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NCT05864846 | ACTIVE NOT RECRUITING | Tuberous Sclerosis Complex Associated Neuropsychiatric Disease

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
Sponsor:

Jazz Pharmaceuticals

Brief Summary:

The purpose of this study is to investigate behavioral and other co-occurring outcomes with EPID(I/Y)OLEX as an add-on therapy in participants aged 1 to 65 years with tuberous sclerosis complex (TSC) who experience seizures.

Condition or disease

Tuberous Sclerosis Complex Associated Neuropsychiatric Disease

Intervention/treatment

Cannabidiol Oral Solution [Epidiolex]

Phase

PHASE4

Study Type : INTERVENTIONAL
Estimated Enrollment : 79 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : A Phase 4, Interventional, Multicenter, Open-Label, Single-Arm Study to Assess Behavioral and Other Co-occurring Outcomes Following Treatment With EPID(I/Y)OLEX as Add-on Therapy in Participants (Aged 1 to 65 Years Old) With Seizures Associated With Tuberous Sclerosis Complex
Actual Study Start Date : 2023-06-29
Estimated Primary Completion Date : 2026-03-26
Estimated Study Completion Date : 2026-03-26

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 1 Year to 65 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
Inclusion Criteria
  • 1. Is within the required age range at the time of signing (or at the time of the participant's parent(s)/Legally Authorized Representative (LAR) signing) the informed consent or providing assent (as applicable)
    • * 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
          • 1. Has a clinically significant unstable medical condition other than epilepsy.
          • 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.
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

Location Details

NCT05864846

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

Not yet recruiting

United States, Colorado

Children's Hospital Colorado

Aurora, Colorado, United States, 80045

Not yet recruiting

United States, Connecticut

Yale University School of Medicine

New Haven, Connecticut, United States, 06519

Not yet recruiting

United States, Florida

University of Florida Health - Department of Neurology

Gainesville, Florida, United States, 32608

Not yet recruiting

United States, Florida

Nicklaus Children's Health, Miami

Miami, Florida, United States, 33155

Not yet recruiting

United States, Massachusetts

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

Not yet recruiting

United States, Minnesota

Minnesota Epilepsy Group

Roseville, Minnesota, United States, 55113

Not yet recruiting

United States, North Carolina

Atrium Health Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, United States, 27157

Not yet recruiting

United States, Ohio

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

Not yet recruiting

United States, Pennsylvania

University of Pennsylvania - Penn Epilepsy Center

Philadelphia, Pennsylvania, United States, 19104

Not yet recruiting

United States, Tennessee

Happiness Children's Hospital

Memphis, Tennessee, United States, 38103

Not yet recruiting

United States, Texas

University of Texas Health Science Center at Houston - Clinical Research Unit

Houston, Texas, United States, 77030

Not yet recruiting

United States, Texas

University of Texas Health Science Center - San Antonio

San Antonio, Texas, United States, 78229

Not yet recruiting

United States, Virginia

University of Virginia, Charlottesville

Charlottesville, Virginia, United States, 22903

Not yet recruiting

Canada, Alberta

Alberta Children's Hospital

Calgary, Alberta, Canada, Tired medicine

Not yet recruiting

Canada, British Columbia

BC Children's Hospital

Vancouver, British Columbia, Canada, V6H 3V4

Not yet recruiting

Canada, Ontario

The Hospital for Sick Children

Toronto, Ontario, Canada, M5G 1X8

Not yet recruiting

Poland,

The Children's Memorial Health Institute

Warsaw, Poland, 04-736

Not yet recruiting

United Kingdom, England

Evelina London Children's Hospital Guys St Thomas' NHS Foundation Trust, the Guy's Hospital

London, England, United Kingdom, SE1 7EH

Not yet recruiting

United Kingdom,

University Hospitals Bristol NHS Foundation Trust

Bristol, United Kingdom, BS1 3NU

Not yet recruiting

United Kingdom,

Sheffield Children's NHS Foundation Trust

Sheffield, United Kingdom, so