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

Phase 4

Recruiting

• Conditions: Tuberous Sclerosis Complex Associated Neuropsychiatric Disease

• Registration Number: NCT05864846
• Lead Sponsor: Jazz Pharmaceuticals

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-5-9
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 75

Inclusion Criteria

Inclusion Criteria:<br><br> 1. Is within the required age range at the time of signing (or at the time of the<br> participant's parent(s)/Legally Authorized Representative (LAR) signing) the<br> informed consent or providing assent (as applicable):<br><br> - Participants based in the US: 1 to 65 years of age, inclusive.<br><br> - Participants based outside the US: 2 to 65 years of age, inclusive.<br><br> 2. Has a confirmed clinical diagnosis of TSC with a history of seizures in accordance<br> with the 2012 International Tuberous Sclerosis Complex Consensus Conference<br> criteria.<br><br> 3. Has behaviors (eg, aggression, impulsivity, temper tantrum, self-injury,<br> hyperactivity, extreme shyness, mood swings, poor eye contact, repetitive behaviors,<br> restlessness, difficulty getting along with peers, rigid/inflexible to procedure<br> and/or change) that are considered moderate or severe per the CareGI-S at Screening.<br><br> 4. Is taking 1 or more anti-seizure medicine (ASM) at a dose that has been stable for<br> at least 4 weeks prior to Screening.<br><br> • All medications or interventions for epilepsy (including ketogenic diet and any<br> neurostimulation devices for epilepsy) must have been stable for 4 weeks prior to<br> screening and any major changes to treatment regimens should be discussed with the<br> medical monitor.<br><br> 5. Is naïve to CBD-OS treatment or has been off CBD-OS treatment for at least 3 months<br> prior to Screening.<br><br> 6. Is willing to maintain any factors expected to affect seizures stable (eg, alcohol<br> consumption, smoking, concomitant medication usage).<br><br> 7. Is male or female<br><br> 1. Male participants:<br><br> • Male participants are eligible to participate if they agree to the following<br> during the intervention period and for at least 2 weeks, corresponding to the<br> time needed to eliminate the study intervention after the last dose of study<br> intervention:<br><br> - Refrain from donating fresh unwashed semen. PLUS<br><br> - Use a male condom in addition to a second method of acceptable<br> contraception used by their female partners when having sexual intercourse<br> with a women of childbearing potential (WOCBP) who is not currently<br> pregnant.<br><br> 2. Female participants:<br><br> - A female participant is eligible to participate if she is not pregnant or<br> breastfeeding and one of the following conditions applies:<br><br> - Is a woman of nonchildbearing potential. OR<br><br> - Is a WOCBP and using a contraceptive method that is highly effective,<br> preferably with low user dependency during the study intervention<br> period and for at least 3 months after the last dose of study<br> intervention. The investigator should evaluate the potential for<br> contraceptive method failure (eg, noncompliance, recently initiated)<br> in relationship to the first dose of study intervention.<br><br> - A WOCBP must have a negative highly sensitive pregnancy test within 28<br> days before the first dose of study intervention.<br><br>Exclusion Criteria:<br><br> 1. Has a clinically significant unstable medical condition other than epilepsy.<br><br> 2. Has an illness during the 4 weeks prior to screening other than epilepsy which, in<br> the investigator's opinion, could affect study outcomes.<br><br> 3. Has TSC-specific tumor growth which, in the investigator's opinion, could affect the<br> effectiveness endpoints.<br><br> 4. Has previously undergone significant surgery for epilepsy that, in the<br> investigator's opinion, may impact the assessment of outcomes.<br><br> 5. Has initiated felbamate within the last 12 months prior to Screening.<br><br> 6. Is currently using or has in the past used recreational or medicinal cannabis or<br> synthetic cannabinoid-based medications within the 3 months prior to Screening and<br> is not willing to undergo a 1-month washout period before being rescreened.<br><br> 7. Has received an investigational medicinal product within the 3 months prior to the<br> Screening Visit.<br><br> 8. Has previously been assigned study intervention for this study or is currently<br> enrolled in any other interventional study.<br><br> 9. Has laboratory values at the Baseline Visit that are abnormal and of clinical<br> significance in the investigator's opinion.<br><br> 10. Participant has significantly impaired hepatic function at the Baseline Visit.<br><br> 11. Has any history of suicidal behavior or any suicidal ideation of type 4 or 5 as<br> evaluated with C-SSRS or Children's C-SSRS at the Screening Visit (for participants<br> = 4 years of age).<br><br> 12. Has any known or suspected hypersensitivity to cannabinoids or any of the excipients<br> of CBD-OS.<br><br> 13. Has a known or suspected history of alcohol or substance abuse.

Exclusion Criteria

Not provided

Study & Design

• Study Type: Interventional
• Study Design: Not specified