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Crohn's Disease: Efficacy, Safety, and Pharmacokinetics of Upadacitinib in Pediatric Subjects With Moderately to Severely Active Crohn's Disease
NCT06332534 · AbbVie
In plain English
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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 Crohn's Disease and Inadequate Response, Intolerance, or Medical Contraindications to Corticosteroids, Immunosuppressants, and/or Biologic Therapy
About this study
Crohn's disease (CD) is a long-lasting disease that causes severe inflammation (redness, swelling), in the digestive tract, most often affecting the bowels. It can cause many different symptoms including abdominal pain, diarrhea, tiredness, and weight loss. This study will assess how safe and effective oral Upadacitinib is in treating moderately to severely active Crohn's Disease in pediatric participants aged 2 to 18 years old who have had inadequate response, loss of response, intolerance, or medical contraindications to corticosteroids, immunosuppressants, and/or biologic therapy.
Upadacitinib (RINVOQ) is a drug approved in adults for moderate- to severely active CD and is being developed for moderate- to severely active CD in pediatric participants. This study is conducted in 2 periods: Period 1 is comprised of two phases: a 12-week open-label induction phase which means that the study doctor and participants know that participants will receive UPA Dose-A (or the adult equivalent based on body weight) followed by a 52-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 156-week open-label extension of Period 1. Approximately 110 pediatric participants with moderate to severely active CD will be enrolled at approximately 92 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 have a safety follow up for 30 days after discontinuation from any time point within the study.
There may be higher treatment burden for participants in this trial compared to their standard of care (due to study procedures). Participants will attend regular (weekly, monthly) 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:
* Weight at Screening and Baseline must be \>= 10 kg
* Moderate to severe Crohn's Disease (CD) defined as Pediatric Crohn's Disease Activity Index (PCDAI) \> 30 and endoscopic evidence of mucosal inflammation as documented by a centrally read SES-CD of \>= 6 (or SES-CD of \>=4 for isolated ileal disease) excluding the presence of narrowing component.
* Documented diagnosis of CD prior to Baseline, confirmed by colonoscopy during the screening period, with exclusion of current infection, colonic dysplasia and/or malignancy. Appropriate documentation of biopsy results consistent with the diagnosis of CD, in the assessment of the investigator, must be available
* Demonstrated an inadequate response, loss of response, or intolerance to corticosteroids, immunomodulators (IMMs), and/or biologic therapy or in whom use of those therapies is medically contraindicated. For participants in the US and South Korea, participants must have demonstrated an inadequate response, loss of response, or intolerance to one or more anti-TNFs (tumor necrosis factor).
Exclusion Criteria:
* History of:
* A diagnosis of CD prior to 2 years of age.
* Currently known complications of CD such as:
* Active abscess (abdominal or perianal);
* Symptomatic bowel strictures;
* More than 2 missing segments of the following 5 intestinal segments: terminal ileum, right colon, transverse colon, sigmoid and left colon, and rectum;
* Ostomy or ileoanal pouch;
* Surgical bowel resection within the past 3 months prior to Baseline, or a history of more than 3 bowel resections.
* Japan participants only: positive result of beta-D-glucan or two consecutive indeterminate results of beta-D-glucan during the Screening period (screening for Pneumocystis jiroveci infection)
* History of any of the following:
* Current diagnosis of ulcerative colitis (UC), indeterminate colitis, or monogenic inflammatory bowel disease (IBD);
* Fulminant colitis or toxic megacolon;
* Gastrointestinal (GI) perforation (other than due to appendicitis or mechanical injury), diverticulitis, or significantly increased risk for GI perforation per investigator judgment including history of volvulus and/or intussusception (telescoping of bowels);
* Current diagnosis of any primary immune deficiency
* Conditions that could interfere with drug absorption including but not limited to short bowel syndrome or gastric bypass surgery; subjects with a history of gastric banding/segmentation are not excluded.
