Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative Colitis
NCT ID: NCT04624230
Last Updated: 2025-10-24
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
120 participants
INTERVENTIONAL
2021-08-12
2029-05-15
Brief Summary
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All eligible participants will initially receive open label tofacitinib at a dose expected to produce equivalent systemic exposure to that observed in adults receiving 5 mg BID with the option for individual dose increase to 10 mg BID adult dose equivalent if dose escalation criteria are met.
The primary objective of this study is to evaluate the efficacy of tofacitinib based on remission in pediatric participants with moderately to severely active UC. The primary endpoint is remission by central read Mayo score following 44 weeks in the maintenance phase. Remission is defined by a Mayo score of 2 points or lower, with no individual subscore exceeding 1 point and a rectal bleeding subscore of 0.
The study Design is an open-label Phase 3 study that includes a screening period of up to 4-weeks duration, an 8-week or 16-week induction phase, a 44-week maintenance phase, and a 24-month extension phase for pediatric participants with moderately to severely active UC. Participants will have a follow-up visit 4 weeks after the last dose of study intervention and a telephone contact 8 weeks later to assess for any adverse events (AEs)/serious adverse events (SAEs). The total maximum duration of this study will be up to 180 weeks.
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Detailed Description
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Conditions
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Study Design
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NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
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tofacitinib
Open label tofacitinib 5 mg BID weight based adult equivalent with the option for individual dose increase to 10 mg BID weight based adult equivalent for a limited time if dose escalation criteria are met, prior to returning to 5 mg BID.
tofacitinib
Open label tofacitinib 5 mg BID weight based adult equivalent with the option for individual dose increase to 10 mg BID weight based adult equivalent for a limited time if dose escalation criteria are met, prior to returning to 5 mg BID.
Interventions
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tofacitinib
Open label tofacitinib 5 mg BID weight based adult equivalent with the option for individual dose increase to 10 mg BID weight based adult equivalent for a limited time if dose escalation criteria are met, prior to returning to 5 mg BID.
Eligibility Criteria
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Inclusion Criteria
* Males and females 2 to less than18 years old and weighing at least 10 kg.
* Having a pathology report that confirms colonic inflammation consistent with UC with a clinical diagnosis of UC for at least 12 weeks prior to baseline, with biopsy report supporting the diagnosis of UC.
* Participants diagnosed with UC at age less than 6 years old, must have had testing and be negative for monogenic disorders associated with very early onset IBD.
* Moderately to severely active UC as defined (via screening colonoscopy) by a Mayo score of at least 6, with a rectal bleeding score of at least 1 and an endoscopic subscore of at least 2.
* Pediatric Ulcerative Colitis Activity Index (PUCAI) score greater or equal to 35 .
* No history of dysplasia or colon cancer.
* No evidence or history of untreated or inadequately treated active or latent infection with Mycobacterium Tuberculosis.
* For participants outside of the United States or the European Union: have had an inadequate response or been intolerant to at least one prior therapy as listed below or have a medical contraindication to such therapies:
* Oral or intravenous (IV) corticosteroids;
* Azathioprine or 6-mercaptopurine;
* TNF inhibitors or anti integrin therapy.
* For participants in the United States and the European Union: have had an inadequate response or intolerance to TNF inhibitors.
* Stable doses of the following therapies for UC:
* Oral 5 Aminosalicyclic acids (ASA) or sulfasalazine
* Oral corticosteroids equivalent to prednisone at most 1 mg/kg up to a maximum of 20 mg/day or budesonide up to 9 mg/day.
* female participant is eligible if she is not pregnant or breastfeeding, If she is a woman of child bearing potential, she needs to be using a contraceptive method that is highly effective (with a failure rate of \<1% per year).
Exclusion Criteria
* History of symptomatic obstructive intestinal strictures or active ostomy, or history of colectomy, extensive small bowel resection ( greater than100 centimetres) or short bowel syndrome, or hospitalization for UC related reason(s) within 2 weeks of baseline visit.
* Any factors or clinical characteristics potentially related to the risk of venous thromboembolism that may increase the risk associated with study participation or study intervention administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
* Participants who have previously received tofacitinib or another Janus Kinase inhibitor.
* Vaccination or exposure to a live or attenuated vaccine within the 6 weeks prior to the first dose of study drug, or who are expected to be vaccinated or to have household exposure to these vaccines during treatment or during the 6 weeks following discontinuation of study drug.
* Participants having received azathioprine, 6-mercaptopurine, methotrexate, thioguanine, infliximab, adalimumab, golimumab, ustekinumab, interferon, cyclosporine, mycophenolate, tacrolimus, IV or rectally administered corticosteroids, natalizumab, vedolizumab, other antiadhesion molecules, or investigational drugs during the specified time periods prior to baseline whereby they may still have pharmacokinetic and/or pharmacodynamic effect in the body of the participant.
* Previous treatment by leukocyte apheresis including selective lymphocyte, monocyte, or granulocyte apheresis, or plasma exchange within 6 months prior to baseline.
* Treatment by specified prohibited concomitant medications, including moderate to potent CYP3A inducers or inhibitors in the specified time periods prior to the first dose of study drug or are expected to receive any of these medications during the study period.
* Chronic and frequent use of antimotility agents for control of diarrhea (ie, diphenoxylate hydrochloride with atropine sulfate or loperamide).
* History of bowel surgery, including cholecystectomy within 6 months prior to baseline, history of appendectomy within 3 months prior to baseline, or significant trauma or major surgery within 4 weeks of screening visit are excluded.
* Participants with the following laboratory values at screening:
* Hemoglobin level lower than 9.0 g/Dl.
* Absolute white blood cell (WBC) count lower than 3000/mm3.
* Absolute neutrophil count lower than 1200/mm3.
* Absolute lymphocyte count lower than 750/mm3.
* Thrombocytopenia as defined by a platelet count lower than 100,000/mm3.
* Estimated bedside Schwartz Glomerular filtration rate (GFR) lower or equal to 40 mL/min/1.73 m2.
* Total bilirubin, aspartate aminostransferase (AST) or alanine aminotransferase (ALT) more than 1.5 times the upper limit of normal.
* Positive stool examinations for enteric pathogens, pathogenic ova or parasites, or C. difficile toxin at screening.
* Participants infected with human immunodeficiency virus (HIV) or hepatitis B or C viruses.
* History of more than one episode of HZ, a history of disseminated HZ or disseminated herpes simplex.
* History or current symptoms of any lymphoproliferative disorder (eg, Epstein Barr Virus (EBV) related lymphoproliferative disorder, lymphoma, leukemia, myeloproliferative disorders, multiple myeloma, or signs and symptoms suggestive of currently lymphatic disease).
* Clinically significant infections currently or within 3 months prior to baseline (eg, those requiring hospitalization or parenteral antimicrobial therapy or opportunistic infections), a history of any infection requiring antimicrobial therapy within 2 weeks of baseline, or a history of any infection otherwise judged by the investigator to have the potential for exacerbation by participation in the study.
* Any malignancies or with a history of malignancies, with the exception of adequately treated or excised nonmetastatic basal cell or squamous cell cancer of the skin.
* Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or participants who are employees of the Sponsor, including their family members, directly involved in the conduct of the study.
* Participation in other studies involving investigational drug(s) within 2 months prior to study entry and/or during study participation.
* Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or study intervention administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
* Pregnant female participants; breastfeeding female participants; fertile female participants of childbearing potential who are unwilling or unable to use a highly effective method of contraception as outlined in this protocol for the duration of the study and through the telephone follow up visit.
* History of allergies, intolerance or hypersensitivity to lactose or tofacitinib, or any other excipients of the investigational medicinal products, including placebos.
