Finding studies
Finding studies
Take this into the appointment.
Saves the questions and what to expect into your notes, next to the visit they belong to.
Weiping Liao, Ph.D
CONTACT
Lead
Second Affiliated Hospital of Guangzhou Medical University
With
This is an open-label, single-center clinical study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of RC001 administered intrathecally in patients aged 2 to 18 years with Dravet syndrome. The study consists of two stages: Stage 1 (intra-subject dose escalation) and Stage 2 (fixed-dose multiple dosing). In Stage 1, three cohorts will be enrolled with a total of three participants. In Stage 2, a total of five participants will be enrolled to receive fixed-dose multiple administrations. Participants in both the dose-escalation stage and the fixed-dose multiple dosing stage may enter an extension phase after completion of the last dose, based on the investigator's assessment of efficacy and safety.
Age
2–18
Sex
ALL
Healthy volunteers
Not accepted
