Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

An Open-label, Single-arm Study to Assess the Safety, Pharmacokinetics, and Efficacy of Adjunctive Cannabidiol Oral Solution (GWP42003‑P) in Participants with Tuberous Sclerosis Complex (1 Month to < 2 Years of Age), Dravet Syndrome (1 Year to < 2 Years of Age), or Lennox-Gastaut Syndrome (1 Year to < 2 Years of Age) who Experience Inadequately‑controlled Seizures

Q: What is Epidyolex?

Epidyolex is a medicine being studied for certain types of severe epilepsy in young children, aiming to reduce seizures.

Q: What age group is this study for?

This study is for children aged between one month and under two years old.

Q: What are the main goals of the study?

The study aims to check the safety of Epidyolex, how it works in the body, and if it helps reduce seizures.

Q: Will my child stop their other epilepsy medicines?

This study is for children still having seizures despite other treatments, suggesting Epidyolex would be added to their current medicines, not replace them.

Q: Can I take my child out of the study if we change our mind?

Yes, you can withdraw your child from the study at any time without it affecting their usual medical care.

This study is investigating a medicine called Epidyolex (also known as GWP42003-P) in very young children, from one month to under two years old, who have specific types of severe epilepsy: Dravet Syndrome, Tuberous Sclerosis Complex, or Lennox-Gastaut Syndrome. These children are still having seizures despite other treatments. The main goals are to see how safe the medicine is, how it moves through the body, and if it can help reduce the number of seizures. This is a 'Phase III' study, which means it's one of the final steps before a medicine might be approved for wider use. Carers will help keep track of seizure diaries and children will have regular check-ups to monitor their health and development throughout the study.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Gw Research Limited, Jazz Pharmaceuticals Research UK Limited

Enrolment target

Start

29 Nov 2023

Dravet Syndrome Tuberous sclerosis complex Lennox-Gastaut Syndrome

What is this study about?

This clinical trial is looking at a medicine called Epidyolex (you might also see it referred to as GWP42003-P) for very young children. Specifically, it's for babies and toddlers between one month and under two years old who have certain types of severe epilepsy. These conditions are Dravet Syndrome, Tuberous Sclerosis Complex, and Lennox-Gastaut Syndrome. Children in this study are still experiencing seizures even though they're already on other seizure medicines.

The main purpose of this study is threefold: first, to check how safe Epidyolex is for these young children; second, to understand how the medicine is absorbed and processed by their bodies; and third, to see how well it works at reducing seizures. This is a 'Phase III' study, which means it's a large and important step in researching new medicines. It's designed to gather a lot of information on safety and effectiveness before a medicine can be considered for general use.

Researchers will closely monitor the children in the study. This includes keeping a close eye on any side effects, checking their overall health with physical exams, heart tests (ECGs), and blood tests. Parents or carers will play a crucial role by regularly recording seizures in a diary. There will also be special assessments of the children's development to ensure everything is being carefully observed.

Key takeaways

This study is for very young children (1 month to < 2 years) with specific severe epilepsies.
It tests a medicine called Epidyolex to see if it reduces seizures and is safe.
Parents/carers will keep seizure diaries to track treatment progress.
Children will have regular health checks, including physical exams, ECGs, and blood tests.
This is a Phase III study, meaning it's a late-stage trial before potential wider approval.
Your child's development will be assessed throughout the study.

Who may be eligible?

This study is specifically for very young children who are still having seizures despite other treatments. To be considered, a child must be diagnosed with one of three specific types of epilepsy: Dravet Syndrome, Tuberous Sclerosis Complex, or Lennox-Gastaut Syndrome.

The age range for children in this study is quite narrow: they must be at least one month old but not yet two years old when the study begins. Both boys and girls can take part. The research team will carefully review each child's medical history to make sure they meet all the necessary requirements to ensure the study is safe and effective 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.

Is your child between 1 month and under 2 years old?
Does your child have a diagnosis of Dravet Syndrome, Tuberous Sclerosis Complex, or Lennox-Gastaut Syndrome?
Is your child still experiencing seizures despite current treatments?
Are you able to commit to regular hospital visits and record seizure information in a diary?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they will receive the study medicine, Epidyolex, as an oral solution. You, as their parent or carer, will play a key role by keeping a detailed record of your child's seizures in a special diary. You'll record how many seizures they have, what they are like, and when they occur. This information will be collected frequently, starting at 12 weeks into the study and then every four weeks after that, right up until the end of treatment.

Throughout the study, your child will have regular check-ups. These will include physical examinations, having their vital signs (like heart rate and breathing) checked, and sometimes an ECG (a test for heart activity). Blood tests will also be done to check their overall health and how the medicine is affecting their body. The study will also involve special assessments of your child's development. This is to ensure that researchers can track any changes over time. Your child will be followed for a significant period to truly understand the effects of the medicine.

Potential risks and benefits

Taking part in any clinical trial involves potential benefits and risks. For your child, a potential benefit could be a reduction in their seizures, which might improve their quality of life. However, there's no guarantee the medicine will work, or that it will work for every child. As with all medicines, Epidyolex can have side effects, and these will be carefully monitored throughout the study. The research team will check your child's health closely with regular tests and exams. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (2)

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

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Unverified
Italy
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Unverified
Spain

Common questions

What is Epidyolex?

Epidyolex is a medicine being studied for certain types of severe epilepsy in young children, aiming to reduce seizures.

What age group is this study for?

This study is for children aged between one month and under two years old.

What are the main goals of the study?