Ongoing, recruitingPhase III and phase IV (Integrated)Interventional

A Phase 3b/4, Interventional, Multicenter, Open-Label, Single-Arm Study to Assess Behavioral and Other Co-occurring Outcomes Following Treatment With EPID(I/Y)OLEX as Add-on Therapy in Participants (Aged 1 to 65 Years Old) With Seizures Associated With Tuberous Sclerosis Complex (EpiCom)

This study is investigating how a medicine called Epidyolex, when added to existing treatments, might help with behaviour problems and other difficulties frequently seen in children, teenagers, and young adults (ages 1 to 65) who have seizures due to Tuberous Sclerosis Complex (TSC). It aims to understand if the treatment can improve these issues over time. Researchers will check things like changes in behaviour, sleep, and overall quality of life at different points over a year. The study also monitors how safe the medicine is and how many seizures participants have. This is an open-label study, meaning both participants and doctors will know they are receiving Epidyolex.

At a glance

Status

Ongoing, recruiting

Phase

Phase III and phase IV (Integrated)

Sponsor

Jazz Pharmaceuticals Research UK Limited, Jazz Pharmaceuticals Research UK Limited, Jazz Pharmaceuticals Research UK Limited

Enrolment target

Start

06 May 2024

Tuberous Scerlosis Complex

What is this study about?

This study, sometimes called EpiCom, is looking into a medicine named Epidyolex. It's for children and young people between 1 and 65 years old who have seizures because of a condition called Tuberous Sclerosis Complex (TSC). The main goal is to see if taking Epidyolex, alongside their usual seizure medicines, can help with behaviour problems and other challenges that often come with TSC, such as difficulties with sleep, attention, or social interactions.

Researchers will observe participants over a full year to track how their behaviour changes, especially looking at the most troublesome behaviours they experience. They will also collect information on other aspects of life, like how well they sleep, their general mood, and their overall quality of life. This helps paint a complete picture of the medicine's effects.

The study is exploring both how effective Epidyolex is and how safe it is to use. This kind of research is important because it helps doctors and patients understand more about how different treatments can improve the lives of people with TSC. By gathering detailed information from many participants, the study aims to provide valuable insights for the wider medical community.

Key takeaways

A study looking at Epidyolex for Tuberous Sclerosis Complex (TSC).
Focuses on improving behaviour and other issues, not just seizures.
For individuals aged 1 to 65 with TSC and seizures.
Epidyolex is given as an 'add-on' to existing treatments.
Involves regular check-ups over about a year to monitor progress and safety.

Who may be eligible?

This study is for both male and female participants aged between 1 and 65 years old.

To be considered for this study, you or your child must have been diagnosed with Tuberous Sclerosis Complex (TSC) and be experiencing seizures.

There may be other specific medical details that determine if someone can join, which the study doctor will discuss with you. These details ensure the study is safe and appropriate for everyone involved.

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 or your child between 1 and 65 years old?
Do you or your child have a diagnosis of Tuberous Sclerosis Complex (TSC)?
Do you or your child experience seizures?
Are you willing to take an additional medicine (Epidyolex) if currently on seizure medication?

Answer every question to see your result.

What does participation involve?

If you or your child decide to take part, you will receive the study medicine, Epidyolex, as an add-on to any current seizure medications. You and your doctor will know what medicine is being given. There will be several visits over a year where doctors will check your progress. These visits will involve discussions about behaviour, sleep, mood, and overall well-being. Doctors will use special questionnaires and assessments to track changes over time. They will also monitor how many seizures you or your child have and any side effects. The total duration of active participation involves regular check-ups over 52 weeks (about a year).

Potential risks and benefits

Taking part in this study could offer several potential benefits, such as a chance to improve behaviour issues, sleep, or overall quality of life related to Tuberous Sclerosis Complex. It also contributes to our understanding of Epidyolex for TSC. As with any medicine, there's always a possibility of side effects, which the study team will monitor closely. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

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

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Unverified
Poland

Common questions

What is Tuberous Sclerosis Complex (TSC)?

TSC is a rare genetic condition that can cause non-cancerous tumours to grow in different parts of the body, including the brain, which can lead to seizures and other health problems.

What is Epidyolex?

Epidyolex is a medicine given as an oral solution (liquid by mouth). It's used to help control certain types of seizures.

What does 'add-on therapy' mean?

It means Epidyolex will be given in addition to any other seizure medicines you or your child are already taking, not as a replacement.

Why are they studying behaviour?

Behavioural difficulties are common in people with TSC. This study aims to see if Epidyolex can help improve these issues, making daily life better for individuals and their families.

Will I know if I'm getting the actual medicine?

Yes, this is an 'open-label' study, which means both you/your child and the study doctors will know that Epidyolex is being given.