An Open-Label Study to Investigate the Safety and Pharmacokinetics of Single Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome

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An Open-Label Study to Investigate the Safety and Pharmacokinetics of Single Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome

Brief description of study

The purpose of this study is to see how safe and effective a drug called STK-001 is for people with Dravet syndrome. This is an experimental drug. “Experimental” means that the study drug is currently being tested and is not approved for sale in the United States by the Food and Drug Administration (FDA). Dravet syndrome is a severe form of epilepsy (a brain disorder that causes seizures). There are several types of therapies available to help reduce the number of seizures. However, most of the current anti-seizure therapies do not work well to reduce the number of seizures or prevent seizures from happening.

Clinical Study Identifier: s20-00617

ClinicalTrials.gov Identifier: NCT04442295

Principal Investigator: Orrin Devinsky.

Other Investigators: Erin K Conway, Cassandra Kazl, Judith Bluvstein, Daniel Friedman.

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