A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome

Radius Pharmaceuticals, Inc.7 sites in 1 country7 target enrollmentMay 9, 2018

ConditionsPrader-Willi Syndrome

InterventionsCannabidiol Placebo

DrugsCannabidiol Placebo

Overview

Phase: Phase 2
Intervention: Cannabidiol
Conditions: Prader-Willi Syndrome
Sponsor: Radius Pharmaceuticals, Inc.
Enrollment: 7
Locations: 7
Primary Endpoint: Change From Baseline in Hyperphagia Behavior as Measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)
Status: Terminated
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

May 9, 2018

End Date

July 31, 2019

Last Updated

2 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Radius Pharmaceuticals, Inc.

Responsible Party

Sponsor

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:
•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.
•Total abstinence from sexual intercourse since the last menses before study drug administration.
•Intrauterine device.
•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.

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.

Arms & Interventions

Cannabidiol

Cannabidiol oral solution (40 milligram/kilogram/day \[40 mg/kg/day\]) divided into two daily doses with a standard meal

Intervention: Cannabidiol

Placebo

Matching placebo solution divided into two daily doses with a standard meal

Intervention: Placebo

Outcomes

Primary Outcomes

Change From Baseline in Hyperphagia Behavior as Measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)

Time Frame: Baseline, Week 13

The HQ-CT measures hyperphagia by Prader-Willi syndrome (PWS)-specialized clinicians. The HQ-CT generates a score ranging from 0 to 36, where a higher score represents more severe abnormal food related behaviors. The change from baseline was calculated from two time points as the value at the later time point minus the value at the earlier time point: value at Week 13 minus value at Baseline.

Secondary Outcomes

Change From Baseline In The Three Factor Eating Questionnaire - 18-item Version (TFEQ-R18)(Baseline, Week 13)
Change From Baseline In Physical Activity (PROMIS Physical Activity And Fatigue Questionnaires)(Baseline, Week 13)
Change From Baseline In Quality Of Life [Patient-Reported Outcomes Measurement Information System (PROMIS) Life Satisfaction And Positive Affect Questionnaires](Baseline, Week 13)
Responder Rate From Baseline Through Study Completion(Baseline up to Week 13)
Change From Baseline In Total Body Weight(Baseline, Week 13)
Change In Patient Global Impression Of Change (PGI-C) Questionnaire(Week 3, Week 13)