Prader-Willi SyndromePhase 2NCT02844933
Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
A Phase 2 study testing Cannabidiol in people with Prader-Willi Syndrome, run by Radius Pharmaceuticals, Inc. at 7 sites in United States.
Trial summary
The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS). The secondary objectives of this study are to assess the efficacy, safety and tolerability, impact on quality of life, and impact on physical activity… 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
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 7 participants
- Where
- 7 sitesUnited States
- Study dates
- 9 May 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
- 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.
- Participants with a genetically confirmed diagnosis of Prader-Willi Syndrome using standard deoxyribonucleic acid methylation test or fluorescent in situ hybridization. Documentation of genetically confirmed diagnosis of Prader-Willi Syndrome is acceptable.
- A score of ≥10 on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT).
- A caregiver is available to complete the HQ-CT.
- If female, is either not of childbearing potential (defined as premenarchal or surgically sterile [bilateral tubal ligation, bilateral oophorectomy, or hysterectomy]) or practicing one of the following medically acceptable methods of birth control:
- Hormonal methods such as oral, implantable, injectable, vaginal ring, or transdermal contraceptives for a minimum of 1 full cycle (based on the participant's usual menstrual cycle period) before study drug administration.
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- Total abstinence from sexual intercourse since the last menses before study drug administration.
- Intrauterine device.
- Double-barrier method (condoms, sponge, or diaphragm with spermicidal jellies or cream).
- Adequate renal function, defined as serum creatinine ≤ 1.5*upper limit of normal (ULN) and urine protein/creatinine ratio ≤0.4. The Investigator may deem the participant eligible if he or she judges the laboratory values to be not clinically significant.
- Adequate hepatic function, defined as total bilirubin ≤ 1.5*ULN and aspartate aminotransferase and alanine aminotransferase levels ≤ 3*ULN.
- Growth hormone treatment will be permitted if doses have been stable for at least 1 month prior to screening.
- Psychotropic treatment will be permitted and should be stable at least 6 weeks prior to screening.
- Any other treatment including thyroid hormones should be stable for at least 6 weeks prior to screening.
Who cannot join
- Known use of cannabis or cannabinoid-containing products for 4 weeks prior to baseline.
- History of chronic liver diseases, such as cirrhosis or chronic hepatitis due to any cause, or suspected alcohol abuse.
- Use of weight loss agents or drugs known to affect appetite (including glucagon-like peptide-1 [GLP-1] analogs) within 2 months prior to screening.
- Uncontrolled Type I and Type II diabetes.
- Currently taking concomitant medication that are strong-moderate inhibitors/inducers/sensitive substrates with a narrow therapeutic index for CYP2C19 or CYP3A.
- Co-morbid condition or disease (such as respiratory disease, heart disease, or psychiatric disorder) diagnosed less than 1 month prior to screening.
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- History or presence of gastrointestinal or any other condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs as determined by the Investigator.
- Participants who have participated in any other trials involving an investigational product or device within 30 days of screening or longer as required by local regulations.
- Clinically significant abnormalities on electrocardiogram at screening or other evidence of heart disease as determined by the Investigator.
- Has screening systolic blood pressure ≥160 millimeters of mercury (mmHg) and diastolic blood pressure >100 mm Hg (may be repeated 1 additional time after 5 minutes rest to verify). Participants with hypertensive levels lower than those specified may be excluded at the Investigator's discretion if deemed to be in the best interest of the participant.
- Currently taking felbamate.
- Uncontrolled sleep apnea.
- Pregnant or lactating female.
- History of hypersensitivity to drugs with a similar chemical structure or class as cannabidiol.
- Unwillingness or inability to follow the procedures outlined in the protocol.
- Participant judged by the investigator or sponsor (or designee) as unable to comply with the treatment protocol, including appropriate supportive care, follow-up and research tests.
- Positive drug screen, including tetrahydrocannabinol, at time of screening.
- Creatinine clearance test of < 30 milliliters/minute (mL/min).
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Johns Hopkins University
Baltimore, Maryland, 21287
University of Iowa
Iowa City, Iowa, 52242
The University of Kansas , Medical Center
Kansas City, Kansas, 66160
Rady Children's, UC San Diego
San Diego, California, 92123
Institute for Research and Innovation | MultiCare Health System
Tacoma, Washington, 98405
University of Arizona
Tucson, Arizona, 85724
The University of Oklahoma Health Sciences Center
Tulsa, Oklahoma, 74135
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS). The secondary objectives of this study are to assess the efficacy, safety and tolerability, impact on quality of life, and impact on physical activity of cannabidiol oral solution in patients with PWS.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Cannabidiol, Placebo
- 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.
CannabidiolDrug
Oral solution
PlaceboDrug
Matching oral solution
| Study group | Role | What they receive |
|---|---|---|
| Cannabidiol | Experimental | Cannabidiol oral solution (40 milligram/kilogram/day [40 mg/kg/day]) divided into two daily doses with a standard meal Receives: Drug: Cannabidiol |
| Placebo | Placebo comparator | Matching placebo solution divided into two daily doses with a standard meal Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Hyperphagia Behavior as Measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)
The HQ-CT measures hyperphagia by Prader-Willi syndrome (PWS)-specialized clinicians. The HQ-CT generates a score ranging from 0 to 36, where a higher score represents more severe abnormal food related behaviors.
The change from baseline was calculated from two time points as the value at the later time point minus the value at the earlier time point: value at Week 13 minus value at Baseline.
Measured: Baseline, Week 13
Secondary outcomes (6)
Change From Baseline In Total Body Weight
Total body weight refers to participants' weight throughout the study. The change from baseline was calculated from two time points as the value at the later time point minus the value at the earlier time point: value at Week 13 minus value at Baseline.
