Clinical trial NCT06865417: 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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: Ulcerative Colitis
Lead sponsor: Alfasigma S.p.A.
Sponsor class: INDUSTRY
First posted: 2025-03-07
Enrollment: 80
Sex: ALL
Minimum age (years): 8
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


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.

Primary outcomes:
measure: Efficacy of filgotinib in induction of remission and maintenance of effect in pediatric subjects with ulcerative colitis (UC)
timeFrame: Week 10
measure: subjects with ulcerative colitis (UC)
timeFrame: Week 58
Secondary outcomes:
measure: Safety and tolerability of filgotinib in pediatric subjects with UC
timeFrame: Week 62
measure: Safety and tolerability of filgotinib in pediatric subjects with UC
timeFrame: Week 58
measure: Safety and tolerability of filgotinib in pediatric subjects with U
timeFrame: Week 58
Other outcomes:
Locations:
city: Brussels
country: Belgium
facility: Association Hospitaliere De Bruxelles Hopital Universitaire Des Enfants Reine Fabiola
city: Liege
country: Belgium
facility: Centre Hospitalier Regional De La Citadelle
city: Namur
country: Belgium
facility: Centre Hospitalier Universitaire Dinant Godinne Sainte-Elisabeth-UCL-Namur
city: Osijek
country: Croatia
facility: Klinicki Bolnicki Centar Osijek
city: Zagreb
country: Croatia
facility: Children's Hospital Zagreb
city: Zagreb
country: Croatia
facility: University Hospital Centre Zagreb
city: Bron
country: France
facility: Hospital Femme Mere Enfant
city: Dijon
country: France
facility: Centre Hospitalier Universitaire De Dijon
city: Lille Cedex
country: France
facility: Groupement Des Hopitaux De L'Institut Catholique De Lille
city: Montpellier Cedex 05
country: France
facility: CHU de Montpellier
city: Rennes
country: France
facility: Centre Hospitalier Universitaire De Rennes
city: Tours
country: France
facility: Centre Hospitalier Regional Universitaire De Tours
city: Aachen
country: Germany
facility: Universitaetsklinikum Aachen AöR
city: Leipzig
country: Germany
facility: Universitaetsklinikum Leipzig AöR
city: Mainz
country: Germany
facility: Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
city: Tuebingen
country: Germany
facility: Universitaetsklinikum Tuebingen AöR
city: Athens
country: Greece
facility: Athens General Children's Hospital Panagioti And Aglaia Kyriakou
city: Athens
country: Greece
facility: University General Hospital Attikon
city: Efkarpia
country: Greece
facility: General Hospital Of Thessaloniki Papageorgiou
city: Thessaloniki
country: Greece
facility: Hippokration Hospital
city: Dublin
country: Ireland
facility: Children's Health Ireland
city: Alessandria
country: Italy
facility: Azienda Ospedaliero-Universitaria Ss Antonio E Biagio E Cesare Arrigo
city: Ancona
country: Italy
facility: Azienda Ospedaliero Universitaria Ospedali Riuniti Umberto I G M Lancisi G Salesi
city: Milan
country: Italy
facility: ASST Fatebenefratelli Sacco
city: Naples
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II Di Napoli
city: Rome
country: Italy
facility: Azienda Ospedaliero-Universitaria Policlinico Umberto I
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambino Gesu
city: Trieste
country: Italy
facility: Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo
city: Oslo
country: Norway
facility: Oslo University Hospital HF
city: Tønsberg
country: Norway
facility: Sykehuset I Vestfold HF
city: Bydgoszcz
country: Poland
facility: In Vivo Sp. z o.o.
city: Gdansk
country: Poland
facility: Copernicus Podmiot Leczniczy Sp. z o.o.
city: Kraków
country: Poland
facility: Uniwersytecki Szpital Dzieciecy W Krakowie
city: Lodz
country: Poland
facility: Instytut Centrum Zdrowia Matki Polki
city: Rzeszów
country: Poland
facility: Gabinet Lekarski Bartosz Korczowski
city: Warsaw
country: Poland
facility: Instytut Pomnik Centrum Zdrowia Dziecka
city: Braga
country: Portugal
facility: CCAB Centro Clinico Academico Braga Associacao
city: Coimbra
country: Portugal
facility: Unidade Local De Saude De Coimbra E.P.E.
city: Viana Do Castelo
country: Portugal
facility: Unidade Local De Saude Do Alto Minho E.P.E.
city: Bucharest
country: Romania
facility: Dr. Victor Gomoiu Clinical Children Hospital
city: Timisoara
country: Romania
facility: Spitalul Clinic De Urgenta Pentru Copii Louis Turcanu Timisoara
city: Badalona
country: Spain
facility: Hospital Germans Trias I Pujol
city: Cordoba
country: Spain
facility: Hospital Universitario Reina Sofia
Study officials:
References: