A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Participants With Prader-Willi Syndrome

A Multicenter, Open-Label Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Patients With Prader-Willi Syndrome

The objective of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in participants with Prader-Willi Syndrome.

Study Overview

• Status: Terminated
• Conditions: Prader-Willi Syndrome
• Intervention / Treatment: Drug: Cannabidiol Oral Solution

• Study Type: Interventional
• Enrollment (Actual): 2
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Washington
    • Tacoma, Washington, United States, 98405
      • Institute for Research and Innovation | MultiCare Health System

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 15 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Completed activities up to and including Visit 10 (Study Completion) of INS011-16- 085.
2. 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.
3. If female, is either not of childbearing potential or practicing medically acceptable methods of birth control.
4. Psychotropic treatment will be permitted if the participant has been on a stable dose during the INS011-16-085 and does not anticipate a dose change during the course of the study.
5. Growth hormone treatment will be permitted if the participant has been on a stable dose during INS01-16-085.
6. Any other treatment including thyroid hormones should be stable prior to entering the INS011-17-115 study.
7. 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.

Exclusion Criteria:

1. Participant or parent(s)/caregiver(s) have commitments during the study duration that would interfere with attending all study visits.
2. Experienced an anoxic episode related to study drug requiring resuscitation during the previous study.
3. Uncontrolled Type I and Type II Diabetes.
4. Developed an adverse event thought to be related to CBD in the previous study and the investigator determines that continuing treatment with CBD would not be in the best interest of the partient.
5. History of clinically significant gastrointestinal, renal, hepatic, neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, psychiatric, or cardiovascular disease, or other condition which would jeopardize safety or impact validity of results (per investigator).
6. Currently taking felbamate.
7. Compromised respiratory function or severe respiratory insufficiency.
8. Pregnant or lactating female.
9. In the opinion of the investigator, the participant is unsuitable in any other way to participate in this study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cannabidiol Oral Solution: 20-40 mg/kg/day; Participants will receive total daily doses between 20 milligrams per kilograms per day (mg/kg/day), 30 mg/kg/day, and 40 mg/kg/day. The two equivalent doses will be administered twice a day with a standard meal approximately every 12 hours.	Drug: Cannabidiol Oral Solution; An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence Of Adverse Events (AEs) And Serious Adverse Events (SAEs) Associated With Cannabidiol Oral Solution	AE is defined as any untoward medical occurrence in a participant administered with a pharmaceutical product which does not necessarily have a causal relationship with the treatment. An SAE is defined as any event that results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or a congenital anomaly/birth defect. A summary of other non-serious AEs and all serious AEs regardless of causality, is located in the Reported AE section.	Up to Week 52

Collaborators and Investigators

• Sponsor: Radius Pharmaceuticals, Inc.
• Collaborators: Benuvia Therapeutics Inc.
• Investigators: Study Director: Ahmed Elkashef, MD, INSYS Therapeutics Inc

Study record dates

Study Major Dates

• Study Start (Actual): September 6, 2018
• Primary Completion (Actual): July 31, 2019
• Study Completion (Actual): July 31, 2019

Study Registration Dates

• First Submitted: March 2, 2018
• First Submitted That Met QC Criteria: March 2, 2018
• First Posted (Actual): March 8, 2018

Study Record Updates

• Last Update Posted (Actual): June 22, 2023
• Last Update Submitted That Met QC Criteria: May 26, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Overnutrition; Nutrition Disorders; Overweight; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Syndrome; Prader-Willi Syndrome; Anticonvulsants; Cannabidiol
• Other Study ID Numbers: INS011-17-115

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No