Crohn's Disease: Efficacy, Safety, and Pharmacokinetics of Upadacitinib in Pediatric Subjects With Moderately to Severely Active Crohn's Disease
This study is looking at a medicine called Upadacitinib (also known as RINVOQ) for children and teenagers aged 2 to 17 who have Crohn's disease. Crohn's disease causes irritation and swelling in the gut, leading to symptoms like tummy pain and tiredness. We're focusing on those whose current treatments, like steroids or other strong medicines, haven't worked well enough or caused problems. Upadacitinib is already approved for adults with Crohn's disease, and this study aims to find out if it's safe and effective for younger people. Participants will take the medicine daily, and doctors will closely monitor their health and progress over several years. The goal is to see if this medicine can help manage their Crohn's symptoms better.
Överblick
Vad handlar denna studie om?
This study is all about helping young people who have Crohn's disease. Crohn's is a condition where parts of the digestive system become inflamed, which means they get red, swollen, and sore. This can lead to tummy aches, diarrhoea, feeling very tired, and losing weight. For many young people with Crohn's, their current treatments might not be working as well as hoped, or they might have problems with side effects.
Researchers are investigating a medicine called Upadacitinib, which is already used for adults with Crohn's disease under the brand name RINVOQ. They want to find out if it can also safely and effectively help children and teenagers between 2 and 17 years old who have moderate to severe Crohn's. The study will carefully check how well the medicine works to reduce symptoms and improve their overall health, as well as if there are any side effects.
Taking part in a study like this is very important because it helps doctors learn more about new ways to treat conditions. If Upadacitinib proves to be safe and effective for younger people, it could offer a new treatment option for those struggling to manage their Crohn's disease, giving them a better quality of life.
Viktiga punkter
- Tests a new medicine (Upadacitinib) for children and teenagers with Crohn's.
- Aimed at those whose existing treatments aren't effective.
- Participants will take the medicine daily, some openly, some blinded.
- Requires regular clinic visits for health checks and tests.
- Study duration can be several years, with close monitoring.
- Potential for symptom improvement, but also risks of side effects.
Vem kan vara berättigad?
This study is looking for children and teenagers aged 2 to 17 years old who have Crohn's disease that is moderately to severely active. This means their Crohn's is causing significant problems, and previous treatments like steroids, other strong medicines, or biologic therapies haven't worked well enough, have stopped working, or caused bad side effects. You must weigh at least 10 kg (about 22 pounds) to join.
To be considered, your child's Crohn's disease must be confirmed by a colonoscopy (an examination of the bowel) and show signs of inflammation. We need to make sure there are no other infections, growths, or serious complications of Crohn's that would prevent participation.
There are also some reasons why someone might not be able to join. For example, if they were diagnosed with Crohn's before they were 2 years old, or if they have certain complications like active abscesses (pus-filled pockets), severe narrowing of the bowel, or have had recent bowel surgery. Also, if they have other types of inflammatory bowel disease like ulcerative colitis, they wouldn't be able to participate in this study.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- Is my child between 2 and 17 years old?
- Does my child have moderate to severe Crohn's disease?
- Have my child's current Crohn's treatments not worked well enough?
- Does my child weigh at least 10 kg (about 22 pounds)?
- Has my child NOT had certain recent bowel surgeries or complications?
- Does my child have a confirmed diagnosis of Crohn's, not other bowel conditions?
Vad innebär deltagande?
If you join this study, your child will take Upadacitinib as a tablet once a day or a liquid medicine twice a day, usually around the same time each day, with or without food. The study is divided into two main parts. For the first 12 weeks, everyone knows they are getting an active dose of the medicine. After that, for about a year, it's a 'blinded' phase, meaning neither you nor the study doctors will know which dose of the medicine your child is receiving. This helps make the study results fair and unbiased. There's also an optional long-term extension of up to three more years where everyone knows they are getting the medicine.
Throughout the study, your child will have regular visits to a hospital or clinic, which could be weekly or monthly, especially at the beginning. During these visits, the medical team will check their health, take blood samples, and ask about any side effects. Your child will also complete questionnaires to help understand how they are feeling. These visits help the doctors monitor how the medicine is working and keep your child safe. After stopping the study medicine, there will be one final safety check approximately 30 days later. The total time a child could be in the study is several years if they participate in all phases.
Potentiella risker och fördelar
Platser (85)
- UCSF Benioff Children's Hospital - Oakland /ID# 262217Verifierat postnummerOakland, USA
- Lucile Packard Children's Hospital /ID# 262193Verifierat postnummerPalo Alto, USA
- Children's Hospital Colorado - Aurora /ID# 262207Verifierat postnummerAurora, USA
- Connecticut Children's Medical Center - Hartford /ID# 262256Verifierat postnummerHartford, USA
- OSF St. Francis Medical Center /ID# 262192Verifierat postnummerPeoria, USA
- Indiana University Health Riley Hospital for Children /ID# 262215Verifierat postnummerIndianapolis, USA
- Boston Children's Hospital /ID# 262191Verifierat postnummerBoston, USA
- MNGI Digestive Health, P. A. /ID# 262204Verifierat postnummerMinneapolis, USA
- Icahn School of Medicine at Mount Sinai /ID# 262216Verifierat postnummerNew York, USA
- Univ NC Chapel Hill /ID# 262198Verifierat postnummerChapel Hill, USA
- UH Cleveland Medical Center /ID# 262188Verifierat postnummerCleveland, USA
- Children's Hospital of Philadelphia - Main /ID# 262197Verifierat postnummerPhiladelphia, USA
Vanliga frågor
Vad är Crohns sjukdom?
Crohn's disease is a condition where parts of your digestive system become inflamed (red, swollen, and sore), causing symptoms like tummy pain, diarrhoea, and tiredness.
What is Upadacitinib?
Upadacitinib (also known as RINVOQ) is a medicine being studied to see if it can help reduce inflammation and symptoms in young people with Crohn's disease.
Vem kan delta i denna studie?
This study is for children and teenagers aged 2 to 17 years old with moderate to severe Crohn's disease whose current treatments aren't working well.
What does taking part involve?
It involves taking medicine daily, having regular check-ups at a hospital or clinic, blood tests, and filling out questionnaires over several years.
Can I stop participating anytime?
Yes, you or your child can leave the study at any point, and it won't affect their regular medical care.
Hur du får reda på mer
Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.
Diskussion
Gemenskapsdiskussion
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