Prader-Willi Syndrome
Conditions
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.
Interventions
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
Sponsors
Benuvia Therapeutics Inc.
Radius Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
8 Years to 17 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence Of Adverse Events (AEs) And Serious Adverse Events (SAEs) Associated With Cannabidiol Oral Solution | Up to Week 52 | 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. |
Countries
United States
Participant flow
Recruitment details
This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.
Pre-assignment details
Participants were enrolled in this long-term safety study after completing the INS011-16-085 study. Only 2 participants enrolled in this study. The participants received cannabidiol oral solution (CBD) doses for up to 48 weeks, and a follow-up period of 30 days.
Participants by arm
| Arm | Count |
|---|---|
| Cannabidiol Oral Solution Participants received 20 mg/kg/day to 40 mg/kg/day of CBD given twice daily with standard meals. | 0 |
| Total | 0 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 0 |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
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.
Time frame: Up to Week 52
Population: This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.