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

Radius Health

Status and phase: Terminated; Phase 2

Conditions: Prader-Willi Syndrome

Treatments: Drug: Cannabidiol Oral Solution

Study type: Interventional

Funder types: Industry

Identifiers

NCT03458416

INS011-17-115

Details and patient eligibility

About

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.

Enrollment: 2 patients

Sex: All

Ages: 8 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Completed activities up to and including Visit 10 (Study Completion) of INS011-16- 085.
2. 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.
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 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.
5. Growth hormone treatment will be permitted if the participant 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. Participant 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 partient.
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 participant is unsuitable in any other way to participate in this study.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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2 participants in 1 patient group

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

Experimental group

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.

Treatment:

Drug: Cannabidiol Oral Solution