Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative Colitis
This study, named A3921210, is for children and young people aged 2 to 17 who have moderate to severe ulcerative colitis, a long-term condition affecting the bowel. We want to find out how effective and safe a medicine called tofacitinib is for them. This medicine is given by mouth. The study is particularly for those who haven't responded well to other treatments or couldn't tolerate them. We'll be checking if the medicine helps put the condition into remission, meaning symptoms are much improved or gone. Participants will take the medicine and be closely monitored over a period, with visits to see how they're doing and to check for any side effects. The total study could last up to 180 weeks.
At a glance
What is this study about?
This study is looking into a medication called tofacitinib for children and young people between the ages of 2 and 17 who have a condition called ulcerative colitis (UC). UC is a long-term illness where the lining of the large bowel (colon) becomes inflamed and develops ulcers, causing symptoms like tummy pain and bloody diarrhoea. Researchers want to understand if this medication, which is taken by mouth, can help these children feel better and reduce their UC symptoms.
The main goal of this study is to see how well tofacitinib works in bringing UC into remission. Remission means that the symptoms of UC are either gone or very mild, and the bowel inflammation has improved significantly. We also want to make sure the medicine is safe and to understand how the body processes it in children. This study is especially for children who haven't gotten better with other common treatments for UC, or who couldn't use those treatments because of side effects.
Participants will start on a specific dose of tofacitinib, and if needed, their doctor might be able to increase the dose slightly. They will be carefully monitored throughout the study to track their UC symptoms and check for any health changes. This clinical trial is important because it could help doctors find effective new ways to manage ulcerative colitis in young people, improving their quality of life.
Key takeaways
- This study is testing a medicine called tofacitinib for children and teens (2-17) with moderate to severe ulcerative colitis.
- It aims to see how well tofacitinib works to get symptoms into remission and how safe it is.
- This is for children whose UC hasn't responded to previous treatments.
- Participation involves taking the medicine and regular clinic visits over several years.
- Your child will be closely monitored for any changes or side effects.
Who may be eligible?
This study is for children and young people aged between 2 and 17 years old who have been diagnosed with moderate to severe ulcerative colitis for at least 12 weeks. They must weigh at least 10 kg. Your child will need to have clear signs of moderate to severe ulcerative colitis, as shown by specific medical scores and a colonoscopy.
For children over 6 years old, they are eligible if their previous treatments for UC, such as certain steroids, azathioprine, 6-mercaptopurine, or a type of medicine called TNF inhibitors, haven't worked well or caused too many side effects. In the UK, USA, and EU, the main focus is on those who haven't responded to TNF inhibitors.
There are also some reasons why a child might not be able to join. For example, if they have a different type of bowel condition, a history of colon cancer, or certain untreated infections like tuberculosis. Female participants must not be pregnant or breastfeeding, and if they could become pregnant, they must be using effective contraception.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child aged between 2 and 17 years old?
- Does your child weigh at least 10 kg?
- Has your child been diagnosed with moderate to severe ulcerative colitis for at least 12 weeks?
- Have your child's previous ulcerative colitis treatments (like certain steroids or TNF inhibitors) not worked well or caused bad side effects?
- If your child is female, are they not pregnant or breastfeeding, and if they could get pregnant, are they using effective birth control?
What does participation involve?
If your child joins the study, they will first go through a screening period of up to 4 weeks to make sure they meet all the requirements. After that, they will start taking the study medication, tofacitinib, which is given as a tablet. Initially, they'll take a set dose, but their doctor might adjust it if needed.
The study involves several phases: an initial 8 to 16-week 'induction' phase to get the condition under control, followed by a 44-week 'maintenance' phase to keep it in remission. There's also an optional longer 'extension' phase that could last up to 24 months. Throughout this time, your child will have regular visits to the clinic for check-ups, assessments, and to discuss how they're feeling and any side effects.
After your child stops taking the study medicine, there will be a follow-up visit about 4 weeks later and a phone call 8 weeks after that. These are important for checking on their health and making sure any potential side effects are monitored. The maximum total time your child could be involved in the study is about 180 weeks (a little over 3 years).
Potential risks and benefits
Locations (68)
- Children's Hospital Los AngelesVerified postcodeLos Angeles, United States
- University of California, San Francisco Benioff Children's HospitalVerified postcodeSan Francisco, United States
- University of California, San Francisco Pediatric Clinical Research Center (PCRC)Verified postcodeSan Francisco, United States
- Connecticut Children's Ambulatory Surgical CenterVerified postcodeFarmington, United States
- Connecticut Children's Infusion CenterVerified postcodeFarmington, United States
- Connecticut Children's Medical CenterVerified postcodeHartford, United States
- Nicklaus Children's HospitalVerified postcodeMiami, United States
- Center for Advanced PediatricsVerified postcodeAtlanta, United States
- Children's Healthcare of Atlanta - Arthur M. Blank HospitalVerified postcodeAtlanta, United States
- Boston Children's HospitalVerified postcodeBoston, United States
- Northwell Health - Cohen Children's Medical CenterVerified postcodeLake Success, United States
- Northwell Health - Cohen Children's Medical CenterVerified postcodeNew Hyde Park, United States
Common questions
What is ulcerative colitis?
Ulcerative colitis is a long-term condition where the lining of the large bowel (colon) becomes inflamed and develops ulcers, causing symptoms like tummy pain and bloody diarrhoea.
What is 'remission' in this study?
Remission means that the symptoms of ulcerative colitis are either gone or very mild, and the bowel inflammation has improved significantly.
What is tofacitinib?
Tofacitinib is a medicine taken by mouth that researchers are studying to see if it can help reduce inflammation and symptoms in children with ulcerative colitis.
Will my child have to stay overnight in hospital?
The study description doesn't mention overnight hospital stays. Most visits would likely be outpatient appointments to the clinic.
How long will my child be in the study?
Your child could be in the study for up to 180 weeks (about 3.5 years), depending on how long they stay in the different phases of the study.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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