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.

Tracking Information

• First Submitted Date: July 22, 2016
• First Posted Date: July 26, 2016
• Last Update Posted Date: November 13, 2020
• Actual Study Start Date: June 6, 2018
• Actual Primary Completion Date: June 10, 2019 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: February 2, 2018): 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) ]

Original Primary Outcome Measures (submitted: July 22, 2016)

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

Current Secondary Outcome Measures (submitted: February 2, 2018)

• Change from Baseline in Total Body-Weight [ Time Frame: Baseline through Study Completion/ Early Withdrawal (within 13 weeks) ]
• 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
• Change from Baseline in Patient Global Impression of Change and Severity (PGI-C) [ Time Frame: Baseline through Study Completion/Early Withdrawal (within 13 weeks) ]
• 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) ]
• 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) ]
• Change from Baseline in Physical Activity (PROMIS Physical Activity and Fatigue questionnaires) [ Time Frame: Baseline through Study Completion/Early Withdrawal (within 13 weeks) ]

Original Secondary Outcome Measures (submitted: July 22, 2016)

• Change in the Three Factor Eating Questionnaire (TFEQ) from Baseline through Study Completion/Withdrawal [ Time Frame: Baseline through Study Completion/Withdrawal (within 13 weeks) ]
• Change in the Development Behavior Checklist (DBC) from Baseline through Study Completion/Withdrawal [ Time Frame: Baseline through Study Completion/Withdrawal (within 13 weeks) ]
• Change in Skin Picking Impact Scale (SPIS) from Baseline through Study Completion/Withdrawal [ Time Frame: Baseline through Study Completion/Withdrawal (within 13 weeks) ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Cannabidiol Oral Solution for the Treatment of Subjects With Prader-Willi Syndrome
• 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
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double (Participant, Investigator); Primary Purpose: Treatment
• Condition: Prader-Willi Syndrome

Intervention

• Drug: Cannabidiol
  Oral solution
• Drug: Placebo
  Matching oral solution

Study Arms

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

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Terminated
• Actual Enrollment (submitted: November 11, 2020): 7
• Original Estimated Enrollment (submitted: July 22, 2016): 66
• Actual Study Completion Date: June 10, 2019
• Actual Primary Completion Date: June 10, 2019 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 8 Years to 17 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02844933
• Other Study ID Numbers: INS011-16-085
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

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

• IPD Sharing Statement: Not Provided
• Responsible Party: Benuvia Therapeutics Inc.
• Study Sponsor: Benuvia Therapeutics Inc.
• Collaborators: Not Provided

Investigators

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

• PRS Account: Benuvia Therapeutics Inc.
• Verification Date: November 2020