Dravet SyndromePhase 3NCT04611438
Research on Cognitive Effect of Cannabidiol on Dravet Syndrome and Lennox-Gastaut SyndromeGastaut Syndrome
A Phase 3 study testing Cannabidiol in people with Dravet Syndrome and Lennox Gastaut Syndrome, run by Yonsei University at 1 site in South Korea.
Trial summary
The clinical trial "A Prospective Single-Center Single-Arm Clinical Trial on Cognitive Effect of Cannabidiol (CBD-OS®) on Dravet syndrome and Lennox-Gastaut Syndrome" is a single-group phase III study done in single tertiary referral center in Seoul, Korea. Chief investigator is Dr. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Dravet Syndrome, Lennox Gastaut Syndrome
- What is being tested?
- Cannabidiol
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 107 participants
- Where
- 1 siteSouth Korea
- Study dates
- 30 Mar 2020to Jun 2021
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age: 2 years old to 18 years old
- Investigation center: Severance hospital, Yonsei University College of Medicine, Seoul, Korea
- (1) Diagnosed as Dravet syndrome by clinical manifestations and/or genetic test (2) Diagnosed as Lennox-Gastaut syndrome by clinical manifestations and electroencephalography
- The diagnosed patients by criteria 3-(1) or 3-(2) with intractable seizures after using over two antiepileptic medications
- Patients and caregivers who agreed to participation in this trial
Who cannot join
- Patients with possibility of progressive central nervous system disease other systemic disease
- The patients who started additional antiepileptic medication during the investigation period, with exception of short-term acute treatment of seizures (e.g. benzodiazepine for rescue therapy)
- Patients with alanine and aspartate aminotransferase and/or total bilirubin levels over 2 times of the normal range, or blood urea nitrogen and creatinine ratio over 3 times of the normal range
- Patient with hypersensitivity or allergic reactions to cannabidiol
- patients who are incapable of executing cognitive tests, or cases whom the investigators consider inappropriate to be enrolled in the trial
Trial locations and contacts
1 site in 1 country.
South Korea1 site, 1 recruiting
- Recruiting
Severance Hospital, Yonsei University College of Medicine
Seoul
Hoon-Chul Kang, MD, 82-2-2228-2050, hipo0207@yuhs.ac
About this study
Official title: A Prospective Multi-Center Single-Arm Clinical Trial on Cognitive Effect of Cannabidiol (CBD-OS®) on Dravet Syndrome and Lennox-Gastaut Syndrome
The clinical trial "A Prospective Single-Center Single-Arm Clinical Trial on Cognitive Effect of Cannabidiol (CBD-OS®) on Dravet syndrome and Lennox-Gastaut Syndrome" is a single-group phase III study done in single tertiary referral center in Seoul, Korea.
Chief investigator is Dr. Hoon-Chul Kang of Severance Hospital, Yonsei University College of Medicine. Associate investigators are Dr. Heung Dong Kim, Joon Soo Lee, Se Hee Kim, Han Som Choi, Ji Hoon Na, Dong Hwa Yang, and Hee Jung Kang, of Severance Hospital, Yonsei University College of Medicine.
Read more
The aim of the study is to evaluate the effect of cannabidiol (CBD-OS®) on cognitive functions in patients aged from 2 to 18 years old diagnosed with Dravet syndrome or Lennox-Gastaut syndrome. The duration of study is planned as one year, after patient recruitment of 6 months. The intervention period in each patient is 24 weeks, with 2 weeks of medication titration, stabilization period of 10 weeks, and maintenance period of 12 weeks. The recruitment goal of patient number is 104, considering the study power of 90 percent.
Primary outcomes are improvement of cognitive and development and improvement of seizure outcome. Secondary outcomes are improvement in behavior and quality of life.
Safety monitoring criteria are adverse event profiles and physician's and caregiver's global assessment.
Statistical analysis of outcomes is subject only to the patients who completed the 24-week medication and 2 times of tests before and after treatment of cannabidiol. Evaluation of seizure outcomes would include all patients who completed the 24-week medication and those who dropped out of the study, either by follow-up loss or discontinuation of medication due to incomplete seizure control or adverse effect of the medication.
To evaluate safety, the investigators would measure adverse events and dropout rates by percentage. The investigators would analyze overall evaluation of the caregivers and investigator. Serious adverse events would be noted after causality evaluation.
Read the detailed description
Selection criteria of the patients are as below:
- Age: 2 years old to 18 years old
- Investigation center: Severance hospital, Yonsei University College of Medicine, Seoul, Korea
- A) Diagnosed as Dravet syndrome by clinical manifestations and/or genetic test B) Diagnosed as Lennox-Gastaut syndrome by clinical manifestations and electroencephalography
- The diagnosed patients by criteria 3-(1) or 3-(2) with intractable seizures after using over two antiepileptic medications
- Patients and caregivers who agreed to participation in this trial
Exclusion criteria of the patients are as below:
- Patients with possibility of progressive central nervous system disease other systemic disease
- The patients who started additional antiepileptic medication during the investigation period, with exception of short-term acute treatment of seizures (e.g. benzodiazepine for rescue therapy)
- Patients with alanine and aspartate aminotransferase and/or total bilirubin levels over 2 times of the normal range, or blood urea nitrogen and creatinine ratio over 3 times of the normal range
- Patient with hypersensitivity or allergic reactions to cannabidiol
- patients who are incapable of executing cognitive tests, or cases whom the investigators consider inappropriate to be enrolled in the trial Overall trial schedule for each patient is as follows:
- Baseline Phase (2 weeks)
- Baseline Phase starts from the screening visit, where the patient and caregiver write permission to the trial and goes through laboratory, electroencephalography, and neuropsychological tests.
- Treatment Period : Titration(2 weeks) + stabilization period (10 weeks) + maintenance period (12 weeks)
- Titration: cannabidiol titration up to 10mg/kg/day with the caregiver monitoring the patient's tolerability
- Stabilization: caregiver monitoring the patient's tolerability, with no change in medication dosage
- Maintenance: no change in medication dosage
- Statistical analysis of outcomes would be done as follows:
T-test would be done to compare each test before and after cannabidiol treatment. For multiple results along the intervention time period, repeated data analyses would be done. Continuous variables would be evaluated by repeated measures analysis of variance or generalized estimating equations. Nominal variables would be evaluation by chi-squared test or trend test.
Evaluation of seizure outcomes would include all patients who completed the 24-week medication and those who dropped out of the study, either by follow-up loss or discontinuation of medication due to incomplete seizure control or adverse effect of the medication.
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome
- Being tested
- Cannabidiol
- Keywords
- cannabidiol, pediatric, Dravet syndrome, Lennox-Gastaut syndrome
- Medical subject headings
- Epilepsies, Myoclonic, Lennox Gastaut Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CannabidiolDrug
Intervention schedule for each patient is as follows:
- Baseline Phase (2 weeks) Baseline Phase starts from the screening visit, where the patient and caregiver write permission to the trial and goes through laboratory, electroencephalography, and neuropsychological tests.
- Treatment Period: Titration (2 weeks) + stabilization period (10 weeks) + maintenance period (12 weeks)
- Titration: The patient would start cannabidiol on 5mg/kg/mg for 1 week and titrate dosage up to 10mg/kg/day for second week with the caregiver monitoring the patient's tolerability
- Stabilization: caregiver monitoring the patient's tolerability, with no change in medication dosage
- Maintenance: no change in medication dosage
| Study group | Role | What they receive |
|---|---|---|
| CBD | Experimental | The patient would be on cannabidiol for 24 weeks and would go through laboratory, electroencephalography, and neuropsychological tests before, during, and after intervention. Receives: Drug: Cannabidiol |
What is being measured
The primary outcome is the main question the study is designed to answer.
development assessment
Assessment of cognition and development would be done by Bayley Scales of Infant Development, Wechsler Preschool and Primary Scale of Intelligence, Wechsler Intelligence Scale for Children, Developmental Test of Visual-Motor Integration, and CCTT according to the patient's age and capability.
