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Due to a change in corporate priorities, this study was voluntarily terminated by the Sponsor for reasons other than safety
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RAD011 40 milligrams per kilogram (mg/kg) | Active Comparator | Participants were administered 40 mg/kg of RAD011 orally daily with food. |
|
| RAD011 20 mg/kg | Active Comparator | Participants were administered 20 mg/kg of RAD011 orally daily with food. |
|
| RAD011 10 mg/kg | Active Comparator | Participants were administered 10 mg/kg of RAD011 orally daily with food. |
|
| Placebo | Placebo Comparator | Participants were administered a placebo matching to RAD011, orally daily with food. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RAD011 | Drug | Cannabidiol Oral Solution (containing synthetic cannabidiol) |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline, Week 34 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States | ||
| Rady Children's Hospital |
In this study, 4 participants were randomized and received at least 1 dose of study drug. No participants were randomized to the placebo group. The 4 participants were randomized in Phase 2 of the study and due to premature termination of the study, phase 3 was not conducted. None of the enrolled participants completed the study due to the study being terminated prematurely.
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| ID | Title | Description |
|---|---|---|
| FG000 | RAD011 10 Milligrams Per Kilogram (mg/kg) | Participants were administered 10 mg/kg of RAD011 orally daily with food. |
| FG001 | RAD011 20 mg/kg | Participants were administered 20 mg/kg of RAD011 orally daily with food. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 10, 2022 | Aug 16, 2023 |
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| Placebo | Drug | Matching Placebo for RAD011 |
|
| 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. | 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. | Baseline, Week 34 |
| San Diego |
| California |
| 92123 |
| United States |
| University of Iowa | Iowa City | Iowa | 52242 | United States |
| Maimonides Medical Center | Brooklyn | New York | 11219 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37212 | United States |
| Research Institute of Dallas | Dallas | Texas | 75231 | United States |
| MultiCare Institute for Research & Innovation | Tacoma | Washington | 98405 | United States |
| FG002 | RAD011 40 mg/kg | Participants were administered 40 mg/kg of RAD011 orally daily with food. |
| FG003 | Placebo | Participants were administered a placebo matching to RAD011, orally daily with food. |
| Received 1 Dose of Study Drug |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
The Intent to Treat (ITT) Population included all randomized participants who received at least 1 dose of study drug. Due to the small sample size, data was not reported for confidentiality reasons.
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| ID | Title | Description |
|---|---|---|
| BG000 | RAD011 10 mg/kg | Participants were administered 10 mg/kg of RAD011 orally daily with food. |
| BG001 | RAD011 20 mg/kg | Participants were administered 20 mg/kg of RAD011 orally daily with food. |
| BG002 | RAD011 40 mg/kg | Participants were administered 40 mg/kg of RAD011 orally daily with food. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median | Full Range | years |
| ||||||||||
| Sex: Female, Male | Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons. | Count of Participants | Participants |
| ||||||||||
| Ethnicity (NIH/OMB) | Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons. | Count of Participants | Participants |
| ||||||||||
| Race (NIH/OMB) | Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons. | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Due to the premature termination of the study, no efficacy analyses were conducted. Therefore, no data was collected for this outcome measure. | Posted | Baseline, Week 34 |
|
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| Secondary | 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. | Due to the premature termination of the study, no efficacy analyses were conducted. Therefore, no data was collected for this outcome measure. | Posted | Baseline, Week 34 |
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| Secondary | 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. | Due to the premature termination of the study, no efficacy analyses were conducted. Therefore, no data was collected for this outcome measure. | Posted | Baseline, Week 34 |
| ||||||||||||||||||||||||||
| Secondary | 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. | Due to the premature termination of the study, no efficacy analyses were conducted. Therefore, no data was collected for this outcome measure. | Posted | Baseline, Week 34 |
|
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Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | RAD011 10 mg/kg | Participants were administered 10 mg/kg of RAD011 orally daily with food. | 0 | 1 | 0 | 1 | 1 | 1 |
| EG001 | RAD011 20 mg/kg | Participants were administered 20 mg/kg of RAD011 orally daily with food. | 0 | 1 | 0 | 1 | 1 | 1 |
| EG002 | RAD011 40 mg/kg | Participants were administered 40 mg/kg of RAD011 orally daily with food. | 0 | 2 | 0 | 2 | 2 | 2 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dermatitis | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Lethargy | Nervous system disorders | Systematic Assessment |
| ||
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Irritability | Psychiatric disorders | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
| ||
| Ear discomfort | Ear and labyrinth disorders | Systematic Assessment |
|
The study has been voluntarily terminated by the sponsor due to change in corporate priorities, for reasons other than safety. Due to early termination of study, no efficacy analyses were done.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Ellie Ratigan | Radius Pharmaceuticals, Inc. | 617-444-1800 | scout015@radiuspharm.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 10, 2022 | Aug 16, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D011218 | Prader-Willi Syndrome |
| ID | Term |
|---|---|
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D000096803 | Imprinting Disorders |
| D009765 | Obesity |
| D050177 | Overweight |
| D044343 | Overnutrition |
| D009748 | Nutrition Disorders |
| D009750 | Nutritional and Metabolic Diseases |
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| Units | Counts |
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| Participants |
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| Participants |
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