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

Back to results

Primary Purpose

Status

Recruiting

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Epidiolex 100 mg/mL Oral Solution

Placebo

About this trial

This is an interventional treatment trial for Electrical Status Epilepticus of Slow-Wave Sleep focused on measuring Neurology, Pediatric, ESES

Eligibility Criteria

2 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Northwell HealthRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

IP

Placebo

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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).

Full Information

NCT ID

NCT04721691

First Posted

September 24, 2020

Last Updated

February 22, 2023

Sponsor

Northwell Health

Collaborators

Jazz Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT04721691

Brief Title

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

2. Study Status

Record Verification Date

February 2023

Overall Recruitment Status

Recruiting

Study Start Date

March 10, 2021 (Actual)

Primary Completion Date

January 2024 (Anticipated)

Study Completion Date

December 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Northwell Health

Collaborators

Jazz Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

No

Product Manufactured in and Exported from the U.S.

No

Data Monitoring Committee

No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Electrical Status Epilepticus of Slow-Wave Sleep

Keywords

Neurology, Pediatric, ESES

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Crossover Assignment

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

ParticipantInvestigator

Masking Description

All study personnel aside from the institution's pharmacist will remain blinded for the duration of this study.

Allocation

Randomized

Enrollment

34 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

IP

Arm Type

Active Comparator

Arm Description

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.

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

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.

Intervention Type

Drug

Intervention Name(s)

Epidiolex 100 mg/mL Oral Solution

Intervention Description

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.

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

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

Primary Outcome Measure Information:

Title

Frequency of Spike Wave Index

Description

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

Time Frame

Up to 20 weeks

Secondary Outcome Measure Information:

Title

Likert Scale Assessment

Description

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).

Time Frame

Up to 20 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

No

Eligibility Criteria

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Sanjeev V. Kothare, MD

Phone

(516) 465-5255

Email

skothare@northwell.edu

First Name & Middle Initial & Last Name or Official Title & Degree

Aaqil Ali

Phone

(516) 465-5255

Email

aali27@northwell.edu

Facility Information:

Facility Name

Northwell Health

City

Lake Success

State/Province

New York

ZIP/Postal Code

11042

Country

United States

Individual Site Status

Recruiting

Electrical Status Epilepticus of Slow-Wave Sleep

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial