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

NCT06332534 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: M14-6712023-509618-12
• Study Type: interventional
• Target: 110
• Locations: 18 countries
• Sites: 83

Brief 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.

Conditions

Crohn's Disease

Keywords

Crohn's Disease; Upadacitinib; RINVOQ

Outcome Measures

Primary Outcomes (3)

• 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

  PCDAI is an index used to measure disease activity of pediatric patients with Crohn's disease assessing abdominal pain, stool frequency, patient functioning, hematocrit, erythrocyte sedimentation rate, albumin, weight, height, abdomen, perirectal disease, and extraintestinal manifestations. It ranges from 0 to 100; higher scores indicate more active disease. Clinical remission was defined as PCDAI ≤ 10.

  At Week 64

• Achievement of endoscopic response at Week 64 in participants who achieved clinical response per PCDAI at Week 12.

  Endoscopic response is defined as \> 50% reduction in Simple Endoscopic Score for Crohn's Disease (SES-CD) score from Baseline (or for participants with a Baseline SES-CD of 4, at least a 2-point reduction from Baseline), as scored by a central reader.

  At Week 64

• Number of Participants with Adverse Events

  An adverse event (AE) is defined as any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. The investigator assessed the relationship of each event to the use of study drug as either probably related, possibly related, probably not related or not related.

  Through Week 156

Secondary Outcomes (7)

• Achievement of clinical remission per PCDAI

  Week 12

• Achievement of endoscopic response

  Week 12

• Achievement of endoscopic remission

  Week 12

• Achievement of clinical response per PCDAI

  Week 12

• Achievement of clinical response per PCDAI at Week 64 in participants who achieved clinical response per PCDAI at Week 12

  Week 64

Study Arms (6)

Period 1: Open Label Induction Phase (Dose A)

EXPERIMENTAL

All participants in the open label induction phase of Period 1 will receive upadacitinib Dose A for 12 weeks based on body weight.

Drug: Upadacitinib

Period 1: Double-Blind Maintenance Phase (Dose B)

EXPERIMENTAL

Clinical responders per PCDAI at the end of open label induction phase of Period 1 will be randomly assigned to receive Dose B or C for 52 weeks (oral solution dose will be based on body weight)

Drug: Upadacitinib

Period 1: Double-Blind Maintenance Phase (Dose C)

EXPERIMENTAL

Clinical responders per PCDAI at the end of open label induction phase of Period 1 will be randomly assigned to receive either upadacitinib Dose C or B for 52 weeks (oral solution dose will be based on body weight)

Drug: Upadacitinib

Period 2: Open Label Long-Term Extension Phase Cohort 1

EXPERIMENTAL

Participants receiving double-blind maintenance therapy with upadacitinib Dose B or upadacitinib Dose C daily in Period 1 who complete the Week 64 visit will receive upadacitinib Dose B daily for up to 156 weeks.

Drug: Upadacitinib

Period 2: Open Label Long-Term Extension Phase Cohort 2

EXPERIMENTAL

Participants who were receiving rescue therapy with open-label upadacitinib Dose C during maintenance phase in Period 1 and completed the Week 64 visit will continue to receive upadacitinib Dose C daily for up to 156 weeks.

Drug: Upadacitinib

Period 2: Open Label Long-Term Extension Phase Cohort 3

EXPERIMENTAL

Participants who did not achieve clinical response per PCDAI at Week 12 of Period 1 will receive an extended treatment with open-label upadacitinib Dose C daily for an additional 12 weeks. If they are responders after 12 weeks extended treatment, they will continue, otherwise they may be discontinued at the discretion of the investigator

Drug: Upadacitinib

Interventions

Upadacitinib DRUG

Oral Solution/ Extended-Release Tablets

Also known as: RINVOQ

Period 1: Double-Blind Maintenance Phase (Dose B); Period 1: Double-Blind Maintenance Phase (Dose C); Period 1: Open Label Induction Phase (Dose A); Period 2: Open Label Long-Term Extension Phase Cohort 1; Period 2: Open Label Long-Term Extension Phase Cohort 2; Period 2: Open Label Long-Term Extension Phase Cohort 3

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• AbbVie: lead

Study Sites (83)

