A Study Evaluating the Effects of Filgotinib in Children and Teenagers With Ulcerative Colitis

Official title

A Multicenter Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Filgotinib, With Single Arm Induction and Maintenance, in Pediatric Subjects (8 to <18 Years of Age) With Moderately to Severely Active Ulcerative Colitis

Overview

The aim of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of filgotinib as a treatment for UC in children and adolescents aged from 8 to less than 18 years. Approximately 80 subjects from 8 to \<18 years of age with moderately to severely active UC, including a minimum of 8 subjects from 8 to \<12 years of age, will be enrolled in this study. During the study, eligible subjects will take the investigational product (IP) on-site at Week 4, Week 10, and Week 22 (in the morning; with or without food). On all other days, subjects will take IP at home (in the morning; with or without food). Subjects who do not achieve mMCS remission and/or MCS response at Week 10 will continue with induction treatment until Week 22. Subjects who do not achieve PUCAI remission at Week 22 will be permanently discontinued from the study. Subjects will all receive a filgotinib dose targeting the same systemic exposure as that observed in adults with UC treated with 200 mg q.d.

Interventions

• Drug Filgotinib
  IP will be provided as commercially developed film-coated tablets (100 and 200 mg strength) or reduced-strength (65 mg) age-appropriate film-coated tablets for use in pediatric subjects aged at least 8 years and needs to be taken orally q.d. at approximately the same time every morning (with or without food).

Primary outcome measures

• Efficacy of filgotinib in induction of remission and maintenance of effect in pediatric subjects with ulcerative colitis (UC) [Time frame: Week 10]
• subjects with ulcerative colitis (UC) [Time frame: Week 58]

Secondary outcome measures (3)

• Safety and tolerability of filgotinib in pediatric subjects with UC [Time frame: Week 62]
• Safety and tolerability of filgotinib in pediatric subjects with UC [Time frame: Week 58]
• Safety and tolerability of filgotinib in pediatric subjects with U [Time frame: Week 58]

Eligibility criteria

Inclusion criteria

• Subject must have a minimum body weight (BW) of 15 kg.
• Subject:

• has documented diagnosis of UC with a minimum duration of 3 months,
• has mMCS of 5 to 9, and an MCS endoscopic score >=2, rectal bleeding >=1, and stool frequency >=1,
• has had an inadequate response, loss of response, intolerance, or has medical contraindications to corticosteroids, immunosuppressants, and/or biologic therapy. This includes subjects who depend on corticosteroids to control their symptoms and who experience worsening of their disease when attempting to wean off corticosteroids.

Exclusion criteria

• Subject has a diagnosis of inflammatory bowel disease -unclassified or indeterminate colitis, isolated proctitis, or toxic megacolon.
• Subject has an active infection.
• Subject 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, herpes zoster, or atypical mycobacteria).
• Subject has a history of colectomy or extensive small bowel resection.
• Subject with psychological or cognitive difficulties that might interfere with study participation.
• Subject has any previous exposure to a Janus kinase inhibitor or medication with a similar mode of action (e.g. tofacitinib, baricitinib, upadacitinib).
• Female subject is pregnant or breast feeding or intending to become pregnant or breastfeed during the study.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

Italy · 7 centers

• Azienda Ospedaliero-Universitaria Ss Antonio E Biagio E Cesare Arrigo — Alessandria
• Azienda Ospedaliero Universitaria dell Marche I G M Lancisi G Salesi — Ancona
• ASST Fatebenefratelli Sacco — Milan
• Azienda Ospedaliera Universitaria Federico II Di Napoli — Naples
• Azienda Ospedaliero-Universitaria Policlinico Umberto I — Rome
• Ospedale Pediatrico Bambino Gesu — Rome
• IRCCS Materno Infantile Burlo Garofolo — Trieste

France · 6 centers

• Hospital Femme Mere Enfant — Bron
• Centre Hospitalier Universitaire De Dijon — Dijon
• Hopital Saint Vincent de Paul - GHICL Lille — Lille
• CHU de Montpellier — Montpellier
• Centre Hospitalier Universitaire De Rennes — Rennes
• Centre Hospitalier Regional Universitaire De Tours — Tours

United Kingdom · 6 centers

• Bristol Royal Infirmary — Bristol
• Ninewells Hospital — Glasgow
• Alder Hey Children's Hospital — Liverpool
• Chelsea and Westminster Hospital — London
• The Newcastle upon Tyne Hospital FT — Newcastle
• Sheffield Children's Hospital — Sheffield

Germany · 4 centers

• Universitaetsklinikum Aachen AöR — Aachen
• Universitaetsklinikum Leipzig AöR — Leipzig
• Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR — Mainz
• Universitaetsklinikum Tuebingen AöR — Tübingen

Greece · 4 centers

• Athens General Children's Hospital Panagioti And Aglaia Kyriakou — Athens
• University General Hospital Attikon — Athens
• General Hospital Of Thessaloniki Papageorgiou — Efkarpia
• Hippokration Hospital — Thessaloniki

Poland · 4 centers

• In Vivo Sp. z o.o. — Bydgoszcz
• Instytut Centrum Zdrowia Matki Polki — Lodz
• Gabinet Lekarski Bartosz Korczowski — Rzeszów
• Instytut Pomnik Centrum Zdrowia Dziecka — Warsaw

Belgium · 3 centers

• Association Hospitaliere De Bruxelles Hopital Universitaire Des Enfants Reine Fabiola — Brussels
• Centre Hospitalier Regional De La Citadelle — Liège
• Centre Hospitalier Universitaire Dinant Godinne Sainte-Elisabeth-UCL-Namur — Namur

Croatia · 3 centers

• Klinicki Bolnicki Centar Osijek — Osijek
• Children's Hospital Zagreb — Zagreb
• University Hospital Centre Zagreb — Zagreb

Portugal · 3 centers

• CCAB Centro Clinico Academico Braga Associacao — Braga
• Hospital Pediátrico de Coimbra — Coimbra
• Unidade Local De Saude Do Alto Minho E.P.E. — Viana do Castelo

Norway · 2 centers

• Oslo University Hospital HF — Oslo
• Sykehuset I Vestfold HF — Tønsberg

Romania · 2 centers

• Dr. Victor Gomoiu Clinical Children Hospital — Bucharest
• Spitalul Clinic De Urgenta Pentru Copii Louis Turcanu Timisoara — Timișoara

Spain · 2 centers

• Hospital Germans Trias I Pujol — Badalona
• Hospital Universitario Reina Sofia — Córdoba

Ireland · 1 center

• Children's Health Ireland — Dublin

Identifiers

NCT: NCT06865417 · GLPG0634-CL-331

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