Condition: Dravet Syndrome (DS) · Sponsor: Second Affiliated Hospital of Guangzhou Medical University
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.
This description comes directly from the study's public registry record.
Weiping Liao, Ph.D · 086-020-34152498 · wpliao@163.net
Always discuss trial participation with your own doctor first.
| The Second Affiliated Hospital of Guangzhou Medical University | Guangzhou, Guangdong, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT07675746