A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex
NCT ID: NCT05864846
Last Updated: 2025-11-13
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE4
79 participants
INTERVENTIONAL
2023-06-29
2026-03-26
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Cannabidiol Oral Solution
Participants who will receive the Cannabidiol Oral Solution (CBD-OS) titrated up to a dose of 12.5 mg/kg administered twice daily for a total dose of up to 25 mg/kg/day for 26 consecutive weeks. Participants will have the option to continue receiving the CBD-OS for an additional 26 weeks, for a total of 52 weeks.
Cannabidiol Oral Solution [Epidiolex]
100 mg/ml Cannabidiol Oral solution
Interventions
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Cannabidiol Oral Solution [Epidiolex]
100 mg/ml Cannabidiol Oral solution
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Participants based in the US: 1 to 65 years of age, inclusive.
* Participants based outside the US: 2 to 65 years of age, inclusive.
2. Has a confirmed clinical diagnosis of TSC with a history of seizures in accordance with the 2012 International Tuberous Sclerosis Complex Consensus Conference criteria.
3. Has behaviors (eg, aggression, impulsivity, temper tantrum, self-injury, hyperactivity, extreme shyness, mood swings, poor eye contact, repetitive behaviors, restlessness, difficulty getting along with peers, rigid/inflexible to procedure and/or change) that are considered moderate or severe per the CareGI-S at Screening.
4. Is taking 1 or more anti-seizure medicine (ASM) at a dose that has been stable for at least 4 weeks prior to Screening.
• All medications or interventions for epilepsy (including ketogenic diet and any neurostimulation devices for epilepsy) must have been stable for 4 weeks prior to screening and any major changes to treatment regimens should be discussed with the medical monitor.
5. Is naïve to CBD-OS treatment or has been off CBD-OS treatment for at least 3 months prior to Screening.
6. Is willing to maintain any factors expected to affect seizures stable (eg, alcohol consumption, smoking, concomitant medication usage).
7. Is male or female
1. Male participants:
• Male participants are eligible to participate if they agree to the following during the intervention period and for at least 2 weeks, corresponding to the time needed to eliminate the study intervention after the last dose of study intervention:
* Refrain from donating fresh unwashed semen. PLUS
* Use a male condom in addition to a second method of acceptable contraception used by their female partners when having sexual intercourse with a women of childbearing potential (WOCBP) who is not currently pregnant.
2. Female participants:
* A female participant is eligible to participate if she is not pregnant or breastfeeding and one of the following conditions applies:
* Is a woman of nonchildbearing potential. OR
* Is a WOCBP and using a contraceptive method that is highly effective, preferably with low user dependency during the study intervention period and for at least 3 months after the last dose of study intervention. The investigator should evaluate the potential for contraceptive method failure (eg, noncompliance, recently initiated) in relationship to the first dose of study intervention.
* A WOCBP must have a negative highly sensitive pregnancy test within 28 days before the first dose of study intervention.
Exclusion Criteria
2. Has an illness during the 4 weeks prior to screening other than epilepsy which, in the investigator's opinion, could affect study outcomes.
3. Has TSC-specific tumor growth which, in the investigator's opinion, could affect the effectiveness endpoints.
4. Has previously undergone significant surgery for epilepsy that, in the investigator's opinion, may impact the assessment of outcomes.
5. Has initiated felbamate within the last 12 months prior to Screening.
6. Is currently using or has in the past used recreational or medicinal cannabis or synthetic cannabinoid-based medications within the 3 months prior to Screening and is not willing to undergo a 1-month washout period before being rescreened.
7. Has received an investigational medicinal product within the 3 months prior to the Screening Visit.
8. Has previously been assigned study intervention for this study or is currently enrolled in any other interventional study.
9. Has laboratory values at the Baseline Visit that are abnormal and of clinical significance in the investigator's opinion.
10. Participant has significantly impaired hepatic function at the Baseline Visit.
11. Has any history of suicidal behavior or any suicidal ideation of type 4 or 5 as evaluated with C-SSRS or Children's C-SSRS at the Screening Visit (for participants ≥ 4 years of age).
12. Has any known or suspected hypersensitivity to cannabinoids or any of the excipients of CBD-OS.
13. Has a known or suspected history of alcohol or substance abuse.
1 Year
65 Years
ALL
No
Sponsors
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Jazz Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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Children's Hospital Colorado
Aurora, Colorado, United States
Yale University School of Medicine
New Haven, Connecticut, United States
University of Florida Health - Department of Neurology
Gainesville, Florida, United States
Nicklaus Children's Health, Miami
Miami, Florida, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Minnesota Epilepsy Group
Roseville, Minnesota, United States
Atrium Health Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Le Bonheur Children's Hospital
Memphis, Tennessee, United States
University of Texas Health Science Center at Houston - Clinical Research Unit
Houston, Texas, United States
University of Texas Health Science Center - San Antonio
San Antonio, Texas, United States
University of Virginia, Charlottesville
Charlottesville, Virginia, United States
Alberta Children's Hospital
Calgary, Alberta, Canada
BC Children's Hospital
Vancouver, British Columbia, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
The Children's Memorial Health Institute
Warsaw, , Poland
Evelina London Children's Hospital Guys St Thomas' NHS Foundation Trust, the Guy's Hospital
London, England, United Kingdom
University Hospitals Bristol NHS Foundation Trust
Bristol, , United Kingdom
Sheffield Children's NHS Foundation Trust
Sheffield, , United Kingdom
Countries
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Other Identifiers
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2023-507426-17
Identifier Type: OTHER
Identifier Source: secondary_id
JZP926-402
Identifier Type: -
Identifier Source: org_study_id