant Flow

The study started to enroll participants in March 2012 and concluded in April 2024.

Participant Flow refers to the Safety Set.

Participant milestones

Participant milestones
Measure	Any CZP Dose - Weight Group: 10 - < 20 kg Participants received Certolizumab Pegol (CZP) subcutaneously (sc) as a fixed dose based on their body weight every 2 weeks (Q2W) or every 4 weeks (Q4W) throughout the study. Participants started with 3 loading doses of CZP at Weeks 0, 2, and 4 followed by a maintenance dose for the duration of the study (maximum up to 12 years).	Any CZP Dose - Weight Group: 20 - <40 kg Participants received CZP sc as a fixed dose based on their body weight Q2W throughout the study. Participants started with 3 loading doses of CZP at Weeks 0, 2, and 4 followed by a maintenance dose for the duration of the study.	Any CZP Dose - Weight Group: >= 40 kg Participants received CZP sc as a fixed dose based on their body weight Q2W throughout the study. Participants started with 3 loading doses of CZP at Weeks 0, 2, and 4 followed by a maintenance dose for the duration of the study.
Overall Study STARTED	18	63	112
Overall Study Original CZP Dose	7	34	64
Overall Study Reduced CZP Dose	11	29	48
Overall Study COMPLETED	0	0	0
Overall Study NOT COMPLETED	18	63	112

Reasons for withdrawal

Reasons for withdrawal
Measure	Any CZP Dose - Weight Group: 10 - < 20 kg Participants received Certolizumab Pegol (CZP) subcutaneously (sc) as a fixed dose based on their body weight every 2 weeks (Q2W) or every 4 weeks (Q4W) throughout the study. Participants started with 3 loading doses of CZP at Weeks 0, 2, and 4 followed by a maintenance dose for the duration of the study (maximum up to 12 years).	Any CZP Dose - Weight Group: 20 - <40 kg Participants received CZP sc as a fixed dose based on their body weight Q2W throughout the study. Participants started with 3 loading doses of CZP at Weeks 0, 2, and 4 followed by a maintenance dose for the duration of the study.	Any CZP Dose - Weight Group: >= 40 kg Participants received CZP sc as a fixed dose based on their body weight Q2W throughout the study. Participants started with 3 loading doses of CZP at Weeks 0, 2, and 4 followed by a maintenance dose for the duration of the study.
Overall Study Adverse Event	0	9	15
Overall Study Lack of Efficacy	4	11	15
Overall Study Protocol Violation	0	3	2
Overall Study Lost to Follow-up	1	4	8
Overall Study Consent withdrawn	2	11	23
Overall Study Missing	1	0	2
Overall Study Study closure as announced by sponsor	7	6	24
Overall Study Protocol Noncompliance	1	0	0
Overall Study Study closure	0	1	1
Overall Study Patient reached adulthood	0	1	0
Overall Study Early discontinuation at request of sponsor	0	2	4
Overall Study Sponsor's order	0	3	3
Overall Study Sponsor's decision	1	5	4
Overall Study Subject mother passed away	0	1	0
Overall Study Discontinuation	0	1	0
Overall Study Subject moved to rheumatology and out of study	0	1	1
Overall Study Site closure	0	2	0
Overall Study Pregnancy	0	1	0
Overall Study By medical decision and approved by the sponsor	0	0	1
Overall Study Subject is in college/unable to come to visits	0	0	1
Overall Study Switching to adult rheumatologist	0	0	1
Overall Study Patient transition to adult care	0	0	1
Overall Study Subject was able to obtain commercial cimzia	0	0	1
Overall Study Treatment or 12 week follow up visit completed	0	0	1
Overall Study Per Sponsor request	1	0	1
Overall Study Patient not compliance	0	0	1
Overall Study Non-compliance	0	0	1
Overall Study Study closed	0	1	0
Overall Study Transitioned to adult rheumatology	0	0	1

Baseline Characteristics

Pediatric Arthritis Study of Certolizumab Pegol

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Any CZP Dose - Weight Group: 10 - <20 kg n=18 Participants Participants received CZP sc as a fixed dose based on their body weight Q2W or Q4W throughout the study. Participants started with 3 loading doses of CZP at Weeks 0, 2, and 4 followed by a maintenance dose for the duration of the study.	Any CZP Dose - Weight Group: 20 - <40 kg n=63 Participants Participants received CZP sc as a fixed dose based on their body weight Q2W throughout the study. Participants started with 3 loading doses of CZP at Weeks 0, 2, and 4 followed by a maintenance dose for the duration of the study.	Any CZP Dose - Weight Group: >=40 kg n=112 Participants Participants received CZP sc as a fixed dose based on their body weight Q2W throughout the study. Participants started with 3 loading doses of CZP at Weeks 0, 2, and 4 followed by a maintenance dose for the duration of the study.	Total n=193 Participants Total of all reporting groups
Age, Continuous	5.4 years STANDARD_DEVIATION 1.3 • n=5 Participants	9.1 years STANDARD_DEVIATION 2.0 • n=7 Participants	14.5 years STANDARD_DEVIATION 2.2 • n=5 Participants	11.9 years STANDARD_DEVIATION 3.8 • n=4 Participants
Age, Customized 24 months - <12 years	18 Participants n=5 Participants	55 Participants n=7 Participants	13 Participants n=5 Participants	86 Participants n=4 Participants
Age, Customized 12 - <18 years	0 Participants n=5 Participants	8 Participants n=7 Participants	99 Participants n=5 Participants	107 Participants n=4 Participants
Sex: Female, Male Female	11 Participants n=5 Participants	43 Participants n=7 Participants	76 Participants n=5 Participants	130 Participants n=4 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	20 Participants n=7 Participants	36 Participants n=5 Participants	63 Participants n=4 Participants
Race/Ethnicity, Customized American Indian/ Alaska Native	0 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants	5 Participants n=4 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	5 Participants n=4 Participants
Race/Ethnicity, Customized Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	6 Participants n=5 Participants	6 Participants n=4 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race/Ethnicity, Customized White	17 Participants n=5 Participants	53 Participants n=7 Participants	85 Participants n=5 Participants	155 Participants n=4 Participants
Race/Ethnicity, Customized Other/Mixed	1 Participants n=5 Participants	7 Participants n=7 Participants	14 Participants n=5 Participants	22 Participants n=4 Participants
Race/Ethnicity, Customized Hispanic or Latino	10 Participants n=5 Participants	18 Participants n=7 Participants	27 Participants n=5 Participants	55 Participants n=4 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	8 Participants n=5 Participants	45 Participants n=7 Participants	85 Participants n=5 Participants	138 Participants n=4 Participants

PRIMARY outcome

Timeframe: Week 16

Population: The Pharmacokinetic Per-Protocol (PK-PP) Set was a subset of the SS consisting of those study participants who took at least 1 dose of study medication, provided measurable plasma CZP concentration samples (with recorded sampling date/time or for which date/time can be reasonably assumed). Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Certolizumab Pegol (CZP) plasma concentration level was measured in micrograms per milliliter (ug/ml).

Outcome measures

Outcome measures
Measure	Reduced CZP Dose -Weight Group: 10 - <20 kg n=10 Participants Participants received CZP 50 milligrams (mg) sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q4W (maintenance dose) from Week 8 onwards.	Reduced CZP Dose - Weight Group: 20 - <40 kg n=24 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Reduced CZP Dose - Weight Group: >=40 kg n=36 Participants Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 10 - <20 kg n=5 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 20 - <40 kg n=29 Participants Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: >=40 kg n=49 Participants Participants received CZP 400 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 200 mg sc Q2W (maintenance dose) from Week 6 onwards.
Certolizumab Pegol (CZP) Plasma Concentration Level at Week 16	1.6166 ug/ml Interval 0.472 to 5.5368	9.2277 ug/ml Interval 5.1453 to 16.5491	13.8928 ug/ml Interval 11.003 to 17.5416	22.9060 ug/ml Interval 13.338 to 39.3374	25.7752 ug/ml Interval 16.9024 to 39.3058	33.5680 ug/ml Interval 25.7883 to 43.6945

PRIMARY outcome

Timeframe: Week 48

Certolizumab Pegol (CZP) plasma concentration level was measured in ug/mL.

Outcome measures

Outcome measures
Measure	Reduced CZP Dose -Weight Group: 10 - <20 kg n=9 Participants Participants received CZP 50 milligrams (mg) sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q4W (maintenance dose) from Week 8 onwards.	Reduced CZP Dose - Weight Group: 20 - <40 kg n=24 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Reduced CZP Dose - Weight Group: >=40 kg n=31 Participants Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 10 - <20 kg n=2 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 20 - <40 kg n=12 Participants Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: >=40 kg n=25 Participants Participants received CZP 400 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 200 mg sc Q2W (maintenance dose) from Week 6 onwards.
Certolizumab Pegol (CZP) Plasma Concentration Level at Week 48	4.7404 ug/ml Interval 1.7596 to 12.771	8.4459 ug/ml Interval 5.0002 to 14.2661	12.2987 ug/ml Interval 8.695 to 17.3962	NA ug/ml As pre-specified in the SAP, Geometric Mean and 95% confidence interval (CI) were not calculated if less than 3 concentration were available or if the below the lower limit of quantification (LLOQ) was greater than (\>) 1/3.	20.7048 ug/ml Interval 11.1744 to 38.3636	25.5940 ug/ml Interval 14.6913 to 44.5878

PRIMARY outcome

Timeframe: Week 16

Population: Safety Set (SS) consisted of all study participants in the Enrolled Set (ES) who have received at least 1 dose of study medication. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Number of participants with anti-CZP antibodies were reported.

Outcome measures

Outcome measures
Measure	Reduced CZP Dose -Weight Group: 10 - <20 kg n=84 Participants Participants received CZP 50 milligrams (mg) sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q4W (maintenance dose) from Week 8 onwards.	Reduced CZP Dose - Weight Group: 20 - <40 kg n=97 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Reduced CZP Dose - Weight Group: >=40 kg Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 10 - <20 kg Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 20 - <40 kg Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: >=40 kg Participants received CZP 400 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 200 mg sc Q2W (maintenance dose) from Week 6 onwards.
Number of Participants With Anti-Certolizumab Pegol (Anti-CZP) Antibody Level at Week 16	69 Participants	77 Participants	—	—	—	—

PRIMARY outcome

Timeframe: Week 48

Number of participants with anti-CZP antibodies were reported.

Outcome measures

Outcome measures
Measure	Reduced CZP Dose -Weight Group: 10 - <20 kg n=72 Participants Participants received CZP 50 milligrams (mg) sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q4W (maintenance dose) from Week 8 onwards.	Reduced CZP Dose - Weight Group: 20 - <40 kg n=60 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Reduced CZP Dose - Weight Group: >=40 kg Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 10 - <20 kg Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 20 - <40 kg Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: >=40 kg Participants received CZP 400 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 200 mg sc Q2W (maintenance dose) from Week 6 onwards.
Number of Participants With Anti-Certolizumab Pegol (Anti-CZP) Antibody Level at Week 48	58 Participants	47 Participants	—	—	—	—

PRIMARY outcome

Timeframe: From Baseline (Week 0) up to the Final Visit (70 days after final dose of CZP) (maximum up to 12 years)

Population: Safety Set (SS) consisted of all study participants in the Enrolled Set (ES) who have received at least 1 dose of study medication.

A Serious Adverse Event (SAE) is any untoward medical occurrence that at any dose: results in deaths, is life-threatening, requires in patient hospitalization or prolongation of existing hospitalization, is a congenital anomaly or birth defect and other important medical events which based on medical or scientific judgement may jeopardize the patients, or may require medical or surgical intervention to prevent any of the above. TEAEs are defined as AEs starting on or after first administration of CZP and up to 70 days after last dose of study medication.

Outcome measures

Outcome measures
Measure	Reduced CZP Dose -Weight Group: 10 - <20 kg n=18 Participants Participants received CZP 50 milligrams (mg) sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q4W (maintenance dose) from Week 8 onwards.	Reduced CZP Dose - Weight Group: 20 - <40 kg n=63 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Reduced CZP Dose - Weight Group: >=40 kg n=112 Participants Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 10 - <20 kg Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 20 - <40 kg Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: >=40 kg Participants received CZP 400 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 200 mg sc Q2W (maintenance dose) from Week 6 onwards.
Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs) During the Study	5 Participants	20 Participants	21 Participants	—	—	—

PRIMARY outcome

Timeframe: From Baseline (Week 0) up to the Final Visit (70 days after final dose of CZP) (maximum up to 12 years)

Population: Safety Set (SS) consisted of all study participants in the Enrolled Set (ES) who have received at least 1 dose of study medication.

An AE is any untoward medical occurrence in a patient or clinical investigation study participant administered a pharmaceutical product which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an IMP, whether or not related to the IMP. TEAEs are defined as AEs starting on or after first administration of CZP and up to 70 days after last dose of study medication. TEAEs leading to permanent withdrawal of the IMP during the study were reported in this outcome measure.

