Lennox-Gastaut SyndromePhase 4EPI-COGNCT04133480

Investigation of Cognitive Outcomes With Cannabidiol Oral Solution

A Phase 4 study testing GWP42003-P in people with Lennox-Gastaut Syndrome, run by Jazz Pharmaceuticals.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Trial summary

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). Read more about this study

Who is it for?: Ages 3 to 10 years
Conditions the study is for: Lennox-Gastaut Syndrome
What is being tested?: GWP42003-P
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 0 participants
Where: No sites listed yet
Study dates: Oct 2020to Dec 2021
Phase: Phase 4
Are there results?: None posted in the registry

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 3 to 10 years
All sexes
No healthy volunteers
Child

Who may be able to join

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.

Show all 9Show fewer

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.

Who cannot join

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.

Show all 18Show fewer

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.

About this study

Official title: 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).

Read the 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.

Conditions: Lennox-Gastaut Syndrome
Being tested: GWP42003-P
Keywords: GWP42003-P, EPIDIOLEX®, cannabidiol, CBD, cognition
Medical subject headings: Lennox Gastaut Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

GWP42003-PDrug
Also called Cannabidiol, CBD, Epidiolex
oral solution of 100 milligrams per milliliter (mg/mL) cannabidiol (CBD)

Study groups in NCT04133480
Study group	Role	What they receive
GWP42003-P	Experimental	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). Receives: Drug: GWP42003-P

What is being measured

The primary outcome is the main question the study is designed to answer.

Change from Baseline to end of treatment (Day 181 [Visit 5]) in processing speed on the National Institutes of Health Toolbox Cognition Battery (NIHTCB)
Measured: Baseline; Day 181

Secondary outcomes (20)

Change from Baseline to Day 91 (Visit 4) in processing speed on the NIHTCB
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: 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
Measured: 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
Measured: Baseline; Days 91 and 181
Number of participants with the indicated type of adverse event
Measured: up to Day 219
Number of participants with clinically significant changes in laboratory parameter values
Measured: Baseline; up to Day 191
Number of participants with clinically significant changes in physical examination findings
Measured: Baseline; up to Day 191
Number of participants with clinically significant changes in vital sign values
Measured: Baseline; up to Day 191

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Oct 2020Dec 2021

First posted21 Oct 2019
Study startOct 2020 (estimated)
Primary completionDec 2021 (estimated)
Study completionDec 2021 (estimated)

Why it stopped: The decision to withdraw the study was based on pandemic-related concerns of conducting a Phase IV study requiring multiple clinic visits when medication is commercially available. The study was withdrawn before any participants were recruited.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Lennox-Gastaut SyndromeStudies of the same condition, open and under way.
More trials run by Jazz PharmaceuticalsThe sponsor's other registered studies.
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 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04133480
Sponsor's protocol ID: GWEP18060
Acronym: EPI-COG
Status last verified: Aug 2022
First submitted: 17 Oct 2019
Last updated: 1 Sep 2022
Results posted: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

RecruitingPhase 3
Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults
Seizures, Lennox Gastaut Syndrome
Not yet recruitingPhase 4
A Study to Investigate the Effect of Gradual Titration to Optimize Cannabidiol Treatment in Adults With LGS
Lennox-Gastaut Syndrome
Enrolling by invitationPhase 3
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)
Developmental and Epileptic Encephalopathy
RecruitingPhase 3
An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome
Lennox Gastaut Syndrome
Enrolling by invitation
Clinical Utility of Reduced EEG Home Monitoring in Fenfluramine Titration for Dravet and LGS
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)

All Lennox-Gastaut Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 1 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-Label Exploratory Investigation of Cognitive Outcomes With Cannabidiol Oral Solution (EPIDIOLEX®; GWP42003-P)
ClinicalTrials.gov ID: NCT04133480
Acronym / sponsor ID: EPI-COG / GWEP18060
Conditions: Lennox-Gastaut Syndrome
Keywords: GWP42003-P, EPIDIOLEX®, cannabidiol, CBD, cognition
Phase: Phase 4
Study type: Interventional
Recruitment status: WithdrawnReason stopped: The decision to withdraw the study was based on pandemic-related concerns of conducting a Phase IV study requiring multiple clinic visits when medication is commercially available. The study was withdrawn before any participants were recruited.
Enrolment: 0 participants (actual)
Lead sponsor: Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Oct 2020 (estimated)
Primary completion: Dec 2021 (estimated)
Study completion: Dec 2021 (estimated)
First posted: 21 Oct 2019 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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).

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
GWP42003-P	Experimental	Drug: GWP42003-PFor 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).

Who may qualify

Ages: 3 to 10 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (9 listed)

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.

Key exclusion criteria (18 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
GWP42003-PAlso: Cannabidiol, CBD, Epidiolexoral solution of 100 milligrams per milliliter (mg/mL) cannabidiol (CBD)	Drug	GWP42003-P

Primary outcome

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

20 secondary outcomes

Change from Baseline to Day 91 (Visit 4) in processing speed on the NIHTCB
Time frame: 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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
Number of participants with the indicated type of adverse event
Time frame: up to Day 219
Number of participants with clinically significant changes in laboratory parameter values
Time frame: Baseline; up to Day 191
Number of participants with clinically significant changes in physical examination findings
Time frame: Baseline; up to Day 191
Number of participants with clinically significant changes in vital sign values
Time frame: Baseline; up to Day 191

Results and safety

The study was withdrawn before enrolling participants, so there are no results.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2022.
Individual participant dataSharing planned: No

Related trials for Lennox-Gastaut Syndrome

RecruitingPhase 3
Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults
Seizures, Lennox Gastaut Syndrome
Not yet recruitingPhase 4
A Study to Investigate the Effect of Gradual Titration to Optimize Cannabidiol Treatment in Adults With LGS
Lennox-Gastaut Syndrome
Enrolling by invitationPhase 3
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)
Developmental and Epileptic Encephalopathy
RecruitingPhase 3
An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome
Lennox Gastaut Syndrome
Enrolling by invitation
Clinical Utility of Reduced EEG Home Monitoring in Fenfluramine Titration for Dravet and LGS
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing GWP42003-P in people with Lennox-Gastaut Syndrome, run by Jazz Pharmaceuticals.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 1 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04133480.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2022.