Research and Clinical Trials

The Division’s research focuses on a wide range of neurological disorders, including mitochondrial disease, movement disorders, epilepsy, Angelman’s syndrome, and cognitive impairment associated with autism spectrum disorders and genetic/metabolic disorders.

Clinical Trials

Phase 3 Clinical Trial for Eteplirsen for Boys with Duchenne Muscular Dystrophy

Eteplirsen is an experimental drug in development to treat about 13 percent of people with Duchenne muscular dystrophy, which is caused by specific mutations (flaws) in the gene for the muscle protein dystrophin. The goal of treatment with eteplirsen — known as an exon-skipping drug — is to coax muscle cells to reinterpret genetic information for the dystrophin protein so that functional dystrophin protein can be made even though the dystrophin gene is flawed.

Eligibility: Boys with Duchenne muscular dystrophy between the ages of 7 and 16

To learn more and to see if your child may quality for the study, contact Dr. Richard Haas at rhaas@rchsd.org or 858-966-6246.

NEOLEV2 Efficacy of Intravenous Levetiracitam in the Treatment of Neonatal Seizures / NEOLEV2

This study hopes to improve treatment of neonatal seizures in this vulnerable and under researched population. Levetiracetam (LEV or trade name Keppra) is a very promising new treatment for neonatal seizures. Current treatments are poorly effective and have significant side effects. This medicine has been used in oral form in older children for over a decade, but experience with use in babies is limited.

The study will compare effects of LEV vs phenobarbital (the traditional treatment for neonatal seizures), obtain essential safety & efficacy data, and develop remote EEG monitoring systems that will facilitate rapid seizure detection for quicker treatment.

To learn more about the study, contact Dr. Richard Haas at rhaas@rchsd.org or 858-966-8068.

T-Force Study (Not currently recruiting): The primary purpose of this study is to determine the safety and tolerability of an investigational medication for Tourette syndrome. Each child who qualifies will receive study medication, study-related medical exams and laboratory tests at no cost.

Eligibility critera: Boys and girls ages 6 to 18 with Tourette syndrome

Compensation for time and travel may be available.