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NCT06332534PHASE3Recruiting

Crohn's Disease: Efficacy, Safety, and Pharmacokinetics of Upadacitinib in Pediatric Subjects With Moderately to Severely Active Crohn's Disease

AbbVie

Start Date

8/8/2024

Completion Date

12/1/2034

Summary

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

Age Range: 2 years to 17 years

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.

Interventions

DRUG

Upadacitinib

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Conditions

Crohn's Disease

Locations

UCSF Benioff Children's Hospital - Oakland /ID# 262217

Oakland, California 94609

United States

Lucile Packard Children's Hospital /ID# 262193

Palo Alto, California 94304

United States

Children's Hospital Colorado - Aurora /ID# 262207

Aurora, Colorado 80045

United States

Connecticut Children's Medical Center - Hartford /ID# 262256

Hartford, Connecticut 06106

United States

OSF St. Francis Medical Center /ID# 262192

Peoria, Illinois 61637-0001

United States

Indiana University Health Riley Hospital for Children /ID# 262215

Indianapolis, Indiana 46202

United States

Boston Children's Hospital /ID# 262191

Boston, Massachusetts 02115

United States

MNGI Digestive Health, P. A. /ID# 262204

Minneapolis, Minnesota 55413-2195

United States

Icahn School of Medicine at Mount Sinai /ID# 262216

New York, New York 10029

United States

Univ NC Chapel Hill /ID# 262198

Chapel Hill, North Carolina 27514-4220

United States

UH Cleveland Medical Center /ID# 262188

Cleveland, Ohio 44106

United States

Children's Hospital of Philadelphia - Main /ID# 262197

Philadelphia, Pennsylvania 19104-4319

United States

Sydney Children's Hospital /ID# 262352

Randwick, New South Wales 2031

Australia

Children's Hospital at Westmead /ID# 262350

Westmead, New South Wales 2145

Australia

Queensland Children's Hospital /ID# 262351

South Brisbane, Queensland 4101

Australia

Monash Health - Monash Medical Centre - Clayton /ID# 262878

Clayton, Victoria 3168

Australia

Perth Children'S Hospital /ID# 272905

Perth, Western Australia 6009

Australia

Uza /Id# 261745

Edegem, Antwerpen 2650

Belgium

Cliniques Universitaires UCL Saint-Luc /ID# 261741

Brussels, Brussels Capital 1200

Belgium

Universitair Ziekenhuis Leuven /ID# 261740

Leuven, Vlaams-Brabant 3000

Belgium

Hospital Universite Enfants Reine Fabiola /ID# 261744

Brussels, 1020

Belgium

CHR de la Citadelle /ID# 261749

Liège, 4000

Belgium

UCL Namur University Hospital, Site Sainte-Elisabeth /ID# 261750

Namur, 5000

Belgium

Galileo Medical Research Ltda /ID# 262602

Juiz de Fora, Minas Gerais 36033-318

Brazil

Hospital Pequeno Príncipe /ID# 262600

Curitiba, Paraná 80250-060

Brazil

Santo Antônio Children's Hospital /ID# 262601

Porto Alegre, Rio Grande do Sul 90240-510

Brazil

Rocco & Nazato Servicos Medicos /ID# 262485

São Paulo, São Paulo 04543-011

Brazil

Hospital Sirio Libanes - Sao Paulo /ID# 262670

São Paulo, 01308-050

Brazil

UMHAT Sveti Georgi /ID# 262590

Plovdiv, 4002

Bulgaria

Specialized Hospital For Active Treatment Of Children Diseases Prof. Ivan Mitev /ID# 262589

Sofia, 1606

Bulgaria

BC Children's Hospital /ID# 262237

Vancouver, British Columbia V6H 3V4

Canada

IWK Health Center /ID# 262543

Halifax, Nova Scotia B3K 6R8

Canada

CHU Sainte-Justine /ID# 262096

Montreal, Quebec H3T 1C5

Canada

Beijing Children's Hospital /ID# 262258

Beijing, Beijing Municipality 100045

China

Guangzhou Medical University Affiliated Women and Children's Medical Center /ID# 262596

