Cannabidiol Oral Solution for the Treatment of Subjects With Prader-Willi Syndrome

• ClinicalTrials.gov Identifier: NCT02844933
• Recruitment Status: Terminated
• First Posted: July 26, 2016
• Last Update Posted: November 13, 2020
• Sponsor: Benuvia Therapeutics Inc.
• Information provided by (Responsible Party): Benuvia Therapeutics Inc.

Study Description

Brief Summary:

The primary objective of this study is to assess the efficacy of Cannabidiol Oral Solution on hyperphagia-related behavior in subjects 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 subjects with PWS.

Condition or disease	Intervention/treatment	Phase
Prader-Willi Syndrome	Drug: Cannabidiol; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 7 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution for the Treatment of Subjects With Prader-Willi Syndrome
• Actual Study Start Date: June 6, 2018
• Actual Primary Completion Date: June 10, 2019
• Actual Study Completion Date: June 10, 2019

Arms and Interventions

Arm	Intervention/treatment
Experimental: Cannabidiol; Cannabidiol oral solution (40 mg/kg/day) divided into two daily doses with a standard meal	Drug: Cannabidiol; Oral solution
Placebo Comparator: Placebo; Matching placebo solution divided into two daily doses with a standard meal	Drug: Placebo; Matching oral solution

Outcome Measures

Primary Outcome Measures :

1. Change from Baseline in the total score of the Hyperphagia Questionnaire for Clinical Trial (HQ-CT) [ Time Frame: Baseline through Study Completion/ Early Withdrawal (within 13 weeks) ]

Secondary Outcome Measures :

1. Change from Baseline in Total Body-Weight [ Time Frame: Baseline through Study Completion/ Early Withdrawal (within 13 weeks) ]
2. Responder Rate from Baseline through Study Completion [ Time Frame: Baseline through Study Completion (within 13 weeks) ]
   Responder is defined as 6-point decrease on the HQ-CT
3. Change from Baseline in Patient Global Impression of Change and Severity (PGI-C) [ Time Frame: Baseline through Study Completion/Early Withdrawal (within 13 weeks) ]
4. Change from Baseline in the Three Factor Eating Questionnaire - 18-item Version (TFEQ-R18) [ Time Frame: Baseline through Study Completion/Early Withdrawal (within 13 weeks) ]
5. Change from Baseline in Quality of Life (PROMIS Life Satisfaction and Positive Affect Questionnaires) [ Time Frame: Baseline through Study Completion/Early Withdrawal (within 13 weeks) ]
6. Change from Baseline in Physical Activity (PROMIS Physical Activity and Fatigue questionnaires) [ Time Frame: Baseline through Study Completion/Early Withdrawal (within 13 weeks) ]

Eligibility Criteria

• Ages Eligible for Study: 8 Years to 17 Years (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Meets protocol-specified criteria for qualification and contraception
• In the opinion of the investigator, the parent(s)/caregiver(s) is (are) willing and able to comply with the study procedures and visit schedules, including venipuncture, and the visit schedules
• Voluntarily consents to participate and provides written informed consent prior to any protocol-specific procedures

Exclusion Criteria:

• History or current use of over-the-counter medications, dietary supplements, or drugs (including nicotine and alcohol) outside protocol-specified parameters

• Signs, symptoms or history of any condition that, per protocol or in the opinion of the investigator, might compromise:

  1. the safety or well-being of the participant or study staff;
  2. the safety or well-being of the participant's offspring (such as through pregnancy or breast-feeding);
  3. the analysis of results

Contacts and Locations

Locations

United States, Arizona

University of Arizona

Tucson, Arizona, United States, 85724

United States, California

Rady Children's, UC San Diego

San Diego, California, United States, 92123

United States, Iowa

University of Iowa

Iowa City, Iowa, United States, 52242

United States, Kansas

The University of Kansas , Medical Center

Kansas City, Kansas, United States, 66160

United States, Maryland

Johns Hopkins University

Baltimore, Maryland, United States, 21287

United States, Oklahoma

The University of Oklahoma Health Sciences Center

Tulsa, Oklahoma, United States, 74135

United States, Washington

Institute for Research and Innovation | MultiCare Health System

Tacoma, Washington, United States, 98405

Sponsors and Collaborators

Benuvia Therapeutics Inc.

Investigators

• Study Director: Ahmed Elkashef, MD; INSYS Therapeutics Inc

More Information

• Responsible Party: Benuvia Therapeutics Inc.
• ClinicalTrials.gov Identifier: NCT02844933
• Other Study ID Numbers: INS011-16-085
• First Posted: July 26, 2016
• Last Update Posted: November 13, 2020
• Last Verified: November 2020

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Prader-Willi Syndrome; Syndrome; Disease; Pathologic Processes; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Obesity; Overnutrition; Nutrition Disorders; Cannabidiol; Anticonvulsants