Study on Ganaxolone for Children and Adults with Epilepsy Related to Tuberous Sclerosis Complex

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What is this study about?

This clinical trial is focused on studying a condition known as Tuberous Sclerosis Complex (TSC)-related epilepsy. TSC is a genetic disorder that causes non-cancerous tumors to form in many different organs, primarily the brain, which can lead to seizures. The treatment being tested in this study is called Ganaxolone, which is a type of medication known as a neurosteroid. Neurosteroids are compounds that can influence brain activity and are being explored for their potential to help manage seizures.

The purpose of this study is to evaluate the long-term safety and tolerability of Ganaxolone when used alongside other treatments for seizures in both children and adults with TSC. Participants in the study will take Ganaxolone in capsule form by mouth. The study will monitor participants over a period to observe any side effects and how well the medication is tolerated. Participants will also continue their usual seizure treatments while taking Ganaxolone.

Throughout the study, various health checks will be conducted, including monitoring vital signs like blood pressure and heart rate, as well as performing physical and neurological exams. The study will also track changes in seizure frequency and assess the overall quality of life for participants. This information will help researchers understand how effective Ganaxolone is in managing seizures associated with TSC and its impact on the daily lives of those affected by this condition.

1 initial visit

Upon joining the study, you will have an initial visit where you will be informed about the study’s nature and risks. You will be asked to provide written consent to participate. If you are unable to provide consent due to age or other factors, a parent or legal representative will do so on your behalf.

During this visit, a blood test will be conducted to ensure you are not pregnant if you are a woman of childbearing potential. You will also be asked about your current method of birth control.

2 medication administration

You will begin taking the study medication, ganaxolone, in the form of capsules. The medication is to be taken orally three times a day with food.

It is important to follow the dosage instructions carefully and maintain a consistent schedule for taking the medication.

3 daily seizure diary

You or your caregiver will be required to keep a daily diary of any seizures you experience. This diary is crucial for tracking the frequency and type of seizures during the study.

4 regular check-ups

Throughout the study, you will have regular check-ups to monitor your health and the effects of the medication. These check-ups will include measurements of vital signs such as blood pressure, heart rate, and body temperature.

You will also undergo physical, neurological, and developmental examinations, as well as an electrocardiogram (ECG) to check your heart’s activity.

5 laboratory tests

Clinical laboratory tests will be conducted periodically to assess your overall health and any potential side effects of the medication.

6 safety and tolerability assessment

The study aims to assess the long-term safety and tolerability of ganaxolone as an additional treatment for seizures associated with tuberous sclerosis complex. Any adverse effects or changes in your condition will be closely monitored.

7 end of study evaluation

At the end of the study, a final evaluation will be conducted to assess the overall impact of the treatment on your seizure frequency and quality of life.

You will be asked to provide feedback on your experience and any changes you have noticed during the study.

Who Can Join the Study?

Must have completed a previous study related to Tuberous Sclerosis Complex (TSC) or continue to meet the requirements of an ongoing study.
The participant or their parent(s)/legal representative(s) must be willing and able to provide written consent after understanding the study’s nature and risks. If the participant cannot provide consent due to age or other factors, the parent(s) or legal representative(s) must agree to the participant’s involvement.
Parents or caregivers must be willing and able to keep a detailed daily record of seizures throughout the study.
Must be willing and able to take the study medication as a liquid with food three times a day.
Women of childbearing potential must use a medically approved method of birth control and have a negative pregnancy test at the start of the study. Childbearing potential refers to females who can become pregnant. Acceptable birth control methods include devices placed inside the body, hormonal methods like pills or patches, and surgical procedures to prevent pregnancy.
Male participants must agree to use highly effective birth control methods during the study and for 30 days after the last dose of the study medication. This includes surgical procedures like a vasectomy or using two forms of barrier protection.

Who Cannot Join the Study?

