Electrical Status Epilepticus of Slow-Wave Sleep
Conditions
Keywords
Neurology, Pediatric, ESES
Brief summary
This study aims to assess the efficacy of Epidiolex in patients with ESES. ESES is characterized by sleep potentiated spikes with a spike index greater than 85% (conventional definition) and 50% (new definition)1. Several drugs including: steroids, intravenous Gama globulin, Clobazam, other benzodiazepines, Valproic acid, and other anti-epileptic drugs have been tried with mixed benefits2,3. Cannabidiol (CBD) would provide a novel mechanism of action to assess for its efficacy in this population. This will be a double-blind placebo-controlled crossover clinical trial.
Detailed description
This study attempts to view the effect of Epidiolex on subjects with Electrical Status Epilepticus of Sleep (ESES). ESES can cause various types of seizures which can fluctuate during sleep. Similar studies have been conducted with Epidiolex with other seizure disorders such as Lennox-Gastaut syndrome (LGS) and Dravet Syndrome. Since there is no data available on the effect of Cannabidiol (CBD) on patients with ESES, this study hopes to fill that void. As such, patients that have been diagnosed with ESES will be eligible to participate in this study to ascertain whether or not Epidiolex can reduce the frequency or intensity of the seizures brought on by ESES during sleep.
Interventions
Epidiolex is a schedule 5 controlled substance that is a colorless to yellow oral liquid solution that is prepackaged into 100mL vials with 5mL syringes for use. It is typically used for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet Syndrome in patients 2 years of age and older.
OTHERPlacebo
Placebo is composed of dehydrated alcohol, sesame seed oil, strawberry flavor, and sucralose.
Sponsors
Jazz Pharmaceuticals
Northwell Health
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Masking description
All study personnel aside from the institution's pharmacist will remain blinded for the duration of this study.
Intervention model description
This study aims to assess the efficacy of Epidiolex in patients with ESES. ESES is characterized by sleep potentiated spikes with a spike index greater than 85% (conventional definition) and 50% (new definition). Several drugs including: steroids, intravenous Gama globulin, Clobazam, other benzodiazepines, Valproic acid, and other anti-epileptic drugs have been tried with mixed benefits. Cannabidiol (CBD) would provide a novel mechanism of action to assess for its efficacy in this population4. This will be a double-blind placebo-controlled crossover clinical trial.
Eligibility
Sex/Gender
ALL
Age
2 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
In order to be eligible to participate in this study, an individual must meet all of the following criteria: 1. Provision of signed and dated informed consent form 2. Stated willingness to comply with all study procedures and availability for the duration of the study 3. Male or female, aged 2-17 years old 4. In good general health as evidenced by medical history or diagnosed with ESES. Good health in relation to this study is understood as stable without current seizures requiring immediate hospitalization. 5. Ability to take oral medication and be willing to adhere to the Epidiolex/Placebo regimen 6. For females of reproductive potential: use of highly effective contraception for at least 1 month prior to screening and agreement to use such a method during study participation and for an additional 1 month after the end of oral Epidiolex administration 7. For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner
Exclusion criteria
An individual who meets any of the following criteria will be excluded from participation in this study: 1. Previous use of cannabidiol within 4 months. 2. Pregnancy or lactation 3. Known allergic reactions to components of the Epidiolex: cannabidiol, sesame seed oil, and sucralose 4. Febrile illness within 1 month of screening 5. Treatment with another investigational drug or other intervention within 6 months 6. Current smoker or tobacco use within 6 months
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Frequency of Spike Wave Index | Up to 20 weeks | Assess the reduction in spike wave index by evaluating the differences in activity from the four 24-hour ambulatory EEGs each participant obtains while enrolled in this study |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Likert Scale Assessment | Up to 20 weeks | The secondary objective is to assess seizure counts and subjective behavior change on a 5 point Likert scale (1= no improvement, 2= some improvement, 3=moderate improvement, 4=significant improvement, 5=extreme improvement). |
Countries
United States
Outcome results
None listed