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Study on the Safety and Effectiveness of Sirolimus and Vigabatrin for Preventing Symptoms in Infants with Tuberous Sclerosis Complex

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of two treatments for infants with Tuberous Sclerosis Complex (TSC), a genetic disorder that can cause non-cancerous tumors to grow in the brain and other vital organs, leading to conditions such as epilepsy. The trial will compare the safety and effectiveness of two medications: Rapamune (also known as sirolimus) and Sabril (also known as vigabatrin). Rapamune is an oral solution used to prevent organ rejection in transplant patients, while Sabril is used to treat seizures in epilepsy. The purpose of this study is to see how well these medications can prevent the symptoms of TSC in infants.

Participants in the study will be randomly assigned to receive either Rapamune, Sabril, or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving which treatment. This helps ensure that the results are not biased. The trial will last for a period of up to two years, during which the infants' health will be closely monitored. Researchers will look at various factors, such as the occurrence of seizures, the growth of TSC-related tumors, and the overall development of the infants.

Throughout the study, the infants will undergo regular check-ups to monitor their physical development, including weight and height, as well as vital signs like body temperature and blood pressure. The study will also assess the risk of developing conditions such as autism and drug-resistant epilepsy. By the end of the study, researchers hope to gather valuable information on the effectiveness and safety of Rapamune and Sabril in preventing the symptoms of TSC in infants, which could lead to better treatment options in the future.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization and initial assessment

    Upon joining the study, infants are randomly assigned to receive either vigabatrin or rapamycin. This process is called randomization and ensures that each participant has an equal chance of receiving either treatment.

    An initial assessment is conducted to confirm the diagnosis of Tuberous Sclerosis Complex (TSC) and to evaluate the presence of cortical dysplasia through a brain MRI.

  2. Step 2

    Medication administration

    Participants receive their assigned medication. Vigabatrin is administered as an oral solution, with a dosage of 500 mg. Rapamycin is provided as a 1 mg/mL oral solution.

    The frequency and duration of administration are determined by the study protocol and are monitored by the study team.

  3. Step 3

    Monitoring and follow-up

    Regular follow-up visits are scheduled to monitor the infant's response to the treatment. This includes checking for any clinical seizures and measuring the volume of TSC-associated tumors.

    Physical development parameters such as weight and height, as well as vital signs like body temperature, pulse rate, respiration rate, and blood pressure, are recorded throughout the study.

  4. Step 4

    Assessment of outcomes

    The primary outcomes assessed include the occurrence of clinical seizures and any increase in the volume of TSC-associated tumors.

    Secondary outcomes include the time to the first epileptic seizure, the risk of autism, developmental quotient, drug-resistant epilepsy, and any adverse events.

  5. Step 5

    Completion of the study

    The study is expected to conclude by March 31, 2026. At the end of the study, a comprehensive evaluation of the safety and efficacy of vigabatrin and rapamycin in preventing TSC symptoms will be conducted.

Who can join the trial?

5 criteria

  • The child must be a boy or girl aged up to 16 weeks old, which means they should be between 41 to 56 weeks from the time they were expected to be born.
  • The parents or caregivers must be willing and able to sign a form that shows they agree to let their child participate in the study.
  • The parents or caregivers must be willing and able to follow all the rules and requirements of the study.
  • The child must have a definite diagnosis of **Tuberous Sclerosis Complex (TSC)**, which is a condition diagnosed using specific guidelines.
  • The child must have at least one area of **cortical dysplasia** found on a brain **MRI**. Cortical dysplasia is a condition where the brain's surface develops abnormally, and an MRI is a type of scan that takes pictures of the inside of the body.

Who cannot join the trial?

5 criteria

  • Patients who do not have a diagnosis of Tuberous Sclerosis Complex cannot participate. Tuberous Sclerosis Complex is a genetic disorder that causes non-cancerous tumors to form in many different organs.
  • Patients who do not have epilepsy associated with Tuberous Sclerosis Complex cannot participate. Epilepsy is a condition that causes repeated seizures.
  • Patients who do not have tumors associated with Tuberous Sclerosis Complex cannot participate. Tumors are abnormal growths of tissue.
  • Patients who are not infants cannot participate. Infants are very young children, typically under one year of age.
  • Patients who are not part of the vulnerable population selected for the study cannot participate. Vulnerable populations may include groups like infants or those with specific health conditions.
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Investigated drugs

  • Vigabatrin

    is a medication used to help prevent seizures. In this trial, it is being tested to see how well it works and how safe it is for infants with Tuberous Sclerosis Complex (TSC). The goal is to see if it can help prevent the onset of seizures in these infants.

  • Rapamycin

    is another medication being tested in this trial. It is used to see if it can prevent seizures in infants with Tuberous Sclerosis Complex (TSC). The study aims to determine its safety and effectiveness in preventing seizures in these young patients.

What is already known about the treatment

  • Vigabatrin

    Vigabatrin is administered orally, typically in the form of tablets or powder for solution. It is currently used in medicine to manage certain types of seizures, particularly in patients with Tuberous Sclerosis Complex (TSC). The main therapeutic indication for vigabatrin is epilepsy, especially when associated with TSC. At the molecular level, vigabatrin works by inhibiting an enzyme called GABA transaminase, which leads to increased levels of the neurotransmitter GABA in the brain, helping to reduce seizure activity. It is classified as an anticonvulsant medication.

  • Rapamycin

    Rapamycin is usually administered orally in tablet or liquid form. It is being studied for its potential use in treating conditions related to Tuberous Sclerosis Complex, such as epilepsy and tumors. The main therapeutic indications include its use in preventing seizures and managing tumor growth in TSC patients. Rapamycin works by inhibiting a protein called mTOR, which plays a role in cell growth and proliferation, thereby helping to control abnormal cell growth. It is classified as an immunosuppressant and antiproliferative agent.

Investigated diseases

  • Tuberous Sclerosis Complex

    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. These tumors can lead to a variety of symptoms, including seizures, developmental delays, and skin abnormalities. The condition is caused by mutations in either the TSC1 or TSC2 genes, which are responsible for controlling cell growth. As the disease progresses, the size and number of tumors may increase, potentially affecting the function of the involved organs. The severity and range of symptoms can vary widely among individuals.

  • Epilepsy

    Epilepsy is a neurological disorder characterized by recurrent, unprovoked seizures. These seizures are caused by abnormal electrical activity in the brain and can vary in type and intensity. Some individuals may experience brief lapses in attention, while others may have full-body convulsions. The frequency and severity of seizures can change over time, and they may be influenced by factors such as stress, sleep deprivation, or hormonal changes. Epilepsy can affect people of all ages and may be associated with other neurological conditions.

  • Tumors associated with Tuberous Sclerosis Complex

    These tumors are benign growths that occur in various organs due to Tuberous Sclerosis Complex. They are most commonly found in the brain, kidneys, heart, lungs, and skin. While these tumors are non-cancerous, their growth can lead to complications by interfering with the normal function of the affected organs. The progression of these tumors can vary, with some remaining stable while others may grow larger over time. The presence and impact of these tumors can contribute to the overall clinical picture of Tuberous Sclerosis Complex.

Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IVTrial ID2024-513068-25-00Protocol codeViRapEstimated enrolment60 patientsSponsorInstytut Pomnik Centrum Zdrowia Dziecka

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