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

NCT07675746 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2026-06-30

No results posted yet for this study

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.

Conditions

  • Dravet Syndrome (DS)

Interventions

DRUG

RC001 injection-Dose Escalation Cohort

RC001 will be administered using a sequential dose-escalation scheme. Participants will receive ascending dose levels of RC001 according to the study protocol. Dose escalation will proceed only after safety data from prior participants have been reviewed and deemed acceptable. This intervention is intended to evaluate safety, tolerability, and preliminary pharmacodynamic effects at increasing dose levels.

DRUG

RC001 injection-Fixed Dose Cohort

RC001 will be administered at a predefined fixed dose level selected based on safety, tolerability, and pharmacological data obtained from the dose-escalation cohort. This intervention is intended to further evaluate safety and preliminary efficacy at the selected dose level in a fixed-dose setting.

Sponsors & Collaborators

  • RecoRNA(Guangzhou) Biotechnology Co., Ltd

    collaborator UNKNOWN

  • Second Affiliated Hospital of Guangzhou Medical University

    lead OTHER

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-12-22
Primary Completion
2027-12-31
Completion
2027-12-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07675746 on ClinicalTrials.gov