Clinical trial NCT05782907: Study to Assess Adverse Events, Change in Disease Activity, and How Oral Upadacitinib Moves Through the Body of Pediatric Participants With Moderately to Severely Active Ulcerative Colitis.
Official title: A Phase 3 Multicenter Study to Evaluate Efficacy, Safety, and Pharmacokinetics of Upadacitinib With Open-Label Induction, Randomized, Double-Blind Maintenance and Open-Label Long-Term Extension in Pediatric Subjects With Moderately to Severely Active Ulcerative Colitis and Inadequate Response, Intolerance, or Medical Contraindications to Corticosteroids, Immunosuppressants, and/or Biologic Therapy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Ulcerative Colitis
Keywords: Upadacitinib
RINVOQ
Ulcerative Colitis
UC
Corticosteroids
Immunosuppressants
Biologic Therapy
Lead sponsor: AbbVie
Sponsor class: INDUSTRY
First posted: 2023-03-24
Enrollment: 110
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: Ulcerative colitis (UC) is a type of inflammatory bowel disease that causes inflammation and bleeding from the lining of the rectum and colon (large intestine). This study will assess how safe and effective Upadacitinib is in treating pediatric participants with UC. Adverse events and change in disease activity will be assessed.

Upadacitinib (RINVOQ) is a drug approved in adults for moderate- to severely active UC and is being developed for moderate- to severely active UC in pediatric participants. This study is conducted in 2 periods: Period 1 is comprised of two phases: an 8-week open-label induction phase which means that the study doctor and patients know that participants will receive UPA Dose-A (or the adult equivalent based on body weight) followed by a 44-week double-blind maintenance phase meaning that neither the participants nor the study doctors will know which dose of upadacitinib will be given(UPA Dose B or Dose C). Period 2 is a 260 week open-label extension (OLE) of Period 1. Approximately 110 pediatric participants with moderate to severely active UC will be enrolled at up to 100 sites worldwide.

Participants will receive upadacitinib oral tablets once daily or oral solution twice daily at approximately the same time each day, with or without food. Participants will be followed up for 30 days after each phase (i.e. after induction, maintenance, OLE) and only if a participant doesn't continue into the next phase.

There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.


Eligibility criteria: Inclusion Criteria:

* Active UC with an AMS of 5 to 9 points and endoscopic subscore of 2 to 3.
* Demonstrate an inadequate response, loss of response, intolerance, or medical contraindications to corticosteroids, immunosuppressants, and/or biologic therapy.

Exclusion Criteria:

* Partcipants with previous exposure to JAK inhibitors (e.g., tofacitinib, baricitinib, filgotinib, upadacitinib).
* Females who are pregnant, breastfeeding, or considering becoming pregnant during the study and for approximately 30 days after the last dose of study drug.

