Trial Outcomes & Findings for Start Time Optimization of Biologics in Polyarticular JIA (NCT NCT02593006)

Last Updated: 2021-02-05

Results Overview

This is a provisional criteria which describes a state of complete disease inactivity in Juvenile Idiopathic Arthritis (JIA). We will assess the proportion of patients achieving CID off glucocorticoids in each treatment arm.

Recruitment status

COMPLETED

Target enrollment

400 participants

Primary outcome timeframe

12 months after baseline

Results posted on

2021-02-05

Participant Flow

Participant milestones

Participant milestones
Measure	Step-Up CTP Consensus Treatment Plan where subjects begin non biologic DMARD (methotrexate, sulfasalazine, or leflunomide) stepping up to biologic if not improved by 3 months	Early Combination CTP Consensus Treatment Plan where patients start a DMARD(methotrexate, sulfasalazine, or leflunomide) and biologic treatment within one month of each other.	Biologic Frist CTP Consensus Treatment Plan where patients begin Biologic treatment
Overall Study STARTED	257	100	43
Overall Study COMPLETED	250	93	38
Overall Study NOT COMPLETED	7	7	5

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Start Time Optimization of Biologics in Polyarticular JIA

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Step-Up CTP n=257 Participants Consensus Treatment Plan where subjects begin non biologic DMARD (methotrexate, sulfasalazine, or leflunomide) stepping up to biologic if not improved by 3 months	Early Combination CTP n=100 Participants Consensus Treatment Plan where patients start a DMARD(methotrexate, sulfasalazine, or leflunomide) and biologic treatment within one month of each other.	Biologic Frist CTP n=43 Participants Consensus Treatment Plan where patients begin Biologic treatment	Total n=400 Participants Total of all reporting groups
Age, Categorical <=18 years	257 Participants n=5 Participants	99 Participants n=7 Participants	43 Participants n=5 Participants	399 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Continuous	11.07 years n=5 Participants	11.38 years n=7 Participants	12.34 years n=5 Participants	11.28 years n=4 Participants
Sex: Female, Male Female	192 Participants n=5 Participants	75 Participants n=7 Participants	27 Participants n=5 Participants	294 Participants n=4 Participants
Sex: Female, Male Male	65 Participants n=5 Participants	25 Participants n=7 Participants	16 Participants n=5 Participants	106 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	3 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	3 Participants n=4 Participants
Race (NIH/OMB) Asian	15 Participants n=5 Participants	4 Participants n=7 Participants	2 Participants n=5 Participants	21 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Black or African American	15 Participants n=5 Participants	5 Participants n=7 Participants	3 Participants n=5 Participants	23 Participants n=4 Participants
Race (NIH/OMB) White	188 Participants n=5 Participants	68 Participants n=7 Participants	27 Participants n=5 Participants	283 Participants n=4 Participants
Race (NIH/OMB) More than one race	9 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants	17 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	27 Participants n=5 Participants	19 Participants n=7 Participants	6 Participants n=5 Participants	52 Participants n=4 Participants
Region of Enrollment Canada	32 participants n=5 Participants	2 participants n=7 Participants	1 participants n=5 Participants	35 participants n=4 Participants
Region of Enrollment United States	225 participants n=5 Participants	98 participants n=7 Participants	42 participants n=5 Participants	365 participants n=4 Participants
Clinical Juvenile Arthritis Disease Activity Score	17.08 units on a scale STANDARD_DEVIATION 4.55 • n=5 Participants	20.18 units on a scale STANDARD_DEVIATION 4.37 • n=7 Participants	19.05 units on a scale STANDARD_DEVIATION 4.29 • n=5 Participants	18.08 units on a scale STANDARD_DEVIATION 4.67 • n=4 Participants

PRIMARY outcome

Timeframe: 12 months after baseline

Population: Subjects are children and adolescents between the ages of 2 and 18 who are newly diagnosed with a polyarticular form of JIA and treated for 12 months with one of the consensus treatment plans. 328 participants had complete data for the primary CID endpoint at 12 months

Outcome measures

Outcome measures
Measure	Step-Up Consensus Treatment Plan (CTP) n=217 Participants Initial therapy with a non biologic Disease Modifying Anti-Rheumatic Drug (DMARD) stepping up to a biologic treatment if needed	Early Combination CTP n=78 Participants Initial therapy with a non-biologic DMARD and biologic treatment at the start of treatment	Biologic First CTP n=33 Participants Initial therapy with a biologic treatment without a non-biologic DMARD
Percentage of Participants With Clinically Inactive Disease (CID) Off Glucocorticoids	32.3 percentage of participants achieving CID Interval 26.2 to 39.0	37.2 percentage of participants achieving CID Interval 26.7 to 48.9	24.2 percentage of participants achieving CID Interval 11.7 to 42.6

SECONDARY outcome

Timeframe: 12 months after baseline

Population: Same description as above for the population for the primary outcome

The Patient Reported Outcomes Measurement Information System (PROMIS) pain interference and mobility scores will be compared between the 3 CTP groups. Pain interference scores range from 0-100 and higher scores are worse. Mobility scores also range from 0-100 and higher scores are better.

Outcome measures

Outcome measures
Measure	Step-Up Consensus Treatment Plan (CTP) n=257 Participants Initial therapy with a non biologic Disease Modifying Anti-Rheumatic Drug (DMARD) stepping up to a biologic treatment if needed	Early Combination CTP n=100 Participants Initial therapy with a non-biologic DMARD and biologic treatment at the start of treatment	Biologic First CTP n=43 Participants Initial therapy with a biologic treatment without a non-biologic DMARD
Comparison of PROMIS Pain and Mobility Scores Between the 3 Consensus Treatment Plan Groups Pain Interference	53.1 score on a scale Standard Deviation 10.0	55.5 score on a scale Standard Deviation 9.2	55.9 score on a scale Standard Deviation 9.3
Comparison of PROMIS Pain and Mobility Scores Between the 3 Consensus Treatment Plan Groups Mobility	38.6 score on a scale Standard Deviation 10.3	35.8 score on a scale Standard Deviation 9.1	37.5 score on a scale Standard Deviation 9.1

Adverse Events

Step-Up CTP

Serious events: 12 serious events

Other events: 19 other events

Deaths: 0 deaths

Early Combination CTP

Serious events: 3 serious events

Other events: 5 other events

Deaths: 0 deaths

Biologic First CTP

Serious events: 5 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Step-Up CTP n=257 participants at risk Initial therapy with a non biologic Disease Modifying Anti-Rheumatic Drug (DMARD) stepping up to a biologic treatment if needed	Early Combination CTP n=100 participants at risk Initial therapy with a non-biologic DMARD and biologic treatment at the start of treatment	Biologic First CTP n=43 participants at risk Initial therapy with a biologic treatment without a non-biologic DMARD
Musculoskeletal and connective tissue disorders Hip Pain	0.78% 2/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Skin and subcutaneous tissue disorders Cellulitis	0.39% 1/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Injury, poisoning and procedural complications Cervical Spine Fracture	0.39% 1/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Injury, poisoning and procedural complications Compund Fracture Ulna & Radius	0.00% 0/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	1.0% 1/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Psychiatric disorders Depression with Suicidal Ideation	0.00% 0/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	2.3% 1/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
General disorders Influenza	0.78% 2/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Infections and infestations Infections treated with IV antibiotics	0.78% 2/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	1.0% 1/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	4.7% 2/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Gastrointestinal disorders Inflammatory Bowel Disease	0.39% 1/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Immune system disorders Drug induced Lupus	0.39% 1/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Musculoskeletal and connective tissue disorders hip effusion	0.39% 1/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Infections and infestations Shingles	0.00% 0/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	2.3% 1/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Nervous system disorders Vertigo	0.00% 0/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	2.3% 1/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Psychiatric disorders Worsening Bipolar Affective Disorder	0.39% 1/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Immune system disorders Macrophage Activation Syndrome	0.00% 0/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	1.0% 1/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year

Other adverse events

Other adverse events
Measure	Step-Up CTP n=257 participants at risk Initial therapy with a non biologic Disease Modifying Anti-Rheumatic Drug (DMARD) stepping up to a biologic treatment if needed	Early Combination CTP n=100 participants at risk Initial therapy with a non-biologic DMARD and biologic treatment at the start of treatment	Biologic First CTP n=43 participants at risk Initial therapy with a biologic treatment without a non-biologic DMARD
Hepatobiliary disorders Hepatitis	1.2% 3/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	2.0% 2/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Immune system disorders Hypersensitivity Reaction	0.39% 1/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Gastrointestinal disorders Inflammatory Bowel Disease	0.39% 1/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	1.0% 1/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Blood and lymphatic system disorders Leukopenia	0.78% 2/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Skin and subcutaneous tissue disorders Psoriasis	1.2% 3/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	0.00% 0/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year
Eye disorders Uveitis (New Onset)	3.5% 9/257 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	2.0% 2/100 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year	2.3% 1/43 • Adverse Events were collected from Baseline through Study completion. For each participant, the time period was on average 1 year

Additional Information

Yukiko Kimura MD

Hackensack University Medical Center

Phone: 551-996-5306

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place