New hope for kids with severe colitis: oral drug shows promise

NCT ID NCT04624230

Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 04, 2026 · Updated 2 times

Summary

This study tests an oral medication called tofacitinib in children aged 2 to 17 with moderate to severe ulcerative colitis, a chronic bowel disease causing inflammation and bleeding. The goal is to see if the drug can bring the disease into remission (no symptoms and healed gut lining) for up to 44 weeks. Children who have not responded to other treatments may be eligible. Since ulcerative colitis is a lifelong condition, this treatment aims to control the disease rather than cure it.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

118 people

The number who actually took part.

Started

Aug 2021

Expected to finish

Jun 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Evidence of a personally signed and dated informed consent document and assent document. * 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: * Diagnosis of indeterminate colitis, isolated proctitis, microscopic colitis, infectious colitis, Crohn's disease, or clinical findings suggestive of Crohn's disease. * 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.

Get updates

Get notified about this study

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

ulcerative colitis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Ulcerative colitis

Contacts and locations

Locations

A.O.U. Federico II

Naples, Naples, 80131, Italy
ASST Papa Giovanni XXIII Epatologia e Gastroenterologia Pediatrica e dei Trapianti

Bergamo, BG, 24127, Italy
Aichi Children's Health and Medical Center

Obu-shi, Aichi-ken, 474-8710, Japan
Amsterdam University Medical Center, VUmc Boelelaan

Amsterdam, North Holland, 1105 AZ, Netherlands
Azienda Ospedaliero - Universitaria Policlinico Umberto I di Roma

Roma, RM, 00161, Italy
Azienda USL di Bologna - IRCCS ISNB - Programma Gastroenterologia Pediatrica

Bologna, 40133, Italy
Bart's Health NHS Trust

London, E1 1BB, United Kingdom
Birmingham Women's and Children's NHS Foundation Trust

Birmingham, WEST Midlands, B4 6NH, United Kingdom
Boston Children's Hospital

Boston, Massachusetts, 02115, United States
British Columbia Children's Hospital

Vancouver, British Columbia, V6H 3N1, Canada
Buerger Center for Advanced Pediatric Care

Philadelphia, Pennsylvania, 19104, United States
CHU Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada
CHU de Lyon - Hôpital Femme Mère Enfant

Bron, 69677, France
Center for Advanced Pediatrics

Atlanta, Georgia, 30329, United States
Children's Healthcare of Atlanta - Arthur M. Blank Hospital

Atlanta, Georgia, 30329, United States
Children's Hospital Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Cliniques Universitaires Saint-Luc

Brussels, 1200, Belgium
Connecticut Children's Ambulatory Surgical Center

Farmington, Connecticut, 06032, United States
Connecticut Children's Infusion Center

Farmington, Connecticut, 06032, United States
Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States
Dr. von Haunersches Kinderspital, LMU

Munich, Bavaria, 80337, Germany
Erasmus Medical Center - Sophia Children's Hospital

Rotterdam, 3015 GD, Netherlands
Gunma University Hospital

Maebashi, Gunma, 371-8511, Japan
Hôpital Necker Enfants Malades

Paris, 75015, France
Hôpital Universitaire Des Enfants Reine Fabiola

Brussels, 1020, Belgium
IWK Health Centre

Halifax, Nova Scotia, B3K 6R8, Canada
Instytut "Centrum Zdrowia Matki Polki"

Lodz, 93-338, Poland
Instytut "Pomnik - Centrum Zdrowia Dziecka"

Warsaw, 04-730, Poland
Jichi Medical University Hospital

Shimotsuke, Tochigi, 329 0498, Japan
Juntendo University Hospital

Bunkyo-ku, Tokyo, 113-8431, Japan
Karolinska Universitetssjukhuset Barngastroenterologi, hepatologi och nutrition

Stockholm, 171 76, Sweden
King's College Hospital NHS Foundation Trust

London, Greater London, SE5 9RS, United Kingdom
Korczowski Bartosz, Gabinet Lekarski

Rzeszów, 35-302, Poland
Kurume University Hospital

Kurume-shi, Fukuoka, 830-0011, Japan
Lady Davis Carmel Medical Center

Haifa, 3436212, Israel
London Health Sciences Centre - Children's Hospital

London, Ontario, N6A 5W9, Canada
London Health Sciences Centre - Children's Hospital

London, Ontario, N6C2R5, Canada
Medical Network Spółka z o.o. WIP Warsaw IBD Point Profesor Kierkuś

Warsaw, Masovian Voivodeship, 04-501, Poland
Miyagi Children's Hospital

Sendai, Miyagi, 989-3126, Japan
Narodny ustav detskych chorob

Bratislava, 833 40, Slovakia
National Center for Child Health and Development

Setagaya-ku, Tokyo, 157-8535, Japan
Nicklaus Children's Hospital

Miami, Florida, 33155, United States
Northwell Health - Cohen Children's Medical Center

Lake Success, New York, 11042, United States
Northwell Health - Cohen Children's Medical Center

New Hyde Park, New York, 11040, United States
Osaka Medical and Pharmaceutical University Hospital

Takatsuki-shi, Osaka, 569-8686, Japan
Osaka Women's and Children's Hospital

Izumi, Osaka, 594-1101, Japan
Roberts Center for Pediatric Research

Philadelphia, Pennsylvania, 19146, United States
Sachsska Children's and Youth Hospital/South General Hospital

Stockholm, 118 83, Sweden
Sahlgrenska Universitetssjukhuset

Gothenburg, 416 50, Sweden
Saitama Prefectural Children's Medical Center

Saitama-shi, Saitama, 330-8777, Japan
Schneider Children's Medical Center of Israel

Petah Tikva, 4920235, Israel
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Shaare Zedek Medical Center

Jerusalem, 9103102, Israel
Shamir Medical Center (Assaf Harofeh)

Be’er Ya‘aqov, 7033001, Israel
Stollery Children's Hospital University of Alberta

Edmonton, Alberta, T6G 1C9, Canada
Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont

Szeged, 6720, Hungary
Tampereen yliopistollinen sairaala

Tampere, 33520, Finland
Tel-Aviv Sourasky Medical Center

Tel Aviv, 6423906, Israel
Texas Children's Hospital

Houston, Texas, 77030, United States
Texas Children's Hospital - RRO Regulatory (Administrative Offices - Regulatory Location)

Houston, Texas, 77030, United States
The Hospital for Sick Children - Division of Gastroenterology, Hepatology and Nutrition

Toronto, Ontario, M5G 1X8, Canada
The Royal Children's Hospital

Parkville, Victoria, 3052, Australia
Universitair Ziekenhuis Brussel

Brussels, 1090, Belgium
Universitaire Ziekenhuizen Leuven

Leuven, Vlaams Brabant, 3000, Belgium
University of California, San Francisco Benioff Children's Hospital

San Francisco, California, 94158, United States
University of California, San Francisco Pediatric Clinical Research Center (PCRC)

San Francisco, California, 94158, United States
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu

Wroclaw, 50-369, Poland

More trials for these conditions

Other studies related to the condition(s) this trial covers.