Efficacy of Epidiolex in Patients With Electrical Status Epilepticus of Sleep (ESES).

NCT04721691 | Completed Phase 1 FDA Drug

• 1 other identifier: 20-0168
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

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.

Conditions

Electrical Status Epilepticus of Slow-Wave Sleep

Keywords

Neurology; Pediatric; ESES

Outcome Measures

Primary Outcomes (1)

• 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

  Up to 20 weeks

Secondary Outcomes (1)

• Likert Scale Assessment

  Up to 20 weeks

Study Arms (2)

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.

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.

Other: Placebo

Interventions

Epidiolex 100 mg/mL Oral Solution DRUG

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.

IP

Placebo OTHER

Placebo is composed of dehydrated alcohol, sesame seed oil, strawberry flavor, and sucralose.

Placebo

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may not qualify if:

• 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

Sponsors & Collaborators

• Northwell Health: lead
• Jazz Pharmaceuticals: collaborator

Study Sites (1)

Northwell Health

Lake Success, New York, 11042, United States

Location

MeSH Terms

Interventions

Cannabidiol; Solutions

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals; Pharmaceutical Preparations

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: All study personnel aside from the institution's pharmacist will remain blinded for the duration of this study.
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Service Line Chief of Child Neurology

Model Details: 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.

Study Record Dates

• First Submitted: September 24, 2020
• First Posted: January 25, 2021
• Study Start: March 10, 2021
• Primary Completion: January 17, 2024
• Study Completion: January 17, 2024
• Last Updated: June 4, 2025

Record last verified: 2025-05

Locations

US (1)