Primary outcomes:
measure: Percentage of Participants Achieving Adapted Mayo score (AMS) Clinical Remission (Period 1)
timeFrame: Week 8
measure: Percentage of Participants Achieving AMS Clinical Remission Among Week 8 Responders per AMS (Period 1)
timeFrame: Week 52
Secondary outcomes:
measure: Percentage of Participants Achieving Endoscopic Improvement (Period 1)
timeFrame: Week 8
measure: Percentage of Participants Achieving Partial Mayo Score (PMS) Clinical Remission (Period 1)
timeFrame: Week 8
measure: Percentage of Participants Achieving AMS Clinical Response (Period 1)
timeFrame: Week 8
measure: Percentage of Participants Achieving Endoscopic Improvement Among Week 8 Responders per AMS (Period 1)
timeFrame: Week 52
measure: Percentage of Participants Achieving PMS Clinical Remission Among Week 8 Responders per AMS (Period 1)
timeFrame: Week 52
measure: Percentage of Participants Achieving AMS Clinical Response Among Week 8 Responders per AMS (Period 1)
timeFrame: Week 52
measure: Percentage of Participants Achieving PMS Clinical Response Among Week 8 Clinical Responders per AMS (Period 1)
timeFrame: Week 52
measure: Percentage of Participants Achieving Corticosteroid-Free AMS Clinical Remission Among Week 8 Responders per AMS (Period 1)
timeFrame: Week 52
measure: Percentage of Participants Achieving AMS Clinical Remission Among Week 8 Remitters per AMS (Period 1)
timeFrame: Week 52
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital /ID# 250135
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children's Hospital /ID# 250106
state: Arkansas
city: Corona
country: United States
facility: Kindred Medical Institute - Corona /ID# 255484
state: California
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital - Oakland /ID# 255067
state: California
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital /ID# 258430
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado - Aurora /ID# 250110
state: Colorado
city: Wilmington
country: United States
facility: Nemours/Alfred duPont Hospital for Children /ID# 255483
state: Delaware
city: Atlanta
country: United States
facility: Childrens Healthcare of Atlanta - Center for Advanced Pediatrics /ID# 255069
state: Georgia
city: Peoria
country: United States
facility: OSF St. Francis Medical Center /ID# 256968
state: Illinois
city: Boston
country: United States
facility: Massachusetts General Hospital /ID# 250142
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital /ID# 250108
state: Massachusetts
city: Mineola
country: United States
facility: NYU Langone Hospital - Long Island /ID# 250136
state: New York
city: New York
country: United States
facility: The Mount Sinai Hospital /ID# 250141
state: New York
city: Chapel Hill
country: United States
facility: Univ NC Chapel Hill /ID# 254541
state: North Carolina
city: Charlotte
country: United States
facility: Levine Children's Hospital /ID# 250131
state: North Carolina
city: Cleveland
country: United States
facility: UH Cleveland Medical Center /ID# 250134
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia - Main /ID# 258773
state: Pennsylvania
city: Norfolk
country: United States
facility: Children's Specialty Group /ID# 256966
state: Virginia
city: Madison
country: United States
facility: University of Wisconsin - Madison /ID# 250632
state: Wisconsin
city: Westmead
country: Australia
facility: Children's Hospital at Westmead /ID# 255556
state: New South Wales
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital /ID# 261032
state: Queensland
city: Clayton
country: Australia
facility: Monash Health - Monash Medical Centre /ID# 254726
state: Victoria
city: Perth
country: Australia
facility: Perth Children'S Hospital /ID# 254727
state: Western Australia
city: Edegem
country: Belgium
facility: Universitair Ziekenhuis Antwerpen /ID# 251184
state: Antwerpen
city: Leuven
country: Belgium
facility: Universitair Ziekenhuis Leuven /ID# 251185
state: Vlaams-Brabant
city: Curitiba
country: Brazil
facility: Hospital Pequeno Príncipe /ID# 251911
state: Parana
city: Campinas
country: Brazil
facility: Hospital e Maternidade Celso Pierro - PUC-Campinas /ID# 251912
state: Sao Paulo
city: São Paulo
country: Brazil
facility: Rocco & Nazato Servicos Medicos /ID# 251910
state: Sao Paulo
city: Plovdiv
country: Bulgaria
facility: UMHAT Sveti Georgi /ID# 251949
city: Sofiya
country: Bulgaria
facility: Specialized Hospital For Active Treatment Of Children Diseases Prof. Ivan Mitev /ID# 251285
city: Varna
country: Bulgaria
facility: UMHAT Sveta Marina /ID# 251948
city: Edmonton
country: Canada
facility: Alberta Health Services /ID# 252088
state: Alberta
city: Halifax
country: Canada
facility: IWK Health Center /ID# 250943
state: Nova Scotia
city: Toronto
country: Canada
facility: Hospital for Sick Children /ID# 250945
state: Ontario
city: Pilsen
country: Czechia
facility: Fakultní Nemocnice Plzeň-Lochotín /ID# 253284
state: Plzen-jih
city: Prague
country: Czechia
facility: Fakultní nemocnice v Motole /ID# 251955
state: Praha 5
city: Bordeaux
country: France
facility: CHU Bordeaux - Hopital Pellegrin /ID# 253182
state: Nouvelle-Aquitaine
city: Bron CEDEX
country: France
facility: HCL - Hopital Femme Mere Enfant /ID# 251502
state: Rhone
city: Paris
country: France
facility: AP-HP - Hopital Necker /ID# 251658
city: Paris
country: France
facility: Hopitaux de Paris (AP-HP) - Hopital Robert Debre - CHU /ID# 252069
city: Toulouse
country: France
facility: CHU Toulouse - Hopital Paule de Viguier /ID# 252070
city: Muenchen
country: Germany
facility: Dr. von Haunerschen Kinderspital /ID# 251440
state: Bayern
city: Muenster
country: Germany
facility: Universitaetsklinikum Muenster /ID# 256763
state: Nordrhein-Westfalen
city: Berlin
country: Germany
facility: Charite Universitaetsklinikum Berlin - Campus Virchow /ID# 251434
city: Athens
country: Greece
facility: Agia Sofia Hospital /ID# 250697
state: Attiki
city: Heraklion
country: Greece
facility: University General Hospital of Heraklion PA.G.N.I /ID# 250696
state: Kriti
city: Debrecen
country: Hungary
facility: Debreceni Egyetem-Klinikai Kozpont /ID# 251835
state: Hajdu-Bihar
city: Budapest
country: Hungary
facility: Semmelweis Egyetem /ID# 251083
city: Petah Tikva
country: Israel
facility: Schneider Children's Medical Center /ID# 254833
state: HaMerkaz
city: Jerusalem
country: Israel
facility: Shaare Zedek Medical Center /ID# 254832
state: Yerushalayim
city: Florence
country: Italy
facility: Azienda Ospedaliero Universitaria Meyer /ID# 251624
state: Firenze
city: Rome
country: Italy
facility: Azienda Ospedaliero-Universitaria Policlinico Umberto I /ID# 251625
state: Roma
city: Bologna
country: Italy
facility: Ospedale Maggiore Carlo Alberto Pizzardi /ID# 251626
city: Kashiwa-shi
country: Japan
facility: Tsujinaka Hospital Kashiwanoha /ID# 251930
state: Chiba
city: Kurume-shi
country: Japan
facility: Kurume University Hospital /ID# 251927
state: Fukuoka
city: Sapporo-shi
country: Japan
facility: Hokkaido P.W.F.A.C. Sapporo-Kosei General Hospital /ID# 251928
state: Hokkaido
city: Sendai-shi
country: Japan
facility: Miyagi Children's Hospital /ID# 251931
state: Miyagi
city: Izumi-Shi
country: Japan
facility: Osaka Women's and Children's Hospital /ID# 252397
state: Osaka
city: Osaka-shi
country: Japan
facility: Osaka General Medical Center /ID# 253678
state: Osaka
city: Saitama-shi
country: Japan
facility: Saitama Children's Medical Center /ID# 252362
state: Saitama
city: Bunkyo-ku
country: Japan
facility: Institute of Science Tokyo Hospital /ID# 251929
state: Tokyo
city: Fuchu-shi
country: Japan
facility: Tokyo Metropolitan Children's Medical Center /ID# 252477
state: Tokyo
city: Setagaya-ku
country: Japan
facility: National Center for Child Health and Development /ID# 251926
state: Tokyo
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital /ID# 252024
state: Seoul Teugbyeolsi
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center /ID# 252023
state: Seoul Teugbyeolsi
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Chilgok Hospital /ID# 252663
city: San Pedro Garza Garcia
country: Mexico
facility: Servicios de Oncologia Medica Integral SA de CV /ID# 252974
state: Nuevo Leon
city: Amsterdam
country: Netherlands
facility: Amsterdam UMC, locatie AMC /ID# 250845
state: Noord-Holland
city: Groningen
country: Netherlands
facility: Universitair Medisch Centrum Groningen /ID# 252003
city: Christchurch
country: New Zealand
facility: Christchurch Hospital /ID# 254703
state: Canterbury
city: Auckland
country: New Zealand
facility: Starship Child Health /ID# 254702
city: Torun
country: Poland
facility: Gastromed Sp. z o.o /ID# 251290
state: Kujawsko-pomorskie
city: Warszawa
country: Poland
facility: Instytut Pomnik - Centrum Zdrowia Dziecka /ID# 251289
state: Mazowieckie
city: San Juan
country: Puerto Rico
facility: Clinical Research Puerto Rico /ID# 266477
city: Ferrol
country: Spain
facility: Hospital Arquitecto Marcide - Complejo Hospitalario Universitario de Ferrol /ID# 252105
state: A Coruna
city: Esplugues de Llobregat
country: Spain
facility: Hospital Sant Joan de Deu /ID# 251194
state: Barcelona
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron /ID# 252104
city: Malaga
country: Spain
facility: Hospital Regional Universitario de Malaga /ID# 251193
city: Taipei City
country: Taiwan
facility: National Taiwan University Hospital /ID# 251650
state: Taipei
city: Taoyuan City
country: Taiwan
facility: Linkou Chang Gung Memorial Hospital /ID# 251654
city: Sheffield
country: United Kingdom
facility: Sheffield Children's Hospital NHS Foundation Trust /ID# 251600
state: England
city: London
country: United Kingdom
facility: Barts Health NHS Trust /ID# 251917
state: Greater London
city: London
country: United Kingdom
facility: Great Ormond Street Children's Hospital /ID# 252126
state: Greater London
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust /ID# 252097
state: Hampshire
city: Birmingham
country: United Kingdom
facility: Birmingham Women's and Children's NHS Foundation Trust /ID# 253072
Study officials:
affiliation: AbbVie
name: ABBVIE INC.
role: STUDY_DIRECTOR
References: