Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Darvadstrocel in the Treatment of Complex Perianal Fistula in Pediatric Subjects with Crohn’s Disease over a Period of 24 Weeks and an Extended Follow-up Period for a Total of up to 52 Weeks

This research study is for children and teenagers who have Crohn's disease and are experiencing complex perianal fistulas. These areChannels that can form near the bottom, often causing discomfort and drainage. The study is testing a new treatment called Darvadstrocel, which is given as an injection. We want to find out if this treatment is effective in healing these fistulas and if it is safe to use. Researchers will follow participants for up to a year, carefully checking for improvements in their fistulas and any side effects. This is a Phase 3 study, meaning it's a key step to see if the treatment could become more widely available.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Takeda Development Center Americas Inc.

Enrolment target

Start

07 Jul 2023

Complex Perianal Fistulas in Crohn's Disease

What is this study about?

This study, called a Phase 3 trial, is about finding better ways to treat a specific problem that some children and teenagers with Crohn's disease face: complex perianal fistulas. Think of fistulas as small, unusual tunnels that can form around the bottom area. For people with Crohn's disease, these can be particularly tricky, causing pain, leakage, and infection. Currently, these can be very challenging to treat effectively.

The main goal of this study is to test a new treatment called Darvadstrocel. It's a special type of cell therapy given by injection. Researchers are trying to see if this treatment can help these complex fistulas heal fully and stay closed. They'll also be looking very carefully to make sure the treatment is safe for young people.

Over the course of the study, which lasts about a year, doctors will regularly check the fistulas. They'll use different methods, including looking at them and sometimes using MRI scans (a special type of scan that takes detailed pictures inside the body), to see if the fistulas are closing up and if any pockets of infection (abscesses) are clearing. This will help them understand how well the treatment is working and if it leads to lasting relief for young patients.

Key takeaways

New treatment (Darvadstrocel) for complex perianal fistulas in young people with Crohn's.
Tests if the treatment helps heal fistulas and is safe.
Study lasts up to one year with regular check-ups.
Involves injections and MRI scans.
Aims for complete healing and no internal infection.

Who may be eligible?

This study is specifically looking for children and teenagers who have Crohn's disease and have complex perianal fistulas that are causing drainage. You would need to have specific types of these fistulas that meet the study's requirements.

There aren't any strict age limits mentioned beyond being a child or teenager, and the study is open to both boys and girls. The medical team would need to review your full health history to ensure you meet all the criteria and that the study would be safe and appropriate for you.

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.

I have Crohn's disease.
I am a child or teenager.
I have complex perianal fistulas that are draining.
I am willing to have injections and MRI scans.

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive the study treatment, which is an injection called Darvadstrocel. You would have regular visits to the clinic over a period of 24 weeks (about six months), where doctors would check your progress and overall health. They will look at your fistulas to see if they are healing and assess for any side effects.

After the initial 24 weeks, you would continue to be followed up for an extended period, making the total study time up to 52 weeks (about one year). During this extended follow-up, there would be further check-ups to see if the healing is lasting and to monitor your health. These visits might include physical examinations and possibly MRI scans to get detailed pictures of the fistulas. The exact number and frequency of visits would be explained fully by the study team.

Potential risks and benefits

Taking part in a study like this might offer the potential benefit of trying a new treatment that could help heal your perianal fistulas, which can be very difficult to treat otherwise. However, as with any new medicine, there could be risks of side effects, which might be mild or more serious. The study team will explain all known or potential risks in detail. It's important to remember that participating is entirely your choice, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

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

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Unverified
Spain
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Unverified
Netherlands
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Unverified
Poland

Common questions

What are 'complex perianal fistulas'?

These are unusual tunnels or channels that form around the bottom area, often causing drainage, pain, and sometimes infection in people with Crohn's disease.

What is Darvadstrocel?

Darvadstrocel is a new type of cell treatment given by injection, being tested to see if it can help these fistulas heal.

How long does the study last?

The study lasts for up to one year, with regular check-ups during this time.

Will I have to have any scans?

Yes, MRI scans will be used to get detailed pictures of the fistulas and see if they are healing inside.

What does 'combined remission' mean in this study?

It means the fistulas have completely closed on the outside, and detailed scans show no significant pockets of infection (abscesses) inside.