Research on Cognitive Effect of Cannabidiol on Dravet Syndrome and Lennox-Gastaut SyndromeGastaut Syndrome

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Brief Title

Research on Cognitive Effect of Cannabidiol on Dravet Syndrome and Lennox-Gastaut SyndromeGastaut Syndrome

Official Title

A Prospective Multi-Center Single-Arm Clinical Trial on Cognitive Effect of Cannabidiol (CBD-OS®) on Dravet Syndrome and Lennox-Gastaut Syndrome

Brief 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. 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:

        1. Age: 2 years old to 18 years old

        2. Investigation center: Severance hospital, Yonsei University College of Medicine, Seoul,
           Korea

        3. A) Diagnosed as Dravet syndrome by clinical manifestations and/or genetic test B)
           Diagnosed as Lennox-Gastaut syndrome by clinical manifestations and
           electroencephalography

        4. The diagnosed patients by criteria 3-(1) or 3-(2) with intractable seizures after using
           over two antiepileptic medications

        5. Patients and caregivers who agreed to participation in this trial

      Exclusion criteria of the patients are as below:

        1. Patients with possibility of progressive central nervous system disease other systemic
           disease

        2. 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)

        3. 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

        4. Patient with hypersensitivity or allergic reactions to cannabidiol

        5. 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:

      A) 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.

      B) 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 Phase

Phase 3

Study Type

Interventional


Primary Outcome

development assessment

Secondary Outcome

 Behavior assessment

Condition

Dravet Syndrome

Intervention

Cannabidiol

Study Arms / Comparison Groups

 CBD
Description:  The patient would be on cannabidiol for 24 weeks and would go through laboratory, electroencephalography, and neuropsychological tests before, during, and after intervention.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

107

Start Date

March 30, 2020

Completion Date

June 2021

Primary Completion Date

March 2021

Eligibility Criteria

        Inclusion Criteria:

          1. Age: 2 years old to 18 years old

          2. Investigation center: Severance hospital, Yonsei University College of Medicine,
             Seoul, Korea

          3. (1) Diagnosed as Dravet syndrome by clinical manifestations and/or genetic test (2)
             Diagnosed as Lennox-Gastaut syndrome by clinical manifestations and
             electroencephalography

          4. The diagnosed patients by criteria 3-(1) or 3-(2) with intractable seizures after
             using over two antiepileptic medications

          5. Patients and caregivers who agreed to participation in this trial

        Exclusion Criteria:

          1. Patients with possibility of progressive central nervous system disease other systemic
             disease

          2. 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)

          3. 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

          4. Patient with hypersensitivity or allergic reactions to cannabidiol

          5. patients who are incapable of executing cognitive tests, or cases whom the
             investigators consider inappropriate to be enrolled in the trial
      

Gender

All

Ages

2 Years - 18 Years

Accepts Healthy Volunteers

No

Contacts

, +82-2-2228-2050, [email protected]

Location Countries

Korea, Republic of

Location Countries

Korea, Republic of

Administrative Informations


NCT ID

NCT04611438

Organization ID

4-2020-0120


Responsible Party

Sponsor

Study Sponsor

Yonsei University


Study Sponsor

, , 


Verification Date

October 2020