Prader-Willi SyndromePhase 2NCT03458416

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

A Phase 2 study testing Cannabidiol Oral Solution in people with Prader-Willi Syndrome, run by Radius Pharmaceuticals, Inc. at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 8 to 17 years
Conditions the study is for: Prader-Willi Syndrome
What is being tested?: Cannabidiol Oral Solution
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 2 participants
Where: 1 siteUnited States
Study dates: 6 Sep 2018to 31 Jul 2019
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 8 to 17 years
All sexes
No healthy volunteers
Child

Who may be able to join

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.

Who cannot join

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.

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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.

Trial locations and contacts

1 site in 1 country.

United States1 site

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

Other trials in United States

About this study

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

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: Prader-Willi Syndrome
Being tested: Cannabidiol Oral Solution
Medical subject headings: Prader-Willi Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Cannabidiol Oral SolutionDrug
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).

Study groups in NCT03458416
Study group	Role	What they receive
Cannabidiol Oral Solution: 20-40 mg/kg/day	Experimental	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. Receives: Drug: Cannabidiol Oral Solution

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Up to Week 52

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

6 Sep 201831 Jul 2019

First posted8 Mar 2018
Study start6 Sep 2018
Primary completion31 Jul 2019
Study completion31 Jul 2019

Why it stopped: Insys Therapeutics filed Chapter 11 and terminated all studies.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

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

IDs, dates and publications

ClinicalTrials.gov ID: NCT03458416
Sponsor's protocol ID: INS011-17-115
Status last verified: May 2023
First submitted: 2 Mar 2018
Last updated: 22 Jun 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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A Study of CSTI-500 in Patients With Prader-Willi Syndrome
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Cerebellar TMS and Satiety in Prader-Willi Syndrome
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Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome
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A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
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All Prader-Willi Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 22 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT03458416
Acronym / sponsor ID: INS011-17-115
Conditions: Prader-Willi Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Insys Therapeutics filed Chapter 11 and terminated all studies.
Enrolment: 2 participants (actual)
Lead sponsor: Radius Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
Collaborators: Benuvia Therapeutics Inc.
Study start: 6 Sep 2018 (actual)
Primary completion: 31 Jul 2019 (actual)
Study completion: 31 Jul 2019 (actual)
First posted: 8 Mar 2018 (actual)
Results: Posted 22 Jun 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Cannabidiol Oral Solution: 20-40 mg/kg/day	Experimental	Drug: Cannabidiol Oral SolutionParticipants 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.

Who may qualify

Ages: 8 to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (7 listed)

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.

Key exclusion criteria (9 listed)

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).

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Cannabidiol Oral SolutionAn oral solution containing pharmaceutical grade cannabidiol (nonplant-based).	Drug	Cannabidiol Oral Solution: 20-40 mg/kg/day

Primary outcome

Incidence Of Adverse Events (AEs) And Serious Adverse Events (SAEs) Associated With Cannabidiol Oral Solution
Time frame: 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.

Results and safety

Results posted to the registry on 22 Jun 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Cannabidiol Oral Solution	0	0

Primary outcome: Incidence Of Adverse Events (AEs) And Serious Adverse Events (SAEs) Associated With Cannabidiol Oral Solution

Time frame: Up to Week 52

Population analysed: 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.

Adverse events

Time frame: Up to 52 Weeks. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cannabidiol Oral Solution	0 of 0	0 of 0	0

Limitations and caveats (from the sponsor): 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.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Prader-Willi Syndrome

RecruitingPhase 2
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Recruiting
Cerebellar TMS and Satiety in Prader-Willi Syndrome
Prader-Willi Syndrome
Enrolling by invitationObservational study
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Active, not recruitingPhase 1/2
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
Hypothalamic Obesity, Prader-Willi Syndrome
Active, not recruiting
Impact of Bright Light Therapy on Prader-Willi Syndrome
Prader-Willi Syndrome, Excessive Daytime Sleepiness

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Ahmed Elkashef, MD study director

INSYS Therapeutics Inc

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Cannabidiol Oral Solution in people with Prader-Willi Syndrome, run by Radius Pharmaceuticals, Inc. at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 22 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 22 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence Of Adverse Events (AEs) And Serious Adverse Events (SAEs) Associated With Cannabidiol Oral Solution (time frame: Up to Week 52). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03458416.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2023.