Trial · NCT03746522
Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity
NCT03746522, Phase 3, Completed, Rhythm Pharmaceuticals, Inc.; results first posted 2023-12-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity. Trials, record NCT03746522. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03746522
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity
- NCT number
- NCT03746522
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 52 participants, actual
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.
- Conditions
- Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
- Interventions
- Setmelanotide, Placebo
- First posted
- 2018-11-19
- Last updated
- 2023-12-01
- Other registries' IDs
- eudract:2018-004058-11
- Results first posted
- 2023-12-01
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants (≥12 Years of Age at Baseline) Who Reached ≥10% Weight Loss Threshold After 1 Year (Period 2): Pivotal Cohort | 52 weeks | The percentage of participants (≥12 years of age at baseline) who achieved a ≥10% reduction from baseline in body weight at Period 2 or after 52 weeks of treatment with setmelanotide were analyzed. There was a 14-week placebo-controlled period at the beginning of the trial. After completion of the placebo-controlled period, all participants from the placebo group switched to setmelanotide treatment. The placebo group was integrated into the 52-week analysis so that the participants who received placebo, had 52 weeks of setmelanotide treatment after the first dose of "active" treatment. Placebo participants were also included in this analysis. |
| Secondary | Mean Percent Change From Baseline in Body Weight (≥12 Years of Age) at Week 52 (Period 2): Pivotal Cohort | Baseline, Week 52 | The mean percent change from baseline in body weight at 52 weeks was analyzed. There was a 14-week placebo-controlled period at the beginning of the trial. After completion of the placebo-controlled period, all participants from the placebo group switched to setmelanotide treatment. The placebo group was integrated into the 52-week analysis so that the participants who received placebo, had 52 weeks of setmelanotide treatment after the first dose of "active" treatment. Placebo participants were also included in this analysis. |
| Secondary | Mean Percent Change From Baseline in the Weekly Average of the Daily Hunger Score (≥12 Years of Age) at Week 52 (Period 2): Pivotal Cohort | Baseline, Week 52 | Mean percent change in hunger scores for participants ≥12 years of age with leptin receptor (LEPR) deficiency obesity in treatment with setmelanotide was evaluated. Hunger score ranged from 0= "not hungry at all" to 10= "hungriest possible" on Likert-type scale. On Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on weekly basis. Weekly average hunger score of daily worst (most) hunger score in 24 hours is the hunger score used to assess this study endpoint. There was a 14-week placebo-controlled period at the beginning of the trial. After completion of the placebo-controlled period, all participants from the placebo group switched to setmelanotide treatment. Placebo group was integrated into the 52-week analysis so that participants who received placebo, had 52 weeks of setmelanotide treatment after the first dose of "active" treatment. Placebo participants were also included in this analysis. |
| Secondary | Number of Participants (≥12 Years of Age With no Cognitive Impairment) Who Achieved a ≥ 25% Improvement in the Weekly Average of the Daily Hunger Score From Baseline at Week 52 (Period 2): Pivotal Cohort | Baseline, Week 52 | Number of participants (≥12 years of age with no cognitive impairment) achieving ≥25% improvement from baseline in hunger score at Week 52 was assessed. Hunger score ranged from 0= "not hungry at all" to 10= "hungriest possible" on Likert-type scale. On Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on weekly basis. Weekly average hunger score of daily worst (most) hunger score in 24 hours is the hunger score used to assess this study endpoint. There was a 14-week placebo-controlled period at the beginning of the trial. After completion of placebo-controlled period, all participants from placebo group switched to setmelanotide treatment. Placebo group was integrated into the 52-week analysis so that participants who received placebo, had 52 weeks of setmelanotide treatment after the first dose of "active" treatment. Placebo participants were also included in this analysis. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants (≥12 Years of Age at Baseline) Who Reached ≥10% Weight Loss Threshold After 1 Year (Period 2): Pivotal Cohort | 52 weeks | Participants in the Full Analysis Set (defined as randomized participants who received at least 1 dose of study drug and had active treatment baseline \[ATB\] data) with available data were analyzed. Participants in the pivotal cohort who received setmelanotide (and were ≥12 years of age) were included in the analysis. | percentage of participants | NUMBER |
