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Vigabatrin

Clinical trials of Vigabatrin are studying whether it can help prevent or reduce seizures in young children at high risk. The trials focus on infants with tuberous sclerosis complex and other high-risk brain injuries, and they measure safety, seizure prevention, and later neurodevelopment.

Table of Contents

Clinical trial overview

The available trials are studying Vigabatrin in young children at high risk for seizures, especially children with tuberous sclerosis complex (TSC) and children with serious brain injury risks.[1][2][3]

Across the trials, researchers are mainly asking whether Vigabatrin can help prevent infantile spasms or other clinical seizures, and whether early treatment may affect later development or tumor growth.[1][2][3]

Main studies and what they measure

The Phase 2 PROTECT study is in children with TSC under 4 months of age and compares neuropsychologic outcome at 24 months with standard of care alone.[1]

The Phase 3 study in high-risk children under 1 year old is designed to prevent infantile spasm syndrome, including in children with tuberous sclerosis, large vascular cortical injury, grade IV intraventricular hemorrhage, and severe periventricular leukomalacia in preterm infants.[2]

The Phase 4 ViRap study compares rapamycin versus Vigabatrin in infants with TSC and looks at seizure occurrence and growth of TSC-associated tumors.[3]

Who the trials are for

These studies focus on infants and very young children, not older children or adults.[1][2][3]

One study includes children with TSC under 4 months of age, another includes children under 1 year old who are at high risk for infantile spasms, and the third trial includes infants with TSC.[1][2][3]

The high-risk group in the Phase 3 study includes children with tuberous sclerosis, major vascular brain injury, severe bleeding in the brain, infection or trauma-related injury, hypoxic-ischemic encephalopathy, and severe white matter injury in preterm infants.[2]

Trial phases and study design

The listed trials are interventional, which means the researchers give a treatment and then measure the results.[1][2][3]

One trial is Phase 2, one is Phase 3, and one is Phase 4, showing that Vigabatrin is being studied at different stages of clinical research.[1][2][3]

The Phase 4 trial is randomized and compares two preventive strategies, rapamycin and Vigabatrin, in infants with TSC.[3]

Main endpoints and outcomes

The main outcome in the Phase 2 study is neuropsychologic outcome at 24 months, measured with blinded testing and the cognitive scale of the Bayley Scales of Infant and Toddler Development III.[1]

The Phase 3 study measures whether the child develops infantile spasm syndrome, which is the key efficacy result for that trial.[2]

The Phase 4 study measures two main results: whether clinical seizures occur during the blinded phase and whether the total volume of TSC-associated tumors increases by at least 25% from the starting value.[3]

What these trials mean for patients

For families, these studies are mainly about early prevention: trying to stop seizures before they start, or learning whether early treatment may protect later development.[1][2][3]

The trials also show that researchers are not only watching seizure control, but also longer-term brain development and tumor changes in children with TSC.[1][3]

Because the studies are focused on specific high-risk infant groups, participation depends on age and the child’s medical condition.[1][2][3]

Trial ID Phase Condition studied Status Enrollment
2022-502332-39-00 Phase 2 Tuberous sclerosis complex (TSC) Authorised 64
2024-517488-22-00 Phase 3 High risk for infantile spasm syndrome in children under 1 year Authorised 140
NCT04987463 Phase 4 Tuberous Sclerosis Complex, Epilepsy, Tumors associated with Tuberous Sclerosis Complex Authorised 60

FAQ

  • What are clinical trials of Vigabatrin studying? These trials are looking at whether Vigabatrin can prevent infantile spasms or other clinical seizures, especially in very young children at high risk. Some studies also measure later brain and learning outcomes.
  • Who may take part in these trials? The studies focus on infants and very young children, often under 1 year of age or under 4 months of age. The target groups include children with tuberous sclerosis complex and children with major brain injuries that raise the risk of infantile spasms.
  • What trial phases are listed for Vigabatrin? The available trials are Phase 2, Phase 3, and Phase 4. This means the research includes both earlier testing and later studies that compare treatment strategies in larger groups.
  • What conditions are being studied with Vigabatrin? The main condition is tuberous sclerosis complex, a genetic disorder linked with seizures and tumors. Another trial studies infants at high risk for infantile spasm syndrome because of serious brain injury, bleeding, infection, or prematurity-related brain damage.
  • What outcomes are measured in these trials? The trials measure whether children develop infantile spasms or clinical seizures, whether tumors grow, and how children do on neuropsychologic testing. One study uses the Bayley Scales of Infant and Toddler Development III to assess cognitive development at 24 months.

Ongoing Clinical Trials on Vigabatrin

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

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Poland

  • Prevention of Infantile Spasms with Vigabatrin in High-Risk Children Under 1 Year Old: A Study for Patients with Tuberous Sclerosis or Large Vascular Brain Injury

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Finland

  • Study on the Effects of Sirolimus and Drug Combination for Children Under 4 Months with Tuberous Sclerosis Complex

    Recruiting

    1 1 1
    Investigated drugs:
    Austria Germany

Glossary

  • Tuberous sclerosis complex (TSC): A genetic condition that can cause seizures, brain changes, and tumors in different parts of the body.
  • Infantile spasms: A type of seizure syndrome in babies that can cause sudden repeated body movements and may affect development.
  • Clinical seizure: A seizure that can be seen or recognized by symptoms, not only by a test.
  • Prophylactic treatment: Treatment given to help prevent a disease or symptom before it starts.
  • Interventional study: A trial where researchers give a treatment and then watch what happens.
  • Phase 2: A trial stage that usually looks more closely at whether a treatment seems to work and how well it is tolerated.
  • Phase 3: A larger trial stage that compares treatments and helps confirm whether they work.
  • Phase 4: A later trial stage done after a treatment is already being studied more widely.
  • Enrollment: The number of participants planned for the study.
  • Primary outcome: The main result the trial is designed to measure.
  • Neuropsychologic testing: Tests that look at thinking, learning, memory, and other brain-related skills.
  • Bayley Scales of Infant and Toddler Development III (BSID-III): A standard test used to measure development in babies and toddlers, including cognitive skills.

References