Texas-led researchers are recruiting children for a Phase 2b clinical trial that will investigate whether vigabatrin can prevent or delay the onset of epileptic seizures in youngsters with tuberous sclerosis.
The McGovern Medical School at The University of Texas Health Science Center at Houston (UTHealth) is heading the study, which will enroll patients at seven other U.S. medical facilities as well.
“It could potentially be a game-changer for epilepsy in general as it is the first trial ever aimed at preventing seizures from developing in a vulnerable population,” Dr. Mary K. Koenig, a professor at UTHealth, said in a press release. She will be the lead investigator of the part of the trial taking place at UTHealth.
Tuberous sclerosis complex (TSC) is a genetic disorder characterized by tumors in the brain and other organs. The tumors can affect the normal functioning of brain neurons, leading to seizures, developmental problems, intellectual disabilities and autism. About 80 percent of children with TSC develop epilepsy in their first three years, according to studies at Harvard.
The three-year trial, called Preventing Epilepsy Using Vigabatrin in Infants with Tuberous Sclerosis Complex (NCT02849457), is expected to cover 80 infants with TSC.
Its main objective is to see whether vigabatrin extends the period between a child developing abnormal brain activity and the onset of seizures. This usually takes only two to three months. Researchers hope early vigabatrin treatment will prevent or delay the onset of seizures.
The team will use electroencephalograms to check for signs of abnormal brain activity. Children who show such activity will receive either vigabatrin, an anticonvulsant, or a placebo.
When a child has a seizure, they will receive a standard therapy for infants with TSC and epileptic seizures.
In addition to assessing the onset and severity of epileptic seizures, researchers will assess the children’s development.
Several molecular mechanisms contribute to the electrical signals in the brain that neurons use to communicate. The signals are faulty in epilepsy, causing a signaling overload that triggers seizures.