Study design
Enrollment target: 110 participants
Allocation: randomized
Masking: quadruple
Age groups: child
Timeline
Starts: 2024-08-08
Estimated completion: 2034-12
Last updated: 2026-04-15
Interventions
Drug: Upadacitinib
Primary outcomes
- • Percentage of participants who achieved clinical response per the Pediatric Crohn's Disease Activity Index (PCDAI) at Week 12, with clinical remission per the PCDAI at Week 64 (At Week 64)
- • Achievement of endoscopic response at Week 64 in participants who achieved clinical response per PCDAI at Week 12. (At Week 64)
- • Number of Participants with Adverse Events (Through Week 156)
Sponsor
AbbVie · industry
Contacts & investigators
ContactABBVIE CALL CENTER · contact · abbvieclinicaltrials@abbvie.com · 844-663-3742
InvestigatorABBVIE INC. · study_director, AbbVie
All locations (83)
UCSF Benioff Children's Hospital - Oakland /ID# 262217Recruiting
Oakland, California, United States
Lucile Packard Children's Hospital /ID# 262193Recruiting
Palo Alto, California, United States
Children's Hospital Colorado - Aurora /ID# 262207Recruiting
Aurora, Colorado, United States
Connecticut Children's Medical Center - Hartford /ID# 262256Recruiting
Hartford, Connecticut, United States
OSF St. Francis Medical Center /ID# 262192Recruiting
Peoria, Illinois, United States
Indiana University Health Riley Hospital for Children /ID# 262215Recruiting
Indianapolis, Indiana, United States
Boston Children's Hospital /ID# 262191Recruiting
Boston, Massachusetts, United States
MNGI Digestive Health, P. A. /ID# 262204Recruiting
Minneapolis, Minnesota, United States
Icahn School of Medicine at Mount Sinai /ID# 262216Recruiting
New York, New York, United States
Univ NC Chapel Hill /ID# 262198Recruiting
Chapel Hill, North Carolina, United States
UH Cleveland Medical Center /ID# 262188Recruiting
Cleveland, Ohio, United States
Children's Hospital of Philadelphia - Main /ID# 262197Recruiting
Philadelphia, Pennsylvania, United States
Sydney Children's Hospital /ID# 262352Recruiting
Randwick, New South Wales, Australia
Children's Hospital at Westmead /ID# 262350Recruiting
Westmead, New South Wales, Australia
Queensland Children's Hospital /ID# 262351Recruiting
South Brisbane, Queensland, Australia
Monash Health - Monash Medical Centre /ID# 262878Recruiting
Clayton, Victoria, Australia
Perth Children'S Hospital /ID# 272905Recruiting
Perth, Western Australia, Australia
Uza /Id# 261745Recruiting
Edegem, Antwerpen, Belgium
Cliniques Universitaires UCL Saint-Luc /ID# 261741Recruiting
Brussels, Brussels Capital, Belgium
Universitair Ziekenhuis Leuven /ID# 261740Recruiting
Leuven, Vlaams-Brabant, Belgium
Hospital Universite Enfants Reine Fabiola /ID# 261744Recruiting
Brussels, Belgium
CHR de la Citadelle /ID# 261749Recruiting
Liège, Belgium
UCL Namur University Hospital, Site Sainte-Elisabeth /ID# 261750Recruiting
Namur, Belgium
Galileo Medical Research Ltda /ID# 262602Recruiting
Juiz de Fora, Minas Gerais, Brazil
Hospital Pequeno Príncipe /ID# 262600Recruiting
Curitiba, Paraná, Brazil
Irmandade da Santa Casa de Misericordia de Porto Alegre /ID# 262601Recruiting
Porto Alegre, Rio Grande do Sul, Brazil
Rocco & Nazato Servicos Medicos /ID# 262485Recruiting
São Paulo, São Paulo, Brazil
Hospital Sirio Libanes /ID# 262670Recruiting
São Paulo, Brazil
UMHAT Sveti Georgi /ID# 262590Recruiting
Plovdiv, Bulgaria
Specialized Hospital For Active Treatment Of Children Diseases Prof. Ivan Mitev /ID# 262589Recruiting
Sofia, Bulgaria
IWK Health Center /ID# 262543Recruiting
Halifax, Nova Scotia, Canada
Beijing Children's Hospital /ID# 262258Recruiting
Beijing, Beijing Municipality, China
Guangzhou Medical University Affiliated Women and Children's Medical Center /ID# 262596Recruiting
Guangzhou, Guangdong, China
The Sixth Affiliated Hospital of Sun Yat-sen University /ID# 272807Recruiting
Guangzhou, Guangdong, China
Henan Children's Hospital Zhengzhou Children's Hospital /ID# 262300Recruiting
Zhengzhou, Henan, China
Hunan Children's Hospital /ID# 262512Recruiting
Changsha, Hunan, China
Jiangxi Provincial Children's Hospital /ID# 262295Recruiting
Nanchang, Jiangxi, China