Measured: Baseline, Week 13
Responder Rate From Baseline Through Study Completion
A responder was defined as having a 6 or more points decrease in HQ-CT score.
Measured: Baseline up to Week 13
Change In Patient Global Impression Of Change (PGI-C) Questionnaire
The change in PGI-C scores at Week 3, Week 9, and Week 13 of the participant are evaluated.
Measured: Week 3, Week 13
Change From Baseline In The Three Factor Eating Questionnaire - 18-item Version (TFEQ-R18)
The change from baseline to Week 13 of the participant's TFEQ-R18 score is evaluated.
Measured: Baseline, Week 13
Change From Baseline In Quality Of Life [Patient-Reported Outcomes Measurement Information System (PROMIS) Life Satisfaction And Positive Affect Questionnaires]
The change from baseline to Week 13 of the participant's quality of life (PROMIS life satisfaction and positive affect questionnaire) score is evaluated.
Measured: Baseline, Week 13
Change From Baseline In Physical Activity (PROMIS Physical Activity And Fatigue Questionnaires)
The change from baseline to Week 13 of the participant's physical activity (PROMIS physical activity and fatigue questionnaires) score is evaluated.
Measured: Baseline, Week 13
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2016 (estimated)
- Study start9 May 2018
- Primary completion17 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Radius Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ahmed Elkashef, MD, Study director, INSYS Therapeutics Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02844933
- Sponsor's protocol ID
- INS011-16-085
- Status last verified
- Jul 2023
- First submitted
- 22 Jul 2016
- Last updated
- 1 Aug 2023
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
- ClinicalTrials.gov ID
- NCT02844933
- Acronym / sponsor ID
- INS011-16-085
- Conditions
- Prader-Willi Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Insys Therapeutics filed Chapter 11 and terminated all studies.
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Radius Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 9 May 2018 (actual)
- Primary completion
- 17 Jul 2019 (actual)
- Study completion
- 31 Jul 2019 (actual)
- First posted
- 26 Jul 2016 (estimated)
- Results
- Posted 1 Aug 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS). The secondary objectives of this study are to assess the efficacy, safety and tolerability, impact on quality of life, and impact on physical activity of cannabidiol oral solution in patients with PWS.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cannabidiol | Experimental | Drug: CannabidiolCannabidiol oral solution (40 milligram/kilogram/day [40 mg/kg/day]) divided into two daily doses with a standard meal |
| Placebo | Placebo comparator | Drug: PlaceboMatching placebo solution divided into two daily doses with a standard meal |
Who may qualify
- Ages
- 8 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (14 listed)
- 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.
- Participants with a genetically confirmed diagnosis of Prader-Willi Syndrome using standard deoxyribonucleic acid methylation test or fluorescent in situ hybridization. Documentation of genetically confirmed diagnosis of Prader-Willi Syndrome is acceptable.
- A score of ≥10 on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT).
- A caregiver is available to complete the HQ-CT.
- If female, is either not of childbearing potential (defined as premenarchal or surgically sterile [bilateral tubal ligation, bilateral oophorectomy, or hysterectomy]) or practicing one of the following medically acceptable methods of birth control:
Key exclusion criteria (18 listed)
- Known use of cannabis or cannabinoid-containing products for 4 weeks prior to baseline.
- History of chronic liver diseases, such as cirrhosis or chronic hepatitis due to any cause, or suspected alcohol abuse.
- Use of weight loss agents or drugs known to affect appetite (including glucagon-like peptide-1 [GLP-1] analogs) within 2 months prior to screening.
- Uncontrolled Type I and Type II diabetes.
- Currently taking concomitant medication that are strong-moderate inhibitors/inducers/sensitive substrates with a narrow therapeutic index for CYP2C19 or CYP3A.
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 |
|---|---|---|
| CannabidiolOral solution | Drug | Cannabidiol |
| PlaceboMatching oral solution | Drug | Placebo |
Primary outcome
Change From Baseline in Hyperphagia Behavior as Measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)
Time frame: Baseline, Week 13
The HQ-CT measures hyperphagia by Prader-Willi syndrome (PWS)-specialized clinicians. The HQ-CT generates a score ranging from 0 to 36, where a higher score represents more severe abnormal food related behaviors. The change from baseline was calculated from two time points as the value at the later time point minus the value at the earlier time point: value at Week 13 minus value at Baseline.
6 secondary outcomes
Change From Baseline In Total Body Weight
Time frame: Baseline, Week 13
Responder Rate From Baseline Through Study Completion
Time frame: Baseline up to Week 13
Change In Patient Global Impression Of Change (PGI-C) Questionnaire
Time frame: Week 3, Week 13
Change From Baseline In The Three Factor Eating Questionnaire - 18-item Version (TFEQ-R18)
Time frame: Baseline, Week 13
Change From Baseline In Quality Of Life [Patient-Reported Outcomes Measurement Information System (PROMIS) Life Satisfaction And Positive Affect Questionnaires]
Time frame: Baseline, Week 13
Change From Baseline In Physical Activity (PROMIS Physical Activity And Fatigue Questionnaires)
Time frame: Baseline, Week 13
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Aug 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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
7 sites 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 in people with Prader-Willi Syndrome, run by Radius Pharmaceuticals, Inc. at 7 sites 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 14 inclusion and 18 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 1 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites 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. Some participants in this study may receive a placebo. 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 1 Aug 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?
Change From Baseline in Hyperphagia Behavior as Measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) (time frame: Baseline, Week 13). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 1 Aug 2023.