Study to Assess Adverse Events, Change in Disease Activity, and How Oral Upadacitinib Moves Through the Body of Pediatric Participants With Moderately to Severely Active Ulcerative Colitis.
This important study is investigating a medicine called Upadacitinib (also known as RINVOQ) for children and young people aged 2 to 17 who have moderate to severe ulcerative colitis. Ulcerative colitis is a long-term condition that causes inflammation and bleeding in the large intestine. Upadacitinib is already approved for adults with this condition, and this study aims to find out if it is safe and works well for younger people. Researchers will be looking at any side effects and how much the medicine improves their symptoms. Participants will take the medicine daily and have regular check-ups at a hospital or clinic over several years to see how they are doing.
At a glance
What is this study about?
This study is about a medicine called Upadacitinib, which also goes by the brand name RINVOQ. It's being tested for children and teenagers aged 2 to 17 who have a condition called ulcerative colitis. Ulcerative colitis is a type of inflammatory bowel disease where the lining of the large bowel (your colon and rectum) becomes inflamed, which can cause symptoms like tummy pain, bloody stools, and tiredness. For many young people with moderate to severe ulcerative colitis, current treatments might not always work well, or they might experience side effects.
Upadacitinib is already a treatment option for adults with ulcerative colitis, and now doctors want to see if it can be a safe and effective option for younger patients too. The main goals of this study are to understand if Upadacitinib helps to improve symptoms of ulcerative colitis in children, if it's safe to use, and how it moves through their bodies. Finding new and better ways to manage ulcerative colitis in children and young people is really important to help them live healthier, more comfortable lives.
Roughly 110 children and young people with moderate to severe ulcerative colitis will take part in this study at various locations worldwide. They will be carefully monitored by doctors and nurses to make sure the treatment is safe and to see how well it's working. The study is designed to gather important information that could help Upadacitinib become a treatment option for children in the future.
Key takeaways
- This study is for children and young people (2-17) with moderate to severe ulcerative colitis.
- It's testing a medicine called Upadacitinib, already used in adults.
- The main goals are to check the medicine's safety and how well it reduces symptoms.
- Participation involves taking daily medicine and regular hospital visits for several years.
- Some parts of the study are 'blinded', meaning you might not know your exact dose.
- Your medical team will monitor you closely, and you can leave the study at any time.
Who may be eligible?
To be considered for this study, children and young people need to have ulcerative colitis that is currently active and moderate to severe. This means their doctors have assessed their condition and found it to be flaring up or causing significant problems. They also need to have tried other treatments for their ulcerative colitis, like certain medicines (corticosteroids, immunosuppressants, or biologic therapies), and found that these treatments either didn't work well enough, stopped working, caused difficult side effects, or couldn't be used for other medical reasons.
However, some individuals would not be able to join. For example, if they have previously taken medicines that work in a similar way to Upadacitinib (called JAK inhibitors, such as tofacitinib or baricitinib), they wouldn't be able to participate. Also, females who are pregnant, breastfeeding, or planning to become pregnant during the study or shortly after would not be eligible to take part.
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.
- Are you between 2 and 17 years old?
- Do you have moderate to severe ulcerative colitis that is currently active?
- Have other treatments for your ulcerative colitis either not worked well, stopped working, or caused difficult side effects?
- Have you never taken medicines similar to Upadacitinib (called JAK inhibitors) before?
- If female, are you not pregnant, not breastfeeding, and not planning to become pregnant during or shortly after the study?
What does participation involve?
If you join this study, you would take Upadacitinib medicine every day. This will either be a tablet that you swallow once a day, or a liquid medicine twice a day, at about the same time, with or without food. The study will last for several years. Initially, for the first 8 weeks, you will know you are receiving Upadacitinib at a specific dose. After that, for 44 weeks, neither you nor your doctor will know if you are receiving one of two different doses of Upadacitinib. Following this, there will be a longer period of up to 260 weeks (about five years) where you will again know you are receiving Upadacitinib.
During the study, you'll need to go to a hospital or clinic regularly for check-ups. These visits will involve medical examinations, blood tests, and discussions about any side effects you might be experiencing. You'll also be asked to complete questionnaires about your well-being and how your ulcerative colitis is affecting you. If you decide to stop the study at certain points, or naturally finish a stage, you'll have a follow-up check about 30 days later to ensure everything is okay.
Potential risks and benefits
Locations (89)
- Phoenix Children's Hospital /ID# 250135Verified postcodePhoenix, United States
- Arkansas Children's Hospital /ID# 250106Verified postcodeLittle Rock, United States
- Kindred Medical Institute - Corona /ID# 255484Verified postcodeCorona, United States
- UCSF Benioff Children's Hospital - Oakland /ID# 255067Verified postcodeOakland, United States
- Lucile Packard Children's Hospital /ID# 258430Verified postcodePalo Alto, United States
- Children's Hospital Colorado - Aurora /ID# 250110Verified postcodeAurora, United States
- Nemours/Alfred duPont Hospital for Children /ID# 255483Verified postcodeWilmington, United States
- Childrens Healthcare of Atlanta - Center for Advanced Pediatrics /ID# 255069Verified postcodeAtlanta, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago /ID# 254505Verified postcodeChicago, United States
- OSF St. Francis Medical Center /ID# 256968Verified postcodePeoria, United States
- Massachusetts General Hospital /ID# 250142Verified postcodeBoston, United States
- Boston Children's Hospital /ID# 250108Verified postcodeBoston, United States
Common questions
What is ulcerative colitis?
Ulcerative colitis is a long-lasting condition where the lining of your large bowel (colon and rectum) becomes inflamed and can bleed, causing symptoms like tummy pain and urgent need to go to the toilet.
What is Upadacitinib?
Upadacitinib is a medicine that's already used for adults with moderate to severe ulcerative colitis, and this study wants to see if it can help children and young people too.
Who can join the study?
Children and teenagers aged 2 to 17 with active, moderate to severe ulcerative colitis who haven't responded well to other treatments might be eligible. There are also specific reasons why someone might not be able to join.
How long will the study last?
The study involves different periods, with the main treatment phase lasting about a year, followed by an optional longer period of up to five years, plus follow-up checks.
Will I know which dose of medicine I'm getting?
For the first 8 weeks, you'll know. After that, for 44 weeks, neither you nor your doctor will know which of two doses you are receiving. Then, for a longer period, you will know again.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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