A Phase 2 interventional study of Cannabidiol and Placebo in Prader-Willi Syndrome, sponsored by Radius Pharmaceuticals, Inc.. Terminated at 7 sites in United States. Open to participants aged 8 Years to 17 Years. Per ClinicalTrials.gov, last updated 2023-08-01.
Sponsored by Radius Pharmaceuticals, Inc. · Phase 2, Interventional, and Treatment
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.
144 studies on the registry are indexed under Prader-Willi Syndrome; 23 are open to participants now.
This study's enrollment of 7 is below the median of 30 across 100 interventional studies indexed under Prader-Willi Syndrome.
Browse Prader-Willi Syndrome studies →Radius Pharmaceuticals, Inc. is the lead sponsor of 9 studies on the registry; none are open to participants now.
Of its 7 completed or terminated interventional studies of FDA-regulated products, 7 (100%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
If female, is either not of (defined as premenarchal or surgically sterile [bilateral tubal ligation, bilateral oophorectomy, or hysterectomy]) or practicing one of the following medically acceptable methods of :
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cannabidiol oral solution (40 milligram/kilogram/day \[40 mg/kg/day\]) divided into two daily doses with a standard meal
Drug: Cannabidiol
Matching placebo solution divided into two daily doses with a standard meal
Drug: Placebo
Oral solution
Matching oral solution
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.
Time frame: Baseline, Week 13
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
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]
The change from baseline to Week 13 of the participant's quality of life (PROMIS life satisfaction and positive affect questionnaire) score is evaluated.
Time frame: 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.
Time frame: Baseline, Week 13
Participants took part in the study at 5 investigative sites in the United States from 09 May 2018 and 31 July 2019.
| Milestone | Cannabidiol | Placebo |
|---|---|---|
| Started | 4 | 3 |
| Received at least 1 dose of study drug | 4 | 3 |
| Completed | 1 | 1 |
| Not completed | 3 | 2 |
| Withdrew: Discontinued due to premature termination of study | 3 | 2 |
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.
| score on a scale | Cannabidiol | Placebo |
|---|---|---|
| Baseline | 23 (10 to 25) | 22 (18 to 26) |
| Week 13 | 14 (7 to 20) | 16 (10 to 28) |
| Change from Baseline at Week 13 | -4.5 (-14 to -3) | -2 (-12 to 2) |
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.
| kg | Cannabidiol | Placebo |
|---|---|---|
| Baseline | 88.35 (47.9 to 112.4) | 56.7 (45.5 to 109.7) |
| Week 13 | 87.6 (47.4 to 110.8) | 59.5 (46.8 to 109.4) |
| Change From Baseline at Week 13 | -1.05 (-1.6 to 0.1) | 1.3 (-0.3 to 1.3) |
A responder was defined as having a 6 or more points decrease in HQ-CT score.
| Participants | Cannabidiol | Placebo |
|---|---|---|
| Yes | 1 | 1 |
| No | 3 | 2 |
The change in PGI-C scores at Week 3, Week 9, and Week 13 of the participant are evaluated.
No measurements were reported for this outcome.
The change from baseline to Week 13 of the participant's TFEQ-R18 score is evaluated.
No measurements were reported for this outcome.
The change from baseline to Week 13 of the participant's quality of life (PROMIS life satisfaction and positive affect questionnaire) score is evaluated.
No measurements were reported for this outcome.
The change from baseline to Week 13 of the participant's physical activity (PROMIS physical activity and fatigue questionnaires) score is evaluated.
No measurements were reported for this outcome.
Collected over Baseline up to Week 13. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cannabidiol | 0/4 (0%) | 0/4 (0%) | 4/4 (100%) |
| Placebo | 0/3 (0%) | 0/3 (0%) | 2/3 (66.7%) |
| Event | Cannabidiol | Placebo |
|---|---|---|
| DiarrheaGastrointestinal disorders | 2/4 | 1/3 |
| RashSkin and subcutaneous tissue disorders | 2/4 | 0/3 |
| Upper respiratory tract infectionInfections and infestations | 1/4 | 1/3 |
| SeizureNervous system disorders | 0/4 | 1/3 |
| Abdominal crampingGastrointestinal disorders | 1/4 | 0/3 |
| ImpetigoInfections and infestations | 1/4 | 0/3 |
| CellulitisInfections and infestations | 1/4 | 0/3 |
| Ear infectionEar and labyrinth disorders | 1/4 | 0/3 |
| Bacterial infectionInfections and infestations | 1/4 | 0/3 |
All participants who were enrolled and received at least 1 dose of study drug.
| Age, Continuous(years) | Cannabidiol | Placebo | Total |
|---|---|---|---|
| Mean | 13.5 (10 to 15) | 11.67 (8 to 15) | 12.7 (8 to 15) |
| Sex: Female, Male(Participants) | Cannabidiol | Placebo | Total |
|---|---|---|---|
| Female | 2 | 1 | 3 |
| Male | 2 | 2 | 4 |
| Ethnicity (NIH/OMB)(Participants) | Cannabidiol | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 1 | 0 | 1 |
| Not Hispanic or Latino | 2 | 3 | 5 |
| Unknown or Not Reported | 1 | 0 | 1 |
| Race (NIH/OMB)(Participants) | Cannabidiol | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 1 | 1 |
| White | 3 | 2 | 5 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 0 | 1 |
| Baseline in Total Body Weight(kg) | Cannabidiol | Placebo | Total |
|---|---|---|---|
| Median | 88.35 (47.9 to 112.4) | 56.7 (45.5 to 109.7) | 72.50 (45.5 to 112.4) |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is terminated, as verified in Jul 2023. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Cannabidiol
Radius Pharmaceuticals, Inc.