Study on Basimglurant for Children, Adolescents, and Young Adults with Tuberous Sclerosis Complex-Related Seizures

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What is this study about?

This clinical trial is focused on studying a condition known as Tuberous Sclerosis Complex (TSC), which is a genetic disorder that causes non-cancerous tumors to form in many different organs, primarily the brain, eyes, heart, kidney, skin, and lungs. The study aims to evaluate the effectiveness and safety of a medication called Basimglurant, which is being tested as an additional treatment alongside ongoing anticonvulsive therapy for managing seizures associated with TSC. Basimglurant is taken in the form of a capsule and is known by the code name NOE-101.

The purpose of the study is to assess how well Basimglurant works in reducing seizures when used together with current seizure medications. Participants in the study will be randomly assigned to receive either Basimglurant or a placebo, which looks like the medication but does not contain the active ingredient. The study will be conducted over a period of 30 weeks, during which participants will not know whether they are receiving Basimglurant or the placebo. After this period, there will be an additional 52-week phase where all participants will receive Basimglurant.

Throughout the study, participants will continue their regular anticonvulsive therapy. The study will monitor the number of seizures, any changes in daily activities, and overall impressions of change from caregivers. Safety will also be closely observed, including any side effects or changes in health. This trial is designed to provide valuable information on whether Basimglurant can be a beneficial addition to existing treatments for those with Tuberous Sclerosis Complex.

1 initial phase

Upon joining the study, the participant will begin a 30-week trial period. This phase is designed to evaluate the effectiveness and safety of the medication basimglurant when used alongside existing seizure treatments.

The participant will be randomly assigned to receive either basimglurant or a placebo. Both are administered in capsule form and taken orally.

2 treatment period 1

During the first 12 weeks, the participant will take the assigned medication daily. The goal is to monitor seizure frequency and any changes in seizure patterns.

The participant will continue their current anticonvulsive therapy throughout this period.

3 evaluation and crossover

After the initial 12 weeks, the participant’s response to the treatment will be evaluated. This includes assessing seizure counts and any side effects.

The participant may then switch to the alternative treatment (from placebo to basimglurant or vice versa) for another 12-week period.

4 treatment period 2

In this phase, the participant will continue with the new treatment regimen for another 12 weeks. The focus remains on monitoring seizure activity and overall health.

Regular assessments will be conducted to track any changes in seizure frequency and to ensure the participant’s safety.

5 open-label extension

Following the 30-week double-blind phase, the participant may enter a 52-week open-label extension. During this time, all participants will receive basimglurant.

The purpose of this phase is to gather long-term data on the medication’s effectiveness and safety.

6 ongoing assessments

Throughout the study, the participant will undergo regular health checks, including physical exams and laboratory tests, to monitor for any adverse effects.

The participant’s seizure activity will be closely tracked, and any changes in health or medication tolerance will be documented.

Who Can Join the Study?

Ability and willingness to provide informed assent or written consent, or consent from their legal representative. This means the patient or their guardian agrees to participate after understanding the study.
Fluency in the language of the study staff. The patient must be able to speak and understand the language used by the study team.
Age between 5 to 30 years at the start of the study.
A documented history of Tuberous Sclerosis Complex (TSC). This means the patient has medical records showing they have TSC.
Refractory seizure history. This means the patient has seizures that are difficult to control with medication.
Currently receiving one or more anti-epileptic drugs (AEDs). These are medications used to help control seizures.
Stable medications or interventions for epilepsy for 30 days before study entry. The patient’s treatment for epilepsy should not have changed in the last 30 days.
Willingness to complete Patient Reported Outcome assessments. The patient agrees to provide feedback on their health and treatment during the study.
For female patients of childbearing potential: Willingness to undergo serum or urinary pregnancy testing at screening and during the trial period. This means they agree to take pregnancy tests before and during the study.
For female patients of childbearing potential: Willingness to use contraception. This means they agree to use birth control methods during the study.

Who Cannot Join the Study?

Patients who are not diagnosed with Tuberous Sclerosis Complex cannot participate. Tuberous Sclerosis Complex is a genetic disorder that causes non-cancerous growths in many parts of the body.
Patients who are not currently receiving anticonvulsive therapy cannot participate. Anticonvulsive therapy involves taking medication to help control seizures.
Patients who are unable to take the study medication as directed cannot participate.
Patients who have a medical condition that might interfere with the study cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have participated in another clinical trial recently may not be eligible.
Patients who have a history of drug or alcohol abuse may not be eligible.
Patients who have a known allergy to the study medication cannot participate.
Patients who are unable to comply with the study procedures cannot participate.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania	Catania	Italy
Amicare Sp. z o.o. S.K.	Lodz	Poland
Azienda Ospedaliera Universitaria Meyer IRCCS	Florence	Italy
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Centrum Medyczne Plejady Magdalena Celinska Loewenhoff Michal Zolnowski sp.k.	Cracow	Poland
ASST Grande Ospedale Metropolitano Niguarda	Milan	Italy
Instytut Pomnik Centrum Zdrowia Dziecka	Warsaw	Poland
Azienda Sociosanitaria Territoriale Santi Paolo E Carlo	Milan	Italy
Hhqaulte Uouwtnhhhlhrh Hltbtwpg Tnwiv y Pgyxmm Iintaari Clyiqj dlpyoblwxkutsshhl (aduh	Badalona	Spain
Cqtlyw do Natkuuufsj Ansutidc	Sevilla	Spain

Trial status

Country	Status	Recruitment Start
Italy	Not recruiting	01.08.2024
Poland	Not recruiting	01.08.2024
Spain	Not recruiting	01.08.2024

Trial locations

Investigated drugs:

Basimglurant

Basimglurant is being studied to see if it can help reduce seizures in children, adolescents, and young adults who have Tuberous Sclerosis Complex. It is given in addition to the anticonvulsive medications that patients are already taking. The study aims to find out if adding basimglurant to their current treatment can improve their condition.

Investigated diseases:

Tuberous sclerosis complex

Tuberous Sclerosis Complex – Tuberous Sclerosis Complex is a genetic disorder characterized by the growth of non-cancerous tumors in various parts of the body, including the brain, skin, kidneys, and heart. These tumors can lead to a range of symptoms, such as seizures, developmental delays, and skin abnormalities. The condition is caused by mutations in either the TSC1 or TSC2 genes, which are responsible for regulating cell growth. As the disease progresses, individuals may experience an increase in the number and size of tumors, potentially affecting organ function. The severity and specific symptoms can vary widely among individuals, even within the same family. Tuberous Sclerosis Complex is considered a rare disease, affecting a small percentage of the population.

Trial ID:

2024-512611-53-00

Protocol code:

NOE-TSC-201

NCT ID:

NCT05059327

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on Basimglurant for Children, Adolescents, and Young Adults with Tuberous Sclerosis Complex-Related Seizures

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?