2 Years
17 Years
ALL
No
Sponsors
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Pfizer
INDUSTRY
Responsible Party
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Principal Investigators
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Pfizer CT.gov Call Center
Role: STUDY_DIRECTOR
Pfizer
Locations
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Children's Hospital Los Angeles
Los Angeles, California, United States
University of California, San Francisco Benioff Children's Hospital
San Francisco, California, United States
University of California, San Francisco Pediatric Clinical Research Center (PCRC)
San Francisco, California, United States
Connecticut Children's Ambulatory Surgical Center
Farmington, Connecticut, United States
Connecticut Children's Infusion Center
Farmington, Connecticut, United States
Connecticut Children's Medical Center
Hartford, Connecticut, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Center for Advanced Pediatrics
Atlanta, Georgia, United States
Children's Healthcare of Atlanta - Arthur M. Blank Hospital
Atlanta, Georgia, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Atlantic Children's Health-Pediatric Gastroenterology & Nutrition
Morristown, New Jersey, United States
Goryeb Children's Hospital (Endoscopy only)
Morristown, New Jersey, United States
Atlantic Health System- Morristown Medical Center (Pharmacy)
Morristown, New Jersey, United States
Northwell Health - Cohen Children's Medical Center
Lake Success, New York, United States
Northwell Health - Cohen Children's Medical Center
New Hyde Park, New York, United States
Columbia University Irving Medical Center
New York, New York, United States
CUIMC Research Pharmacy - Milstein Hospital (Pharmacy Only)
New York, New York, United States
Morgan Stanley Children's Hospital, CUIMC
New York, New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Buerger Center for Advanced Pediatric Care
Philadelphia, Pennsylvania, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Roberts Center for Pediatric Research
Philadelphia, Pennsylvania, United States
Texas Children's Hospital
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
The Royal Children's Hospital
Parkville, Victoria, Australia
Universitaire Ziekenhuizen Leuven
Leuven, Vlaams Brabant, Belgium
Hôpital Universitaire Des Enfants Reine Fabiola
Brussels, , Belgium
Universitair Ziekenhuis Brussel
Brussels, , Belgium
Cliniques Universitaires Saint-Luc
Brussels, , Belgium
Stollery Children's Hospital University of Alberta
Edmonton, Alberta, Canada
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
IWK Health Centre
Halifax, Nova Scotia, Canada
London Health Sciences Centre - Children's Hospital
London, Ontario, Canada
The Hospital for Sick Children - Division of Gastroenterology, Hepatology and Nutrition
Toronto, Ontario, Canada
CHU Sainte-Justine
Montreal, Quebec, Canada
Tampereen yliopistollinen sairaala
Tampere, , Finland
CHU de Lyon - Hôpital Femme Mère Enfant
Bron, , France
Hôpital Necker Enfants Malades
Paris, , France
Dr. von Haunersches Kinderspital, LMU
Munich, Bavaria, Germany
Debreceni Egyetem Klinikai Kozpont
Debrecen, Hajdú-Bihar, Hungary
Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont
Szeged, , Hungary
Shamir Medical Center (Assaf Harofeh)
Be’er Ya‘aqov, , Israel
Lady Davis Carmel Medical Center
Haifa, , Israel
Shaare Zedek Medical Center
Jerusalem, , Israel
Schneider Children's Medical Center of Israel
Petah Tikva, , Israel
Tel-Aviv Sourasky Medical Center
Tel Aviv, , Israel
ASST Papa Giovanni XXIII Epatologia e Gastroenterologia Pediatrica e dei Trapianti
Bergamo, BG, Italy
A.O.U. Federico II
Napoli, Naples, Italy
Azienda Ospedaliero - Universitaria Policlinico Umberto I di Roma
Roma, RM, Italy
Azienda USL di Bologna - IRCCS ISNB - Programma Gastroenterologia Pediatrica
Bologna, , Italy
Aichi Children's Health and Medical Center
Obu-shi, Aichi-ken, Japan
Kurume University Hospital
Kurume-shi, Fukuoka, Japan
Gunma University Hospital
Maebashi, Gunma, Japan
Miyagi Children's Hospital
Sendai, Miyagi, Japan
Osaka Women's and Children's Hospital
Izumi, Osaka, Japan
Osaka Medical and Pharmaceutical University Hospital
Takatsuki-shi, Osaka, Japan
Saitama Prefectural Children's Medical Center
Saitama-shi, Saitama, Japan
Jichi Medical University Hospital
Shimotsuke, Tochigi, Japan
Juntendo University Hospital
Bunkyo-ku, Tokyo, Japan
