Clinical trial NCT06222034: Study to Measure Filgotinib in the Blood of Children and Teenagers With Arthritis Taking Filgotinib (SCALESIA)
Official title: An Open-label, Multiple Dose, Multicenter Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Filgotinib in Children and Adolescents From 8 to Less Than 18 Years of Age With Juvenile Idiopathic Arthritis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Juvenile Idiopathic Arthritis
Lead sponsor: Alfasigma S.p.A.
Sponsor class: INDUSTRY
First posted: 2024-01-24
Enrollment: 10
Sex: ALL
Minimum age (years): 8
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A Study to evaluate the pharmacokinetics, safety, and tolerability in paediatric population for treating juvenile idiopathic arthritis (JIA).


Eligibility criteria: Key Inclusion Criteria:

* Participant with a body mass index (BMI) within the 5th to 95th percentiles for the age and gender (based on World Health Organization BMI charts). Participant must have a minimum weight of 15 kg.
* Participant must meet the International League of Associations for Rheumatology classification for 1 of the following categories and have, according to the investigator's judgment, moderately to severely active disease that is not adequately controlled with his/her current therapy.

  * Rheumatoid factor (RF)-positive polyarthritis
  * RF-negative polyarthritis
  * Oligoarthritis
  * Psoriatic arthritis
  * Enthesis-related arthritis (ERA) Note: Historical Human leukocyte antigen B-27 (HLA-B27) results are considered appropriate for ERA diagnosis during screening.
  * Systemic JIA with active arthritis without active systemic features, or with active systemic features that are stable in the prior 6 months of time of enrollment
* Participant with intolerance or a history of inadequate response to at least one of the following medications for the treatment of JIA, administered for at least 12 weeks, based on current treatment guidelines: conventional synthetic disease-modifying antirheumatic drugs and biological disease-modifying antirheumatic drugs (including methotrexate) and non-steroidal anti-inflammatory drugs for ERA and psoriatic arthritis.
* Female participants of childbearing potential (i.e. who have passed menarche) must have a negative highly sensitive urine pregnancy test.

Key Exclusion Criteria:

* Participant with persistent oligoarthritis.
* Participant with undifferentiated arthritis.
* Participant with any other any other rheumatic, inflammatory, or immunologic disease (e.g. inflammatory bowel disease, hypogammaglobulinemia, systemic lupus erythematosus, or uncontrolled uveitis).
* Active infection that is clinically significant, as per judgment of the investigator.
* Participant with a history of complicated herpes zoster infection (with multi-dermatomal, disseminated, ophthalmic, or central nervous system involvement).
* Currently on any therapy for chronic infection (such as pneumocystis, cytomegalovirus, herpes simplex, or atypical mycobacteria).

Note: Other protocol defined Inclusion/ Exclusion criteria may apply.

Primary outcomes:
measure: Maximum observed plasma concentration at steady state of filgotinib (Cmax,ss)
timeFrame: Pre-dose on Day 10, 0.5 hour, 1 hour, 2 hours, 3 hours, 4 hours, 8 hours post-dose on Day 10
measure: Cmax,ss of GS-829845, major active metabolite
timeFrame: Pre-dose on Day 10, 0.5 hour, 1 hour, 2 hours, 3 hours, 4 hours, 8 hours post-dose on Day 10
measure: Area under the plasma concentration-time curve over the dosing interval at steady state of filgotinib (AUC0-24,ss)
timeFrame: Pre-dose on Day 10, 0.5 hour, 1 hour, 2 hours, 3 hours, 4 hours, 8 hours post-dose on Day 10
measure: AUC0-24,ss of GS-829845, major active metabolite
timeFrame: Pre-dose on Day 10, 0.5 hour, 1 hour, 2 hours, 3 hours, 4 hours, 8 hours post-dose on Day 10
measure: Area under the plasma concentration time curve over the dosing interval at steady state or the effective exposure of filgotinib (AUCeff,ss)
timeFrame: Pre-dose on Day 10, 0.5 hour, 1 hour, 2 hours, 3 hours, 4 hours, 8 hours post-dose on Day 10
measure: AUCeff,ss of GS-829845, major active metabolite
timeFrame: Pre-dose on Day 10, 0.5 hour, 1 hour, 2 hours, 3 hours, 4 hours, 8 hours post-dose on Day 10
Secondary outcomes:
measure: Number of participants with treatment-emergent adverse events (TEAEs), TEAEs of interest, serious TEAEs, and TEAEs leading to treatment discontinuation.
timeFrame: Baseline (Day 1) up to week 96
measure: Acceptability of the commercially developed film-coated tablets and of the minitablets as measured by the Pediatric Oral Medicine Acceptability Questionnaire for Patients (POMAQ-P).
timeFrame: Week 4 and week 12
Other outcomes:
Locations:
city: Amiens Cedex 1
country: France
facility: CHU Amiens - Hopital Nord
city: Le Kremlin Bicêtre
country: France
facility: Bicêtre University Hospital
city: Berlin
country: Germany
facility: Children's university hospital Charité, Campus Virchow, SPZ
city: Hamburg
country: Germany
facility: Hamburger Zentrum fur Kinder und Jugendrheumatologie
city: Krakow
country: Poland
facility: Malopolskie Badania Kliniczne
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Deu
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe
city: London
country: United Kingdom
facility: Great Ormond Street Hospital
Study officials:
affiliation: Alfasigma S.p.A.
name: Alfasigma Study Director
role: STUDY_DIRECTOR
References: