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Study · Non-randomized trial · n = 21 · 2020

12 results

Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials.

Clément K, van den Akker E, Argente J, Bahm A, Chung WK, Connors H, De Waele K, Farooqi IS, Gonneau-Lejeune J, Gordon G, Kohlsdorf K, Poitou C and 6 more

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Puder L, Swain J, Stewart M, Yuan G, Wabitsch M, Kühnen P

The lancet. Diabetes & endocrinology30 Oct 2020Cited by 363 (Europe PMC)

PMID33137293 (opens in a new tab)DOI10.1016/s2213-8587(20)30364-8 (opens in a new tab)PepBankPUB-33137293
Publisher pagePubMedAsk about this study
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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study12

12 results from studies that gave the peptide. 12 results from people.

  1. 01HumanNon-randomized trialEfficacy outcome

    In LEPR deficiency obesity, five participants (45%) achieved at least 10% weight loss from baseline at approximately 1 year with setmelanotide.

    In plain wordsIn LEPR deficiency obesity, 45% achieved at least 10% weight loss from baseline at approximately 1 year with setmelanotide.

    Peptide
    Setmelanotide
    Outcome
    DecreaseAt least 10% weight loss in LEPR deficiency
    Study

    n = 11

    Comparator
    Baseline
    Analysis
    Primary
    Outcome group
    Body weight · Metabolic and weight
    Studied dose
    Dose not stated

    Measurements

    Participants with at least 10% weight loss
    45%
    LEPR trial, approximately 1 year
    Participants with at least 10% weight loss
    5 participants
    LEPR trial, approximately 1 year

    Source sentences

    Summary
    Eight (80%) participants in the POMC trial and five (45%) participants in the LEPR trial achieved at least 10% weight loss at approximately 1 year.
    4 context sentences
    Summary · context
    Between Feb 14, 2017, and Sept 7, 2018, ten participants were enrolled in the POMC trial and 11 participants were enrolled in the LEPR trial, and included in the full analysis and safety sets.
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.
  2. 02HumanNon-randomized trialEfficacy outcome

    In POMC deficiency obesity, eight participants (80%) achieved at least 10% weight loss from baseline at approximately 1 year with setmelanotide.

    In plain wordsIn POMC deficiency obesity, 80% achieved at least 10% weight loss from baseline at approximately 1 year with setmelanotide.

    Peptide
    Setmelanotide
    Outcome
    DecreaseAt least 10% weight loss in POMC deficiency
    Study

    n = 10

    Comparator
    Baseline
    Analysis
    Primary
    Outcome group
    Body weight · Metabolic and weight
    Studied dose
    Dose not stated

    Measurements

    Participants with at least 10% weight loss
    80%
    POMC trial, approximately 1 year
    Participants with at least 10% weight loss
    8 participants
    POMC trial, approximately 1 year

    Source sentences

    Summary
    Eight (80%) participants in the POMC trial and five (45%) participants in the LEPR trial achieved at least 10% weight loss at approximately 1 year.
    4 context sentences
    Summary · context
    Between Feb 14, 2017, and Sept 7, 2018, ten participants were enrolled in the POMC trial and 11 participants were enrolled in the LEPR trial, and included in the full analysis and safety sets.
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.
  3. 03HumanNon-randomized trialEfficacy outcome

    In POMC deficiency obesity, setmelanotide changed the most hunger score by -27·1% (90% CI -40·6 to -15·0; p=0·0005).

    In plain wordsIn POMC deficiency obesity, the most hunger score decreased by 27·1% from baseline with setmelanotide.

    Peptide
    Setmelanotide
    Outcome
    DecreaseMost hunger score in POMC deficiency
    Study

    n = 7

    Analysis
    Secondary
    Outcome group
    Appetite and food intake · Metabolic and weight
    Studied dose
    Dose not stated

    Measurements

    Mean percentage change in most hunger score
    −27.1%
    POMC trial, n=7
    90% CI of mean percentage change in most hunger score
    −40.6 to −15%
    POMC trial, n=7

    Source sentences

    Summary
    The mean percentage change in the most hunger score was -27·1% (n=7; 90% CI -40·6 to -15·0; p=0·0005) in the POMC trial and -43·7% (n=7; -54·8 to -29·1; p<0·0001) in the LEPR trial.
    4 context sentences
    Summary · context
    A key secondary endpoint was mean percentage change in the most hunger score of the 11-point Likert-type scale at approximately 1 year on the therapeutic dose,
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.
  4. 04HumanNon-randomized trialEfficacy outcome

    In LEPR deficiency obesity, setmelanotide changed the most hunger score by -43·7% (90% CI -54·8 to -29·1; p<0·0001).

    In plain wordsIn LEPR deficiency obesity, the most hunger score decreased by 43·7% from baseline with setmelanotide.

    Peptide
    Setmelanotide
    Outcome
    DecreaseMost hunger score in LEPR deficiency
    Study

    n = 7

    Analysis
    Secondary
    Outcome group
    Appetite and food intake · Metabolic and weight
    Studied dose
    Dose not stated

    Measurements

    Mean percentage change in most hunger score
    −43.7%
    LEPR trial, n=7
    90% CI of mean percentage change in most hunger score
    −54.8 to −29.1%
    LEPR trial, n=7

    Source sentences

    Summary
    The mean percentage change in the most hunger score was -27·1% (n=7; 90% CI -40·6 to -15·0; p=0·0005) in the POMC trial and -43·7% (n=7; -54·8 to -29·1; p<0·0001) in the LEPR trial.
    4 context sentences
    Summary · context
    A key secondary endpoint was mean percentage change in the most hunger score of the 11-point Likert-type scale at approximately 1 year on the therapeutic dose,
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.
  5. 05HumanNon-randomized trialSafety outcome

    No serious treatment-related adverse events occurred in either setmelanotide trial in POMC or LEPR deficiency obesity.

    In plain wordsNo serious treatment-related adverse events occurred in either setmelanotide trial in POMC or LEPR deficiency obesity.

    Peptide
    Setmelanotide
    Outcome
    Not applicableSerious treatment-related adverse events
    Study

    n = 21

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    No serious treatment-related adverse events occurred in both trials.
    4 context sentences
    Summary · context
    Between Feb 14, 2017, and Sept 7, 2018, ten participants were enrolled in the POMC trial and 11 participants were enrolled in the LEPR trial, and included in the full analysis and safety sets.
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.
  6. 06HumanNon-randomized trialSafety outcome

    All 11 participants with LEPR deficiency receiving setmelanotide reported treatment-related injection site reaction.

    In plain wordsAll 11 participants with LEPR deficiency receiving setmelanotide reported treatment-related injection site reaction.

    Peptide
    Setmelanotide
    Outcome
    Not applicableInjection-site reaction in LEPR deficiency
    Study

    n = 11

    Outcome group
    Adverse events · Skin and wound
    Studied dose
    Dose not stated

    Measurements

    Participants with treatment-related injection site reaction
    11 participants
    LEPR trial, all 11 participants

    Source sentences

    Summary
    In the LEPR trial, the most commonly reported treatment-related adverse events were injection site reaction in all 11 participants, skin disorders in five participants, and nausea in four participants.
    3 context sentences
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.
  7. 07HumanNon-randomized trialSafety outcome

    Five participants with LEPR deficiency receiving setmelanotide reported treatment-related skin disorders.

    In plain wordsFive participants with LEPR deficiency receiving setmelanotide reported treatment-related skin disorders.

    Peptide
    Setmelanotide
    Outcome
    Not applicableSkin disorders in LEPR deficiency
    Study

    n = 11

    Outcome group
    Skin and hair · Skin and wound
    Studied dose
    Dose not stated

    Measurements

    Participants with treatment-related skin disorders
    5 participants
    LEPR trial

    Source sentences

    Summary
    In the LEPR trial, the most commonly reported treatment-related adverse events were injection site reaction in all 11 participants, skin disorders in five participants, and nausea in four participants.
    3 context sentences
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.
  8. 08HumanNon-randomized trialSafety outcome

    Four participants with LEPR deficiency receiving setmelanotide reported treatment-related nausea.

    In plain wordsFour participants with LEPR deficiency receiving setmelanotide reported treatment-related nausea.

