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

Primary Purpose

Prader-Willi Syndrome

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Cannabidiol Oral Solution
Sponsored by
Radius Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Prader-Willi Syndrome

Eligibility Criteria

8 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Completed activities up to and including Visit 10 (Study Completion) of INS011-16- 085.
  2. Patient and/or parent(s)/caregiver(s) fully comprehend the informed consent form (ICF) 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 subject 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 subject 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. Patient 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 patient.
  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 patient is unsuitable in any other way to participate in this study.

Sites / Locations

  • Institute for Research and Innovation | MultiCare Health System

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

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

Arm Description

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Full Information

First Posted
March 2, 2018
Last Updated
May 26, 2023
Sponsor
Radius Pharmaceuticals, Inc.
Collaborators
Benuvia Therapeutics Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03458416
Brief Title
A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Participants With Prader-Willi Syndrome
Official Title
A Multicenter, Open-Label Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Patients With Prader-Willi Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Terminated
Why Stopped
Insys Therapeutics filed Chapter 11 and terminated all studies.
Study Start Date
September 6, 2018 (Actual)
Primary Completion Date
July 31, 2019 (Actual)
Study Completion Date
July 31, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Radius Pharmaceuticals, Inc.
Collaborators
Benuvia Therapeutics Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cannabidiol Oral Solution: 20-40 mg/kg/day
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Cannabidiol Oral Solution
Intervention Description
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
Primary Outcome Measure Information:
Title
Incidence Of Adverse Events (AEs) And Serious Adverse Events (SAEs) Associated With Cannabidiol Oral Solution
Description
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.
Time Frame
Up to Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ahmed Elkashef, MD
Organizational Affiliation
INSYS Therapeutics Inc
Official's Role
Study Director
Facility Information:
Facility Name
Institute for Research and Innovation | MultiCare Health System
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98405
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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