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Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A phase 3, prospective, open-label, multisite, extension of phase 3 studies to assess the long-term safety and tolerability of soticlestat as adjunctive therapy in subjects with Dravet Syndrome or Lennox-Gastaut Syndrome (ENDYMION 2)

This study, called ENDYMION 2, is looking at a medicine called soticlestat for people who have either Dravet Syndrome or Lennox-Gastaut Syndrome. These are serious types of epilepsy. The main goal is to understand the long-term safety of soticlestat and how well people tolerate it over a longer period. Participants in this study will have already been part of an earlier study looking at soticlestat. Researchers will be checking for any side effects, how a person's general health is changing, and looking at things like blood tests and heart scans. They will also be monitoring seizure frequency and quality of life for those taking part.

At a glance

Status
Ongoing, recruiting
Phase
Therapeutic confirmatory (Phase III)
Sponsor
Takeda Development Center Americas Inc.
Enrolment target
127
Start
14 Feb 2024

What is this study about?

This study is called ENDYMION 2 and it's looking at a medicine called soticlestat. It's for people with two types of epilepsy: Dravet Syndrome and Lennox-Gastaut Syndrome. These conditions often cause severe seizures and other health challenges, and families are always looking for ways to manage them better.

This particular study is designed to follow up with people who have already been taking soticlestat in an earlier study. The main aim is to understand how safe the medicine is over a longer period and if people can continue to take it without too many problems. Researchers will be carefully checking for any side effects and how the medicine affects a person's general health, including things like their heart, blood tests, and even their mood. For children and young people, they will also be looking at growth and development.

While the main focus is on safety, the study will also track how often seizures happen and how severe they are. It will also look at how the medicine impacts a person's daily life and overall well-being. This information is really important because it helps doctors and patients understand the long-term effects of soticlestat and decide if it's a good treatment option.

Key takeaways

  • This study is for people with Dravet Syndrome or Lennox-Gastaut Syndrome who have previously taken soticlestat.
  • The main goal is to understand the long-term safety of soticlestat.
  • Researchers will closely monitor side effects, general health, and seizure activity.
  • It's an extended follow-up to gather more information over a longer period.
  • Participation involves regular clinic visits for various health checks and discussions.

Who may be eligible?

This study is designed for adults aged 18 and over, of any sex, who have either Dravet Syndrome or Lennox-Gastaut Syndrome. Importantly, you must have already taken part in a previous study looking at the medicine soticlestat.

Because this is an 'extension' study, it means it's a follow-up for people who have already started treatment with soticlestat. This helps researchers understand the longer-term effects of the medicine.

If you haven't been in one of the earlier soticlestat studies, or if you are under 18 years old, this particular study would not be suitable 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.

  1. I have Dravet Syndrome or Lennox-Gastaut Syndrome.
  2. I am 18 years old or older.
  3. I have taken part in a previous study involving the medicine soticlestat.
  4. I am generally able to attend regular clinic appointments.
  5. I am willing to undergo various health checks and give blood samples.
Answer every question to see your result.

What does participation involve?

Taking part in this study means you would continue to receive the medicine soticlestat, which you would already be familiar with from a previous study. You would have regular appointments at a study clinic. During these visits, the study team would carry out various assessments. This includes checking your general health, taking blood and urine samples, performing heart scans (ECGs), and checking your vital signs like blood pressure and pulse. They would also ask questions about your seizures and how you are feeling, including any side effects. For younger participants, growth and development would also be monitored. The study duration is long-term, as it's designed to gather information over an extended period.

Potential risks and benefits

Potential benefits of participating could include continued access to soticlestat, which might help manage your seizures, and close monitoring of your health by medical professionals. Potential risks might include experiencing side effects from the medicine, which will be carefully monitored, and the time commitment for regular study visits. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (10)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Unverified
    Latvia
  • Unverified
    Germany
  • Unverified
    Netherlands
  • Unverified
    France
  • Unverified
    Spain
  • Unverified
    Italy
  • Unverified
    Hungary
  • Unverified
    Belgium
  • Unverified
    Poland
  • Unverified
    Greece

Common questions

What is Dravet Syndrome or Lennox-Gastaut Syndrome?

These are serious types of epilepsy that often start in childhood and can cause frequent, hard-to-control seizures and other developmental challenges.

What is soticlestat?

Soticlestat is a medicine being studied as an 'add-on' treatment for people with these types of epilepsy, meaning it's taken alongside other medicines.

Why is this study only for people who were in a previous study?

This is an 'extension' study. It's designed to collect long-term information about the safety and effects of soticlestat from people who have already started taking it.

What does 'long-term safety' mean?

It means the study will check for any side effects or health changes that might happen over many months or even years of taking the medicine.

Will I know if the medicine is helping my seizures?

While the main focus is safety, the study will also track your seizure frequency and severity, so you and your doctor will have information about its effects.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Discussion

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