Safety, tolerability, pharmacokinetics, and efficacy of filgotinib for the treatment of polyarticular-course juvenile idiopathic arthritis in children and adolescents

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Alfasigma S.p.A.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

27 Apr 2026 → ongoing

Decision date (initial)

2026-04-07

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Alfasigma S.p.A.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Pharmacokinetic, Efficacy, Safety

The main aim of the study is to evaluate the safety, tolerability, PK, and efficacy of filgotinib in subjects with pJIA.

Secondary objectives 4

1. To evaluate the efficacy of filgotinib in achieving clinical response in subjects from 8 to less than 18 years of age with pJIA
2. To assess the effect of filgotinib on the incidence of uveitis in subjects from 8 to less than 18 years of age with pJIA
3. To characterize the PK of filgotinib and its primary metabolite, GS-829845 in subjects from 8 to less than 18 years of age with pJIA
4. To evaluate the acceptability of the filgotinib pediatric film-coated tablet formulation and the commercially developed film-coated tablet formulation in subjects from 8 to less than 18 years of age with pJIA

Conditions and MedDRA coding

Polyarticular-course juvenile idiopathic arthritis

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-001619-PIP04-17

Plan to share IPD

Yes

IPD plan description

Only after approval of the Marketing Authorization, at Alfasigma’s discretion

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Subject and/or parent/legal guardian must be able and willing to comply with the clinical study protocol requirements and must sign and date the ICF and assent (if required per local regulation) as approved by the Independent Ethics Committee / Institutional Review Board, prior to any screening evaluations.
2. Female or male subject 8 to <18 years of age, on the date of signing the informed consent and assent (per local regulation).
3. Subject must meet the ILAR classification and have moderately to severely active disease for one of the following categories that is not adequately controlled with his/her current therapy (see Protocol Appendix 1 for disease activity assessment criteria): • Extended oligoarthritis (i.e. affecting a total of more than 4 joints after the first 6 months of disease) • RF-positive polyarthritis • RF-negative polyarthritis • PsA • ERA
4. Subject with intolerance or a history of inadequate response to at least one of the following medications for the treatment of pJIA, administered for at least 3 months, based on current treatment guidelines: conventional synthetic disease modifying anti-rheumatic drugs (csDMARDs; including methotrexate) and/or biologic disease modifying anti-rheumatic drugs (bDMARDS) administered per local label, and/or non-steroidal anti-inflammatory drugs for ERA and PsA subtypes.
5. Female subject of childbearing potential who is sexually active and at risk for pregnancy must agree to use contraception/preventive exposure measures as described in the protocol.

Exclusion criteria 9

1. Subject with a body weight <15 kg.
2. Subject with persistent oligoarthritis (i.e. affecting not more than 4 joints throughout the disease course).
3. Subject with undifferentiated arthritis.
4. Subject with anterior uveitis (active or uncontrolled) ≤12 weeks prior to baseline.
5. Subject with systemic JIA.
6. Subject with any other rheumatic disease, inflammatory, or immunologic disease (e.g. inflammatory bowel disease, hypogammaglobulinemia, or systemic lupus erythematosus).
7. Subject has any condition or circumstances (including abnormalities in laboratory parameters) that, in the opinion of the investigator, may make a subject unlikely or unable to complete the study or comply with study procedures and requirements.
8. Subject has an active infection.
9. Subject with a history of complicated herpes zoster infection (with multi-dermatomal, disseminated, ophthalmic, or central nervous system involvement).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Frequency and severity of treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (SAEs), and TEAEs leading to treatment discontinuation (up to approximately Week 22 or throughout the duration of the study)

Secondary endpoints 4

1. − Percentage of subjects with juvenile idiopathic arthritis (JIA) American College of Rheumatology (ACR) 30 response at Week 12 and Week 18 − Percentage of subjects with JIA ACR inactive disease at Week 12 and Week 18 − Change from baseline in Juvenile Arthritis Disease Activity Score (JADAS)-27 erythrocyte sedimentation rate (ESR) and JADAS-27 C-reactive protein (CRP) at Week 12 and Week 18
2. Incidence of uveitis at various timepoints (including occurrence, type, and severity)
3. PK parameters of filgotinib and its primary metabolite GS-829845 (including maximum observed plasma concentration at steady-state [Cmax,ss], area under the plasma concentration-time curve over the dosing interval at steady-state [AUC0-24,ss], and area under the plasma concentration-time curve over the dosing interval at steady-state for the effective exposure [AUCeff,ss])
4. Acceptability of the age-appropriate pediatric formulation and the adult commercially developed film-coated tablet formulation assessed by Pediatric Oral Medicine Acceptability Questionnaire for Patients (POMAQ-P)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Alfasigma S.p.A.

Sponsor organisation

Alfasigma S.p.A.

Address

Via Ragazzi Del '99 5

City

Bologna

Postcode

40133

Country

Italy

Scientific contact point

Organisation

Alfasigma S.p.A.

Contact name

Medical Information

Public contact point

Organisation

Alfasigma S.p.A.

Contact name

Medical Information

Third parties 8

Locations

10 EU/EEA countries · 45 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 188 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications