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Study · Non-randomized trial · n = 22 · 2025

3 results

Impact of Setmelanotide on Metabolic Syndrome Risk in Patients With Bardet-Biedl Syndrome.

Haqq AM, Poitou C, Chung WK, Forsythe E, Conroy R, Dollfus H, Malhotra S, Touchot N, Okorie U, Beales P, Clément K, Argente J

The Journal of clinical endocrinology and metabolism1 Sept 2025Cited by 4 (Europe PMC)

PMID39919037 (opens in a new tab)PMCIDPMC12448622 (opens in a new tab)DOI10.1210/clinem/dgaf079 (opens in a new tab)PepBankPUB-39919037
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Summary

ContextBardet-Biedl syndrome (BBS) is a rare genetic disease associated with disruptions in melanocortin-4 receptor pathway signaling that can contribute to increased risk for metabolic syndrome and obesity-related comorbidities.ObjectiveHere, MetS-Z-BMI scores, a continuous measure based on body mass index (BMI), were calculated to determine metabolic syndrome severity and response to treatment with the melanocortin-4 receptor agonist setmelanotide in BBS.MethodsAll patients from a phase 3 study (NCT03746522) of setmelanotide with data required for the calculation of MetS-Z-BMI scores were included. Mean MetS-Z-BMI score was determined at baseline and Week 52; subgroup analyses were conducted by sex, age, genotype, and response to setmelanotide.ResultsMetS-Z-BMI scores were evaluable for 22 of 32 patients enrolled. Baseline mean (SD) MetS-Z-BMI score across patients was 1.1 (0.5); baseline mean (SD) odds ratios of future cardiovascular disease or type 2 diabetes were 3.1 (1.5) and 3.7 (1.7), respectively, for adults and 10.2 (4.7) and 2.8 (1.3), respectively, for pediatric patients. Overall, mean (SD) MetS-Z-BMI score at Week 52 was reduced by 0.34 (0.62). Mean (SD) MetS-Z-BMI scores significantly differed depending on achievement of predetermined weight-based thresholds of ≥10% weight loss (patients aged ≥18 years) or ≥0.3-point reduction in BMI Z score (patients aged <18 years) at Week 52 (achievers, -0.64 [0.54]; nonachievers, 0.08 [0.47]; P = .0043). No significant difference was observed with other subgroup comparisons.ConclusionMetS-Z-BMI score reductions were observed after 52 weeks of treatment, suggesting that setmelanotide may decrease metabolic syndrome severity and risk of future obesity-related comorbidities in those with BBS.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study3

3 results from studies that gave the peptide. 3 results from people.

  1. 01HumanNon-randomized trialEfficacy outcome

    In BBS patients receiving setmelanotide, mean MetS-Z-BMI decreased by 0.34 (SD 0.62) at Week 52.

    In plain wordsIn BBS patients receiving setmelanotide, metabolic syndrome severity decreased compared with baseline at Week 52.

    Peptide
    Setmelanotide
    Outcome
    DecreaseMetS-Z-BMI reduction
    Study

    n = 22

    Comparator
    Baseline
    Outcome group
    Cardiometabolic risk factors · Metabolic and weight
    Studied dose
    Dose not stated · 52 weeksAs given in this study (human).

    Measurements

    Mean MetS-Z-BMI score reduction
    0.34 MetS-Z-BMI score
    Week 52, change from baseline
    SD of MetS-Z-BMI score reduction
    0.62 MetS-Z-BMI score
    Week 52

    Source sentences

    Summary
    Overall, mean (SD) MetS-Z-BMI score at Week 52 was reduced by 0.34 (0.62).
    2 context sentences
    Summary · context
    All patients from a phase 3 study (NCT03746522) of setmelanotide with data required for the calculation of MetS-Z-BMI scores were included.
    Summary · context
    Mean MetS-Z-BMI score was determined at baseline and Week 52; subgroup analyses were conducted by sex, age, genotype, and response to setmelanotide.
  2. 02HumanObservational studyEfficacy outcome

    In setmelanotide-treated BBS patients, MetS-Z-BMI changes differed between weight-threshold achievers (-0.64 [0.54]) and nonachievers (0.08 [0.47]; P = .0043).

    In plain wordsIn setmelanotide-treated BBS patients, weight-response achievers had greater metabolic syndrome score reductions than nonachievers.

    Peptide
    Setmelanotide
    Outcome
    DecreaseMetS-Z-BMI change by weight response
    Study

    n: not reported in source

    Comparator
    Weight-threshold nonachievers
    Outcome group
    Cardiometabolic risk factors · Metabolic and weight
    Studied dose
    Dose not stated

    Measurements

    Mean MetS-Z-BMI score, achievers
    −0.64 MetS-Z-BMI score
    Weight-threshold achievers, Week 52
    SD, achievers
    0.54 MetS-Z-BMI score
    Weight-threshold achievers
    Mean MetS-Z-BMI score, nonachievers
    0.08 MetS-Z-BMI score
    Weight-threshold nonachievers, Week 52
    SD, nonachievers
    0.47 MetS-Z-BMI score
    Weight-threshold nonachievers
    P value
    0.0043
    Achievers versus nonachievers

    Source sentences

    Summary
    Mean (SD) MetS-Z-BMI scores significantly differed depending on achievement of predetermined weight-based thresholds of ≥10% weight loss (patients aged ≥18 years) or ≥0.3-point reduction in BMI Z score (patients aged <18 years) at Week 52
    3 context sentences
    Summary · context
    (achievers, -0.64 [0.54]; nonachievers, 0.08 [0.47]; P = .0043).
    Summary · context
    All patients from a phase 3 study (NCT03746522) of setmelanotide with data required for the calculation of MetS-Z-BMI scores were included.
    Summary · context
    Mean MetS-Z-BMI score was determined at baseline and Week 52; subgroup analyses were conducted by sex, age, genotype, and response to setmelanotide.
  3. 03HumanObservational studyEfficacy outcome

    In setmelanotide-treated BBS patients, other subgroup comparisons showed no significant differences in MetS-Z-BMI.

    In plain wordsIn setmelanotide-treated BBS patients, metabolic syndrome scores did not differ significantly in the other subgroup comparisons.

    Peptide
    Setmelanotide
    Outcome
    No significant changeMetS-Z-BMI differences in other subgroups
    Study

    n: not reported in source

    Comparator
    Other sex, age and genotype subgroups
    Outcome group
    Cardiometabolic risk factors · Metabolic and weight
    Studied dose
    Dose not stated

    Source sentences

    Summary
    No significant difference was observed with other subgroup comparisons.
    2 context sentences
    Summary · context
    All patients from a phase 3 study (NCT03746522) of setmelanotide with data required for the calculation of MetS-Z-BMI scores were included.
    Summary · context
    Mean MetS-Z-BMI score was determined at baseline and Week 52; subgroup analyses were conducted by sex, age, genotype, and response to setmelanotide.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

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.

  1. Version 2Current

    28 Oct 2026, 02:03 UTC

    Study results updated

  2. Version 1

    27 Oct 2026, 16:36 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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