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

• Sponsor: Benuvia Therapeutics Inc. (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT03458416
• Collaborator: (none)
• Enrollment: 7
• Location: 1
• Arm: 1
• Actual Duration (Months): 9.1
• Patients Per Site Per Month: 0.8

Study Details

Study Description

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 Design

• Study Type: Interventional
• Actual Enrollment: 7 participants
• Allocation: N/A
• 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: Sep 6, 2018
• Actual Primary Completion Date: Jun 10, 2019
• Actual Study Completion Date: Jun 10, 2019

Arms and Interventions

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).

Outcome Measures

Primary Outcome Measures

1. Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) Associated with Cannabidiol Oral Solution [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 [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 [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) [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 [Baseline (Visit 10 of INS011-16-085) up to Visit 7 (approximately Week 48)]

6. Number of Participants with Change from Baseline in Weight [Baseline (Visit 10 of INS011-16-085) up to Visit 7 (approximately Week 48)]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 8 Years to 17 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1. Completed activities up to and including Visit 10 (Study Completion) of INS011-16-

3. 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.

4. If female, is either not of childbearing potential or practicing medically acceptable methods of birth control.

5. 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.

6. Growth hormone treatment will be permitted if the subject has been on a stable dose during INS01-16-085.

7. Any other treatment including thyroid hormones should be stable prior to entering the INS011-17-115 study.

8. 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Institute for Research and Innovation | MultiCare Health System	Tacoma	Washington	United States	98405

Sponsors and Collaborators

• Benuvia Therapeutics Inc.

Investigators

• Study Director: Ahmed Elkashef, MD, INSYS Therapeutics Inc

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Benuvia Therapeutics Inc.
• ClinicalTrials.gov Identifier: NCT03458416
• Other Study ID Numbers: INS011-17-115
• First Posted: Mar 8, 2018
• Last Update Posted: Nov 13, 2020
• Last Verified: Nov 1, 2020

• 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; Cannabidiol

Study Results

No Results Posted as of Nov 13, 2020