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.
- Registration Number
- NCT05782907
- Lead Sponsor
- AbbVie
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 110
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Period 2- Long Term Extension Phase Arm C Upadacitinib Clinical responders who complete Period 1 through Week 52 will receive upadacitinib Dose C daily for up to 260 weeks in OLE Period 2. Period 1- Open Label Induction Phase Upadacitinib All participants in open label induction phase of Period 1 will receive upadacitinib Dose A for 8 weeks based on body weight. Period 1- Double Blind Maintenance Phase Upadacitinib Clinical responders at the end of open label induction phase of Period 1 will be randomly assigned to receive either upadacitinib Dose B or Dose C for 44 weeks based on body weight. Period 2- Open Label Long Term Extension Phase Arm B Upadacitinib Clinical non-responders in US after Period 1 induction phase or clinical responders with loss of response during maintenance phase will receive upadacitinib Dose B daily for up to 260 weeks in OLE Period 2. Period 2- Open Label Long Term Extension Phase Arm A Upadacitinib Clinical non-responders outside of US after Period 1 induction phase will receive upadacitinib Dose A daily for 8 week extended induction phase in open label long term extension (OLE) Period 2. Clinical responders from extended induction phase in OLE will receive upadacitinib Dose B daily for up to 252 weeks in OLE period 2.
- Primary Outcome Measures
Name Time Method Percentage of Participants Achieving Adapted Mayo score (AMS) Clinical Remission (Period 1) Week 8 The Mayo score is a tool designed to measure disease activity for ulcerative colitis. The AMS is a composite of the following subscores: stool frequency subscore (SFS), rectal bleeding subscore (RBS) and endoscopy subscore (MES). AMS ranges from 0 to 9 where higher scores represent more severe disease. Clinical remission is defined as an AMS \< or = 2, with SFS \< or = 1 and not higher than baseline, RBS of 0, and MES \< or = 1.
Percentage of Participants Achieving AMS Clinical Remission Among Week 8 Responders per AMS (Period 1) Week 52 The Mayo score is a tool designed to measure disease activity for ulcerative colitis. AMS ranges from 0 to 9 where higher scores represent more severe disease. Clinical remission is defined as an AMS ≤ 2, with SFS ≤ 1 and not higher than Baseline, RBS of 0, and MES ≤ 1.
- Secondary Outcome Measures
Name Time Method Percentage of Participants Achieving Endoscopic Improvement (Period 1) Week 8 Endoscopic Improvement is defined as MES \< or = 1.
Percentage of Participants Achieving AMS Clinical Remission Among Week 8 Remitters per AMS (Period 1) Week 52 The AMS is a composite of the following subscores: SFS, RBS and MES. Adapted Mayo score ranges from 0 to 9 where higher scores represent more severe disease. Clinical remission is defined as an AMS \< or = 2, with SFS \< or = 1 and not higher than baseline, RBS of 0, and MES \< or = 1.
Percentage of Participants Achieving Partial Mayo Score (PMS) Clinical Remission (Period 1) Week 8 The Mayo score is a tool designed to measure disease activity for ulcerative colitis. The PMS is a composite of the following subscores: SFS, RBS and physician's global assessment (PGA). The PMS ranges from 0 to 9 with higher scores representing more severe disease. PMS clinical remission is defined as a PMS \< or = 2 and no individual subscore \> 1.
Percentage of Participants Achieving AMS Clinical Response (Period 1) Week 8 The adapted mayo score (AMS) is a composite of the following subscores: SFS, RBS and MES. AMS clinical response is defined as decrease in AMS by \> or = 2 points and \> or = 30% from baseline with a decrease in RBS of \> or = 1 or an absolute RBS of 0 or 1.
Percentage of Participants Achieving Endoscopic Improvement Among Week 8 Responders per AMS (Period 1) Week 52 Endoscopic Improvement is defined as MES of \< or = 1. The AMS is a composite of the following subscores: SFS, RBS and MES.
Percentage of Participants Achieving PMS Clinical Remission Among Week 8 Responders per AMS (Period 1) Week 52 The Mayo score is a tool designed to measure disease activity for ulcerative colitis. The PMS ranges from 0 to 9 with higher scores representing more severe disease. PMS clinical remission is defined as a PMS \< or = 2 and no individual subscore \> 1. The AMS is a composite of the following subscores: SFS, RBS and MES.
Percentage of Participants Achieving AMS Clinical Response Among Week 8 Responders per AMS (Period 1) Week 52 The AMS is a composite of the following subscores: SFS, RBS and MES. AMS clinical response is defined as decrease in AMS by \> or = 2 points and \> or = 30% from baseline with a decrease in RBS of \> or = 1 or an absolute RBS of 0 or 1.
Percentage of Participants Achieving PMS Clinical Response Among Week 8 Clinical Responders per AMS (Period 1) Week 52 The AMS is a composite of the following subscores: SFS, RBS and endoscopy MES. PMS clinical response is defined as decrease in PMS by \> or = 2 points and \> or = 30% from baseline with a decrease in RBS \> or = 1 or an absolute RBS of 0 or 1.
