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

NCT02844933 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: INS011-16-085
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 7

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.

Conditions

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

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

  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.

  Baseline, Week 13

Secondary Outcomes (6)

• Change From Baseline In Total Body Weight

  Baseline, Week 13

• Responder Rate From Baseline Through Study Completion

  Baseline up to Week 13

• Change In Patient Global Impression Of Change (PGI-C) Questionnaire

  Week 3, Week 13

• Change From Baseline In The Three Factor Eating Questionnaire - 18-item Version (TFEQ-R18)

  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

Study Arms (2)

Cannabidiol

EXPERIMENTAL

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

Drug: Cannabidiol

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

Cannabidiol DRUG

Oral solution

Cannabidiol

Placebo DRUG

Matching oral solution

Placebo

Eligibility Criteria

• Age: 8 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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 ≤0.4. 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Radius Pharmaceuticals, Inc.: lead

Study Sites (7)

University of Arizona

Tucson, Arizona, 85724, United States

Location

Rady Children's, UC San Diego

San Diego, California, 92123, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

The University of Kansas , Medical Center

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

The University of Oklahoma Health Sciences Center

Tulsa, Oklahoma, 74135, United States

Location

Institute for Research and Innovation | MultiCare Health System

Tacoma, Washington, 98405, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Limitations and Caveats

This study was prematurely terminated by the previous Sponsor (Insys Therapeutics Inc.). Only 7 participants were enrolled, and the planned number of participants was not achieved.

Results Point of Contact

• Title: Tarek El Akkad, Head of Clinical Development
• Organization: Radius Pharmaceuticals, Inc.

telakkad@radiuspharm.com

6175514000

Study Officials

• Ahmed Elkashef, MD

  INSYS Therapeutics Inc

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 22, 2016
• First Posted: July 26, 2016
• Study Start: May 9, 2018
• Primary Completion: July 17, 2019
• Study Completion: July 31, 2019
• Last Updated: August 1, 2023
• Results First Posted: August 1, 2023

Record last verified: 2023-07

Locations

US (7)