A Study to Investigate the Effect of Gradual Titration to Optimize Cannabidiol Treatment in Adults With LGS (EpiGOAL)

A Phase 3b/4, Interventional, Prospective, Multicenter, Open-Label Study Evaluating the Effectiveness of a Gradual Titration Regimen to Optimize CBD-OS as Add-On Therapy in Adults With Lennox-Gastaut Syndrome

This is a Phase 3b/Phase 4 study. The purpose of this study is to learn more about the efficacy of using CBD-OS as an add-on therapy for the treatment of LGS in adults.

Study Overview

• Status: Not yet recruiting
• Conditions: Lennox-Gastaut Syndrome
• Intervention / Treatment: Drug: Cannabidiol Oral Solution

• Study Type: Interventional
• Enrollment (Estimated): 40
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Clinical Trial Disclosure & Transparency; Phone Number: 215-832-3750; Email: ClinicalTrialDisclosure@JazzPharma.com

Study Locations

• United States
  • Louisiana
    • New Orleans, Louisiana, United States, 70112
      • Louisiana State University Health Sciences Center New Orleans

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Has a confirmed clinical diagnosis of LGS with a history of seizures and actively experiencing seizures within 6 months of screening.
2. Is naïve to CBD-OS treatment or has been off CBD-OS treatment for at least 3 months prior to screening.
3. Is willing to maintain any factors expected to affect seizures (eg, alcohol consumption, smoking, concomitant medication usage) stable for the duration of the study.
4. Is currently treated with at least 1 ASM, but no more than 3 ASMs, and is on a stable regimen for at least 4 weeks prior to screening.
5. Adequate contraceptive precautions
6. Participant has a primary caregiver/legally authorized representative (LAR) who is willing and able, in the investigator's opinion, to participate throughout the duration of the study and to comply with all study requirements
7. Experienced at least 4 or more countable major motor seizures (isolated seizures or seizures occurring in a cluster) during the 5-week Baseline Period preceding treatment.
8. Caregiver completed at least 80% of seizure diary entries during the Baseline Period with no more than 3 consecutive days of missing entries.

Exclusion Criteria:

1. Has a significant psychiatric illness
2. Has an illness during the 4 weeks prior to screening other than epilepsy which, in the investigator's opinion, could affect study outcomes or seizure frequency.
3. Has history of SE in the 3 months prior to screening.
4. Has any other significant disease other than epilepsy, which, in the investigator's opinion, may either put the participant, other participants, or site staff at risk because of participation in the study, may influence the result of the study, or may affect the participant's ability to take part in the study.
5. Has previously undergone significant surgery for epilepsy in the 6 months prior to screening or planned to undergo surgery for epilepsy during the study.
6. Is currently using or has in the past used recreational or medicinal cannabis or synthetic cannabinoid-based medications, products, or supplements within the 3 months prior to screening and is not willing to undergo a 1-month washout period before being rescreened.
7. Has initiated felbamate within the 12 months prior to screening.
8. Is not willing to adjust dosage of CBD-OS if needed when using strong inducers of CYP3A4 and/or strong inducers of CYP2C19 concomitantly with CBD-OS
9. Previously discontinued cannabidiol due to severe adverse events (SAE), hypersensitivity or lack of efficacy
10. Has clinically significant abnormal lab values
11. Has clinically impaired hepatic or renal function
12. History of, current risk or presence of actual suicidal ideations
13. Has any known or suspected hypersensitivity to cannabinoids or any of the excipients of CBD-OS, such as sesame oil.
14. Has a known or suspected history of alcohol or substance abuse disorder
15. Is currently consuming, and unwilling to stop consumption, or planning to consume tonic water on a regular basis throughout the study.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cannabidiol Oral Solution Treatment group; Participants who will receive the Cannabidiol Oral Solution (CBD-OS) titrated up to a dose of 20 mg/kg per day or the maximum tolerated dose for 24 consecutive weeks.	Drug: Cannabidiol Oral Solution; 100 mg/ml Cannabidiol Oral solution (CBD-OS); Other Names: CBD-OS; Epidiolex; Epidyolex; JZP926