Measured: 24 weeks
Seizure outcome
Caregivers would report seizure outcome by Caregiver Global Impression of Change in Seizure Duration or Caregiver/patient Global Impression of Change. (CGIC)
Measured: 24 weeks
Secondary outcomes (2)
Behavior assessment
Behavior would be evaluated by Child Behavior Checklist (K-CBCL)
Measured: 24 weeks
Quality of life assessment
Tool for evaluation quality of life is Quality of Life in Childhood Epilepsy questionnaire(K-QOLCE).
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2020
- Study start30 Mar 2020
- Primary completionMar 2021 (estimated)
- Study completionJun 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Dravet SyndromeStudies of the same condition, open and under way.
- More trials run by Yonsei UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Patients would be from 2 to 18 years old diagnosed with Dravet syndrome or Lennox-Gastaut syndrome, who are intractable to more than 2 antiepileptic medications. Caregivers would purchase cannabidiol and give it to the patient according to titration schedule. Patients would go through laboratory, neuropsychologic, and electroencephalographic tests before, during and after the intervention.
- Masking
- None (open label)
- Enrolment
- 107 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yonsei University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04611438
- Sponsor's protocol ID
- 4-2020-0120
- Status last verified
- Oct 2020
- First submitted
- 16 Sep 2019
- Last updated
- 2 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Devinsky O, Cross JH, Laux L, Marsh E, Miller I, Nabbout R, Scheffer IE, Thiele EA, Wright S; Cannabidiol in Dravet Syndrome Study Group. Trial of Cannabidiol for Drug-Resistant Seizures in the Dravet Syndrome. N Engl J Med. 2017 May 25;376(21):2011-2020. doi: 10.1056/NEJMoa1611618.
PubMed 28538134Devinsky O, Patel AD, Cross JH, Villanueva V, Wirrell EC, Privitera M, Greenwood SM, Roberts C, Checketts D, VanLandingham KE, Zuberi SM; GWPCARE3 Study Group. Effect of Cannabidiol on Drop Seizures in the Lennox-Gastaut Syndrome. N Engl J Med. 2018 May 17;378(20):1888-1897. doi: 10.1056/NEJMoa1714631.
PubMed 29768152
Study at a glance
From the registry record, last updated by the sponsor on 2 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Multi-Center Single-Arm Clinical Trial on Cognitive Effect of Cannabidiol (CBD-OS®) on Dravet Syndrome and Lennox-Gastaut Syndrome
- ClinicalTrials.gov ID
- NCT04611438
- Acronym / sponsor ID
- 4-2020-0120
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome
- Keywords
- cannabidiol, pediatric, Dravet syndrome, Lennox-Gastaut syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 107 participants (estimated)
- Lead sponsor
- Yonsei UniversityUniversity, hospital or other
- Study start
- 30 Mar 2020 (actual)
- Primary completion
- Mar 2021 (estimated)
- Study completion
- Jun 2021 (estimated)
- First posted
- 2 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The clinical trial "A Prospective Single-Center Single-Arm Clinical Trial on Cognitive Effect of Cannabidiol (CBD-OS®) on Dravet syndrome and Lennox-Gastaut Syndrome" is a single-group phase III study done in single tertiary referral center in Seoul, Korea.
Chief investigator is Dr. Hoon-Chul Kang of Severance Hospital, Yonsei University College of Medicine. Associate investigators are Dr. Heung Dong Kim, Joon Soo Lee, Se Hee Kim, Han Som Choi, Ji Hoon Na, Dong Hwa Yang, and Hee Jung Kang, of Severance Hospital, Yonsei University College of Medicine.
