Prader-Willi SyndromePhase 2/3NCT05098509
A Study to Assess RAD011 (Cannabidiol Oral Solution) for the Treatment of Participants With Prader-Willi Syndrome
A Phase 2/3 study testing RAD011 in people with Prader-Willi Syndrome, run by Radius Pharmaceuticals, Inc. at 8 sites in United States.
Trial summary
This was a study investigating RAD011 in participants diagnosed with Prader-Willi Syndrome (PWS). The primary objective of the Phase 2 part of this study was to assess the safety and tolerability of multiple dose levels of RAD011 in order to select 1 or 2 dose level(s) for further evaluation in the Phase 3 part of the… Read more about this study
- Who is it for?
- Ages 8 to 65 years
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- RAD011
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 4 participants
- Where
- 8 sitesUnited States
- Study dates
- 13 Apr 2022to 31 Oct 2022
- Phase
- Phase 2/3
- 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 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Males and females between 8 and 65 years of age (inclusive) at Screening.
- Genetically confirmed diagnosis of PWS. Documentation of genetically confirmed diagnosis of PWS is acceptable.
- The same caregiver is available to complete the questionnaire throughout the duration of the study.
- After completion of the Tolerability period, participants will have a mean Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score ≥13 and a decrease of HQ-CT score no more than 7 during Tolerability (run-in) period.
- If receiving growth hormone, psychotropic therapy, metabolic treatments that could affect appetite (including metformin), and other treatment including thyroid hormone, must be on the same medication and dose for at least 90 days prior to consent/assent
Who cannot join
- Known use of cannabis or cannabinoid containing products (including topical products) within 90 days prior to consent/ assent.
- Use of prescription or over-the-counter weight loss agents within 90 days prior to consent/assent.
- Implementation of new food or environmental restrictions within 90 days of consent/ assent.
- If living in a group home, participant spends less than 25 waking hours with their caregiver per week.
- Uncontrolled chronic conditions (diabetes, sleep apnea, etc.) as determined by the Investigator.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Maimonides Medical Center
Brooklyn, New York, 11219
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Research Institute of Dallas
Dallas, Texas, 75231
University of Iowa
Iowa City, Iowa, 52242
Children's Hospital Los Angeles
Los Angeles, California, 90027
Vanderbilt University Medical Center
Nashville, Tennessee, 37212
Rady Children's Hospital
San Diego, California, 92123
MultiCare Institute for Research & Innovation
Tacoma, Washington, 98405
About this study
Official title: A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study of RAD011 (Cannabidiol Oral Solution) for the Treatment of Patients With Prader- Willi Syndrome
This was a study investigating RAD011 in participants diagnosed with Prader-Willi Syndrome (PWS). The primary objective of the Phase 2 part of this study was to assess the safety and tolerability of multiple dose levels of RAD011 in order to select 1 or 2 dose level(s) for further evaluation in the Phase 3 part of the study. In Phase 3, the primary objective was to assess the effect of RAD011 on hyperphagia-related behavior in participants with PWS.
- Conditions
- Prader-Willi Syndrome
- Being tested
- RAD011, 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.
RAD011Drug
Cannabidiol Oral Solution (containing synthetic cannabidiol)
PlaceboDrug
Matching Placebo for RAD011
| Study group | Role | What they receive |
|---|---|---|
| RAD011 40 milligrams per kilogram (mg/kg) | Active comparator | Participants were administered 40 mg/kg of RAD011 orally daily with food. Receives: Drug: RAD011 |
| RAD011 20 mg/kg | Active comparator | Participants were administered 20 mg/kg of RAD011 orally daily with food. Receives: Drug: RAD011 |
| RAD011 10 mg/kg | Active comparator | Participants were administered 10 mg/kg of RAD011 orally daily with food. Receives: Drug: RAD011 |
| Placebo | Placebo comparator | Participants were administered a placebo matching to RAD011, orally daily with food. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline to Week 34 in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Questionnaire
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.
Measured: Baseline, Week 34
Secondary outcomes (3)
Change From Baseline to Week 34 in Prader-Willi Syndrome (PWS)- Associated Irritability Using the Aberrant Behavior Checklist (ABC) Questionnaire - Irritability Subscale (ABC-I)
The ABC questionnaire is an informant-rated questionnaire assessing severity of behavioral symptoms. The Irritability subscale of the ABC covers symptoms such as agitation, aggression, meltdowns, and self-harm. The ABC-I contains 15 items and each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). The total score is the sum of individual items scores which ranges from 0 (no problem) to 45 (severe problem), with higher score indicating more severe condition.
Measured: Baseline, Week 34
Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Change (CGI-C)
The CGI-C of hyperphagia is a single-item, clinician-rated measure, assessing the clinician's impression about changes in the patient's hyperphagia condition since the start of taking the study medication at the initiation of the Tolerability Period. The CGI-C of hyperphagia utilizes a 5-point response scale: 1=Much better; 2=A little better; 3=No change; 4=A little worse; 5=Much worse. Higher scores mean a worse outcome.
Measured: Baseline, Week 34
Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Severity (CGI-S)
The CGI-S of hyperphagia is a single-item, clinician-rated measure, assessing the clinician's impression of the severity of a patient's hyperphagia condition. The CGI-S of hyperphagia utilizes a 5-point response scale: 1=None; 2=Mild; 3=Moderate; 4=Severe; 5=Very Severe. Higher scores mean a worse outcome.
Measured: Baseline, Week 34
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 2021
- Study start13 Apr 2022
- Primary completion6 Oct 2022
- Study completion31 Oct 2022
Why it stopped: Due to a change in corporate priorities, this study was voluntarily terminated by the Sponsor for reasons other than safety
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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Radius Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05098509
- Sponsor's protocol ID
- SCOUT-015
- Status last verified
- Sep 2023
- First submitted
- 22 Sep 2021
- Last updated
- 19 Oct 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study of RAD011 (Cannabidiol Oral Solution) for the Treatment of Patients With Prader- Willi Syndrome
- ClinicalTrials.gov ID
- NCT05098509
- Acronym / sponsor ID
- SCOUT-015
- Conditions
- Prader-Willi Syndrome
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to a change in corporate priorities, this study was voluntarily terminated by the Sponsor for reasons other than safety
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Radius Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Apr 2022 (actual)
- Primary completion
- 6 Oct 2022 (actual)
- Study completion
- 31 Oct 2022 (actual)
- First posted
- 28 Oct 2021 (actual)
- Results
- Posted 19 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a study investigating RAD011 in participants diagnosed with Prader-Willi Syndrome (PWS). The primary objective of the Phase 2 part of this study was to assess the safety and tolerability of multiple dose levels of RAD011 in order to select 1 or 2 dose level(s) for further evaluation in the Phase 3 part of the study. In Phase 3, the primary objective was to assess the effect of RAD011 on hyperphagia-related behavior in participants with PWS.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| RAD011 40 milligrams per kilogram (mg/kg) | Active comparator | Drug: RAD011Participants were administered 40 mg/kg of RAD011 orally daily with food. |
| RAD011 20 mg/kg | Active comparator | Drug: RAD011Participants were administered 20 mg/kg of RAD011 orally daily with food. |
| RAD011 10 mg/kg | Active comparator | Drug: RAD011Participants were administered 10 mg/kg of RAD011 orally daily with food. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants were administered a placebo matching to RAD011, orally daily with food. |
Who may qualify
- Ages
- 8 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Males and females between 8 and 65 years of age (inclusive) at Screening.
- Genetically confirmed diagnosis of PWS. Documentation of genetically confirmed diagnosis of PWS is acceptable.
- The same caregiver is available to complete the questionnaire throughout the duration of the study.
- After completion of the Tolerability period, participants will have a mean Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score ≥13 and a decrease of HQ-CT score no more than 7 during Tolerability (run-in) period.
- If receiving growth hormone, psychotropic therapy, metabolic treatments that could affect appetite (including metformin), and other treatment including thyroid hormone, must be on the same medication and dose for at least 90 days prior to consent/assent
Key exclusion criteria (5 listed)
- Known use of cannabis or cannabinoid containing products (including topical products) within 90 days prior to consent/ assent.
- Use of prescription or over-the-counter weight loss agents within 90 days prior to consent/assent.
- Implementation of new food or environmental restrictions within 90 days of consent/ assent.
- If living in a group home, participant spends less than 25 waking hours with their caregiver per week.
- Uncontrolled chronic conditions (diabetes, sleep apnea, etc.) as determined by the 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 |
|---|---|---|
| RAD011Cannabidiol Oral Solution (containing synthetic cannabidiol) | Drug | RAD011 10 mg/kg, RAD011 20 mg/kg, RAD011 40 milligrams per kilogram (mg/kg) |
| PlaceboMatching Placebo for RAD011 | Drug | Placebo |
Primary outcome
Change From Baseline to Week 34 in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Questionnaire
Time frame: Baseline, Week 34
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.
3 secondary outcomes
Change From Baseline to Week 34 in Prader-Willi Syndrome (PWS)- Associated Irritability Using the Aberrant Behavior Checklist (ABC) Questionnaire - Irritability Subscale (ABC-I)
Time frame: Baseline, Week 34
Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Change (CGI-C)
Time frame: Baseline, Week 34
Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Severity (CGI-S)
Time frame: Baseline, Week 34
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 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.
The record lists no contact. Use the official record to reach the sponsor.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing RAD011 in people with Prader-Willi Syndrome, run by Radius Pharmaceuticals, Inc. at 8 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 5 inclusion and 5 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 19 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 19 Oct 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 to Week 34 in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Questionnaire (time frame: Baseline, Week 34). 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 19 Oct 2023.