Percentage of Participants (≥12 Years of Age at Baseline) Who Reached ≥10% Weight Loss Threshold After 1 Year (Period 2): Pivotal Cohort
| Group | Value (percentage of participants) |
|---|---|
| Pivotal Cohort | 32.3 |
Trial · Interventional
CompletedPhase 3Results postedRegistry dataUnited States, Canada, France, Puerto Rico, Spain, United Kingdom
1 trial · 1 completed · 1 with posted results
Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity
NCT0374652223 Nov 2018 to 8 Mar 2021
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
This pivotal, phase 3 study is designed to confirm the efficacy and safety of setmelanotide, a potent melanocortin receptor type 4 (MC4R) agonist, for the treatment of obesity and hyperphagia in participants with Bardet Biedl syndrome (BBS) or Alström syndrome (AS). The study's primary efficacy endpoint is to evaluate the proportion of participants (≥ 12 years of age at baseline) who lose ≥ 10% of their baseline body weight following approximately (\~) 52 weeks of treatment with setmelanotide compared to a historical control rate.
Experimental
Setmelanotide (Double-Blind)
Participants received once daily SC injection of setmelanotide for 14 weeks in a double-blind placebo-controlled treatment period (Period 1). Participants ≥16 years of age started on setmelanotide 2.0 mg with dose escalation to 3.0 mg. Participants \<16 years of age started on setmelanotide 1.0 mg with dose escalation to 3.0 mg.
Placebo comparator
Placebo (Double-Blind)
Participants received once daily SC injection of placebo (matching setmelanotide) for 14 weeks in a double-blind placebo-controlled treatment period (Period 1).
Experimental
Setmelanotide (Open-label)
After the initial 14-week double-blind treatment period, all participants immediately transitioned to open-label setmelanotide once daily SC injection for 38 weeks (Period 2) and then continued to receive setmelanotide in the 14-week open-label treatment period (Period 3).
Outcomes the sponsor listed as primary.
Percentage of Participants (≥12 Years of Age at Baseline) Who Reached ≥10% Weight Loss Threshold After 1 Year (Period 2): Pivotal Cohort
Time frame · 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.
Mean Percent Change From Baseline in Body Weight (≥12 Years of Age) at Week 52 (Period 2): Pivotal Cohort
Time frame · 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.
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
Time frame · 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.
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
Time frame · 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.
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
| Group | Value (percentage of participants) |
|---|---|
| Pivotal Cohort | 32.3 |
| Group | Value (percent change) | Spread |
|---|---|---|
| Pivotal Cohort | -5.21 | 7.895 |
| Group | Value (percent change) | Spread |
|---|---|---|
| Pivotal Cohort | -30.91 | 24.733 |
| Group | Value (Participants) |
|---|---|
| Pivotal Cohort | 10 |
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| Setmelanotide (Double-Blind Period)Participants received once daily SC injection of setmelanotide for 14 weeks in a double-blind placebo-controlled treatment period (Period 1). | 0 / 27 | 1 / 27 | 26 / 27 |
| Placebo (Double-Blind Period)Participants received once daily SC injection of placebo (matching setmelanotide) for 14 weeks in a double-blind placebo-controlled treatment period (Period 1). | 0 / 25 | 2 / 25 | 24 / 25 |
| Setmelanotide (Open-label)After the initial 14-week double-blind treatment period, all participants immediately transitioned to open-label setmelanotide once daily SC injection for 38 weeks (Period 2) and then continued to receive setmelanotide in the 14-week open-label treatment period (Period 3). | 0 / 50 | 0 / 50 | 45 / 50 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies that cite this trial.
2 studies on this page
PubMed tag: none · 2026
Clément et al. 2026Advances in therapy
PMID 42821228DOI 10.1007/s12325-026-03741-xPUB-42821228
Indexing hint: Clinical trial · 2023
Forsythe et al. 2023Orphanet journal of rare diseasescited by 34
PMID 36647077PMC9841937DOI 10.1186/s13023-022-02602-4PUB-36647077
PubMed tag: none · 2023
Ervin et al. 2023Advances in therapycited by 19
PMID 36961653PMC10036981DOI 10.1007/s12325-023-02443-yPUB-36961653
3 studies on this page
Indexing hint: Clinical trial · 2026
Argente et al. 2026Obesity (Silver Spring, Md.)cited by 3
PMID 41703984PMC12933228DOI 10.1002/oby.70125PUB-41703984
Indexing hint: Randomized controlled trial · 2022
Haqq et al. 2022The lancet. Diabetes & endocrinologycited by 149
PMID 36356613PMC9847480DOI 10.1016/s2213-8587(22)00277-7PUB-36356613
PubMed tag: none · 2021
Haws et al. 2021Contemporary clinical trials communicationscited by 32
PMID 34013094PMC8114053DOI 10.1016/j.conctc.2021.100780PUB-34013094
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