The aim of the study is to evaluate the effect of cannabidiol (CBD-OS®) on cognitive functions in patients aged from 2 to 18 years old diagnosed with Dravet syndrome or Lennox-Gastaut syndrome. The duration of study is planned as one year, after patient recruitment of 6 months. The intervention period in each patient is 24 weeks, with 2 weeks of medication titration, stabilization period of 10 weeks, and maintenance period of 12 weeks. The recruitment goal of patient number is 104, considering the study power of 90 percent.
Primary outcomes are improvement of cognitive and development and improvement of seizure outcome. Secondary outcomes are improvement in behavior and quality of life.
Safety monitoring criteria are adverse event profiles and physician's and caregiver's global assessment.
Statistical analysis of outcomes is subject only to the patients who completed the 24-week medication and 2 times of tests before and after treatment of cannabidiol. Evaluation of seizure outcomes would include all patients who completed the 24-week medication and those who dropped out of the study, either by follow-up loss or discontinuation of medication due to incomplete seizure control or adverse effect of the medication.
To evaluate safety, the investigators would measure adverse events and dropout rates by percentage. The investigators would analyze overall evaluation of the caregivers and investigator. Serious adverse events would be noted after causality evaluation.
Detailed description
Selection criteria of the patients are as below:
- Age: 2 years old to 18 years old
- Investigation center: Severance hospital, Yonsei University College of Medicine, Seoul, Korea
- A) Diagnosed as Dravet syndrome by clinical manifestations and/or genetic test B) Diagnosed as Lennox-Gastaut syndrome by clinical manifestations and electroencephalography
- The diagnosed patients by criteria 3-(1) or 3-(2) with intractable seizures after using over two antiepileptic medications
- Patients and caregivers who agreed to participation in this trial
Exclusion criteria of the patients are as below:
- Patients with possibility of progressive central nervous system disease other systemic disease
- The patients who started additional antiepileptic medication during the investigation period, with exception of short-term acute treatment of seizures (e.g. benzodiazepine for rescue therapy)
- Patients with alanine and aspartate aminotransferase and/or total bilirubin levels over 2 times of the normal range, or blood urea nitrogen and creatinine ratio over 3 times of the normal range
- Patient with hypersensitivity or allergic reactions to cannabidiol
- patients who are incapable of executing cognitive tests, or cases whom the investigators consider inappropriate to be enrolled in the trial Overall trial schedule for each patient is as follows:
- Baseline Phase (2 weeks)
- Baseline Phase starts from the screening visit, where the patient and caregiver write permission to the trial and goes through laboratory, electroencephalography, and neuropsychological tests.
- Treatment Period : Titration(2 weeks) + stabilization period (10 weeks) + maintenance period (12 weeks)
- Titration: cannabidiol titration up to 10mg/kg/day with the caregiver monitoring the patient's tolerability
- Stabilization: caregiver monitoring the patient's tolerability, with no change in medication dosage
- Maintenance: no change in medication dosage
- Statistical analysis of outcomes would be done as follows:
T-test would be done to compare each test before and after cannabidiol treatment. For multiple results along the intervention time period, repeated data analyses would be done. Continuous variables would be evaluated by repeated measures analysis of variance or generalized estimating equations. Nominal variables would be evaluation by chi-squared test or trend test.
Evaluation of seizure outcomes would include all patients who completed the 24-week medication and those who dropped out of the study, either by follow-up loss or discontinuation of medication due to incomplete seizure control or adverse effect of the medication.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Patients would be from 2 to 18 years old diagnosed with Dravet syndrome or Lennox-Gastaut syndrome, who are intractable to more than 2 antiepileptic medications. Caregivers would purchase cannabidiol and give it to the patient according to titration schedule. Patients would go through laboratory, neuropsychologic, and electroencephalographic tests before, during and after the intervention.
| Arm | Type | Receives |
|---|---|---|
| CBD | Experimental | Drug: CannabidiolThe patient would be on cannabidiol for 24 weeks and would go through laboratory, electroencephalography, and neuropsychological tests before, during, and after intervention. |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Age: 2 years old to 18 years old
- Investigation center: Severance hospital, Yonsei University College of Medicine, Seoul, Korea
- (1) Diagnosed as Dravet syndrome by clinical manifestations and/or genetic test (2) Diagnosed as Lennox-Gastaut syndrome by clinical manifestations and electroencephalography
- The diagnosed patients by criteria 3-(1) or 3-(2) with intractable seizures after using over two antiepileptic medications
- Patients and caregivers who agreed to participation in this trial
Key exclusion criteria (5 listed)
- Patients with possibility of progressive central nervous system disease other systemic disease
- The patients who started additional antiepileptic medication during the investigation period, with exception of short-term acute treatment of seizures (e.g. benzodiazepine for rescue therapy)
- Patients with alanine and aspartate aminotransferase and/or total bilirubin levels over 2 times of the normal range, or blood urea nitrogen and creatinine ratio over 3 times of the normal range
- Patient with hypersensitivity or allergic reactions to cannabidiol
- patients who are incapable of executing cognitive tests, or cases whom the investigators consider inappropriate to be enrolled in the trial
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CannabidiolIntervention schedule for each patient is as follows: 1. Baseline Phase (2 weeks) Baseline Phase starts from the screening visit, where the patient and caregiver write permission to the trial and goes through laboratory, electroencephalography, and neuropsychological tests. 2. Treatment Period: Titration (2 weeks) + stabilization period (10 weeks) + maintenance period (12 weeks) * Titration: The patient would start cannabidiol on 5mg/kg/mg for 1 week and titrate dosage up to 10mg/kg/day for second week with the caregiver monitoring the patient's tolerability * Stabilization: caregiver monitoring the patient's tolerability, with no change in medication dosage * Maintenance: no change in medication dosage | Drug | CBD |
Primary outcome
development assessment
Time frame: 24 weeks
Assessment of cognition and development would be done by Bayley Scales of Infant Development, Wechsler Preschool and Primary Scale of Intelligence, Wechsler Intelligence Scale for Children, Developmental Test of Visual-Motor Integration, and CCTT according to the patient's age and capability.
Seizure outcome
Time frame: 24 weeks
Caregivers would report seizure outcome by Caregiver Global Impression of Change in Seizure Duration or Caregiver/patient Global Impression of Change. (CGIC)
2 secondary outcomes
Behavior assessment
Time frame: 24 weeks
Quality of life assessment
Time frame: 24 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Nov 2020.
- Results publication: PubMed 28538134Devinsky O, Cross JH, Laux L, Marsh E, Miller I, Nabbout R, Scheffer IE, Thiele EA, Wright S; Cannabidiol in Dravet Syndrome Study Group. Trial of Cannabidiol for Drug-Resistant Seizures in the Dravet Syndrome. N Engl J Med. 2017 May 25;376(21):2011-2020. doi: 10.1056/NEJMoa1611618.
- Results publication: PubMed 29768152Devinsky O, Patel AD, Cross JH, Villanueva V, Wirrell EC, Privitera M, Greenwood SM, Roberts C, Checketts D, VanLandingham KE, Zuberi SM; GWPCARE3 Study Group. Effect of Cannabidiol on Drop Seizures in the Lennox-Gastaut Syndrome. N Engl J Med. 2018 May 17;378(20):1888-1897. doi: 10.1056/NEJMoa1714631.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Cannabidiol in people with Dravet Syndrome and Lennox Gastaut Syndrome, run by Yonsei University at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 2 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Hoon-Chul Kang, MD, Ph.D (+82-2-2228-2050, hipo0207@yuhs.ac), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
development assessment (time frame: 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04611438, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Nov 2020.