Trial · NCT02311673
Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
NCT02311673, Phase 2, Completed, Rhythm Pharmaceuticals, Inc.; results first posted 2023-07-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome. Trials, record NCT02311673. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02311673
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
- NCT number
- NCT02311673
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 40 participants, actual
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.
- Conditions
- Prader-Willi Syndrome
- Interventions
- Setmelanotide, Placebo
- First posted
- 2014-12-08
- Last updated
- 2023-07-27
- Results first posted
- 2023-07-27
Source ClinicalTrials.gov
Registered outcomes26
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 | Days 15 to 41 | An adverse event (AE) was any untoward medical occurrence in a clinical trial participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE is considered to be treatment-emergent if the onset date/time is during or after administration of double-blind study drug or, in the event that onset time precedes double-blind study drug administration, the AE increases in severity during or after administration of double-blind study drug; in either case through 1-week after the last treatment dose. A serious adverse event (SAE) was any untoward medical occurrence that, at any dose: * Resulted in death; * Was life-threatening; * Required inpatient hospitalization or prolongation of existing hospitalization; * Resulted in persistent disability/incapacity; * Was a congenital anomaly/birth defect |
| Primary | Number of Participants Who Experienced a TEAE - Period 3 | Days 42 to 55 | An AE was any untoward medical occurrence in a clinical trial participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE is considered to be treatment-emergent if the onset date/time is during or after administration of double-blind study drug or, in the event that onset time precedes double-blind study drug administration, the AE increases in severity during or after administration of double-blind study drug; in either case through 1-week after the last treatment dose. An SAE was any untoward medical occurrence that, at any dose: * Resulted in death; * Was life-threatening; * Required inpatient hospitalization or prolongation of existing hospitalization; * Resulted in persistent disability/incapacity; * Was a congenital anomaly/birth defect |
| Primary | Number of Participants Who Experienced a TEAE - Period 4 | Days 56 to 69 | An AE was any untoward medical occurrence in a clinical trial participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE is considered to be treatment-emergent if the onset date/time is during or after administration of double-blind study drug or, in the event that onset time precedes double-blind study drug administration, the AE increases in severity during or after administration of double-blind study drug; in either case through 1-week after the last treatment dose. An SAE was any untoward medical occurrence that, at any dose: * Resulted in death; * Was life-threatening; * Required inpatient hospitalization or prolongation of existing hospitalization; * Resulted in persistent disability/incapacity; * Was a congenital anomaly/birth defect |
| Primary | Mean Body Weight - Period 2 | Baseline (Day 15) | |
| Primary | Percent Change From Baseline in Body Weight - Period 2 | Baseline (Day 15) and Day 42 | |
| Primary | Overall Score of Prader-Willi Syndrome (PWS) Hyperphagia Questionnaire - Period 2 | Baseline (Day 15) | The hyperphagia questionnaire is a 10-item instrument designed to measure food-related preoccupations and problems in PWS, as well as the severity of these concerns. Three factors identified from this questionnaire are: Hyperphagic Drive, Hyperphagic Behaviors, and Hyperphagic Severity. Items are rated by care providers on a 5-point scale (1=not a problem to 5=a severe and/or frequent problem). Raw scores for each factor were used in data analyses, and the 3 domains were summed for an overall summary index of hyperphagia. Possible scores on the questionnaire range from a minimum score of 10 (no hyperphagia) to a maximum score of 50 (greater hyperphagia). |
| Primary | Percent Change From Baseline in Overall Score of Prader-Willi Syndrome (PWS) Hyperphagia Questionnaire - Period 2 | Baseline (Day 15) and Day 42 | The hyperphagia questionnaire is a 10-item instrument designed to measure food-related preoccupations and problems in PWS, as well as the severity of these concerns. Three factors identified from this questionnaire are: Hyperphagic Drive, Hyperphagic Behaviors, and Hyperphagic Severity. Items are rated by care providers on a 5-point scale (1=not a problem to 5=a severe and/or frequent problem). Raw scores for each factor were used in data analyses, and the 3 domains were summed for an overall summary index of hyperphagia. Possible scores on the questionnaire range from a minimum score of 10 (no hyperphagia) to a maximum score of 50 (greater hyperphagia). Percent change from baseline in overall score of PWS hyperphagia questionnaire is presented. |
| Secondary | Percent Change From Baseline in Hyperphagic Drive Score of PWS Hyperphagia Questionnaire - Period 2 | Baseline (Day 15) and Day 42 | The hyperphagia questionnaire is a 10-item instrument designed to measure food-related preoccupations and problems in PWS, as well as the severity of these concerns. Hyperphagic drive score assesses the persistence in asking for food based on 4 items. All 4 items are rated by care providers on a 5-point scale (1=not a problem to 5=a severe and/or frequent problem). Possible scores for hyperphagic drive range from a minimum score of 4 (no hyperphagic drive) to a maximum score of 20 (greater hyperphagic drive). Percent change from baseline in hyperphagic drive score of PWS hyperphagia questionnaire is presented. |
| Secondary | Percent Change From Baseline in Hyperphagic Behaviors Score of PWS Hyperphagia Questionnaire - Period 2 | Baseline (Day 15) and Day 42 | The hyperphagia questionnaire is a 10-item instrument designed to measure food-related preoccupations and problems in PWS, as well as the severity of these concerns. Hyperphagic behavior factor score assesses food seeking behaviors based on 4 items. All 4 items are rated by care providers on a 5-point scale (1=not a problem to 5=a severe and/or frequent problem). Possible scores for hyperphagic behaviors range from a minimum score of 4 (no hyperphagic behavior) to a maximum score of 20 (greater hyperphagic behavior). Percent change from baseline in hyperphagic behaviors score of PWS hyperphagia questionnaire is presented. |
| Secondary | Percent Change From Baseline in Hyperphagic Severity Score of PWS Hyperphagia Questionnaire - Period 2 | Baseline (Day 15) and Day 42 | The hyperphagia questionnaire is a 10-item instrument designed to measure food-related preoccupations and problems in PWS, as well as the severity of these concerns. Hyperphagic severity factor score assesses the severity of hyperphagia based on 2 items. Both items are rated by care providers on a 5-point scale (1=not a problem to 5=a severe and/or frequent problem). Possible scores for hyperphagic severity range from a minimum score of 2 (no hyperphagic severity) to a maximum score of 10 (greater hyperphagic severity). Percent change from baseline in hyperphagic severity score of PWS hyperphagia questionnaire is presented. |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 registered outcomesOutcomes26
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 | Days 15 to 41 | Safety Set. Participants were classified into groups based on actual treatment received. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Who Experienced a TEAE - Period 3 | Days 42 to 55 | Participants in the Safety Set with available data were analyzed. Participants were classified into groups based on actual treatment received. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Who Experienced a TEAE - Period 4 | Days 56 to 69 | Participants in the Safety Set with available data were analyzed. Participants were classified into groups based on actual treatment received. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Mean Body Weight - Period 2 | Baseline (Day 15) | Full Analysis Set (FAS) included all randomized participants who received at least 1 dose of randomized study medication with a baseline and at least 1 postbaseline efficacy observation within Period 2. Participants were classified based on randomized treatment group, regardless of the actual treatment received. Participants in the FAS with available data were analyzed. | Kilograms (kg) | MEAN |
| PRIMARY | Percent Change From Baseline in Body Weight - Period 2 | Baseline (Day 15) and Day 42 | Participants in the FAS with available data were analyzed. Participants were classified based on randomized treatment group, regardless of the actual treatment received. | percent change | LEAST_SQUARES_MEAN |
| PRIMARY | Overall Score of Prader-Willi Syndrome (PWS) Hyperphagia Questionnaire - Period 2 | Baseline (Day 15) | Participants in the FAS with available data were analyzed. Participants were classified based on randomized treatment group, regardless of the actual treatment received. | units on a scale | MEAN |
| PRIMARY | Percent Change From Baseline in Overall Score of Prader-Willi Syndrome (PWS) Hyperphagia Questionnaire - Period 2 | Baseline (Day 15) and Day 42 | Participants in the FAS with available data were analyzed. Participants were classified based on randomized treatment group, regardless of the actual treatment received. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Hyperphagic Drive Score of PWS Hyperphagia Questionnaire - Period 2 | Baseline (Day 15) and Day 42 | Participants in the FAS with available data were analyzed. Participants were classified based on randomized treatment group, regardless of the actual treatment received. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Hyperphagic Behaviors Score of PWS Hyperphagia Questionnaire - Period 2 | Baseline (Day 15) and Day 42 | Participants in the FAS with available data were analyzed. Participants were classified based on randomized treatment group, regardless of the actual treatment received. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Hyperphagic Severity Score of PWS Hyperphagia Questionnaire - Period 2 | Baseline (Day 15) and Day 42 | Participants in the FAS with available data were analyzed. Participants were classified based on randomized treatment group, regardless of the actual treatment received. | percent change | LEAST_SQUARES_MEAN |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 outcomesResults by group94
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 | Setmelanotide 0.5 mg (Double-blind Randomized Treatment) | Participants with at least 1 TEAE | 4 | Participants | |
| Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 | Setmelanotide 1.5 mg (Double-blind Randomized Treatment) | Participants with at least 1 TEAE | 11 | Participants | |
| Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 | Setmelanotide 2.5 mg (Double-blind Randomized Treatment) | Participants with at least 1 TEAE | 6 | Participants | |
| Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 | Placebo (Double-blind Randomized Treatment) | Participants with at least 1 TEAE | 11 | Participants | |
| Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 | Setmelanotide 0.5 mg (Double-blind Randomized Treatment) | Participants with at least 1 SAE | 0 | Participants | |
| Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 | Setmelanotide 1.5 mg (Double-blind Randomized Treatment) | Participants with at least 1 SAE | 0 | Participants | |
| Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 | Setmelanotide 2.5 mg (Double-blind Randomized Treatment) | Participants with at least 1 SAE | 0 | Participants | |
| Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 | Placebo (Double-blind Randomized Treatment) | Participants with at least 1 SAE | 0 | Participants | |
| Number of Participants Who Experienced a TEAE - Period 3 | Setmelanotide 0.5 mg (Randomized Withdrawal Period) | Participants with at least 1 TEAE | 3 | Participants | |
| Number of Participants Who Experienced a TEAE - Period 3 | Setmelanotide 1.5 mg (Randomized Withdrawal Period) | Participants with at least 1 TEAE | 7 | Participants |
1 to 10 of 94Source ClinicalTrials.gov
See all 94 results by groupParticipant flow48
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Placebo Run-in Period 1 (2 Weeks) | STARTED | Setmelanotide 0.5 mg | 0 | |
| Placebo Run-in Period 1 (2 Weeks) | STARTED | Setmelanotide 1.5 mg | 0 | |
| Placebo Run-in Period 1 (2 Weeks) | STARTED | Setmelanotide 2.5 mg | 0 | |
| Placebo Run-in Period 1 (2 Weeks) | STARTED | Placebo | 40 | |
| Placebo Run-in Period 1 (2 Weeks) | COMPLETED | Setmelanotide 0.5 mg | 0 | |
| Placebo Run-in Period 1 (2 Weeks) | COMPLETED | Setmelanotide 1.5 mg | 0 | |
| Placebo Run-in Period 1 (2 Weeks) | COMPLETED | Setmelanotide 2.5 mg | 0 | |
| Placebo Run-in Period 1 (2 Weeks) | COMPLETED | Placebo | 40 | |
| Placebo Run-in Period 1 (2 Weeks) | NOT COMPLETED | Setmelanotide 0.5 mg | 0 | |
| Placebo Run-in Period 1 (2 Weeks) | NOT COMPLETED | Setmelanotide 1.5 mg | 0 |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 participant flowAdverse events693
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Injection site erythema | General disorders | Setmelanotide 0.5 mg (Double-blind Randomized Treatment) | 3 | 4 | |
| other | Injection site erythema | General disorders | Setmelanotide 1.5 mg (Double-blind Randomized Treatment) | 5 | 13 | |
| other | Injection site erythema | General disorders | Setmelanotide 2.5 mg (Double-blind Randomized Treatment) | 4 | 8 | |
| other | Injection site erythema | General disorders | Placebo (Double-blind Randomized Treatment) | 6 | 15 | |
| other | Injection site erythema | General disorders | Setmelanotide 0.5 mg (Randomized Withdrawal Period) | 2 | 3 | |
| other | Injection site erythema | General disorders | Setmelanotide 1.5 mg (Randomized Withdrawal Period) | 4 | 10 | |
| other | Injection site erythema | General disorders | Setmelanotide 2.5 mg (Randomized Withdrawal Period) | 4 | 5 | |
| other | Injection site erythema | General disorders | Placebo (Randomized Withdrawal Period) | 4 | 20 | |
| other | Injection site erythema | General disorders | Setmelanotide 1.5 mg (Open-Label Extension) | 8 | 22 | |
| other | Injection site erythema | General disorders | Setmelanotide 2.5 mg (Open-Label Extension) | 6 | 16 |
1 to 10 of 693Source ClinicalTrials.gov
See all 693 adverse eventsAdverse event totals33
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Setmelanotide 0.5 mg (Double-blind Randomized Treatment) | 0 | 4 |
| Total, all-cause mortality | Setmelanotide 1.5 mg (Double-blind Randomized Treatment) | 0 | 13 |
| Total, all-cause mortality | Setmelanotide 2.5 mg (Double-blind Randomized Treatment) | 0 | 8 |
| Total, all-cause mortality | Placebo (Double-blind Randomized Treatment) | 0 | 15 |
| Total, all-cause mortality | Setmelanotide 0.5 mg (Randomized Withdrawal Period) | 0 | 3 |
| Total, all-cause mortality | Setmelanotide 1.5 mg (Randomized Withdrawal Period) | 0 | 10 |
| Total, all-cause mortality | Setmelanotide 2.5 mg (Randomized Withdrawal Period) | 0 | 5 |
| Total, all-cause mortality | Placebo (Randomized Withdrawal Period) | 0 | 20 |
| Total, all-cause mortality | Setmelanotide 1.5 mg (Open-Label Extension) | 0 | 22 |
| Total, all-cause mortality | Setmelanotide 2.5 mg (Open-Label Extension) | 0 | 16 |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R01FD005094 | Phase II clinical evaluation of the melanocortin 4 receptor agonist RM-493 for the treatment of Prader-Willi syndrome | GOTTESDIENER, KEITH | RHYTHM PHARMACEUTICALS, INC. | FOOD AND DRUG ADMINISTRATION | 2015 to 2017 |
Source NIH RePORTER
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
Setmelanotide 1.5 mg (Open-Label Extension) vs Setmelanotide 2.5 mg (Open-Label Extension): Number of Participants With Clinically Significant Percent Change From Baseline in Body Mass Measured Using DEXA - Period 4 | Trial comparisons |
Setmelanotide 1.5 mg (Open-Label Extension) vs Setmelanotide 2.5 mg (Open-Label Extension): Number of Participants With Clinically Significant Percent Change From Baseline in Body Fat Measured Using DEXA - Period 4 | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02311673
GET https://conductscience.org/api/trials/v1/NCT02311673/registered?page=2