Shengjing Hospital of China Medical University /ID# 262301Recruiting
Shenyang, Liaoning, China
Children's Hospital of Shanghai /ID# 262356Recruiting
Shanghai, Shanghai Municipality, China
Ruijin Hospital, Shanghai Jiaotong University School of Medicine /ID# 262502Recruiting
Shanghai, Shanghai Municipality, China
The Children's Hospital of Zhejiang University School of Medicine /ID# 262337Recruiting
Hangzhou, Zhejiang, China
CHU de CAEN - Hopital de la Cote de Nacre /ID# 262311Recruiting
Caen, Calvados, France
Hospices Civils de Lyon - Hôpital Femme Mère Enfant /ID# 263422Recruiting
Bron, Rhone, France
Hopitaux de Paris (AP-HP) - Hopital Robert Debre - CHU /ID# 262308Recruiting
Paris, France
Agia Sofia Hospital /ID# 261792Recruiting
Athens, Attica, Greece
General Hospital of Chest Diseases of Athens SOTIRIA /ID# 261790Recruiting
Athens, Attica, Greece
University General Hospital of Heraklion PA.G.N.I /ID# 261791Recruiting
Heraklion, Crete, Greece
Fondazione di Religione e di Culto Casa Sollievo della Sofferenza /ID# 262384Recruiting
San Giovanni Rotondo, Foggia, Italy
Ospedale Pediatrico Bambino Gesù /ID# 262379Recruiting
Rome, Roma, Italy
Azienda Ospedaliera Universitaria Gaetano Martino /ID# 262380Recruiting
Messina, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico /ID# 275276Recruiting
Milan, Italy
Tsujinaka Hospital - Kashiwanoha /ID# 262454Recruiting
Kashiwa-shi, Chiba, Japan
Kurume University Hospital /ID# 262455Recruiting
Kurume-shi, Fukuoka, Japan
Miyagi Children's Hospital /ID# 262459Recruiting
Sendai, Miyagi, Japan
Osaka Women's and Children's Hospital /ID# 262549Recruiting
Izumi-Shi, Osaka, Japan
Saga University Hospital /ID# 262753Recruiting
Saga, Saga-ken, Japan
Saitama Children's Medical Center /ID# 262461Recruiting
Saitama-shi, Saitama, Japan
Institute of Science Tokyo Hospital /ID# 262510Recruiting
Bunkyo-ku, Tokyo, Japan
Tokyo Metropolitan Children's Medical Center /ID# 262550Recruiting
Fuchu-shi, Tokyo, Japan
National Center For Child Health And Development /ID# 262456Recruiting
Setagaya City, Tokyo, Japan
Toyama Prefectural Central Hospital /ID# 262739Recruiting
Toyama, Toyama, Japan
Christchurch Hospital. /ID# 262577Recruiting
Christchurch, Canterbury, New Zealand
Starship Child Health /ID# 262576Completed
Auckland, New Zealand
Gastromed Sp. z o.o /ID# 262367Recruiting
Torun, Kuyavian-Pomeranian Voivodeship, Poland
Instytut Pomnik - Centrum Zdrowia Dziecka /ID# 262366Recruiting
Warsaw, Masovian Voivodeship, Poland
Clinical Research Investigator Group, LLC /ID# 262357Recruiting
Bayamón, Puerto Rico
Puerto Rico Health Institute /ID# 262358Recruiting
Dorado, Puerto Rico
Clinical Research Puerto Rico /ID# 279595Recruiting
San Juan, Puerto Rico
Seoul National University Hospital /ID# 262324Recruiting
Seoul, Seoul Teugbyeolsi, South Korea
Yonsei University Health System Severance Hospital /ID# 262721Recruiting
Seoul, Seoul Teugbyeolsi, South Korea
Samsung Medical Center /ID# 262323Recruiting
Seoul, Seoul Teugbyeolsi, South Korea
Hospital Arquitecto Marcide - Complejo Hospitalario Universitario de Ferrol /ID# 262226Recruiting
Ferrol, A Coruna, Spain
Hospital Sant Joan de Deu /ID# 262599Recruiting
Esplugues de Llobregat, Barcelona, Spain
Hospital Regional Universitario de Malaga /ID# 262228Recruiting
Málaga, Spain
Hospital Universitario Virgen del Rocio /ID# 262712Recruiting
Seville, Spain
Hospital Clinico Universitario Lozano Blesa /ID# 262227Completed
Zaragoza, Spain
National Taiwan University Hospital /ID# 261695Recruiting
Taipei City, Taipei, Taiwan
Linkou Chang Gung Memorial Hospital /ID# 261696Recruiting
Taoyuan, Taiwan
Addenbrookes Hospital /ID# 262707Recruiting
Cambridge, Cambridgeshire, United Kingdom
Sheffield Children's Hospital NHS Foundation Trust /ID# 261832Recruiting
Sheffield, England, United Kingdom
Disc_Barts Health NHS Trust - The Royal London Hospital /ID# 262811Recruiting
London, Greater London, United Kingdom
Norfolk and Norwich University Hospitals NHS Foundation Trust /ID# 262778Recruiting
Norwich, Norfolk, United Kingdom
Royal Hospital for Children and Young People /ID# 262388Recruiting
Edinburgh, United Kingdom