ClinicalTrials.gov record NCT07675746

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

An Investigator-Initiated Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of RC001 in Patients With Dravet Syndrome Aged 2 to 18 Years

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhaseEarly phase 1A very early study, usually in a small number of people, to learn how the body handles a treatment. It is not designed to show whether the treatment works.
Age2 years old to 18 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This open-label study administers RC001 into the spinal fluid of children and adolescents aged 2–18 years with Dravet syndrome. It has two stages: a small intra-subject dose-escalation stage (three participants) followed by a fixed-dose multiple-administration stage (five participants); some may enter an extension phase. The study collects safety data, measures drug levels in blood and cerebrospinal fluid, and gathers early information on seizures and developmental measures.

What it is studying. The study is testing the safety and tolerability of RC001 given into the spinal fluid, how the drug is absorbed and distributed (pharmacokinetics) in plasma and cerebrospinal fluid, and collecting preliminary data on seizure frequency and clinical/developmental measures after treatment in children with Dravet syndrome.

What the researchers hope to learn. To evaluate treatment-emergent and serious adverse events and clinically significant changes in vital signs, exams, labs, and ECGs; to characterize pharmacokinetic measures (plasma Cmax and Tmax, CSF trough concentrations); to collect preliminary measures of change in countable seizure frequency at 12 and 24 weeks after the last dose; and to assess clinician- and caregiver-rated global impression scores and changes on adaptive and cognitive scales.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • RC001 injection-Dose Escalation Cohort
  • RC001 injection-Fixed Dose Cohort

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Patients aged 2-18 years with Dravet syndrome caused by SCN1A mutations, with onset before 12 months of age characterized by focal seizures, hemiclonic seizures, generalized tonic-clonic seizures, or myoclonic seizures, and with MRI excluding progressive neurological disease either historically or at screening. Enrolled participants will be assigned as follows: 1 participant aged 13-18 years, 1 aged 7-12 years, and 1 aged 2-6 years will undergo intra-subject dose escalation; 5 participants aged 2-12 years will receive fixed-dose multiple administrations.
  • Seizure frequency requirements: at least 6 cumulative seizures within 12 weeks prior to the day of signing the ICF, and at least 2 seizures within 4 weeks prior to ICF signing. For participants in Stage 2 (fixed-dose multiple administration), seizure frequency must also be ≥4 within 4 weeks after ICF signing.
  • Documented pathogenic or likely pathogenic variants in the SCN1A gene associated with Dravet syndrome.
  • Prior treatment with at least one anti-epileptic intervention, including anti-seizure medications (ASM), ketogenic diet, or vagus nerve stimulation (VNS), with inadequate seizure control or discontinuation due to adverse events (AEs).
  • Use of at least one ASM prior to screening, with a stable dose for at least 4 weeks before screening.
  • All epilepsy-related treatments, including ASM and other interventions (ketogenic diet and VNS), must be stable for at least 4 weeks prior to screening and are expected to remain stable throughout the study (medications adjusted by body weight are allowed).
  • Willingness to participate and provision of written informed consent.

Exclusion criteria

  • Presence of other known pathogenic gene mutations causing Dravet syndrome, or SCN1A gain-of-function mutations reported in the literature and/or experimentally validated, including but not limited to: Ala23Glu, Thr162Ile, Thr226Met, Ser228Pro, Val229Leu, Ile236Val, Ile236Thr, Val250Leu, Leu263Val, Thr398Met, Ala420Val, Val422Leu, Ile883Thr, Leu893Phe, Ala989Thr, Thr1174Ser, Trp1204Arg, Ala1339Asp, Pro1345Ser, Pro1345Leu, Ser1346Pro, Ile1347Val, Val1481Ile, Ile1483Met, Gln1489Lys, Ile1498Thr, Ile1498Met, Phe1499Leu, Met1500Val, Arg1575Cys, Val1611Phe, Leu1624Pro, Arg1636Gln, Arg1648Cys, Leu1649Gln, Leu1660Ile, Phe1661Leu, Ala1669Glu, Leu1670Trp, Gly1674Arg, Phe1774Ser, Asp1866Tyr.
  • Current maintenance treatment with anti-epileptic drugs primarily acting as sodium channel blockers, including but not limited to carbamazepine, oxcarbazepine, lamotrigine, lacosamide, or rufinamide.
  • Ongoing neuromodulation therapy (e.g., responsive neurostimulation or deep brain stimulation), excluding vagus nerve stimulation (VNS).
  • Receipt of gene therapy or cell therapy within 1 year prior to screening.
  • Receipt of any vaccination within 12 weeks prior to screening.
  • Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2× the upper limit of normal (ULN), or total bilirubin >1.5× ULN; renal insufficiency or serum creatinine >1.2× ULN.
  • Presence of any severe uncontrolled disease other than Dravet syndrome.
  • History of autoimmune disease, or uncontrolled infectious disease within 1 week prior to screening.
  • History of brain or spinal cord disease (other than epilepsy, Dravet syndrome, or trauma), or history of bacterial meningitis.
  • Spinal deformity or other conditions that may interfere with normal cerebrospinal fluid (CSF) flow, or implantation of a CSF shunt.
  • Pregnant or breastfeeding females.
  • Any other significant disease or condition that, in the investigator's judgment, may pose a risk to the patient, interfere with study results, or affect the patient's ability to participate in the study.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will receive RC001 by intrathecal administration (spinal injection/lumbar puncture) in a small, open‑label dose‑escalation group or a fixed‑dose group, with possible entry into an extension phase after the last dose. They will have repeated clinic visits for safety monitoring (physicals, vital signs, ECGs, laboratory tests), blood and cerebrospinal fluid sampling related to drug levels, and recording of seizures. Caregivers and clinicians will complete seizure diaries and global impression questionnaires; selected developmental and cognitive tests (e.g., Vineland, age‑appropriate Wechsler scale) will be done at baseline and follow‑up. Follow‑up for safety and study assessments continues for weeks after dosing as specified in the protocol.

How the study is structured. Interventional, open-label (no masking), non-randomized, single-group study. Treatment is given intrathecally (into the spinal fluid). Stage 1 is intra-subject dose escalation with three participants; Stage 2 enrols five participants at a fixed dose for multiple administrations. Safety and clinical assessments are collected during treatment and for weeks after the last dose as defined in the protocol.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry lists this study under Dravet Syndrome (DS). Does it apply to my diagnosis?
  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on CDKL5 deficiency disorder

This trial is also listed on this site under Dravet syndrome.

Clinical trials22 trials listed for CDKL5 deficiency disorderEvery trial the registry lists for CDKL5 deficiency disorder, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in CDKL5 deficiency disorder researchLatest monthly update: September 2026. Includes: EEG background changes. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Dec 22, 2025
Primary completion
Dec 31, 2027 (estimated)
Study completion
Dec 31, 2027 (estimated)
Target enrollment
8 participants (estimated)
Last updated on registry
Jun 30, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Second Affiliated Hospital of Guangzhou Medical University
Collaborators
  • RecoRNA(Guangzhou) Biotechnology Co., Ltd

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
pharmacokinetics
How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
intrathecal
Given by injection into the fluid around the spinal cord, usually through a lumbar puncture.
dose-escalation
Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
MRI
A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
ICF
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Prior treatment
Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
adverse events
Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
stable dose
A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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