Outcome measures

Outcome measures
Measure	Reduced CZP Dose -Weight Group: 10 - <20 kg n=18 Participants Participants received CZP 50 milligrams (mg) sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q4W (maintenance dose) from Week 8 onwards.	Reduced CZP Dose - Weight Group: 20 - <40 kg n=63 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Reduced CZP Dose - Weight Group: >=40 kg n=112 Participants Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 10 - <20 kg Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 20 - <40 kg Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: >=40 kg Participants received CZP 400 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 200 mg sc Q2W (maintenance dose) from Week 6 onwards.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of the Investigational Medicinal Product (IMP) During the Study	0 Participants	9 Participants	16 Participants	—	—	—

SECONDARY outcome

Timeframe: Week 16

Population: Full analysis set (FAS): all study participants in SS who have no more than one of 6 core components missing at baseline (count of joints with active arthritis, count of joints with limitation of range of motion, Physician's Global Assessment of Disease Activity score, CHAQ score, Parent's Global Assessment of Overall Well-Being score and CRP result) for calculating PedACR30/50/70/90.

PedACR30-at least 30% improvement from baseline in 3 of any 6 core set measures, with no more than 1 of remaining variables worsening by \>30%: * Number of joints with active arthritis * Number of joints with limitation of range of motion * Physician's Global Assessment of Disease Activity (using visual analog scale (VAS): 100mm; 0= very good, and 100= very poor) * CHAQ (30 questions, 8 domains, scores for each domain are averaged to calculate total score \[ 0= no disability to 3= very severe disability\]) * Parent's Global Assessment of Overall Well-Being (using VAS: 100mm; 0= Very well to 100= Very poor) * C-reactive protein (CRP)

Outcome measures

Outcome measures
Measure	Reduced CZP Dose -Weight Group: 10 - <20 kg n=18 Participants Participants received CZP 50 milligrams (mg) sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q4W (maintenance dose) from Week 8 onwards.	Reduced CZP Dose - Weight Group: 20 - <40 kg n=63 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Reduced CZP Dose - Weight Group: >=40 kg n=112 Participants Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 10 - <20 kg Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 20 - <40 kg Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: >=40 kg Participants received CZP 400 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 200 mg sc Q2W (maintenance dose) from Week 6 onwards.
Percentage of Participants Meeting American College of Rheumatology Pediatric 30 % (PedACR30) Response Criteria at Week 16	83.3 percentage of participants Interval 58.6 to 96.4	77.8 percentage of participants Interval 65.5 to 87.3	79.5 percentage of participants Interval 70.8 to 86.5	—	—	—

SECONDARY outcome

Timeframe: Week 16

Population: FAS:all study participants in SS who have no more than one of 6 core components missing at baseline (count of joints with active arthritis, count of joints with limitation of range of motion, PGA of Disease Activity score, CHAQ score, Parent's Global Assessment of Overall Well-Being score and CRP result) for calculating PedACR30/50/70/90.

PedACR50- at least 50% improvement from baseline in 3 of any 6 core set measures, with no more than 1 of remaining variables worsening by \>30%: * Number of joints with active arthritis * Number of joints with limitation of range of motion * Physician's Global Assessment (PGA) of Disease Activity (using VAS: 100mm; 0= very good, and 100= very poor) * Childhood Health Assessment Questionnaire (CHAQ) (30 questions, 8 domains, scores for each domain are averaged to calculate total score \[ 0= no disability to 3= very severe disability\]) * Parent's Global Assessment of Overall Well-Being (using VAS: 100mm; 0= Very well to 100= Very poor) * CRP

Outcome measures

Outcome measures
Measure	Reduced CZP Dose -Weight Group: 10 - <20 kg n=18 Participants Participants received CZP 50 milligrams (mg) sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q4W (maintenance dose) from Week 8 onwards.	Reduced CZP Dose - Weight Group: 20 - <40 kg n=63 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Reduced CZP Dose - Weight Group: >=40 kg n=112 Participants Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 10 - <20 kg Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 20 - <40 kg Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: >=40 kg Participants received CZP 400 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 200 mg sc Q2W (maintenance dose) from Week 6 onwards.
Percentage of Participants Meeting American College of Rheumatology Pediatric 50 % (PedACR50) Response Criteria at Week 16	66.7 percentage of participants Interval 41.0 to 86.7	71.4 percentage of participants Interval 58.7 to 82.1	74.1 percentage of participants Interval 65.0 to 81.9	—	—	—

SECONDARY outcome

Timeframe: Week 16

Population: FAS consisted of all study participants in SS who have no more than one of 6 core components missing at baseline (count of joints with active arthritis, count of joints with limitation of range of motion, Physician's Global Assessment of Disease Activity score, CHAQ score, Parent's Global Assessment of Overall Well-Being score and CRP result) for calculating PedACR30/50/70/90.

