Ongoing, recruitingPhase II and Phase III (Integrated)Interventional

A Phase 2/3, Multicenter, Randomized, Double-blind Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Oral Ozanimod (RPC1063) in Pediatric Participants with Moderately to Severely Active Crohn’s Disease with an Inadequate Response to Conventional Therapy

Doctors are running a study to test a new medicine called Zeposia for children and young people who have moderately to severely active Crohn's disease. This is for those whose condition hasn't improved enough with their current treatments. The main goal is to find out if Zeposia is effective in reducing Crohn's symptoms and helping the gut heal. They also want to understand how safe it is and how the body uses and processes the medicine. This is an important step in finding better ways to manage Crohn's disease in younger patients, potentially offering a new treatment option for those who need it most.

At a glance

Status

Ongoing, recruiting

Phase

Phase II and Phase III (Integrated)

Sponsor

Celgene International II S.a.r.l.

Enrolment target

Start

01 Feb 2024

Moderately to Severely Active Crohn's Disease

What is this study about?

Imagine your child has Crohn's disease, and despite trying different medicines, they're still really struggling with symptoms. That's a tough situation, and this study aims to explore a new option called Zeposia. Crohn's disease is a long-term condition where parts of the digestive system become inflamed and swollen, causing pain, diarrhoea, and other issues. In children, it can also affect their growth and development.

This research project is designed to carefully look at how well Zeposia works and if it's safe for children and young people with Crohn's. The 'Phase 2/3' part means it's a combined study. Phase 2 usually checks if a medicine has an effect and helps find the right dose, while Phase 3 compares it against existing treatments or a dummy pill to confirm its effectiveness over a larger group. In this study, some participants will receive a dummy pill (placebo) or a different dose of Zeposia, but no one involved in the study – neither the patient nor the doctor – will know who is getting what until the study ends. This helps ensure the results are fair and accurate.

The main things the researchers want to find out are whether Zeposia can significantly reduce Crohn's symptoms after about a year, and if it can help the gut lining to look healthier when examined. They will be using specific scoring systems to measure these improvements in a clear and consistent way. They'll also check if these improvements are seen sooner, after just a few months. This type of research is crucial because it helps doctors understand if a new medicine can really make a difference and provide relief for young patients living with Crohn's disease.

Key takeaways

This study is testing a new medicine, Zeposia, for children and young people with Crohn's disease.
It's for those whose Crohn's is still active despite other treatments.
Participants will receive Zeposia or a dummy pill, with neither patient nor doctor knowing which.
The study aims to see if Zeposia reduces symptoms and helps the gut heal.
Close medical monitoring will be provided throughout the study.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is specifically looking for children and young people who have been diagnosed with Crohn's disease. Your child's Crohn's needs to be considered 'moderately to severely active,' meaning they are still experiencing significant symptoms even with their current treatments.

Crucially, your child must have tried conventional treatments for Crohn's disease, but these haven't worked well enough for them. This means their condition hasn't improved as much as doctors would hope, or they haven't been able to tolerate the side effects of those treatments.

While the study doesn't have specific age limits mentioned, it's designed for 'pediatric participants,' which generally means children and adolescents. Both boys and girls are welcome 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.

Does my child have a confirmed diagnosis of Crohn's disease?
Is my child's Crohn's disease still active, even with current treatments?
Has my child tried other standard Crohn's treatments that haven't worked well enough?
Is my child a 'pediatric participant' (child or young person)?
Is my child able to take capsules by mouth?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they would be given either one of three different doses of Zeposia capsules (0.23 mg, 0.46 mg, or 0.92 mg) or a dummy capsule (placebo). The study is 'double-blind,' meaning neither you, your child, nor the study doctors will know which treatment your child is receiving. This is to ensure the results are as unbiased as possible.

Your child would need to attend regular clinic visits over a period of time, where doctors will check their symptoms, health, and how they are responding to the treatment. These visits will involve various tests, which might include blood tests, stool (poo) samples, and possibly scans or procedures to look at their gut. The total duration of the study and how often visits are needed would be fully explained by the study team.

Throughout the study, your child will be carefully monitored for any side effects or changes in their health. You will have regular contact with the study team to discuss any concerns or questions. At the end of the treatment period, there will be follow-up assessments to see how your child is doing.

Potential risks and benefits

Taking part in a clinical study could offer potential benefits, such as access to a new medicine that isn't widely available yet, which might help your child's Crohn's disease improve if other treatments haven't worked. Your child would also receive very close medical supervision and care throughout the study, which can be reassuring. However, there are potential risks, including side effects from the study medicine, which might not be fully known, or the possibility that the medicine doesn't work for your child. Some participants might also receive a dummy pill that offers no direct treatment benefit. It's important to remember that participation is always voluntary, and you have the right to withdraw your child from the study at any time, for any reason, without it affecting their future medical care.

Locations (6)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
France
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Unverified
Poland
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Unverified
Spain
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Unverified
Germany
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Unverified
Hungary
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Unverified
Belgium

Common questions

What is Zeposia?

Zeposia is a new medicine being tested in this study to see if it can help improve Crohn's disease symptoms in children and young people.

Will my child definitely get the new medicine?

No, this is a 'blinded' study. Your child will either receive Zeposia at one of three doses or a dummy capsule (placebo). Neither you nor the doctors will know which one.

How long will the study last?

The main part of the study measures outcomes for over a year (64 weeks). The full duration and number of visits will be explained by the study team.

Can we stop participating if we want to?

Yes, you can withdraw your child from the study at any time, for any reason, without affecting their future medical care.

What kind of tests will my child have?

Your child will have regular check-ups, which may include blood tests, stool samples, and possibly other procedures to monitor their Crohn's disease and how they're responding to the treatment.