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

Oakland, California, 94609, United States

RECRUITING

Lucile Packard Children's Hospital /ID# 262193

Palo Alto, California, 94304, United States

RECRUITING

Children's Hospital Colorado - Aurora /ID# 262207

Aurora, Colorado, 80045, United States

RECRUITING

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

Hartford, Connecticut, 06106, United States

RECRUITING

OSF St. Francis Medical Center /ID# 262192

Peoria, Illinois, 61637-0001, United States

RECRUITING

Indiana University Health Riley Hospital for Children /ID# 262215

Indianapolis, Indiana, 46202, United States

RECRUITING

Boston Children's Hospital /ID# 262191

Boston, Massachusetts, 02115, United States

RECRUITING

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

Minneapolis, Minnesota, 55413-2195, United States

RECRUITING

Icahn School of Medicine at Mount Sinai /ID# 262216

New York, New York, 10029, United States

RECRUITING

Univ NC Chapel Hill /ID# 262198

Chapel Hill, North Carolina, 27514-4220, United States

RECRUITING

UH Cleveland Medical Center /ID# 262188

Cleveland, Ohio, 44106, United States

RECRUITING

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

Philadelphia, Pennsylvania, 19104-4319, United States

RECRUITING

Sydney Children's Hospital /ID# 262352

Randwick, New South Wales, 2031, Australia

RECRUITING

Children's Hospital at Westmead /ID# 262350

Westmead, New South Wales, 2145, Australia

RECRUITING

Queensland Children's Hospital /ID# 262351

South Brisbane, Queensland, 4101, Australia

RECRUITING

Monash Health - Monash Medical Centre /ID# 262878

Clayton, Victoria, 3168, Australia

RECRUITING

Perth Children'S Hospital /ID# 272905

Perth, Western Australia, 6009, Australia

RECRUITING

Uza /Id# 261745

Edegem, Antwerpen, 2650, Belgium

RECRUITING

Cliniques Universitaires UCL Saint-Luc /ID# 261741

Brussels, Brussels Capital, 1200, Belgium

RECRUITING

Universitair Ziekenhuis Leuven /ID# 261740

Leuven, Vlaams-Brabant, 3000, Belgium

RECRUITING

Hospital Universite Enfants Reine Fabiola /ID# 261744

Brussels, 1020, Belgium

RECRUITING

CHR de la Citadelle /ID# 261749

Liège, 4000, Belgium

RECRUITING

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

Namur, 5000, Belgium

RECRUITING

Galileo Medical Research Ltda /ID# 262602

Juiz de Fora, Minas Gerais, 36033-318, Brazil

RECRUITING

Hospital Pequeno Príncipe /ID# 262600

Curitiba, Paraná, 80250-060, Brazil

RECRUITING

Irmandade da Santa Casa de Misericordia de Porto Alegre /ID# 262601

Porto Alegre, Rio Grande do Sul, 90020-090, Brazil

RECRUITING

Rocco & Nazato Servicos Medicos /ID# 262485

São Paulo, São Paulo, 04543-011, Brazil

RECRUITING

Hospital Sirio Libanes /ID# 262670

São Paulo, 01308-050, Brazil

RECRUITING

UMHAT Sveti Georgi /ID# 262590

Plovdiv, 4002, Bulgaria

RECRUITING

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

Sofia, 1606, Bulgaria

RECRUITING

IWK Health Center /ID# 262543

Halifax, Nova Scotia, B3K 6R8, Canada

RECRUITING

Beijing Children's Hospital /ID# 262258

Beijing, Beijing Municipality, 100045, China

RECRUITING

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

Guangzhou, Guangdong, 510620, China

RECRUITING

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

Guangzhou, Guangdong, 510655, China

RECRUITING

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

Zhengzhou, Henan, 450018, China

RECRUITING

Hunan Children's Hospital /ID# 262512

Changsha, Hunan, 410007, China

RECRUITING

Jiangxi Provincial Children's Hospital /ID# 262295

Nanchang, Jiangxi, 330006, China

RECRUITING

Shengjing Hospital of China Medical University /ID# 262301

Shenyang, Liaoning, 110022, China

RECRUITING

Children's Hospital of Shanghai /ID# 262356

Shanghai, Shanghai Municipality, 200062, China

RECRUITING

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

Shanghai, Shanghai Municipality, 200065, China

RECRUITING

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

Hangzhou, Zhejiang, 310003, China

RECRUITING

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

Caen, Calvados, 14033, France

RECRUITING

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

Bron, Rhone, 69500, France

RECRUITING

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

Paris, 75019, France

RECRUITING

Agia Sofia Hospital /ID# 261792

Athens, Attica, 11527, Greece

RECRUITING

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

Athens, Attica, 11527, Greece

RECRUITING

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

Heraklion, Crete, 71500, Greece

