Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
Condition: Prader-Willi Syndrome
MeSH terms: Prader-Willi Syndrome; Syndrome
Intervention: Cannabidiol; Placebo
Intervention description: Oral solution; Matching oral solution
Intervention MeSH terms: Cannabidiol
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 7.0
Sex: All
Minimum age: 8 Years
Maximum age: 17 Years
Healthy volunteers: No

Summary:

      The primary objective of this study is to assess the efficacy of cannabidiol oral solution on
      hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS). The secondary
      objectives of this study are to assess the efficacy, safety and tolerability, impact on
      quality of life, and impact on physical activity of cannabidiol oral solution in patients
      with PWS.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Participant and/or parent(s)/caregiver(s) fully comprehend the informed consent form
             and assent form, understand all study procedures, and can communicate satisfactorily
             with the investigator and study coordinator, in accordance with applicable laws,
             regulations, and local requirements.

          -  Participants with a genetically confirmed diagnosis of Prader-Willi Syndrome using
             standard deoxyribonucleic acid methylation test or fluorescent in situ hybridization.
             Documentation of genetically confirmed diagnosis of Prader-Willi Syndrome is
             acceptable.

          -  A score of ≥10 on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT).

          -  A caregiver is available to complete the HQ-CT.

          -  If female, is either not of childbearing potential (defined as premenarchal or
             surgically sterile [bilateral tubal ligation, bilateral oophorectomy, or
             hysterectomy]) or practicing one of the following medically acceptable methods of
             birth control:

               1. Hormonal methods such as oral, implantable, injectable, vaginal ring, or
                  transdermal contraceptives for a minimum of 1 full cycle (based on the
                  participant's usual menstrual cycle period) before study drug administration.

               2. Total abstinence from sexual intercourse since the last menses before study drug
                  administration.

               3. Intrauterine device.

               4. Double-barrier method (condoms, sponge, or diaphragm with spermicidal jellies or
                  cream).

          -  Adequate renal function, defined as serum creatinine ≤ 1.5*upper limit of normal (ULN)
             and urine protein/creatinine ratio ≤0.4. The Investigator may deem the participant
             eligible if he or she judges the laboratory values to be not clinically significant.

          -  Adequate hepatic function, defined as total bilirubin ≤ 1.5*ULN and aspartate
             aminotransferase and alanine aminotransferase levels ≤ 3*ULN.

          -  Growth hormone treatment will be permitted if doses have been stable for at least 1
             month prior to screening.

          -  Psychotropic treatment will be permitted and should be stable at least 6 weeks prior
             to screening.

          -  Any other treatment including thyroid hormones should be stable for at least 6 weeks
             prior to screening.

        Exclusion Criteria:

          -  Known use of cannabis or cannabinoid-containing products for 4 weeks prior to
             baseline.

          -  History of chronic liver diseases, such as cirrhosis or chronic hepatitis due to any
             cause, or suspected alcohol abuse.

          -  Use of weight loss agents or drugs known to affect appetite (including glucagon-like
             peptide-1 [GLP-1] analogs) within 2 months prior to screening.

          -  Uncontrolled Type I and Type II diabetes.

          -  Currently taking concomitant medication that are strong-moderate
             inhibitors/inducers/sensitive substrates with a narrow therapeutic index for CYP2C19
             or CYP3A.

          -  Co-morbid condition or disease (such as respiratory disease, heart disease, or
             psychiatric disorder) diagnosed less than 1 month prior to screening.

          -  History or presence of gastrointestinal or any other condition known to interfere with
             the absorption, distribution, metabolism, or excretion of drugs as determined by the
             Investigator.

          -  Participants who have participated in any other trials involving an investigational
             product or device within 30 days of screening or longer as required by local
             regulations.

          -  Clinically significant abnormalities on electrocardiogram at screening or other
             evidence of heart disease as determined by the Investigator.

          -  Has screening systolic blood pressure ≥160 millimeters of mercury (mmHg) and diastolic
             blood pressure >100 mm Hg (may be repeated 1 additional time after 5 minutes rest to
             verify). Participants with hypertensive levels lower than those specified may be
             excluded at the Investigator's discretion if deemed to be in the best interest of the
             participant.

          -  Currently taking felbamate.

          -  Uncontrolled sleep apnea.

          -  Pregnant or lactating female.

          -  History of hypersensitivity to drugs with a similar chemical structure or class as
             cannabidiol.

          -  Unwillingness or inability to follow the procedures outlined in the protocol.

          -  Participant judged by the investigator or sponsor (or designee) as unable to comply
             with the treatment protocol, including appropriate supportive care, follow-up and
             research tests.

          -  Positive drug screen, including tetrahydrocannabinol, at time of screening.

          -  Creatinine clearance test of < 30 milliliters/minute (mL/min).