Patients who do not have Tuberous Sclerosis Complex (TSC) related epilepsy cannot participate. TSC is a genetic disorder that causes non-cancerous tumors to form in many different organs.
Patients who are not experiencing seizures associated with TSC cannot participate. Seizures are sudden, uncontrolled electrical disturbances in the brain.
Patients who are not within the specified age range for the study cannot participate. The study includes children and adults.
Patients who are not able to take the study medication safely cannot participate. This means if the medication could cause harm or serious side effects, the patient cannot join.
Patients who are pregnant or breastfeeding cannot participate. This is to ensure the safety of both the mother and the baby.
Patients who are participating in another clinical trial cannot participate. This is to avoid any interference with the study results.
Patients who have a history of drug or alcohol abuse cannot participate. This is to ensure the safety and accuracy of the study results.
Patients who have a serious medical condition that could interfere with the study cannot participate. This includes conditions that might make it unsafe for the patient to take part.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Centre Hospitalier Universitaire De Lille	Lille	France
Hospital Universitario Y Politecnico La Fe	Valencia	Spain

Other Sites

Site Name	City	Country
Robert Debre University Hospital	Paris	France
Epilepsiezentrum Kleinwachau gGmbH	Radeberg	Germany
Les Hopitaux Universitaires De Strasbourg	STRASBOURG, Alsace	France
Rheinische Friedrich-Wilhelms-Universitaet Bonn	Bonn	Germany
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Hospices Civils De Lyon	Lyon	France
Centre Hospitalier Universitaire De Rennes	Rennes	France
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Azienda Ospedaliera Universitaria Meyer IRCCS	Florence	Italy
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Hospital Ruber Internacional	Madrid	Spain
Vrije Universiteit Brussel	Jette	Belgium
Gemeinschaftskrankenhaus Herdecke gGmbH	Herdecke	Germany
Gesellschaft Fuer Epilepsieforschung	Bielefeld	Germany
Fundacio Institut De Recerca De L’Hospital De La Santa Creu I Sant Pau	Barcelona	Spain
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Hôpital Pontchaillou-CHU Rennes	Rennes	France
Gobhui Ujwmtktjsj Fpjfxqbpz	Frankfurt	Germany
Uwpazifled Og Aqmjvyz	Edegem	Belgium
Uwaedlyygv Dsawv Sgfly Do Rcqo Lh Snkrgfhr	Rome	Italy
Hdikqyue Vuay doylqgym	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	15.07.2023
France	Not recruiting	15.07.2023
Germany	Not recruiting	15.07.2023
Italy	Not recruiting	15.07.2023
Spain	Not recruiting	15.07.2023

Trial locations

Investigated drugs:

Ganaxolone

Ganaxolone (GNX) is a medication being studied for its potential to help people with epilepsy related to Tuberous Sclerosis Complex (TSC). It is used as an additional treatment to help control seizures. Ganaxolone works by calming the brain’s activity, which can help reduce the number of seizures a person experiences. This study is focused on understanding how safe and tolerable Ganaxolone is when used over a long period in both children and adults with TSC-related epilepsy.

Tuberous Sclerosis Complex (TSC) related epilepsy – Tuberous Sclerosis Complex is a genetic disorder that causes non-cancerous tumors to form in many different organs, primarily the brain, eyes, heart, kidney, skin, and lungs. When these tumors affect the brain, they can lead to epilepsy, which is characterized by recurrent seizures. The seizures in TSC-related epilepsy can vary in type and severity, including focal seizures, which affect one part of the brain, and generalized seizures, which affect the whole brain. Over time, the frequency and intensity of seizures can change, potentially impacting cognitive and developmental functions. The progression of epilepsy in TSC is influenced by the location and size of the brain lesions. Managing the condition often involves monitoring and adjusting to the evolving nature of the seizures.

Trial ID:

2022-503067-15-00

Protocol code:

1042-TSC-3002

NCT ID:

NCT05604170

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Ganaxolone for Children and Adults with Epilepsy Related to Tuberous Sclerosis Complex

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