Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

An Open-label, Non-randomized, Phase 3 Study to Evaluate the Efficacy and Safety of Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine.

This research study is looking at a new medicine called Soticlestat. It's for adults (aged 18 and over) who live with serious forms of epilepsy called Dravet Syndrome or Lennox-Gastaut Syndrome. These conditions often cause frequent and difficult-to-control seizures. The main goal is to find out if Soticlestat is effective at reducing the number of seizures people have, and to check if it's safe to use. Specifically, the study is interested in participants who have already tried another epilepsy medicine called Fenfluramine. This is a follow-up study, building on previous research, to gather more information on Soticlestat in real-world settings over a longer period.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Takeda Development Center Americas Inc.

Enrolment target

Start

14 Nov 2023

Dravet Syndrome/Lennox-Gastaut Syndrome

What is this study about?

This study is about an investigational medicine called Soticlestat. Investigational means it's still being tested and isn't yet available as a standard treatment. The goal is to see if Soticlestat can help reduce seizures in adults who have either Dravet Syndrome or Lennox-Gastaut Syndrome.

Dravet Syndrome and Lennox-Gastaut Syndrome are both types of severe epilepsy that can be very challenging to manage. They often lead to different kinds of seizures that can significantly impact daily life. This study is considered a 'Phase 3' study, which means it's one of the final stages of testing before a medicine might be considered for wider use. It's designed to confirm whether the medicine works well and is safe over a longer period, building on what was learned in earlier studies.

Researchers are particularly interested in people who have previously used a different epilepsy medicine called Fenfluramine. They want to understand how Soticlestat works in this group, including how it might compare or combine with previous treatments. The study aims to provide more information to doctors and patients about potential new options for managing these conditions.

Key takeaways

Tests new medicine, Soticlestat, for severe epilepsy.
For adults aged 18+ with Dravet or Lennox-Gastaut Syndrome.
Focuses on people who have used Fenfluramine previously.
Aims to check if Soticlestat reduces seizures and is safe.
Participation involves taking the medicine and regular checks.
You can stop participating at any time.

Who may be eligible?

This study is open to adults aged 18 and older. Both men and women are welcome to take part.

To be considered for this study, you must have a confirmed diagnosis of either Dravet Syndrome or Lennox-Gastaut Syndrome. An important part of the eligibility is that you should have previously taken Fenfluramine, another medicine used for epilepsy.

There will be other specific medical checks to make sure the study is a safe and suitable option for you. Your doctor will be able to discuss all the detailed requirements and help you understand if you might be eligible.

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 18 years old or older?
Have you been diagnosed with Dravet Syndrome or Lennox-Gastaut Syndrome?
Have you previously taken the medicine Fenfluramine for your epilepsy?
Are you generally willing and able to follow study procedures?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would first have a screening visit to see if you meet all the requirements. This would involve medical checks and discussions about your health history. If eligible, you would then begin taking the study medicine, Soticlestat. The main part of the study involves an initial 12-week period where researchers will carefully monitor how often you have seizures. You would need to keep track of your seizures, and there would be regular visits to the clinic for assessments, which might include physical examinations, blood tests, and discussions about any side effects you might experience. The total duration of your participation would be explained fully by the study team, and it would involve ongoing medication, follow-up appointments, and possibly additional tests.

Potential risks and benefits

Taking part in a clinical trial offers potential benefits, such as access to a new medicine that isn't widely available. It could also mean more frequent medical monitoring and expert care. However, there are potential risks, as Soticlestat is still under investigation. You might experience side effects, some of which may not be fully known yet, or it might not work effectively for you. It's important to remember that you can stop participating in the study at any time, for any reason, without affecting your current or future 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
Denmark

Common questions

What is Soticlestat?

Soticlestat is a new medicine being tested to see if it can help reduce seizures in people with certain types of severe epilepsy.

What are Dravet Syndrome and Lennox-Gastaut Syndrome?

These are two severe types of epilepsy that start in childhood and can cause frequent and various kinds of seizures, often difficult to control.

Why is this study looking at people who took Fenfluramine?

Researchers want to understand how Soticlestat works in people who have already tried another epilepsy medicine, Fenfluramine, to get more complete information.

Will I get the actual study medicine or a dummy pill?

This study is 'open-label', which means both you and your doctor will know you are receiving Soticlestat, not a dummy pill (placebo).

How long will the study last?

The initial period of close monitoring will be 12 weeks, but your full participation, including follow-up, will be explained by the study team.