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Percentage of participants reporting 50% or more reduction in their 28-day average countable major motor seizure frequency	End of Treatment Period, Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Retention Rate	Retention Rate is defined as percentage of participants completing or continuing CBD-OS treatment at different timepoints in the study	At Weeks 8, 12, 16 and 24
Percentage of participants experiencing worsening, change, no change or improvement in in their 28-day average countable major motor and total seizure frequency	As reported in participants' electronic diaries	Baseline to Week 4, Baseline to Week 8, Baseline to Week 12, Baseline to Week 16, Baseline to Week 24
Percentage of participants considered treatment responders	Treatment responders will be categorized as those experiencing a 50% or more reduction, a 75% or more reduction or 100% reduction in their 28-day average seizure frequency as reported in participants' electronic diaries	Baseline to Week 4, Baseline to Week 8, Baseline to Week 12, Baseline to Week 16, Baseline to Week 24
Change in 28-day average frequency of countable major motor and total seizures	Assessed per 28 days from electronic diary entries	Baseline to Week 4, Baseline to Week 8, Baseline to Week 12, Baseline to Week 16, Baseline to Week 24
Change in number of seizure-free days	Assessed per 28 days from electronic diary entries	Baseline to Week 4, Baseline to Week 8, Baseline to Week 12, Baseline to Week 16, Baseline to Week 24
Change in CaGI-S severity of seizure scores	Caregiver Global Impression of Severity (CaGI-S) is a single-item, 5-point Likert-type rating scale (ranging from 0 "not severe at all" to 5 "very severe"). Using this scale, caregivers will be asked to rate their impression of the participant's severity of seizures, and the severity of the impact caused by the seizure.	Baseline to Week 16, Baseline to Week 24
Change in CaGI-S severity of impact of seizures scores	Caregiver Global Impression of Severity (CaGI-S) is a single-item, 5-point Likert-type rating scale (ranging from 0 "not severe at all" to 5 "very severe"). Using this scale, caregivers will be asked to rate their impression of the participant's severity of the impact caused by the seizure.	Baseline to Week 16, Baseline to Week 24
Change in VAS scores	Visual Analog Scale (VAS) is a caregiver-reported tool used to quantify the severity of impact of subjective non-seizure symptoms and assesses sleep, communication, and disruptive behavior. The VAS enables sensitive detection of changes in non-seizure domains (disruptive behavior, Sleep, and Communication) important to patients with LGS and their caregivers. The VAS has a total score ranging from 0 to 100 with the higher score indicating worse conditions.	Baseline to Week 16, Baseline to Week 24
Change in CaGI-S severity of level of alertness scores	CaGI-S Alertness is a caregiver single item assessment on a 5-point Likert-type rating scale (ranging from 0 "not severe at all" to 5 "very severe") assessing their impression of participant's severity of level of awareness	Baseline to Week 16, Baseline to Week 24
Change in CaGI-S severity of overall condition scores	CaGI-S Overall Condition is a caregiver single item assessment on a 5-point Likert-type rating scale (ranging from 0 "not severe at all" to 5 "very severe") assessing their impression of participant's severity of overall condition	Baseline to Week 16, Baseline to Week 24
CaGI-C level of alertness scores	Caregiver Global Impression of Change (CaGI-C) Alertness is a caregiver single item assessment on a 7-point Likert-type rating scale (ranging from 1 "very much improved" to 7 "very severe") assessing their impression of participant's severity of level of awareness	At Week 16, at week 24
CaGI-C overall condition scores	Caregiver Global Impression of Change (CaGI-C) overall condition is a caregiver single item assessment on a 7-point Likert-type rating scale (ranging from 1 "very much improved" to 7 "very severe") assessing their impression of participant's severity of overall condition	At Week 16, at week 24
Change in CGI-S overall condition scores	Clinician Global Impression of severity (CGI-S) Overall Condition is a clinician single item assessment on a 5-point Likert-type rating scale (ranging from 1 "not severe at all" to 5 "very severe") assessing their impression of participant's severity of level of overall condition	Baseline to Week 16, Baseline to Week 24
CGI-C overall condition scores	Clinician Global Impression of Change (CGI-C) Overall Condition is a clinician single item assessment on a 7-point Likert-type rating scale (ranging from 1 "very much improved" to 5 "very much worse") assessing their impression of participant's change of overall condition	At Week 16, At Week 24
Change in ELDQOL Behavior subscale scores	Epilepsy and Learning Disabilities Quality of Life (ELDQOL) is designed for the caregiver to rate the quality of life (QOL) in children with severe epilepsy and learning disabilities over the last month. The questionnaire has a total of 28 main questions, grouped into 4 subscales: behavior, seizure severity, mood, and side effects. The ELDQOL behavior subscale includes 9 of the 28 total main questions with a total score ranging from 9 to 36 with the higher score indicating greater behavioral problems	Baseline to Week 16, Baseline to Week 24
Change in ELDQOL seizure severity subscale scores	Epilepsy and Learning Disabilities Quality of Life (ELDQOL) is designed for the caregiver to rate the quality of life (QOL) in children with severe epilepsy and learning disabilities over the last month. The questionnaire has a total of 28 main questions, grouped into 4 subscales: behavior, seizure severity, mood, and side effects. The ELDQOL seizure severity subscale includes 14 of the 28 total main questions with a total score ranging from 10 to 56 with the higher score indicating greater seizure severity	Baseline to Week 16, Baseline to Week 24
Change in ELDQOL mood scale subscale scores	Epilepsy and Learning Disabilities Quality of Life (ELDQOL) is designed for the caregiver to rate the quality of life (QOL) in children with severe epilepsy and learning disabilities over the last month. The questionnaire has a total of 28 main questions, grouped into 4 subscales: behavior, seizure severity, mood, and side effects. The ELDQOL mood scale subscale includes 1 of the 28 total questions with a total score ranging from 16 to 64 with the higher score indicating greater mood problems	Baseline to Week 16, Baseline to Week 24
Change in ELDQOL side-effects profile subscale scores	Epilepsy and Learning Disabilities Quality of Life (ELDQOL) is designed for the caregiver to rate the quality of life (QOL) in children with severe epilepsy and learning disabilities over the last month. The questionnaire has a total of 28 main questions, grouped into 4 subscales: behavior, seizure severity, mood, and side effects. The ELDQOL side-effects profile subscale includes 2 of the 28 total main questions with a score ranging from 19 to 76 with the higher score indicating greater side effects	Baseline to Week 16, Baseline to Week 24
Number of participants reporting adverse events leading to discontinuation		Up to week 24
Time the adverse events started from baseline		Up to week 24
Number of Participants with Abnormal Laboratory Values		Up to week 24
Change in suicidal ideation scores	As evaluated by the Columbia-Suicide Severity Rating Scale (C-SSRS). The questionnaire has a total score ranging from 0 to 5 with a score of 0 indicating no suicidal ideation is present.	Baseline, up to week 24
Change in number of suicide attempts	As per the response in the C-SSRS	Baseline, up to week 24
Percentage of participants requiring inpatient hospitalization due to epilepsy		Up to week 24
Change in number of usage of rescue medication		Baseline, Up to week 24

