ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03458416
Recruitment Status : Recruiting
First Posted : March 8, 2018
Last Update Posted : November 2, 2018
Sponsor:
Information provided by (Responsible Party):
INSYS Therapeutics Inc

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

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: Cannabidiol Oral Solution Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 66 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : September 6, 2018
Estimated Primary Completion Date : May 2020
Estimated Study Completion Date : May 2020

Resource links provided by the National Library of Medicine


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).




Primary Outcome Measures :
  1. Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) Associated with Cannabidiol Oral Solution [ Time Frame: Baseline (Visit 10 of INS011-16-085) up to Visit 9 (approximately Week 52) ]
  2. Number of Participants with Change from Baseline in Vital Signs [ Time Frame: Baseline (Visit 10 of INS011-16-085) up to Visit 7 (approximately Week 48) ]
  3. Number of Participants with Change from Baseline in Physical Exam [ Time Frame: Baseline (Visit 10 of INS011-16-085) up to Visit 7 (approximately Week 48) ]
  4. Number of Participants with Change from Baseline in Electrocardiogram (ECG) [ Time Frame: Baseline (Visit 10 of INS011-16-085) up to Visit 7 (approximately Week 48) ]
  5. Number of Participants with Change from Baseline in Laboratory Values [ Time Frame: Baseline (Visit 10 of INS011-16-085) up to Visit 7 (approximately Week 48) ]
  6. Number of Participants with Change from Baseline in Weight [ Time Frame: Baseline (Visit 10 of INS011-16-085) up to Visit 7 (approximately Week 48) ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   8 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03458416


Locations
United States, Washington
Institute for Research and Innovation | MultiCare Health System Recruiting
Tacoma, Washington, United States, 98405
Contact: Research Coordinator    253-403-2699    ccanorro@multicare.org   
Sponsors and Collaborators
INSYS Therapeutics Inc
Investigators
Study Director: Giovanni DeCastro INSYS Therapeutics Inc

Responsible Party: INSYS Therapeutics Inc
ClinicalTrials.gov Identifier: NCT03458416     History of Changes
Other Study ID Numbers: INS011-17-115
First Posted: March 8, 2018    Key Record Dates
Last Update Posted: November 2, 2018
Last Verified: November 2018

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

Additional relevant MeSH terms:
Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Pharmaceutical Solutions