drugset / Trial / NCT07675746
A Study to Evaluate the Safety and Pharmacokinetics of RC001 in Children With Dravet Syndrome
Phase 0
Recruiting
8 enrolled
Second Affiliated Hospital of Guangzhou Medical University
RecoRNA(Guangzhou) Biotechnology Co., Ltd · collab
Non-randomizedSingle-groupOpen-labelTreatment
Summary
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.
Timeline
- Start
- 2025-12-22
- Primary completion
- 2027-12-31
- Completion
- 2027-12-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | RC001 | Unknown | — | Intrathecal |