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Study of Tofacitinib for Children with Moderate to Severe Ulcerative Colitis

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called tofacitinib in children who have a condition known as Ulcerative Colitis. Ulcerative Colitis is a disease that causes inflammation and sores in the lining of the large intestine, which can lead to symptoms like abdominal pain and diarrhea. The medication being tested, tofacitinib, is taken orally, meaning it is swallowed in the form of a tablet or solution. In this study, the medication is sometimes referred to by its code name, CP-690550, and is also known as XELJANZ when in solution form.

The purpose of the study is to evaluate how effective tofacitinib is in helping children with moderate to severe Ulcerative Colitis achieve remission, which means reducing or eliminating the symptoms of the disease. Participants in the study will receive either the medication or a placebo, and their progress will be monitored over a period of time. The study will involve regular visits to the clinic, where participants will take the medication and undergo various tests to track their health and response to the treatment.

The study will last for a maximum of 39 weeks, during which the participants' health will be closely monitored. The goal is to see if tofacitinib can help improve the condition of children with Ulcerative Colitis and to gather information on its safety and effectiveness. This research is important for understanding how to better treat this condition in young patients and potentially improve their quality of life.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the participant must have a signed informed consent document. This indicates that the participant or their legal guardian has been informed of all important aspects of the study.

    Participants must be between 2 and 18 years old and weigh at least 10 kg. They should have a clinical diagnosis of ulcerative colitis (UC) for at least 12 weeks before the study begins.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to confirm the diagnosis of UC. This includes a pathology report confirming colonic inflammation and a Mayo score assessment, which evaluates the severity of UC.

    Participants must have a Pediatric Ulcerative Colitis Activity Index (PUCAI) score of 35 or higher at baseline.

  3. Step 3

    Induction phase

    During the induction phase, participants will receive tofacitinib orally. The specific dosage and frequency will be determined by the study protocol.

    The induction phase includes assessments at Week 8 and Week 16 to evaluate the response to the medication using the Mayo score and PUCAI score.

  4. Step 4

    Maintenance phase

    Participants who respond to the treatment will enter the maintenance phase, continuing to receive tofacitinib orally.

    The primary goal is to achieve remission by Week 44, as measured by the Mayo score.

  5. Step 5

    Ongoing assessments

    Throughout the study, regular assessments will be conducted to monitor the participant's condition. This includes changes in Mayo and PUCAI scores, endoscopic evaluations, and laboratory tests.

    Participants will also be evaluated for any side effects or changes in their condition.

  6. Step 6

    Completion of study

    The study is estimated to end in March 2029. Participants will have a final assessment to determine the overall effectiveness of the treatment.

    The study aims to provide insights into the long-term management of UC in children using tofacitinib.

Who can join the trial?

13 criteria

  • Must have a signed and dated consent form from the participant or their legal guardian, showing they understand the study.
  • Must be between 2 and under 18 years old and weigh at least 10 kg.
  • Must have been diagnosed with Ulcerative Colitis (UC) for at least 12 weeks before starting the study, with a report confirming inflammation in the colon.
  • If diagnosed with UC before age 6, must have tested negative for specific genetic disorders linked to early onset inflammatory bowel disease.
  • Must have moderately to severely active UC, shown by a Mayo score of 6 or higher, a rectal bleeding score of 1 or higher, and an endoscopic subscore of 2 or higher. An endoscopy must be done within 14 days before starting the study.
  • Must have a Pediatric Ulcerative Colitis Activity Index (PUCAI) score of 35 or higher at the start of the study.
  • Must not have a history of abnormal cell growth (dysplasia) or colon cancer.
  • Must not have untreated or inadequately treated active or hidden infection with Mycobacterium tuberculosis (TB).
  • For participants in the US and the EU, must have had a poor response or intolerance to TNF inhibitors.
  • For participants outside the US or the EU, must have had a poor response or intolerance to at least one prior therapy, such as oral or intravenous corticosteroids, azathioprine or 6-MP, TNF inhibitors, or anti-integrin therapy.
  • If taking certain medications for UC, such as oral 5-Aminosalicyclic acids (ASA) or sulfasalazine, or oral corticosteroids, doses must be stable for a specified time before starting the study.
  • Must be willing and able to attend scheduled visits, follow the treatment plan, and undergo tests and procedures required by the study.
  • No contraception methods are required for male participants. Female participants must not be pregnant or breastfeeding and must use effective birth control if they are of childbearing potential.
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Investigated drugs

Tofacitinib is a medication being studied for its effectiveness in treating children with moderately to severely active ulcerative colitis. It is taken orally and works by reducing inflammation in the body, which can help manage the symptoms of ulcerative colitis. The goal of the trial is to see if tofacitinib can help achieve remission, meaning a significant reduction or disappearance of symptoms, in pediatric patients.

What is already known about the treatment

Tofacitinib – Tofacitinib is administered orally in tablet form. It is currently being studied in clinical trials for its effectiveness in treating children with moderately to severely active ulcerative colitis. The medication works by inhibiting certain enzymes known as Janus kinases, which play a role in the inflammatory process. Tofacitinib is classified as a Janus kinase inhibitor and is being evaluated for its ability to induce remission in pediatric patients with this condition.

Investigated diseases

Ulcerative Colitis – Ulcerative Colitis is a chronic inflammatory condition affecting the colon and rectum. It is characterized by inflammation and ulceration of the innermost lining of the large intestine, leading to symptoms such as abdominal pain, diarrhea, and rectal bleeding. The disease often progresses with periods of exacerbation and remission, where symptoms may worsen and then improve. Over time, the inflammation can lead to complications such as strictures or increased risk of colon cancer. The exact cause of Ulcerative Colitis is not fully understood, but it is believed to involve an abnormal immune response in genetically predisposed individuals. Environmental factors and gut microbiota may also play a role in its development and progression.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2023-509694-22-00Protocol codeA3921210Estimated enrolment114 patientsSponsorPfizer Inc.

sourced from the EU Clinical Trials Register and site verification

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).