A Study to Evaluate the Safety and Pharmacokinetics of RC001 in Children With Dravet Syndrome

This is an open-label, single-center study to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of intrathecal RC001 in patients with Dravet syndrome aged 2 to 18 years. The study includes a dose-escalation part followed by a fixed dose treatment part, with participant progression based on investigator-assessed safety and efficacy.

NCT Number	NCT07675746
Lead Sponsor	Second Affiliated Hospital of Guangzhou Medical University Collaborators: RecoRNA(Guangzhou) Biotechnology Co., Ltd
Conditions	Dravet Syndrome (DS)
Enrollment	8 participants
Start Date	2025-12-22
Primary Completion	2027-12-31 (estimated)
Study Completion	2027-12-31 (estimated)
Updated on ClinicalTrials.gov	2026-06-30