Investigation of Cognitive Outcomes With Cannabidiol Oral Solution (EPI-COG)

August 31, 2022 updated by: Jazz Pharmaceuticals

An Open-Label Exploratory Investigation of Cognitive Outcomes With Cannabidiol Oral Solution (EPIDIOLEX®; GWP42003-P)

This study is being conducted to evaluate the effects of GWP42003-P on cognition in pediatric participants, aged 3 to 10 years, with Lennox-Gastaut Syndrome (LGS).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Withdrawn

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This trial is a 30-week (4-week baseline period; 26-week treatment period) open-label exploratory investigation of the effects of GWP42003-P on cognitive abilities in participants with LGS who reside in the United States.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 4

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

3 years to 10 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Key Inclusion Criteria:

  • Participant is male or female aged 3-10 years.
  • Participants' parent(s)/legal representative is willing and able to give informed consent for participation in the trial; where the participant possesses adequate understanding, informed assent should also be taken.
  • Participant and their caregiver are willing and able (in the investigator's opinion) to comply with all trial requirements.
  • Participant must have a clinical diagnosis of Lennox-Gastaut Syndrome (LGS), with onset within the last 5 years. This includes certification from the investigator of prior electroencephalogram (EEG) documenting slow spike wave (< 3 Hertz [Hz]) during the participant's history and evidence of more than 1 type of generalized seizure, including drop seizures (atonic, tonic, or tonic-clonic), for at least 6 months.
  • Investigator can confirm that the addition of GWP42003-P to the participant's existing antiepileptic drug (AED) regimen is warranted.
  • Participant must have at least 1 drop seizure each week during the first 28 days of the baseline period.
  • A minimum level of general intellectual functioning as assessed at screening with the Peabody Picture Vocabulary Test
  • Participant's parent(s)/legal representative is willing to allow the responsible authorities to be notified of participation in the trial, if mandated by local law.
  • Participant's parent(s)/legal representative is willing to allow his or her primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the trial, if the primary care practitioner/consultant is different to the investigator.

Key Exclusion Criteria:

  • Participant has clinically significant unstable medical conditions other than epilepsy.
  • Participant experiences > 300 total seizures within the first 28 days of the baseline period.
  • Participant has any prior exposure to GWP42003-P.
  • Participant has initiated felbamate within the last 12 months.
  • Participant has initiated mammalian target of rapamycin (mTOR) inhibitors for epilepsy within the last 4 weeks.
  • Participant is currently using or has in the past used recreational or medicinal cannabis or synthetic cannabinoid-based medications (including Sativex®) within the 3 months prior to trial entry.
  • Participant has had clinically relevant symptoms or a clinically significant illness, other than epilepsy, in the 4 weeks prior to screening or Visit 2.
  • Participant has laboratory values at screening or Visit 2 that are clinically significantly abnormal in the investigator's opinion.
  • Participant tests positive for Δ9-tetrahydrocannabinol (THC) or cannabidiol (CBD) at screening.
  • Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of GWP42003-P.

  • Participant has significantly impaired hepatic function at the screening visit, defined as any of the following:

    • Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 5 × upper limit of normal (ULN);
    • Serum ALT or AST > 3 × ULN and (total bilirubin [TBL] > 2 × ULN or international normalized ratio [INR] > 1.5);
    • Serum ALT or AST > 3 × ULN with the presence of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia (> 5%).
  • Participant has received an investigational medical product within the 3 months prior to the screening visit.
  • Participant has any other significant disease or disorder, which, in the opinion of the investigator, may either put the participant at risk because of participation in the trial, may influence the result of the trial, or may affect the participant's ability to take part in the trial.
  • Any abnormalities identified following a physical examination of the participant that, in the opinion of the investigator, would jeopardize the safety of the participant if he/she took part in the trial
  • Participant has been previously enrolled into this trial.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: GWP42003-P
For the first 7 days of the treatment period, participants are to take GWP42003-P at a dose of 5 milligrams per kilogram per day (mg/kg/day), administered as 2 equally divided doses (i.e., 2.5 mg/kg in the morning and 2.5 mg/kg in the evening). On Day 8, participants are to increase the dose to 10 mg/kg/day, administered as 2 equally divided doses (i.e., 5 mg/kg in the morning and 5 mg/kg in the evening). The 10 mg/kg/day dose should be maintained for the remainder of the treatment period; however, per labeling, investigators may increase the dose to a maximum of 20 mg/kg/day if clinically warranted by titrating an additional 5 mg/kg/day each week until reaching the maximum dose. GWP42003-P will be taken b.i.d. (morning and evening).
Drug: GWP42003-P
oral solution of 100 milligrams per milliliter (mg/mL) cannabidiol (CBD)
Other Names:
  • Cannabidiol
  • CBD
  • Epidiolex

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Change from Baseline to end of treatment (Day 181 [Visit 5]) in processing speed on the National Institutes of Health Toolbox Cognition Battery (NIHTCB)
Time Frame: Baseline; Day 181
Baseline; Day 181

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Change from Baseline to Day 91 (Visit 4) in processing speed on the NIHTCB
Time Frame: Baseline; Day 91
Baseline; Day 91
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in executive function and attention on the NIHTCB
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in episodic memory on the NIHTCB
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in language on the NIHTCB
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in the NIHTCB Childhood Composite Score
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in the Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2) or BRIEF, Preschool (BRIEF-P) for participants aged 5 years or younger
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in weekly seizure frequency
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in behavior using the Aberrant Behavior Checklist, Second Edition Community Forms (ABC-2-3)
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in sleep characteristics using the Children's Sleep Habits Questionnaire (CSHQ)
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 181 (end of treatment; Visit 5) in quality of life using the Pediatric Quality of Life Inventory (PEDS-QL4)
Time Frame: Baseline; Day 181
Baseline; Day 181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in generic health status using the EQ-5D-Y Proxy Version 1
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in the Caregiver Global Impression of Change (CGIC) score
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in Patient-Reported Outcomes Measurement Information System (PROMIS®) - Parent Proxy Short Form Anxiety and Depression Subscales
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in Parenting Stress Index, Fourth Edition (PSI-4)
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Change from Baseline to Day 181 (end of treatment; Visit 5) in the participant's ability to perform day-to-day tasks
Time Frame: Baseline; Day181
Baseline; Day181
Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in the Physician Global Impression of Change (PGIC) score
Time Frame: Baseline; Days 91 and 181
Baseline; Days 91 and 181
Number of participants with the indicated type of adverse event
Time Frame: up to Day 219
up to Day 219
Number of participants with clinically significant changes in laboratory parameter values
Time Frame: Baseline; up to Day 191
Baseline; up to Day 191
Number of participants with clinically significant changes in physical examination findings
Time Frame: Baseline; up to Day 191
Baseline; up to Day 191
Number of participants with clinically significant changes in vital sign values
Time Frame: Baseline; up to Day 191
Baseline; up to Day 191

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Jazz Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 1, 2020

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2021

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 17, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 17, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 21, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 1, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 31, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GWEP18060

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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