Percentage of Participants Achieving Corticosteroid-Free AMS Clinical Remission Among Week 8 Responders per AMS (Period 1) Week 52 Corticosteroid-free AMS clinical remission is defined as being in AMS clinical remission and free of corticosteroids for \>= 90 days immediately preceeding the timepoint of endpoint assessment. The AMS is a composite of the following subscores: SFS, RBS and MES.
Trial Locations
- Locations (85)
Phoenix Children's Hospital /ID# 250135
🇺🇸Phoenix, Arizona, United States
Arkansas Children's Hospital /ID# 250106
🇺🇸Little Rock, Arkansas, United States
Kindred Medical Institute - Corona /ID# 255484
🇺🇸Corona, California, United States
UCSF Benioff Children's Hospital - Oakland /ID# 255067
🇺🇸Oakland, California, United States
Lucile Packard Children's Hospital /ID# 258430
🇺🇸Palo Alto, California, United States
Children's Hospital Colorado - Aurora /ID# 250110
🇺🇸Aurora, Colorado, United States
Nemours/Alfred duPont Hospital for Children /ID# 255483
🇺🇸Wilmington, Delaware, United States
Childrens Healthcare of Atlanta - Center for Advanced Pediatrics /ID# 255069
🇺🇸Atlanta, Georgia, United States
OSF St. Francis Medical Center /ID# 256968
🇺🇸Peoria, Illinois, United States
Massachusetts General Hospital /ID# 250142
🇺🇸Boston, Massachusetts, United States
Boston Children's Hospital /ID# 250108
🇺🇸Boston, Massachusetts, United States
NYU Langone Hospital - Long Island /ID# 250136
🇺🇸Mineola, New York, United States
The Mount Sinai Hospital /ID# 250141
🇺🇸New York, New York, United States
Univ NC Chapel Hill /ID# 254541
🇺🇸Chapel Hill, North Carolina, United States
Levine Children's Hospital /ID# 250131
🇺🇸Charlotte, North Carolina, United States
UH Cleveland Medical Center /ID# 250134
🇺🇸Cleveland, Ohio, United States
Children's Hospital of Philadelphia - Main /ID# 258773
🇺🇸Philadelphia, Pennsylvania, United States
Children's Specialty Group /ID# 256966
🇺🇸Norfolk, Virginia, United States
University of Wisconsin - Madison /ID# 250632
🇺🇸Madison, Wisconsin, United States
Children's Hospital at Westmead /ID# 255556
🇦🇺Westmead, New South Wales, Australia
Queensland Children's Hospital /ID# 261032
🇦🇺South Brisbane, Queensland, Australia
Monash Health - Monash Medical Centre /ID# 254726
🇦🇺Clayton, Victoria, Australia
Perth Children'S Hospital /ID# 254727
🇦🇺Perth, Western Australia, Australia
Universitair Ziekenhuis Antwerpen /ID# 251184
🇧🇪Edegem, Antwerpen, Belgium
Universitair Ziekenhuis Leuven /ID# 251185
🇧🇪Leuven, Vlaams-Brabant, Belgium
Hospital Pequeno Príncipe /ID# 251911
🇧🇷Curitiba, Parana, Brazil
Hospital e Maternidade Celso Pierro - PUC-Campinas /ID# 251912
🇧🇷Campinas, Sao Paulo, Brazil
Rocco & Nazato Servicos Medicos /ID# 251910
🇧🇷São Paulo, Sao Paulo, Brazil
UMHAT Sveti Georgi /ID# 251949
🇧🇬Plovdiv, Bulgaria
Specialized Hospital For Active Treatment Of Children Diseases Prof. Ivan Mitev /ID# 251285
🇧🇬Sofiya, Bulgaria
UMHAT Sveta Marina /ID# 251948
🇧🇬Varna, Bulgaria
Alberta Health Services /ID# 252088
🇨🇦Edmonton, Alberta, Canada
IWK Health Center /ID# 250943
🇨🇦Halifax, Nova Scotia, Canada
Hospital for Sick Children /ID# 250945
🇨🇦Toronto, Ontario, Canada
Fakultní Nemocnice Plzeň-Lochotín /ID# 253284
🇨🇿Pilsen, Plzen-jih, Czechia
Fakultní nemocnice v Motole /ID# 251955
🇨🇿Prague, Praha 5, Czechia
CHU Bordeaux - Hopital Pellegrin /ID# 253182
🇫🇷Bordeaux, Nouvelle-Aquitaine, France
HCL - Hopital Femme Mere Enfant /ID# 251502
🇫🇷Bron CEDEX, Rhone, France
AP-HP - Hopital Necker /ID# 251658
🇫🇷Paris, France
Hopitaux de Paris (AP-HP) - Hopital Robert Debre - CHU /ID# 252069
🇫🇷Paris, France
