Clinical trial NCT04721691: Efficacy of Epidiolex in Patients With Electrical Status Epilepticus of Sleep (ESES).
Official title: Efficacy of Epidiolex in Patients With Electrical Status Epilepticus of Sleep (ESES).
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Electrical Status Epilepticus of Slow-Wave Sleep
Keywords: Neurology
Pediatric
ESES
Lead sponsor: Northwell Health
Sponsor class: OTHER
Collaborators: name: Jazz Pharmaceuticals
First posted: 2021-01-25
Enrollment: 16
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
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.
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Frequency of Spike Wave Index
timeFrame: Up to 20 weeks
Secondary outcomes:
measure: Likert Scale Assessment
timeFrame: Up to 20 weeks
Other outcomes:
Locations:
city: Lake Success
country: United States
facility: Northwell Health
state: New York
Study officials:
References: