A Phase 2/3 interventional study of RAD011 and Placebo in Prader-Willi Syndrome, sponsored by Radius Pharmaceuticals, Inc.. Terminated at 8 sites in United States. Open to participants aged 8 Years to 65 Years. Per ClinicalTrials.gov, last updated 2023-10-19.
Sponsored by Radius Pharmaceuticals, Inc. · Phase 2/3, Interventional, and Treatment
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.
144 studies on the registry are indexed under Prader-Willi Syndrome; 23 are open to participants now.
This study's enrollment of 4 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.
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Participants were administered 40 mg/kg of RAD011 orally daily with food.
Drug: RAD011
Participants were administered 20 mg/kg of RAD011 orally daily with food.
Drug: RAD011
Participants were administered 10 mg/kg of RAD011 orally daily with food.
Drug: RAD011
Participants were administered a placebo matching to RAD011, orally daily with food.
Drug: Placebo
Cannabidiol Oral Solution (containing synthetic cannabidiol)
Matching Placebo for RAD011
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.
Time frame: Baseline, Week 34
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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline, Week 34
| Milestone | RAD011 10 Milligrams Per Kilogram (mg/kg) | RAD011 20 mg/kg | RAD011 40 mg/kg | Placebo |
|---|---|---|---|---|
| Started | 1 | 1 | 2 | 0 |
| Received 1 dose of study drug | 1 | 1 | 2 | 0 |
| Completed | 0 | 0 | 0 | 0 |
| Not completed | 1 | 1 | 2 | 0 |
| Withdrew: Withdrawal by subject | 0 | 0 | 1 | 0 |
| Withdrew: Early study termination | 1 | 1 | 1 | 0 |
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.
No measurements were reported for this outcome.
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.
No measurements were reported for this outcome.
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.
No measurements were reported for this outcome.
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.
No measurements were reported for this outcome.
Collected over Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| RAD011 10 mg/kg | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| RAD011 20 mg/kg | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| RAD011 40 mg/kg | 0/2 (0%) | 0/2 (0%) | 2/2 (100%) |
| Event | RAD011 10 mg/kg | RAD011 20 mg/kg | RAD011 40 mg/kg |
|---|---|---|---|
| DermatitisSkin and subcutaneous tissue disorders | 1/1 | 0/1 | 0/2 |
| LethargyNervous system disorders | 0/1 | 1/1 | 0/2 |
| DiarrheaGastrointestinal disorders | 0/1 | 0/1 | 2/2 |
| IrritabilityPsychiatric disorders | 0/1 | 0/1 | 1/2 |
| HeadacheNervous system disorders | 0/1 | 0/1 | 1/2 |
| FatigueGeneral disorders | 0/1 | 0/1 | 1/2 |
| Ear discomfortEar and labyrinth disorders | 0/1 | 0/1 | 1/2 |
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.
| Age, Continuous(years) | RAD011 10 mg/kg | RAD011 20 mg/kg | RAD011 40 mg/kg | Total |
|---|---|---|---|---|
| Median | NA (NA to NA) | NA (NA to NA) | NA (NA to NA) | NA (NA to NA) |
| Sex: Female, Male(Participants) | RAD011 10 mg/kg | RAD011 20 mg/kg | RAD011 40 mg/kg | Total |
|---|---|---|---|---|
| Female | 0 | 0 | 0 | 0 |
| Male | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB)(Participants) | RAD011 10 mg/kg | RAD011 20 mg/kg | RAD011 40 mg/kg | Total |
|---|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 | 0 |
| Not Hispanic or Latino | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | RAD011 10 mg/kg | RAD011 20 mg/kg | RAD011 40 mg/kg | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 | 0 |
| White | 0 | 0 | 0 | 0 |
| More than one race | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Apr 2022 to primary completion in Oct 2022, 5 months.
Results were first posted in Oct 2023, 1 year after primary completion.
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Radius Pharmaceuticals, Inc.