TerminatedNCT03458416Updated Jun 22, 2023Results posted

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

A Phase 2 interventional study of Cannabidiol Oral Solution in Prader-Willi Syndrome, sponsored by Radius Pharmaceuticals, Inc.. Terminated at 1 site in United States. Open to participants aged 8 Years to 17 Years. Per ClinicalTrials.gov, last updated 2023-06-22.

Sponsored by Radius Pharmaceuticals, Inc. · Phase 2, Interventional, and Treatment

Why this study was terminated

Insys Therapeutics filed Chapter 11 and terminated all studies.

Phase: Phase 2
Study type: Interventional
Enrollment: 2
Allocation: Not applicable
Ages: 8 Years to 17 Years
Sex: All

Study 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.

Conditions studied

Prader-Willi Syndrome

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Who can participate

Ages eligible: 8 Years to 17 Years
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Completed activities up to and including Visit 10 (Study Completion) of INS011-16- 085.
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.
If female, is either not of childbearing potential or practicing medically acceptable methods of birth control.
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.
Growth hormone treatment will be permitted if the participant has been on a stable dose during INS01-16-085.
Any other treatment including thyroid hormones should be stable prior to entering the INS011-17-115 study.
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

Exclusion Criteria:

Participant or parent(s)/caregiver(s) have commitments during the study duration that would interfere with attending all study visits.
Experienced an anoxic episode related to study drug requiring resuscitation during the previous study.
Uncontrolled Type I and Type II Diabetes.
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.
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).
Currently taking felbamate.
Compromised respiratory function or severe respiratory insufficiency.
Pregnant or lactating female.
In the opinion of the investigator, the participant is unsuitable in any other way to participate in this study.

Study design

Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 2 participants (actual)

Study arms

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

Interventions

DrugCannabidiol Oral Solution
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).

What researchers measure

Primary outcomes

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

Results

Posted Jun 22, 2023

Limitations and caveats

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.

Participant flow

Participant flow — Overall Study
Milestone	Cannabidiol Oral Solution
Started	0
Completed	0
Not completed	0

Outcome measures

PrimaryIncidence 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

No measurements were reported for this outcome.

Adverse events

Collected over Up to 52 Weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Cannabidiol Oral Solution	—	—	—

Baseline characteristics

Age, Continuous
Age, Continuous	Cannabidiol Oral Solution

Sex: Female, Male
Sex: Female, Male	Cannabidiol Oral Solution
Female	—
Male	—

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)	Cannabidiol Oral Solution
Hispanic or Latino	—
Not Hispanic or Latino	—
Unknown or Not Reported	—

Study locations

1 site

Institute for Research and Innovation | MultiCare Health System
Tacoma, Washington 98405, United States

References and documents

Study documents

Study protocol · Feb 27, 2018
Statistical analysis plan · Oct 4, 2018

Documents are hosted by the registry — open the source record to download them.

Registry details

Key details

Study ID: NCT03458416
Lead sponsor: Radius Pharmaceuticals, Inc.
Collaborators: Benuvia Therapeutics Inc.
Responsible party: Sponsor
First posted: Mar 8, 2018
Start date: Sep 6, 2018
Primary completion: Jul 31, 2019
Completion: Jul 31, 2019
Results posted: Jun 22, 2023
Last update: Jun 22, 2023

Study contacts

Ahmed Elkashef, MD

study director · INSYS Therapeutics Inc

Oversight

Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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Discussion

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