National Center for Child Health and Development
Setagaya-ku, Tokyo, Japan
Amsterdam UMC location AMC
Amsterdam, North Holland, Netherlands
Amsterdam UMC, location VUmc Boelelaan
Amsterdam, , Netherlands
Erasmus Medical Center - Sophia Children's Hospital
Rotterdam, , Netherlands
Medical Network Spółka z o.o. WIP Warsaw IBD Point Profesor Kierkuś
Warsaw, Masovian Voivodeship, Poland
Instytut "Centrum Zdrowia Matki Polki"
Lodz, , Poland
Korczowski Bartosz, Gabinet Lekarski
Rzeszów, , Poland
Instytut "Pomnik - Centrum Zdrowia Dziecka"
Warsaw, , Poland
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu
Wroclaw, , Poland
Narodny ustav detskych chorob
Bratislava, , Slovakia
Hospital Infantil Universitario Niño Jesus
Madrid, , Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, , Sweden
Sachsska Children's and Youth Hospital/South General Hospital
Stockholm, , Sweden
Karolinska Universitetssjukhuset Barngastroenterologi, hepatologi och nutrition
Stockholm, , Sweden
King's College Hospital NHS Foundation Trust
London, Greater London, United Kingdom
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, WEST Midlands, United Kingdom
NHS Lothian
Edinburgh, , United Kingdom
Bart's Health NHS Trust
London, , United Kingdom
Countries
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Central Contacts
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References
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Benchimol EI, Bernstein CN, Bitton A, Carroll MW, Singh H, Otley AR, Vutcovici M, El-Matary W, Nguyen GC, Griffiths AM, Mack DR, Jacobson K, Mojaverian N, Tanyingoh D, Cui Y, Nugent ZJ, Coulombe J, Targownik LE, Jones JL, Leddin D, Murthy SK, Kaplan GG. Trends in Epidemiology of Pediatric Inflammatory Bowel Disease in Canada: Distributed Network Analysis of Multiple Population-Based Provincial Health Administrative Databases. Am J Gastroenterol. 2017 Jul;112(7):1120-1134. doi: 10.1038/ajg.2017.97. Epub 2017 Apr 18.
Konstantinides SV, Meyer G, Becattini C, Bueno H, Geersing GJ, Harjola VP, Huisman MV, Humbert M, Jennings CS, Jimenez D, Kucher N, Lang IM, Lankeit M, Lorusso R, Mazzolai L, Meneveau N, Ni Ainle F, Prandoni P, Pruszczyk P, Righini M, Torbicki A, Van Belle E, Zamorano JL; ESC Scientific Document Group. 2019 ESC Guidelines for the diagnosis and management of acute pulmonary embolism developed in collaboration with the European Respiratory Society (ERS). Eur Heart J. 2020 Jan 21;41(4):543-603. doi: 10.1093/eurheartj/ehz405. No abstract available.
Kelsen JR, Sullivan KE, Rabizadeh S, Singh N, Snapper S, Elkadri A, Grossman AB. North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition Position Paper on the Evaluation and Management for Patients With Very Early-onset Inflammatory Bowel Disease. J Pediatr Gastroenterol Nutr. 2020 Mar;70(3):389-403. doi: 10.1097/MPG.0000000000002567.
Turner D, Otley AR, Mack D, Hyams J, de Bruijne J, Uusoue K, Walters TD, Zachos M, Mamula P, Beaton DE, Steinhart AH, Griffiths AM. Development, validation, and evaluation of a pediatric ulcerative colitis activity index: a prospective multicenter study. Gastroenterology. 2007 Aug;133(2):423-32. doi: 10.1053/j.gastro.2007.05.029. Epub 2007 May 21.
Otley A, Smith C, Nicholas D, Munk M, Avolio J, Sherman PM, Griffiths AM. The IMPACT questionnaire: a valid measure of health-related quality of life in pediatric inflammatory bowel disease. J Pediatr Gastroenterol Nutr. 2002 Oct;35(4):557-63. doi: 10.1097/00005176-200210000-00018.
Marcovitch L, Nissan A, Mack D, Otley A, Hussey S, Mclean B, Lewis M, Croft N, Barakat FM, Griffiths AM, Turner D. Item Generation and Reduction Toward Developing a Patient-reported Outcome for Pediatric Ulcerative Colitis (TUMMY-UC). J Pediatr Gastroenterol Nutr. 2017 Mar;64(3):373-377. doi: 10.1097/MPG.0000000000001259.
Taylor SJ, Whincup PH, Hindmarsh PC, Lampe F, Odoki K, Cook DG. Performance of a new pubertal self-assessment questionnaire: a preliminary study. Paediatr Perinat Epidemiol. 2001 Jan;15(1):88-94. doi: 10.1046/j.1365-3016.2001.00317.x.
Related Links
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Other Identifiers
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OVATION
Identifier Type: OTHER
Identifier Source: secondary_id
2023-509694-22-00
Identifier Type: REGISTRY
Identifier Source: secondary_id
A3921210
Identifier Type: -
Identifier Source: org_study_id
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