An Early Phase 1 interventional study of RC001 injection-Dose Escalation Cohort and RC001 injection-Fixed Dose Cohort in Dravet Syndrome (DS), sponsored by Second Affiliated Hospital of Guangzhou Medical University. Recruiting at 1 site in China. Open to participants aged 2 Years to 18 Years. Per ClinicalTrials.gov, last updated 2026-06-30.
Sponsored by Second Affiliated Hospital of Guangzhou Medical University · Early Phase 1, Interventional, and Treatment
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 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.
92 studies on the registry are indexed under Epilepsies, Myoclonic; 21 are open to participants now.
This study's planned enrollment of 8 is below the median of 31 across 56 interventional studies indexed under Epilepsies, Myoclonic.
Browse Epilepsies, Myoclonic studies →Second Affiliated Hospital of Guangzhou Medical University is the lead sponsor of 74 studies on the registry; 53 are open to participants now.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive RC001 in a dose-escalation manner to evaluate the safety, tolerability, and preliminary pharmacodynamic effects. Dose levels will be administered sequentially, and escalation decisions will be based on safety data from previously treated participants. This cohort includes 3 participants.
Drug: RC001 injection-Dose Escalation Cohort
Participants will receive a predefined fixed dose of RC001 selected based on safety, tolerability, and pharmacological data obtained from the dose-escalation cohort. This cohort is designed to further evaluate safety and preliminary efficacy at the selected dose level. This cohort includes 5 participants.
Drug: RC001 injection-Fixed Dose 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.
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.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
A treatment-emergent adverse event is defined as any adverse event that occurs or worsens after the first dose of RC001 through the end of follow-up. TEAEs will be summarized by system organ class, preferred term, severity, seriousness, and relationship to study drug or study procedure.
Time frame: From first dose to 24 weeks after the last dose
Number of Participants With Serious Adverse Events (SAEs)
Serious adverse events will be collected and summarized throughout the study.
Time frame: From signing informed consent to 24 weeks after the last dose
Number of Participants With Clinically Significant Abnormalities in Vital Signs
Vital signs include body temperature, heart rate, respiratory rate, systolic blood pressure, and diastolic blood pressure. Clinically significant abnormalities will be determined by the investigator.
Time frame: From baseline to 24 weeks after the last dose
Number of Participants With Clinically Significant Abnormal Physical Examination Findings
Physical examination findings will be assessed for clinically significant abnormalities as determined by the investigator.
Time frame: From baseline to 24 weeks after the last dose
Number of Participants With Clinically Significant Abnormal Laboratory Test Results
Laboratory assessments may include hematology, serum chemistry, coagulation, urinalysis, and cerebrospinal fluid laboratory tests, as applicable. Clinically significant abnormalities will be determined by the investigator.
Time frame: From baseline to 24 weeks after the last dose
Number of Participants With Clinically Significant Abnormal 12-Lead Electrocardiogram (ECG) Findings
ECG parameters may include heart rate, PR interval, QRS duration, QT interval, and corrected QT interval. Clinically significant abnormalities will be determined by the investigator.
Time frame: From baseline to 24 weeks after the last dose
Maximum Observed Plasma Concentration (Cmax) of RC001
Cmax of RC001 in plasma following intrathecal administration.
Time frame: From first dose to last dose up to 12 weeks
Time to Maximum Observed Plasma Concentration (Tmax) of RC001
Tmax of RC001 in plasma following intrathecal administration.
Time frame: From first dose through 12 weeks
Trough Concentration (Ctrough) of RC001 in Cerebrospinal Fluid
Trough concentration of RC001 in cerebrospinal fluid before each intrathecal dose.
Time frame: Prior to each dose through 12 weeks
Percentage Change From Baseline in Countable Seizure Frequency at 12 Weeks After the Last Dose
Countable seizure frequency will be calculated as the number of countable seizures during the 28-day period preceding 12 weeks after the last dose of RC001.Baseline countable seizure frequency is defined as the number of countable seizures during baseline observation period. Percentage change from baseline will be calculated as: (post-baseline 28-day seizure frequency - baseline 28-day seizure frequency) / baseline 28-day seizure frequency x 100%.
Time frame: Baseline and the 28-day period preceding 12 weeks after the last dose
Percentage Change From Baseline in Countable Seizure Frequency at 24 Weeks After the Last Dose
Countable seizure frequency will be calculated as the number of countable seizures during the 28-day period preceding 24 weeks after the last dose of RC001. Baseline countable seizure frequency is defined as the number of countable seizures during baseline observation period. Percentage change from baseline will be calculated as: (post-baseline 28-day seizure frequency - baseline 28-day seizure frequency) / baseline 28-day seizure frequency x 100%.
Time frame: Baseline and the 28-day period preceding 24 weeks after the last dose
Clinical Global Impression of Change (CGI-C) Score at 24 Weeks After the Last Dose
The Clinical Global Impression of Change (CGI-C) is a clinician-rated 7-point scale assessing overall clinical change relative to baseline. Scores range from 1 to 7: 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse. Lower scores indicate greater improvement.
Time frame: 24 weeks after the last dose
Caregiver Global Impression of Change (CaGI-C) Score at 24 Weeks After the Last Dose
The Caregiver Global Impression of Change (CaGI-C) is a caregiver-rated 7-point scale assessing overall clinical change relative to baseline. Scores range from 1 to 7: 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse. Lower scores indicate greater improvement.
Time frame: 24 weeks after the last dose
Change From Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Expressive Communication Raw Score at 24 Weeks After the Last Dose
The Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) measures adaptive behavior. The Expressive Communication subdomain raw score will be assessed. Raw scores have a minimum value of 0, and the maximum value varies by subdomain according to the Vineland-3 scoring manual. Higher scores indicate better adaptive functioning.
Time frame: Baseline and 24 weeks after the last dose
Change From Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Receptive Communication Raw Score at 24 Weeks After the Last Dose
The Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) measures adaptive behavior. The Receptive Communication subdomain raw score will be assessed. Raw scores have a minimum value of 0, and the maximum value varies by subdomain according to the Vineland-3 scoring manual. Higher scores indicate better adaptive functioning.
Time frame: Baseline and 24 weeks after the last dose
Change From Baseline in Age-Appropriate Wechsler Intelligence Scale Score at 24 Weeks After the Last Dose
Cognitive function will be assessed using an age-appropriate Wechsler intelligence scale. The selected composite or total score will be analyzed as the change from baseline. Higher scores indicate better cognitive functioning. The applicable scale version and score range will be defined according to the participant's age and the study assessment manual.
Time frame: Baseline and 24 weeks after the last dose
Plan to share: No — Individual participant data (IPD) from this early-phase study will not be shared due to the sensitive nature of patient data and the need to protect participant privacy.
No publications or documents are linked to this record.
Started Dec 2025 (actual). Primary completion is estimated for Dec 2027, 24 months after the start.
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Second Affiliated Hospital of Guangzhou Medical University