CHU Toulouse - Hopital Paule de Viguier /ID# 252070
🇫🇷Toulouse, France
Dr. von Haunerschen Kinderspital /ID# 251440
🇩🇪Muenchen, Bayern, Germany
Universitaetsklinikum Muenster /ID# 256763
🇩🇪Muenster, Nordrhein-Westfalen, Germany
Charite Universitaetsklinikum Berlin - Campus Virchow /ID# 251434
🇩🇪Berlin, Germany
Agia Sofia Hospital /ID# 250697
🇬🇷Athens, Attiki, Greece
University General Hospital of Heraklion PA.G.N.I /ID# 250696
🇬🇷Heraklion, Kriti, Greece
Debreceni Egyetem-Klinikai Kozpont /ID# 251835
🇭🇺Debrecen, Hajdu-Bihar, Hungary
Semmelweis Egyetem /ID# 251083
🇭🇺Budapest, Hungary
Schneider Children's Medical Center /ID# 254833
🇮🇱Petah Tikva, HaMerkaz, Israel
Shaare Zedek Medical Center /ID# 254832
🇮🇱Jerusalem, Yerushalayim, Israel
Azienda Ospedaliero Universitaria Meyer /ID# 251624
🇮🇹Florence, Firenze, Italy
Azienda Ospedaliero-Universitaria Policlinico Umberto I /ID# 251625
🇮🇹Rome, Roma, Italy
Ospedale Maggiore Carlo Alberto Pizzardi /ID# 251626
🇮🇹Bologna, Italy
Tsujinaka Hospital Kashiwanoha /ID# 251930
🇯🇵Kashiwa-shi, Chiba, Japan
Kurume University Hospital /ID# 251927
🇯🇵Kurume-shi, Fukuoka, Japan
Hokkaido P.W.F.A.C. Sapporo-Kosei General Hospital /ID# 251928
🇯🇵Sapporo-shi, Hokkaido, Japan
Miyagi Children's Hospital /ID# 251931
🇯🇵Sendai-shi, Miyagi, Japan
Osaka Women's and Children's Hospital /ID# 252397
🇯🇵Izumi-Shi, Osaka, Japan
Osaka General Medical Center /ID# 253678
🇯🇵Osaka-shi, Osaka, Japan
Saitama Children's Medical Center /ID# 252362
🇯🇵Saitama-shi, Saitama, Japan
Institute of Science Tokyo Hospital /ID# 251929
🇯🇵Bunkyo-ku, Tokyo, Japan
Tokyo Metropolitan Children's Medical Center /ID# 252477
🇯🇵Fuchu-shi, Tokyo, Japan
National Center for Child Health and Development /ID# 251926
🇯🇵Setagaya-ku, Tokyo, Japan
Seoul National University Hospital /ID# 252024
🇰🇷Seoul, Seoul Teugbyeolsi, Korea, Republic of
Samsung Medical Center /ID# 252023
🇰🇷Seoul, Seoul Teugbyeolsi, Korea, Republic of
Kyungpook National University Chilgok Hospital /ID# 252663
🇰🇷Daegu, Korea, Republic of
Servicios de Oncologia Medica Integral SA de CV /ID# 252974
🇲🇽San Pedro Garza Garcia, Nuevo Leon, Mexico
Amsterdam UMC, locatie AMC /ID# 250845
🇳🇱Amsterdam, Noord-Holland, Netherlands
Universitair Medisch Centrum Groningen /ID# 252003
🇳🇱Groningen, Netherlands
Christchurch Hospital /ID# 254703
🇳🇿Christchurch, Canterbury, New Zealand
Starship Child Health /ID# 254702
🇳🇿Auckland, New Zealand
Gastromed Sp. z o.o /ID# 251290
🇵🇱Torun, Kujawsko-pomorskie, Poland
Instytut Pomnik - Centrum Zdrowia Dziecka /ID# 251289
🇵🇱Warszawa, Mazowieckie, Poland
Clinical Research Puerto Rico /ID# 266477
🇵🇷San Juan, Puerto Rico
Hospital Arquitecto Marcide - Complejo Hospitalario Universitario de Ferrol /ID# 252105
🇪🇸Ferrol, A Coruna, Spain
Hospital Sant Joan de Deu /ID# 251194
🇪🇸Esplugues de Llobregat, Barcelona, Spain
Hospital Universitario Vall d'Hebron /ID# 252104
🇪🇸Barcelona, Spain
Hospital Regional Universitario de Malaga /ID# 251193
🇪🇸Malaga, Spain
National Taiwan University Hospital /ID# 251650
🇨🇳Taipei City, Taipei, Taiwan
Linkou Chang Gung Memorial Hospital /ID# 251654
🇨🇳Taoyuan City, Taiwan
Sheffield Children's Hospital NHS Foundation Trust /ID# 251600
🇬🇧Sheffield, England, United Kingdom
Barts Health NHS Trust /ID# 251917
🇬🇧London, Greater London, United Kingdom
Great Ormond Street Children's Hospital /ID# 252126
🇬🇧London, Greater London, United Kingdom
University Hospital Southampton NHS Foundation Trust /ID# 252097
🇬🇧Southampton, Hampshire, United Kingdom
Birmingham Women's and Children's NHS Foundation Trust /ID# 253072
🇬🇧Birmingham, United Kingdom