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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03458416
• Date of registration: 02/03/2018
• Prospective Registration: Yes
• Primary sponsor: Benuvia Therapeutics Inc.
• Public title: A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Patients With Prader-Willi Syndrome
• Scientific 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
• Date of first enrolment: September 6, 2018
• Target sample size: 7
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03458416
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Ahmed Elkashef, MD
• Affiliation: INSYS Therapeutics Inc

Key inclusion & exclusion 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.



Age minimum: 8 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Prader-Willi Syndrome

Intervention(s)

Drug: Cannabidiol Oral Solution

Primary Outcome(s)

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)]

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)]

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)]

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)]

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)]

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)]

Secondary ID(s)

INS011-17-115

Source(s) of Monetary Support

Please refer to primary and secondary sponsors