PedACR70- at least 70% improvement from baseline in 3 of any 6 following core set measures, with no more than 1 of remaining variables worsening by \>30%: * Number of joints with active arthritis * Number of joints with limitation of range of motion * Physician's Global Assessment of Disease Activity (using VAS: 100mm; 0= very good, and 100= very poor) * CHAQ (30 questions, 8 domains, scores for each domain are averaged to calculate total score \[ 0= no disability to 3= very severe disability\]) * Parent's Global Assessment of Overall Well-Being (using VAS: 100mm; 0= Very well to 100= Very poor) * CRP

Outcome measures

Outcome measures
Measure	Reduced CZP Dose -Weight Group: 10 - <20 kg n=18 Participants Participants received CZP 50 milligrams (mg) sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q4W (maintenance dose) from Week 8 onwards.	Reduced CZP Dose - Weight Group: 20 - <40 kg n=63 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Reduced CZP Dose - Weight Group: >=40 kg n=112 Participants Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 10 - <20 kg Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 20 - <40 kg Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: >=40 kg Participants received CZP 400 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 200 mg sc Q2W (maintenance dose) from Week 6 onwards.
Percentage of Participants Meeting American College of Rheumatology Pediatric 70 % (PedACR70) Response Criteria at Week 16	44.4 percentage of participants Interval 21.5 to 69.2	57.1 percentage of participants Interval 44.0 to 69.5	54.5 percentage of participants Interval 44.8 to 63.9	—	—	—

SECONDARY outcome

Timeframe: Week 16

PedACR90- at least 90% improvement from baseline in 3 of any 6 following core set measures, with no more than 1 of remaining variables worsening by \>30%: * Number of joints with active arthritis * Number of joints with limitation of range of motion * Physician's Global Assessment of Disease Activity (using VAS: 100mm; 0= very good, and 100= very poor) * CHAQ (30 questions, 8 domains, scores for each domain are averaged to calculate total score \[ 0= no disability to 3= very severe disability\]) * Parent's Global Assessment of Overall Well-Being (using VAS: 100mm; 0= Very well to 100= Very poor) * CRP

Outcome measures

Outcome measures
Measure	Reduced CZP Dose -Weight Group: 10 - <20 kg n=18 Participants Participants received CZP 50 milligrams (mg) sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q4W (maintenance dose) from Week 8 onwards.	Reduced CZP Dose - Weight Group: 20 - <40 kg n=63 Participants Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Reduced CZP Dose - Weight Group: >=40 kg n=112 Participants Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 10 - <20 kg Participants received CZP 100 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 50 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: 20 - <40 kg Participants received CZP 200 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 100 mg sc Q2W (maintenance dose) from Week 6 onwards.	Original CZP Dose - Weight Group: >=40 kg Participants received CZP 400 mg sc Q2W at Weeks 0, 2, and 4 (loading dose) followed by CZP 200 mg sc Q2W (maintenance dose) from Week 6 onwards.
Percentage of Participants Meeting American College of Rheumatology Pediatric 90 % (PedACR90) Response Criteria at Week 16	22.2 percentage of participants Interval 6.4 to 47.6	25.4 percentage of participants Interval 15.3 to 37.9	29.5 percentage of participants Interval 21.2 to 38.8	—	—	—

Adverse Events

Any CZP Dose - Weight Group: 10 - <20 kg

Serious events: 5 serious events

Other events: 16 other events

Deaths: 0 deaths

Any CZP Dose - Weight Group: 20 - <40 kg

Serious events: 20 serious events

Other events: 59 other events

Deaths: 0 deaths

Any CZP Dose - Weight Group: >=40 kg

Serious events: 21 serious events

Other events: 98 other events

Deaths: 3 deaths

Serious adverse events

Other adverse events

Additional Information

UCB

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Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60