| SECONDARY | Mean Percent Change From Baseline in Body Weight (≥12 Years of Age) at Week 52 (Period 2): Pivotal Cohort | Baseline, Week 52 | Participants in the Full Analysis Set with available data were analyzed. Participants in pivotal cohort who received setmelanotide (and were ≥12 years of age) were included in the analysis. | percent change | MEAN |
| SECONDARY | Mean Percent Change From Baseline in the Weekly Average of the Daily Hunger Score (≥12 Years of Age) at Week 52 (Period 2): Pivotal Cohort | Baseline, Week 52 | Participants in the Full Analysis Set with available data were analyzed. Participants in pivotal cohort who received setmelanotide (and were ≥12 years of age) were included in the analysis. | percent change | MEAN |
| SECONDARY | Number of Participants (≥12 Years of Age With no Cognitive Impairment) Who Achieved a ≥ 25% Improvement in the Weekly Average of the Daily Hunger Score From Baseline at Week 52 (Period 2): Pivotal Cohort | Baseline, Week 52 | Participants in Full Analysis Set with available data were analyzed. Participants in pivotal cohort who received setmelanotide (and were ≥12 years of age) were included in the analysis. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group4
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants (≥12 Years of Age at Baseline) Who Reached ≥10% Weight Loss Threshold After 1 Year (Period 2): Pivotal Cohort | Pivotal Cohort | 32.3 | 95% Confidence Interval 16.7 to 51.4 | percentage of participants | |
| Mean Percent Change From Baseline in Body Weight (≥12 Years of Age) at Week 52 (Period 2): Pivotal Cohort | Pivotal Cohort | -5.21 | Standard Deviation 7.895 | percent change | |
| Mean Percent Change From Baseline in the Weekly Average of the Daily Hunger Score (≥12 Years of Age) at Week 52 (Period 2): Pivotal Cohort | Pivotal Cohort | -30.91 | Standard Deviation 24.733 | percent change | |
Number of Participants (≥12 Years of Age With no Cognitive Impairment) Who Achieved a ≥ 25% Improvement in the Weekly Average of the Daily Hunger Score From Baseline at Week 52 (Period 2): Pivotal Cohort | Pivotal Cohort | 10 | Participants |
Source ClinicalTrials.gov
Participant flow27
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Period 1: Double-Blind (14 Weeks) | STARTED | Setmelanotide (Double-Blind) | 27 | |
| Period 1: Double-Blind (14 Weeks) | STARTED | Placebo (Double-Blind) | 25 | |
| Period 1: Double-Blind (14 Weeks) | STARTED | Setmelanotide (Open-label) | 0 | |
| Period 1: Double-Blind (14 Weeks) | COMPLETED | Setmelanotide (Double-Blind) | 27 | |
| Period 1: Double-Blind (14 Weeks) | COMPLETED | Placebo (Double-Blind) | 23 | |
| Period 1: Double-Blind (14 Weeks) | COMPLETED | Setmelanotide (Open-label) | 0 | |
| Period 1: Double-Blind (14 Weeks) | NOT COMPLETED | Setmelanotide (Double-Blind) | 0 | |
| Period 1: Double-Blind (14 Weeks) | NOT COMPLETED | Placebo (Double-Blind) | 2 | |
| Period 1: Double-Blind (14 Weeks) | NOT COMPLETED | Setmelanotide (Open-label) | 0 | |
| Period 2: Open-Label (38 Weeks) | STARTED | Setmelanotide (Double-Blind) | 0 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 participant flowAdverse events102
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 | High density lipoprotein decreased | Investigations | Setmelanotide (Double-Blind Period) | 4 | 27 | |
| other | High density lipoprotein decreased | Investigations | Placebo (Double-Blind Period) | 0 | 25 | |
| other | High density lipoprotein decreased | Investigations | Setmelanotide (Open-label) | 0 | 50 | |
| other | Melanocytic naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Setmelanotide (Double-Blind Period) | 1 | 27 | |
| other | Melanocytic naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Placebo (Double-Blind Period) | 0 | 25 | |
| other | Melanocytic naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Setmelanotide (Open-label) | 5 | 50 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Setmelanotide (Double-Blind Period) | 0 | 27 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Placebo (Double-Blind Period) | 2 | 25 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Setmelanotide (Open-label) | 3 | 50 | |
| other | Nasal congestion | Respiratory, thoracic and mediastinal disorders | Setmelanotide (Double-Blind Period) | 3 | 27 |
1 to 10 of 102Source ClinicalTrials.gov
See all 102 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Setmelanotide (Double-Blind Period) | 0 | 27 |
| Total, all-cause mortality | Placebo (Double-Blind Period) | 0 | 25 |
| Total, all-cause mortality | Setmelanotide (Open-label) | 0 | 50 |
| Total, other adverse events | Setmelanotide (Double-Blind Period) | 26 | 27 |
| Total, other adverse events | Placebo (Double-Blind Period) | 24 | 25 |
| Total, other adverse events | Setmelanotide (Open-label) | 45 | 50 |
| Total, serious adverse events | Setmelanotide (Double-Blind Period) | 1 | 27 |
| Total, serious adverse events | Placebo (Double-Blind Period) | 2 | 25 |
| Total, serious adverse events | Setmelanotide (Open-label) | 0 | 50 |
Source ClinicalTrials.gov
Papers6
The papers the trial lists on ClinicalTrials.gov.
Grants0
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.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT03746522
GET https://conductscience.org/api/trials/v1/NCT03746522/flow?page=2