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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

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What is this trial about?

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

This clinical trial focuses on infantile spasm syndrome, a serious condition that can affect children under one year of age. The study specifically looks at children who are at high risk of developing this condition, including those with tuberous sclerosis (a rare genetic disorder) or those who have experienced significant brain injuries such as large blood vessel damage, severe brain bleeds, or infections affecting the brain tissue.

The study uses vigabatrin, which is given as granules that are mixed with liquid and taken by mouth. This medication will be given as a preventive treatment before any signs of infantile spasms appear. The maximum daily dose is 150 milligrams per kilogram of body weight, and the treatment can continue for up to 11 months.

The main goal of this research is to determine if giving vigabatrin early to high-risk infants can prevent the development of infantile spasm syndrome. The study also looks at how the children's brain development progresses and uses special eye tracking methods to monitor their development. MRI scans of the brain, which have been performed as part of regular medical care, are used to identify children who might benefit from participating in this research.

The research process

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

Step 1
Initial assessment
Your doctor will confirm if you meet the study requirements based on your medical condition and brain MRI results
The study is for children under 1 year old with specific brain conditions, including tuberous sclerosis or large brain injuries
Step 2
Medication administration
You will receive Sabrilex (vigabatrin) in the form of granules that need to be dissolved in liquid
The medication will be taken by mouth (oral route)
Your doctor will provide specific instructions about the dosage and how to prepare the solution
Step 3
Monitoring for infantile spasms
Your doctor will regularly check for signs of infantile spasm syndrome
This monitoring helps determine if the preventive treatment is effective
Step 4
Development assessment
Your child's brain development will be evaluated throughout the study
Eye tracking analysis will be performed to assess development
EEG (brain wave) measurements will be taken to monitor brain activity
Step 5
Long-term follow-up
The study continues until December 2033
Regular check-ups will monitor your child's progress and development
Your doctor will assess how well the medication prevents infantile spasms

Who can join the trial?

10 criteria

Child must be under 1 year old
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Child must have one of these conditions:
- Tuberous sclerosis (a rare genetic condition affecting various body parts)
- Large vascular brain injury (significant damage to brain tissue due to bleeding, infection, trauma, or lack of oxygen)
- Grade IV intraventricular hemorrhage (severe bleeding inside brain spaces, including damage to brain tissue)
- Grade III-IV PVL (severe damage to white matter in the brain, including fluid-filled spaces)
- Child must have had a brain MRI scan (magnetic resonance imaging) as part of regular medical care
- Parents must be able to communicate in Finnish, Swedish, or English (or the native language of the research center)
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  For cases of brain injury, the damage must affect either:
  - Almost the entire area supplied by the front or back brain arteries
  - About half the area supplied by the middle brain artery
  - Brain MRI must be performed within the first 5 days for cases of oxygen deprivation injury

Who cannot join the trial?

11 criteria

Children over 1 year of age
Children who do not have tuberous sclerosis (a rare genetic condition causing non-cancerous tumors)
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Children who do not have large vascular brain injuries such as:
- Major strokes
- Brain bleeding
- Brain infections
- Severe brain trauma
- Children without severe brain oxygen deprivation at birth (HIE grades II-III)
- Children without grade IV intraventricular hemorrhage (severe bleeding inside brain cavities)
- Premature babies without grade III-IV PVL (severe brain white matter damage)
- Children without porencephalic cysts (fluid-filled cavities in the brain)
- Patients who started treatment after November 2023 for tuberous sclerosis

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Helsinki University Central Hospital
Helsinki, Finland
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Kuopio University Hospital
Kuopio, Finland
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Oulu University Hospital
Oulu, Finland

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Investigated drugs

Vigabatrin is a medication used to treat and prevent seizures. In this trial, it is being used preventively in infants who are at high risk of developing infantile spasms. The medication works by increasing the levels of a natural calming substance in the brain called GABA. It is particularly effective in treating infantile spasms, especially in children with a condition called tuberous sclerosis. The medication comes in a form that can be given to infants and young children.

Vigabatrin

What is already known about the treatment

Vigabatrin – An anticonvulsant medication administered orally in tablet or powder form that is primarily used to prevent infantile spasms, particularly in high-risk infants under one year of age with conditions such as tuberous sclerosis or large vascular brain injuries. The medication works by increasing the levels of gamma-aminobutyric acid (GABA) in the brain by irreversibly inhibiting the enzyme GABA transaminase, which helps reduce abnormal electrical activity in the brain that can lead to seizures. As a specialized antiepileptic drug, vigabatrin is particularly effective in pediatric epilepsy management and is often used as a preventive treatment in cases where there is a high risk of developing infantile spasm syndrome, though it requires careful monitoring due to potential side effects including vision problems.

Investigated diseases

Infantile Spasm Syndrome
A rare epileptic disorder that typically begins in the first year of life, characterized by sudden, brief stiffening of the body, arms, and legs. The spasms tend to occur in clusters and are most common in the morning or after naps. This condition can affect brain development and is often associated with underlying brain conditions.
Tuberous Sclerosis
A genetic disorder that causes non-cancerous tumors to grow in various parts of the body, particularly the brain, kidneys, heart, lungs, and skin. The condition typically becomes apparent in early childhood. It can cause patches on the skin, seizures, and developmental issues. The severity and symptoms vary widely among affected individuals.
Hypoxic-Ischemic Encephalopathy (HIE)
A type of brain dysfunction that occurs when the brain doesn't receive enough oxygen or blood flow. It can affect various areas of the brain and may lead to developmental challenges. The condition develops shortly after birth and can vary in severity.
Periventricular Leukomalacia (PVL)
A type of brain injury that affects white matter in the brain, most commonly seen in premature infants. The condition involves the death of small areas of brain tissue around the fluid-filled ventricles. It can affect the transmission of nerve signals in the brain.