Collaborators and Investigators

• Sponsor: Jazz Pharmaceuticals
• Collaborators: Jazz Pharmaceuticals Research UK Limited

Study record dates

Study Major Dates

• Study Start (Estimated): November 18, 2026
• Primary Completion (Estimated): December 28, 2028
• Study Completion (Estimated): December 28, 2028

Study Registration Dates

• First Submitted: July 22, 2026
• First Submitted That Met QC Criteria: July 24, 2026
• First Posted (Actual): July 27, 2026

Study Record Updates

• Last Update Posted (Actual): July 27, 2026
• Last Update Submitted That Met QC Criteria: July 24, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: CBD-OS; LGS; Cannabidiol; Epidiolex; Epidyolex
• Additional Relevant MeSH Terms: Epileptic Syndromes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Epilepsy; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lennox Gastaut Syndrome; Organic Chemicals; Hydrocarbons; Terpenes; Cannabinoids; Cannabidiol
• Other Study ID Numbers: JZP926-403

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: In accordance with ICMJE requirements, Jazz Pharmaceuticals may provide qualified external researchers access to individual participant data (IPD) and clinical trial data that underlie the results of this trial upon request. Qualified researchers can submit a request on https://www.jazzpharma.com/science/clinical-trial-data-sharing/ as outlined. Jazz Pharmaceuticals reserves the right not to consider a request. For inquiries about Jazz's data sharing policy contact clinicaldatasharing@jazzpharma.com.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No