Comparing treatment strategies for childhood arthritis

Comparison of STep-up and Step-down Therapeutic Strategies in Childhood ARthritiS

This study is testing two different treatment plans for kids with juvenile idiopathic arthritis to see which one helps them feel better faster and stay healthy longer.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 260 (estimated)
• Ages: 2 Years to 17 Years
• Sex: All
• Sponsor: Istituto Giannina Gaslini (Academic / other)
• Locations: 1 site (Genova, GE)
• Trial ID: NCT03728478 on ClinicalTrials.gov

What this trial studies

This study compares two treatment strategies for children with juvenile idiopathic arthritis: a conventional step-up approach that escalates treatment based on disease response, and an early aggressive step-down approach that starts with a combination of conventional and biological DMARDs. Both strategies aim to achieve rapid disease control and maintain it over time, leveraging the 'window of opportunity' for better long-term outcomes. The study will include newly diagnosed children aged 2 to 17 years with specific types of arthritis and will assess the effectiveness of treatments like Etanercept and Methotrexate.

Who should consider this trial

Good fit: Ideal candidates are newly diagnosed children aged 2 to 17 years with active juvenile idiopathic arthritis who have not previously received DMARD treatment.

Not a fit: Patients with chronic arthritis who have already undergone treatment with DMARDs or those with other forms of arthritis may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved treatment protocols that enhance disease control and reduce long-term joint damage in children with arthritis.

How similar studies have performed: Previous studies have shown promising results with early aggressive treatment strategies in similar patient populations, indicating potential for success in this approach.

Eligibility criteria

Inclusion Criteria

Each patient must meet all the following criteria in order to be enrolled in the trial:

I. Newly-diagnosed and synthetic or biologic DMARD-naïve children (only treatment with 1 NSAID is allowed and no corticosteroid joint injections prior to randomization ) with a JIA classified according to the following ILAR categories:

i. Oligoarthritis ii. Rheumatoid factor negative polyarthritis

II. Active arthritis

III. Onset of JIA symptoms no more than 6 months before randomization

IV. Age 2 to 17 years at enrolment.

V. Female of child-bearing potential must have a negative pregnancy test at the beginning of the trial. If sexually active, they must agree to use highly effective contraceptive measures, throughout study participation, and must have no intention of conceiving during the course of the study. Post-pubertal males must have no plans to father a child during the study and agree to use highly effective contraceptive measures if sexually active.

VI. Ability to comply with the entire study procedures, ability to communicate meaningfully with the investigational staff, competence to give written informed consent; to be applied to the parents and/or patients, as appropriate

VII. Duly executed, written, informed consent/assent obtained from the parents/patient.

Exclusion criteria

I. Classification in one of the following JIA categories: systemic arthritis, RF-positive polyarthritis, psoriatic arthritis, enthesitis-related arthritis, undifferentiated arthritis

II. Patients who need systemic treatment for uveitis

III. Tuberculosis related issues: patients are excluded from the study if they have:

1. Active TB or a history of incompletely treated TB
2. PPD or QuantiFERON-TB positive patients (with no active disease) unless it is documented by a specialist that the patient has been adequately treated for TB and can start treatment with a biologic agent, based on the medical judgment of the study investigator and / or an infectious disease specialist.
3. Suspected extrapulmonary TB infection
4. Patients at high risk of contracting TB, such as close contact with individual with active or latent TB

IV. Previous treatment with any synthetic or biologic DMARD

V. Any live attenuated vaccine within 4 weeks prior to the baseline visit, such as varicella-zoster, oral polio, measles, mumps or rubella vaccines and throughout the study. Killed or inactive vaccine may be permitted based on the investigator's judgment

VI. Prior or current history of malignancy or any other significant concomitant illness(es) as per the treating physician evaluation

VII. Any of the following laboratory abnormalities based on the most recent laboratory results:

1. White blood cell (WBC) count \<3.50 x 103/mm3 (SI units: \<3.50 x 109/L) and neutrophils \< 1x109/L;
2. Hemoglobin \< 8.5 g/dL (SI units: \<85 g/L);
3. Platelet Count \< 125,0000/mm3 or ≥1,000,000/mm3 (SI units: \<125 x 109/L or ≥1,000 x 109/L
4. Aspartate aminotransaminase (AST) or alanine aminotransaminase (ALT) ≥ 2.0 x upper limit of normal (ULN).

Where this trial is running

Genova, GE

• IRCCS Istituto Giannina Gaslini — Genova, Ge, Italy (Recruiting)

Study contacts

• Study coordinator: Alessandro Consolaro, MD, PhD

Conditions Oligoarthritis, Juvenile; Polyarthritis, Juvenile, Rheumatoid Factor Negative; juvenile idiopathic arthritis; treatment; treat to target