Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Phase 2B, Multicenter, 30-week, Prospective, Cross-over, Double-blind, Randomized, Placebo-controlled Study Followed by a 52-Week Open-label Extension Study to Evaluate the Efficacy and Safety of Basimglurant Adjunctive to Ongoing Anticonvulsive Therapy in Children, Adolescents, and Young Adults with Seizures Associated with Tuberous Sclerosis Complex

This research is a trial for a new medicine, Basimglurant, for children, teenagers, and young adults who have seizures because of Tuberous Sclerosis Complex (TSC). The main goal is to find out if taking Basimglurant alongside their current seizure medicines can help reduce the number of seizures they have. Scientists will compare it to a dummy pill (placebo) to see if it works better. They also want to understand if the medicine is safe and what side effects it might have. This study will last about 30 weeks, with a longer follow-up period of 52 weeks to keep checking on participants.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Noema Pharma AG

Enrolment target

Start

06 Sep 2024

Tuberous Sclerosis Complex

What is this study about?

This study is investigating a new medicine called Basimglurant. It's designed for children, teenagers, and young adults who have seizures as part of a condition called Tuberous Sclerosis Complex (TSC). The main purpose is to see how well Basimglurant works to reduce the number of seizures when added to the seizure medications people are already taking. Researchers will also be carefully checking for any side effects to make sure the medicine is safe.

The study involves comparing Basimglurant to a 'dummy' pill, called a placebo. This helps researchers understand if any changes in seizure numbers are truly due to the new medicine or if they might happen anyway. Participants won't know if they're receiving the actual medicine or the placebo, and neither will their doctor, which is important for getting clear results. After an initial period, all participants will get the Basimglurant medicine.

Understanding how new medicines like Basimglurant work and if they are safe is really important for people living with TSC. If this study shows that Basimglurant is effective and safe, it could become another treatment option to help manage seizures for children, teenagers, and young adults with this condition, potentially improving their quality of life.

Key takeaways

This study evaluates a new medicine, Basimglurant, for seizures in Tuberous Sclerosis Complex.
It aims to see if Basimglurant reduces seizures and is safe when added to current medication.
The study involves comparing Basimglurant to a placebo (dummy pill) initially.
Participants will eventually receive the active Basimglurant medicine during a longer follow-up.
The study includes regular health checks, seizure tracking, and safety monitoring.
It's for adults aged 18 and over who have seizures due to TSC.

Who may be eligible?

To join this study, people need to be at least 18 years old. There's no upper age limit, meaning adults of any age can potentially take part.

The study is open to both males and females. The most important condition for joining is that participants have seizures that are caused by Tuberous Sclerosis Complex (TSC).

Other specific health requirements or conditions might also apply, such as what other medications they are currently taking or their overall health. These details would be discussed fully with a healthcare professional to see if the study is a good fit.

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?
Do you have seizures that are caused by Tuberous Sclerosis Complex (TSC)?
Are you currently taking other medication for your seizures?
Are you able to attend regular clinic visits and record your seizures?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will essentially be helping researchers understand a new medicine. The study has two main parts: a core part lasting about 30 weeks, and then a longer follow-up period of 52 weeks.

During the core part, you would receive either the study medicine (Basimglurant) or a placebo (a dummy pill that looks the same). You and your doctor won't know which one you're getting at first. Your usual seizure medication will continue as normal. You'll need to keep a careful record of your seizures. There will be regular visits to the clinic, where doctors will check your health, do some tests, and ask questions about how you're feeling and how your seizures are controlled. They will also look at things like your vital signs (blood pressure, heart rate), perform physical examinations, and take blood for lab tests. After the initial 30 weeks, all participants will receive the active study medicine for the additional 52 weeks, with ongoing monitoring.

Throughout the study, doctors will be checking various things such as changes in how many seizures you have, how the medicine affects your daily life, and any side effects. You'll also be asked about your mood and any thoughts you might have. You'll need to take the study medicine as directed. The total duration of active participation receiving either the study medicine or placebo, and then the open-label medicine, will be around 82 weeks, including follow-up assessments.

Potential risks and benefits

Taking part in a clinical trial offers potential benefits, such as gaining access to a new treatment before it's widely available and receiving close medical attention. The goal of this study is to see if Basimglurant can reduce seizures in people with Tuberous Sclerosis Complex, which could improve quality of life. However, like all medicines, Basimglurant may have side effects, and some participants might experience no benefit from the treatment. There's also the chance of unknown risks. You will be fully informed of all known and potential risks before deciding to join. It's very important to remember that participating is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

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

—
Unverified
Spain
—
Unverified
Italy
—
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.

What is Basimglurant?

Basimglurant is a new medicine being tested to see if it helps reduce seizures in people with Tuberous Sclerosis Complex.

What does 'placebo-controlled' mean?

It means some people will get the active medicine, and others will get a dummy pill (placebo) that looks the same but contains no medicine. This helps researchers compare the effects fairly.

Will I know if I'm getting the real medicine or the dummy pill?

No, during the first part of the study, neither you nor your doctors will know if you're getting Basimglurant or the placebo. This is called 'double-blind'.

How long will the study last?

The main part of the study is about 30 weeks, followed by a longer period of 52 weeks where everyone gets the active medicine, making the total about 82 weeks.