Guangzhou, Guangdong 510620

China

The Sixth Affiliated Hospital of Sun Yat-sen University /ID# 272807

Guangzhou, Guangdong 510655

China

Henan Children's Hospital Zhengzhou Children's Hospital /ID# 262300

Zhengzhou, Henan 450018

China

Hunan Children's Hospital /ID# 262512

Changsha, Hunan 410007

China

Jiangxi Provincial Children's Hospital /ID# 262295

Nanchang, Jiangxi 330006

China

Shengjing Hospital of China Medical University /ID# 262301

Shenyang, Liaoning 110022

China

Children's Hospital of Shanghai /ID# 262356

Shanghai, Shanghai Municipality 200062

China

Ruijin Hospital, Shanghai Jiaotong University School of Medicine /ID# 262502

Shanghai, Shanghai Municipality 200065

China

The Children's Hospital of Zhejiang University School of Medicine /ID# 262337

Hangzhou, Zhejiang 310003

China

CHU de CAEN - Hopital de la Cote de Nacre /ID# 262311

Caen, Calvados 14033

France

Hospices Civils de Lyon - Hôpital Femme Mère Enfant /ID# 263422

Bron, Rhone 69500

France

Hopitaux de Paris (AP-HP) - Hopital Robert Debre - CHU /ID# 262308

Paris, 75019

France

Agia Sofia Hospital /ID# 261792

Athens, Attica 11527

Greece

General Hospital of Chest Diseases of Athens SOTIRIA /ID# 261790

Athens, Attica 11527

Greece

University General Hospital of Heraklion PA.G.N.I /ID# 261791

Heraklion, Crete 71500

Greece

Fondazione di Religione e di Culto Casa Sollievo della Sofferenza /ID# 262384

San Giovanni Rotondo, Foggia 71013

Italy

Ospedale Pediatrico Bambino Gesù /ID# 262379

Rome, Roma 00165

Italy

Azienda Ospedaliera Universitaria Gaetano Martino /ID# 262380

Messina, 98125

Italy

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico /ID# 275276

Milan, 20122

Italy

Tsujinaka Hospital - Kashiwanoha /ID# 262454

Kashiwa-shi, Chiba 277-0871

Japan

Kurume University Hospital /ID# 262455

Kurume-shi, Fukuoka 830-0011

Japan

Miyagi Children's Hospital /ID# 262459

Sendai, Miyagi 989-3126

Japan

Osaka Women's and Children's Hospital /ID# 262549

Izumi-Shi, Osaka 594-1101

Japan

Saga University Hospital /ID# 262753

Saga, Saga-ken 849-8501

Japan

Saitama Children's Medical Center /ID# 262461

Saitama-shi, Saitama 330-8777

Japan

Institute of Science Tokyo Hospital /ID# 262510

Bunkyo-ku, Tokyo 113-8519

Japan

Tokyo Metropolitan Children's Medical Center /ID# 262550

Fuchu-shi, Tokyo 183-8561

Japan

National Center For Child Health And Development /ID# 262456

Setagaya City, Tokyo 157-8535

Japan

Toyama Prefectural Central Hospital /ID# 262739

Toyama, Toyama 930-8550

Japan

Starship Child Health /ID# 262576

Grafton, Auckland 1023

New Zealand

Christchurch Hospital. /ID# 262577

Christchurch, Canterbury 8011

New Zealand

Gastromed Sp. z o.o /ID# 262367

Torun, Kuyavian-Pomeranian Voivodeship 87-100

Poland

Instytut Pomnik - Centrum Zdrowia Dziecka /ID# 262366

Warsaw, Masovian Voivodeship 04-730

Poland

Clinical Research Investigator Group /ID# 262357

Bayamón, 00960

Puerto Rico

Puerto Rico Health Institute /ID# 262358

Dorado, 00646

Puerto Rico

Clinical Research Puerto Rico /ID# 279595

San Juan, 00909-1711

Puerto Rico

Seoul National University Hospital /ID# 262324

Seoul, Seoul Teugbyeolsi 03080

South Korea

Yonsei University Health System Severance Hospital /ID# 262721

Seoul, Seoul Teugbyeolsi 03722

South Korea

Samsung Medical Center /ID# 262323

Seoul, Seoul Teugbyeolsi 06351

South Korea

Hospital Arquitecto Marcide - Complejo Hospitalario Universitario de Ferrol /ID# 262226

Ferrol, A Coruna 15405

Spain

Hospital Sant Joan de Deu /ID# 262599

Esplugues de Llobregat, Barcelona 08950

Spain

Hospital Regional Universitario de Malaga /ID# 262228

Málaga, 29011

Spain

Hospital Universitario Virgen del Rocio /ID# 262712

Seville, 41013

Spain

Hospital Clinico Universitario Lozano Blesa /ID# 262227

Zaragoza, 50009

Spain

National Taiwan University Hospital /ID# 261695

Taipei, 100

Taiwan

Linkou Chang Gung Memorial Hospital /ID# 261696

Taoyuan City, 333

Taiwan

Addenbrookes Hospital /ID# 262707

Cambridge, Cambridgeshire CB2 2QQ

United Kingdom

Sheffield Children's Hospital NHS Foundation Trust /ID# 261832

Sheffield, England S10 2TH

United Kingdom

Disc_Barts Health NHS Trust - The Royal London Hospital /ID# 262811

London, Greater London E1 2ES

United Kingdom

Norfolk and Norwich University Hospitals NHS Foundation Trust /ID# 262778

Norwich, Norfolk NR4 7UY

United Kingdom

Royal Hospital for Children and Young People /ID# 262388

Edinburgh, EH16 4TJ

United Kingdom