RECRUITING

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

San Giovanni Rotondo, Foggia, 71013, Italy

RECRUITING

Ospedale Pediatrico Bambino Gesù /ID# 262379

Rome, Roma, 00165, Italy

RECRUITING

Azienda Ospedaliera Universitaria Gaetano Martino /ID# 262380

Messina, 98125, Italy

RECRUITING

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico /ID# 275276

Milan, 20122, Italy

RECRUITING

Tsujinaka Hospital - Kashiwanoha /ID# 262454

Kashiwa-shi, Chiba, 277-0871, Japan

RECRUITING

Kurume University Hospital /ID# 262455

Kurume-shi, Fukuoka, 830-0011, Japan

RECRUITING

Miyagi Children's Hospital /ID# 262459

Sendai, Miyagi, 989-3126, Japan

RECRUITING

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

Izumi-Shi, Osaka, 594-1101, Japan

RECRUITING

Saga University Hospital /ID# 262753

Saga, Saga-ken, 849-8501, Japan

RECRUITING

Saitama Children's Medical Center /ID# 262461

Saitama-shi, Saitama, 330-8777, Japan

RECRUITING

Institute of Science Tokyo Hospital /ID# 262510

Bunkyo-ku, Tokyo, 113-8519, Japan

RECRUITING

Tokyo Metropolitan Children's Medical Center /ID# 262550

Fuchu-shi, Tokyo, 183-8561, Japan

RECRUITING

National Center For Child Health And Development /ID# 262456

Setagaya City, Tokyo, 157-8535, Japan

RECRUITING

Toyama Prefectural Central Hospital /ID# 262739

Toyama, Toyama, 930-8550, Japan

RECRUITING

Christchurch Hospital. /ID# 262577

Christchurch, Canterbury, 8011, New Zealand

RECRUITING

Starship Child Health /ID# 262576

Auckland, 0629, New Zealand

COMPLETED

Gastromed Sp. z o.o /ID# 262367

Torun, Kuyavian-Pomeranian Voivodeship, 87-100, Poland

RECRUITING

Instytut Pomnik - Centrum Zdrowia Dziecka /ID# 262366

Warsaw, Masovian Voivodeship, 04-730, Poland

RECRUITING

Clinical Research Investigator Group, LLC /ID# 262357

Bayamón, 00960, Puerto Rico

RECRUITING

Puerto Rico Health Institute /ID# 262358

Dorado, 00646, Puerto Rico

RECRUITING

Clinical Research Puerto Rico /ID# 279595

San Juan, 00909-1711, Puerto Rico

RECRUITING

Seoul National University Hospital /ID# 262324

Seoul, Seoul Teugbyeolsi, 03080, South Korea

RECRUITING

Yonsei University Health System Severance Hospital /ID# 262721

Seoul, Seoul Teugbyeolsi, 03722, South Korea

RECRUITING

Samsung Medical Center /ID# 262323

Seoul, Seoul Teugbyeolsi, 06351, South Korea

RECRUITING

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

Ferrol, A Coruna, 15405, Spain

RECRUITING

Hospital Sant Joan de Deu /ID# 262599

Esplugues de Llobregat, Barcelona, 08950, Spain

RECRUITING

Hospital Regional Universitario de Malaga /ID# 262228

Málaga, 29011, Spain

RECRUITING

Hospital Universitario Virgen del Rocio /ID# 262712

Seville, 41013, Spain

RECRUITING

Hospital Clinico Universitario Lozano Blesa /ID# 262227

Zaragoza, 50009, Spain

COMPLETED

National Taiwan University Hospital /ID# 261695

Taipei City, Taipei, 100, Taiwan

RECRUITING

Linkou Chang Gung Memorial Hospital /ID# 261696

Taoyuan, 333, Taiwan

RECRUITING

Addenbrookes Hospital /ID# 262707

Cambridge, Cambridgeshire, CB2 2QQ, United Kingdom

RECRUITING

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

Sheffield, England, S10 2TH, United Kingdom

RECRUITING

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

London, Greater London, E1 2ES, United Kingdom

RECRUITING

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

Norwich, Norfolk, NR4 7UY, United Kingdom

RECRUITING

Royal Hospital for Children and Young People /ID# 262388

Edinburgh, EH16 4TJ, United Kingdom

RECRUITING

Related Links

• Related Info

MeSH Terms

Conditions

Crohn Disease

Interventions

upadacitinib

Condition Hierarchy (Ancestors)

Inflammatory Bowel Diseases; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Study Officials

• ABBVIE INC.

  AbbVie

  STUDY DIRECTOR

Central Study Contacts

ABBVIE CALL CENTER

CONTACT

844-663-3742; abbvieclinicaltrials@abbvie.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 19, 2024
• First Posted: March 27, 2024
• Study Start: August 8, 2024
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): December 1, 2034
• Last Updated: April 15, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: For details on when studies are available for sharing visit https://vivli.org/ourmember/abbvie/
• Access Criteria: To learn more about the process, or to submit a request, visit the following link https://www.abbvieclinicaltrials.com/hcp/data-sharing/

Locations

FR (3); PR (3); ES (5); CA (1); GR (3); CN (10); PL (2); KR (3); US (12); AU (5); BR (5); JP (10); GB (5); BE (6); BU (2); IT (4); TW (2); NZ (2)