Stoke Therapeutics recently began the Monarch clinical trial and just announced that they have successfully administered STK-001 to the first patient in their trial. STK-001 is a novel disease-modifying therapy that utilizes RNA antisense oligonucleotides (ASOs) to restore levels of SCN1A gene expression in the affected neurons of individuals with Dravet syndrome. STK-001 is administered via intrathecal injection, similar to a spinal tap, and researchers expect patients will need regular injections (likely every 3-6 months). While it is important for the integrity of a clinical trial that enrolled participants usually remain anonymous, the protocol for the Monarch trial was to begin with the lower dose of STK-001 in two patients aged 13-18 years, followed by two patients aged 2-12 years. Every step of the clinical trial will be closely monitored, but given no adverse events, an additional 3 patients may be included in this lower dose group. Future plans will be to repeat this age grouping in a second arm of the study at a higher dose of STK-001. This is a Phase 1/2a study, which means a primary focus will be evaluating the safety and pharmacokinetics of STK-001. However, as all participants will be patients with Dravet syndrome, the study will also be evaluating the effect of STK-001 on seizure frequency, clinical presentation, and quality of life.
For more details, you can re-watch a webinar by Stoke Therapeutics for the DSF community that aired August 13,2020.
More information on participating in this study can be found here or at clinicaltrials.gov.
For more information on the basic science behind the development of STK-001, see this research review of Stoke Therapeutics’ recent publication in Nature Communications.