Study of Tofacitinib for Children with Moderate to Severe Ulcerative Colitis

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What is this study about?

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.

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 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 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 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 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 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 Study?

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.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Hopital Necker Enfants Malades	Paris	France
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy
Uniwersytecki Szpital Kliniczny Im Jana Mikulicza Radeckiego We Wroclawiu	Wroclaw	Poland
Katholieke Universiteit te Leuven	Leuven	Belgium

Other Sites

Site Name	City	Country
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Instytut Centrum Zdrowia Matki Polki	Lodz	Poland
Dr. von Haunersches Kinderspital, LMU	Munich	Germany
University Of Debrecen	Debrecen	Hungary
Soedersjukhuset AB	Stockholm	Sweden
Instytut Pomnik Centrum Zdrowia Dziecka	Warsaw	Poland
Pirkanmaan hyvinvointialue	Tampere	Finland
Centre Hospitalier Lyon Sud	Pierre Benite	France
Vrije Universiteit Brussel	Jette	Belgium
University Childrens Hospital Queen Fabiola	Brussels	Belgium
University Of Szeged	Szeged	Hungary
MD Korczowski Bartosz Gabinet Lekarski	Rzeszow	Poland
Sahlgrenska Universitetssjukhuset/Östra, Prövningsenheten barn	Gothenburg	Sweden
Ccifcuekj Uumzgbineetjzt Sgvgxvhcn	Woluwe-Saint-Lambert	Belgium
Kxcvdxzvcm Urnvxbkbmtgojefkipwyt Bhdlotvtxloywszgxpeqh hpxqwqmqxh och nxgxgugks	Stockholm	Sweden
Aggilbwou Uqs	Amsterdam	The Netherlands
Evexafu Ununogjkxghi Mpwsvkx Cdvzdcy Rgixuvbkf (suftscf Mow	Rotterdam	The Netherlands
Awogmms Otbaalyghks Pjad Gwwqvlpa Xxben	Bergamo	Italy
Ugcxkyrtlx Dgcwj Scssd Dg Rnem Lx Skebkcks	Rome	Italy
Wgf Wboczc Ibr Pazng Pqnmzjur Kegpuyw	Warsaw	Poland

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	18.06.2021
Finland	Not recruiting	18.06.2021
France	Not recruiting	18.06.2021
Germany	Not recruiting	18.06.2021
Hungary	Not recruiting	18.06.2021
Italy	Not recruiting	18.06.2021
Poland	Not recruiting	18.06.2021
Spain	Not recruiting	18.06.2021
Sweden	Not recruiting	18.06.2021
The Netherlands	Not recruiting	18.06.2021

Trial locations

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.

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 ID:

2023-509694-22-00

Protocol code:

A3921210

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study of Tofacitinib for Children with Moderate to Severe Ulcerative Colitis

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