A Phase 3 Trial in Participants With Obesity Due to Bardet-Biedl Syndrome or Alström Syndrome: Efficacy and Safety of the Melanocortin 4 Receptor Agonist Setmelanotide

Haws R, Clément K, Dollfus H, Han J, Haqq A, Martos-Moreno G, Mittleman R, Stewart M, Webster M, Yanovski J, Yuan G, Argente J

Journal of the Endocrine Society3 May 2021

PMCIDPMC8089587PepBankPUB-95849668

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Summary

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Text rights: CC BY-NC-ND. Results still quote short attributed sentences.

Results from this study5

5 results from studies that gave the peptide. 5 results from people.

01HumanNon-randomized trialEfficacy outcome
After ~52 weeks of setmelanotide, 34.5% achieved ≥10% weight reduction from baseline (95% CI 17.5%-51.6%, P=0.0024); all observed responders had BBS.
In plain wordsAfter setmelanotide, 34.5% achieved at least 10% weight reduction from baseline. All observed responders had BBS.
Peptide
Setmelanotide
Outcome
DecreaseAt least 10% weight reduction
Study
n: not reported in source
Comparator
Baseline
Analysis
Primary
Outcome group
Body weight · Metabolic and weight
Studied dose
Dose not stated · ~52 weeksAs given in this study (human).
Measurements
Participants with at least 10% body weight reduction
34.5%
Participants 12 years and older with BBS or AS, about 52 weeks
95% CI of proportion with at least 10% body weight reduction
17.5 to 51.6%
Participants 12 years and older with BBS or AS, about 52 weeks
Source sentences
Summary
After ~52 weeks of setmelanotide, 34.5% (95% CI, 17.5%-51.6%; P=0.0024) of participants achieved ≥10% reduction in body weight from baseline.
3 context sentences(design, population, supporting numbers)
Summary · context
All observed responders had BBS.
Summary · context
Individuals were randomized and received setmelanotide or placebo for 14 weeks, followed by open-label setmelanotide so that all participants received at least 1 year of drug.
Summary · context
Efficacy analyses (including change in body weight, body mass index Z score, and hunger) were conducted in participants ≥12 years old at baseline.
Report a problemFND-9584966801
02HumanNon-randomized trialEfficacy outcome
In participants with BBS aged ≤17 years receiving setmelanotide, BMI Z score changed by −24.5% ± 22.3% from baseline.
In plain wordsIn participants with BBS aged ≤17 years, BMI Z score decreased from baseline during setmelanotide treatment.
Peptide
Setmelanotide
Outcome
DecreaseBMI Z score change in younger BBS participants
Study
n = 14
Comparator
Baseline
Outcome group
Body weight · Metabolic and weight
Studied dose
Dose not stated
Measurements
Mean percent change in BMI Z score
−24.5%
BBS participants aged 17 years or younger (n=14)
SD of percent change in BMI Z score
22.3%
BBS participants aged 17 years or younger (n=14)
Source sentences
Summary
In participants with BBS aged ≤17 years (n=14), mean ± SD percent change in body mass index Z score from baseline was −24.5% ± 22.3%.
2 context sentences(design, population, supporting numbers)
Summary · context
Individuals were randomized and received setmelanotide or placebo for 14 weeks, followed by open-label setmelanotide so that all participants received at least 1 year of drug.
Summary · context
Efficacy analyses (including change in body weight, body mass index Z score, and hunger) were conducted in participants ≥12 years old at baseline.
Report a problemFND-9584966803
03HumanNon-randomized trialEfficacy outcome
In participants with BBS or AS receiving setmelanotide, mean body weight changed by −6.2% ± 8.6% from baseline (P<0.0001).
In plain wordsIn participants with BBS or AS, body weight decreased from baseline during setmelanotide treatment.
Peptide
Setmelanotide
Outcome
DecreaseBody weight change
Study
n: not reported in source
Comparator
Baseline
Outcome group
Body weight · Metabolic and weight
Studied dose
Dose not stated
Measurements
Mean percent change in body weight
−6.2%
From baseline, mean
SD of percent change in body weight
8.6%
From baseline
Source sentences
Summary
Mean ± SD percent change in body weight from baseline was −6.2% ± 8.6% (P<0.0001).
2 context sentences(design, population, supporting numbers)
Summary · context
Individuals were randomized and received setmelanotide or placebo for 14 weeks, followed by open-label setmelanotide so that all participants received at least 1 year of drug.
Summary · context
Efficacy analyses (including change in body weight, body mass index Z score, and hunger) were conducted in participants ≥12 years old at baseline.
Report a problemFND-9584966802
04HumanNon-randomized trialEfficacy outcome
In participants with BBS or AS receiving setmelanotide, maximal daily hunger score changed by −30.8% ± 25.0% from baseline (P<0.0001).
In plain wordsIn participants with BBS or AS, maximal daily hunger decreased from baseline during setmelanotide treatment.
Peptide
Setmelanotide
Outcome
DecreaseMaximal daily hunger score
Study
n: not reported in source
Comparator
Baseline
Outcome group
Appetite and food intake · Metabolic and weight
Studied dose
Dose not stated
Measurements
Mean percent change in maximal daily hunger score
−30.8%
From baseline
SD of percent change in maximal daily hunger score
25%
From baseline
Source sentences
Summary
Mean ± SD percent Job #11307-1 1/27/2021Haws BBS AS Phase 3 ENDO 2021 EncorePage 2 change in maximal daily hunger score (based on participant responses to scoring their “most” hunger during the day) from baseline was −30.8% ± 25.0% (P<0.0001)
2 context sentences(design, population, supporting numbers)
Summary · context
Individuals were randomized and received setmelanotide or placebo for 14 weeks, followed by open-label setmelanotide so that all participants received at least 1 year of drug.
Summary · context
Efficacy analyses (including change in body weight, body mass index Z score, and hunger) were conducted in participants ≥12 years old at baseline.
Report a problemFND-9584966804
05HumanNon-randomized trialEfficacy outcome
With setmelanotide, 60.2% achieved ≥25% reduction in weekly average daily hunger score from baseline (95% CI 35.3%-85.1%, P<0.0001).
In plain wordsIn participants with BBS or AS, 60.2% achieved at least 25% hunger score reduction from baseline during setmelanotide treatment.
Peptide
Setmelanotide
Outcome
DecreaseAt least 25% hunger score reduction
Study
n: not reported in source
Comparator
Baseline
Outcome group
Appetite and food intake · Metabolic and weight
Studied dose
Dose not stated
Measurements
Participants with at least 25% reduction in weekly average daily hunger score
60.2%
From baseline
95% CI of proportion with at least 25% hunger score reduction
35.3 to 85.1%
From baseline
Source sentences
Summary
60.2% (95% CI, 35.3%-85.1%; P<0.0001) of participants achieved ≥25% reduction in weekly average daily hunger score from baseline.
2 context sentences(design, population, supporting numbers)
Summary · context
Individuals were randomized and received setmelanotide or placebo for 14 weeks, followed by open-label setmelanotide so that all participants received at least 1 year of drug.
Summary · context
Efficacy analyses (including change in body weight, body mass index Z score, and hunger) were conducted in participants ≥12 years old at baseline.
Report a problemFND-9584966805

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Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
28 Oct 2026, 02:03 UTC
Study results updated
Version 1
27 Oct 2026, 16:36 UTC
First added · source update from Europe PMC

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Abstract

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