Electrical Status Epilepticus of Slow-Wave SleepPhase 1NCT04721691
Efficacy of Epidiolex in Patients With Electrical Status Epilepticus of Sleep (ESES).
A Phase 1 study testing Epidiolex 100 mg/mL Oral Solution in people with Electrical Status Epilepticus of Slow-Wave Sleep, run by Northwell Health at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Electrical Status Epilepticus of Slow-Wave Sleep
- What is being tested?
- Epidiolex 100 mg/mL Oral Solution
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Mar 2021to 17 Jan 2024
- Phase
- Phase 1
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 17 years
- All sexes
- No healthy volunteers
- Child
In order to be eligible to participate in this study, an individual must meet all of the following criteria:
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female, aged 2-17 years old
- 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.
- Ability to take oral medication and be willing to adhere to the Epidiolex/Placebo regimen
- 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
- 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:
- Previous use of cannabidiol within 4 months.
- Pregnancy or lactation
- Known allergic reactions to components of the Epidiolex: cannabidiol, sesame seed oil, and sucralose
- Febrile illness within 1 month of screening
- Treatment with another investigational drug or other intervention within 6 months
- Current smoker or tobacco use within 6 months
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- Being tested
- Epidiolex 100 mg/mL Oral Solution, Placebo
- Keywords
- Neurology, Pediatric, ESES
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Epidiolex 100 mg/mL Oral SolutionDrug
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.
PlaceboOther
Placebo is composed of dehydrated alcohol, sesame seed oil, strawberry flavor, and sucralose.
| Study group | Role | What they receive |
|---|---|---|
| IP | Active comparator | Epidiolex (Cannabidiol) is a colorless to yellow solution in a 100mL vial that is to be administered with a 5mL syringe.The starting dosage is 2.5 mg/kg twice daily, or 5 mg/kg/day. After one week, the dosage can be increased to a maintenance dosage of 5mg/kg twice daily, or 10 mg/kg/day. Patients who are tolerating Epidiolex at 10mg/kg per day and require further reduction of seizures may benefit from a dosage increase up to a maximum recommended maintenance. Dosage of 10mg/kg twice daily (20mg/kg/day), in weekly increments of 2.5 mg/kg twice daily(5mg/kg/day), as tolerated. For patients in whom a more rapid titration from 10 mg/kg/day to 20 mg/kg/day is warranted, the dosage may be increased to no more frequently than every other day administration of the 20 mg/kg/day. Dosage resulted in somewhat greater reductions in seizure rates than the recommended maintenance dosage of 10 mg/kg/day, but with an increase in adverse reactions. Receives: Drug: Epidiolex 100 mg/mL Oral Solution |
| Placebo | Placebo comparator | Placebo is a colorless to yellow solution in a 100mL vial that is to be administered with a 5mL syringe. The starting dosage is 2.5 mg/kg twice daily, or 5 mg/kg/day. After one week, the dosage can be increased to a maintenance dosage of 5mg/kg twice daily, or 10 mg/kg/day. Patients who are tolerating Placebo at 10mg/kg per day and require further reduction of seizures may benefit from a dosage increase up to a maximum recommended maintenance. Dosage of 10mg/kg twice daily (20mg/kg/day), in weekly increments of 2.5 mg/kg twice daily(5mg/kg/day), as tolerated. For patients in whom a more rapid titration from 10 mg/kg/day to 20 mg/kg/day is warranted, the dosage may be increased to no more frequently than every other day administration of the 20 mg/kg/day. Dosage resulted in somewhat greater reductions in seizure rates than the recommended maintenance dosage of 10 mg/kg/day, but with an increase in adverse reactions. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Spike Wave Index
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
Measured: Up to 20 weeks
Secondary outcomes (1)
Likert Scale Assessment
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).
Measured: Up to 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2021
- Study start10 Mar 2021
- Primary completion17 Jan 2024
- Study completion17 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Electrical Status Epilepticus of Slow-Wave SleepStudies of the same condition, open and under way.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- 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.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwell Health
- Sponsor type
- University, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Responsible party
- Principal investigator, Sanjeev Vithal Kothare, Northwell Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04721691
- Sponsor's protocol ID
- 20-0168
- Status last verified
- May 2025
- First submitted
- 24 Sep 2020
- Last updated
- 28 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No