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

NCT03458416 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: INS011-17-115
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

Cannabidiol Oral Solution: 20-40 mg/kg/day

EXPERIMENTAL

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

Interventions

Cannabidiol Oral Solution DRUG

An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).

Cannabidiol Oral Solution: 20-40 mg/kg/day

Eligibility Criteria

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

You may qualify if:

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

You may not qualify if:

• Participant or parent(s)/caregiver(s) have commitments during the study duration that would interfere with attending all study visits.
• Experienced an anoxic episode related to study drug requiring resuscitation during the previous study.
• Uncontrolled Type I and Type II Diabetes.
• 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.
• 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).
• Currently taking felbamate.
• Compromised respiratory function or severe respiratory insufficiency.
• Pregnant or lactating female.
• In the opinion of the investigator, the participant is unsuitable in any other way to participate in this study.

Sponsors & Collaborators

• Radius Pharmaceuticals, Inc.: lead
• Benuvia Therapeutics Inc.: collaborator

Study Sites (1)

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 terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.

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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 2, 2018
• First Posted: March 8, 2018
• Study Start: September 6, 2018
• Primary Completion: July 31, 2019
• Study Completion: July 31, 2019
• Last Updated: June 22, 2023
• Results First Posted: June 22, 2023

Record last verified: 2023-05

Locations

US (1)