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RecrutamentoExploratório terapêutico (Fase II)Intervencionista

A Phase 2, Prospective, Interventional, Open-Label, Multisite, Extension Study to Assess the Long-Term Safety and Tolerability of Soticlestat (TAK-935) as Adjunctive Therapy in Subjects with Developmental Epileptic Encephalopathies Including Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome (ENDYMION 1)

This study, called ENDYMION 1, is designed to check the long-term safety and how well people tolerate a medicine called soticlestat (also known as TAK-935). It's for individuals with specific forms of severe epilepsy, such as Dravet Syndrome, Lennox-Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome, along with other similar conditions in adults. The medicine is given alongside other treatments already being used. This is an 'extension' study, meaning that participants would have generally taken soticlestat in an earlier trial and are continuing to receive it to see its effects over a longer period. Researchers will be carefully monitoring any side effects and looking at how the medicine might affect daily life and behaviour.

Em resumo

Status
Recrutamento
Fase
Exploratório terapêutico (Fase II)
Patrocinador
Centro de Desenvolvimento Takeda Américas Inc.
Meta de inscrição
51
Iniciar
23 Ago 2023

Sobre o que é este estudo?

This research study, known as ENDYMION 1, is focused on a medicine called soticlestat (or TAK-935) and is designed for people who have certain severe forms of epilepsy. These conditions include Dravet Syndrome, Lennox-Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome, as well as other similar epilepsy types in adults. The study aims to look closely at the long-term safety of this medicine and how well people cope with it over time when it's used alongside their current treatments.

Because this is an 'extension' study, it means that many participants will have already been taking soticlestat in a previous, shorter study. This helps researchers understand the medicine's effects over a much longer period. They will be very carefully recording any side effects that occur.

Beyond safety, the study also plans to observe how the medicine might affect daily life. This includes looking at changes in behaviour, adaptive skills (how well someone copes with everyday demands), and seizure frequency. Understanding these aspects helps give a more complete picture of the medicine's overall impact.

Pontos principais

  • This study investigates the long-term safety of soticlestat (TAK-935) for severe epilepsies.
  • It's an extension study, meaning participants likely used the drug in a prior trial.
  • It covers specific severe epilepsies like Dravet Syndrome and Lennox-Gastaut Syndrome.
  • Researchers will monitor side effects, behaviour, and daily living skills.
  • Regular health checks, including blood tests and ECGs, will be part of participation.
  • A participação é voluntária, e você pode se retirar a qualquer momento.

Quem pode ser elegível?

This study is open to both adults and children aged 18 years and above, regardless of gender. You would generally be considered for this study if you have already participated in an earlier study involving the medication soticlestat (TAK-935) and have certain types of severe epilepsy.

The specific epilepsy conditions include Dravet Syndrome, Lennox-Gastaut Syndrome, CDKL5 Deficiency Disorder, Chromosome 15 Duplication Syndrome, or other similar severe epilepsies in adults. The main idea is that this study is a continuation for people who have already started treatment with this specific medicine in a past trial.

Este estudo pode ser adequado para você?

Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.

  1. Você tem 18 anos ou mais?
  2. Do you have Dravet Syndrome, Lennox-Gastaut Syndrome, CDKL5 Deficiency Disorder, Chromosome 15 Duplication Syndrome, or another similar severe epilepsy in adults?
  3. Have you previously participated in a clinical study involving the investigational medicine soticlestat (TAK-935)?
  4. Are you able to attend regular study visits and complete necessary assessments?
Responda a todas as perguntas para ver o seu resultado.

O que envolve a participação?

If you take part in this study, you'll continue to receive the study medication, soticlestat, as an add-on to any other epilepsy medicines you're already taking. You would have regular check-ups which involve various assessments. These assessments will include keeping track of any side effects you might experience, and looking at changes in your behaviour and how you manage everyday tasks. Doctors will also check your general health through blood tests, measurements like blood pressure and heart rate, body weight, and heart traces (ECGs).

There will also be questionnaires to understand your mood and behaviour. For children aged 6 and over, there are specific assessments for behaviour and, if aged 6 or over, questions about thoughts of self-harm. The study will also track how often seizures occur. The total duration of your participation will depend on how long the study continues, as it's an 'extension' study designed to gather long-term information.

Riscos e benefícios potenciais

Participating in this study might offer a potential benefit by allowing you to continue receiving soticlestat, which might help manage your seizures or improve daily function, although this is not guaranteed. As with any medication, there are always potential risks, including side effects from the study drug. These will be carefully monitored by the study team. You will be told about all known risks before you decide to join. It's very important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Localizações (3)

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    Espanha
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    Portugal
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    Polônia

Perguntas frequentes

O que é um 'estudo de extensão'?

An 'extension study' means that participants have usually taken the study medicine in a previous, shorter trial and are continuing to take it to see its effects over a longer period, often to gather more information on long-term safety.

What is soticlestat (TAK-935)?

Soticlestat (TAK-935) is the name of the medicine being studied. It's being investigated as a possible additional treatment for certain severe forms of epilepsy.

Will I stop my other epilepsy medicines if I join?

No, this study uses soticlestat as an 'adjunctive therapy', meaning it's given alongside your existing epilepsy medicines, not instead of them.

What kind of tests will I have?

You'll have regular checks including blood tests, measurements of your heart rate and blood pressure, weight checks, heart traces (ECGs), and questionnaires about your behaviour and how you're feeling.

E se eu experimentar efeitos colaterais?

The study team will carefully monitor you for any side effects. It's very important to tell them about anything new or unusual you experience, so they can assess it and provide appropriate care.

Como saber mais

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