    Peptide
    Setmelanotide
    Outcome
    Not applicableNausea in LEPR deficiency
    Study

    n = 11

    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated

    Measurements

    Participants with treatment-related nausea
    4 participants
    LEPR trial

    Source sentences

    Summary
    In the LEPR trial, the most commonly reported treatment-related adverse events were injection site reaction in all 11 participants, skin disorders in five participants, and nausea in four participants.
    3 context sentences
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.
  9. 09HumanNon-randomized trialSafety outcome

    All ten participants with POMC deficiency receiving setmelanotide reported injection site reaction.

    In plain wordsAll ten participants with POMC deficiency receiving setmelanotide reported injection site reaction.

    Peptide
    Setmelanotide
    Outcome
    Not applicableInjection-site reaction in POMC deficiency
    Study

    n = 10

    Outcome group
    Adverse events · Skin and wound
    Studied dose
    Dose not stated

    Measurements

    Participants with injection site reaction
    10 participants
    POMC trial, all ten participants

    Source sentences

    Summary
    The most common adverse events were injection site reaction and hyperpigmentation, which were reported in all ten participants in the POMC trial; nausea was reported in five participants and vomiting in three participants.
    3 context sentences
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.
  10. 10HumanNon-randomized trialSafety outcome

    All ten participants with POMC deficiency receiving setmelanotide reported hyperpigmentation.

    In plain wordsAll ten participants with POMC deficiency receiving setmelanotide reported increased skin pigmentation.

    Peptide
    Setmelanotide
    Outcome
    Not applicableHyperpigmentation in POMC deficiency
    Study

    n = 10

    Outcome group
    Pigmentation · Skin and wound
    Studied dose
    Dose not stated

    Measurements

    Participants with hyperpigmentation
    10 participants
    POMC trial, all ten participants

    Source sentences

    Summary
    The most common adverse events were injection site reaction and hyperpigmentation, which were reported in all ten participants in the POMC trial; nausea was reported in five participants and vomiting in three participants.
    3 context sentences
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.
  11. 11HumanNon-randomized trialSafety outcome

    Five participants with POMC deficiency receiving setmelanotide reported nausea.

    In plain wordsFive participants with POMC deficiency receiving setmelanotide reported nausea.

    Peptide
    Setmelanotide
    Outcome
    Not applicableNausea in POMC deficiency
    Study

    n = 10

    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated

    Measurements

    Participants with nausea
    5 participants
    POMC trial

    Source sentences

    Summary
    The most common adverse events were injection site reaction and hyperpigmentation, which were reported in all ten participants in the POMC trial; nausea was reported in five participants and vomiting in three participants.
    3 context sentences
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.
  12. 12HumanNon-randomized trialSafety outcome

    Three participants with POMC deficiency receiving setmelanotide reported vomiting.

    In plain wordsThree participants with POMC deficiency receiving setmelanotide reported vomiting.

    Peptide
    Setmelanotide
    Outcome
    Not applicableVomiting in POMC deficiency
    Study

    n = 10

    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated

    Measurements

    Participants with vomiting
    3 participants
    POMC trial

    Source sentences

    Summary
    The most common adverse events were injection site reaction and hyperpigmentation, which were reported in all ten participants in the POMC trial; nausea was reported in five participants and vomiting in three participants.
    3 context sentences
    Summary · context
    These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK.
    Summary · context
    Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks.
    Summary · context
    Participants with at least 5 kg weight loss (or ≥5% if weighing <100 kg at baseline) entered an 8-week placebo-controlled withdrawal sequence (including 4 weeks each of blinded setmelanotide and placebo treatment) followed by 32 additional weeks of open-label treatment.

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

Registered trials2

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

2 trials · 2 completed · 2 with posted results · source ClinicalTrials.gov (opens in a new tab)

CompletedPhase 3Results postedNCT03287960

Setmelanotide for the Treatment of Leptin Receptor (LEPR) Deficiency Obesity

Rhythm Pharmaceuticals, Inc.n = 1530 Jan 2018 to 25 Sept 2020

Leptin Receptor Deficiency Obesity

Trial pageClinicalTrials.govUpdated 23 May 2023
CompletedPhase 3Results postedNCT02896192

Setmelanotide for the Treatment of Early-Onset Pro-Opiomelanocortin (POMC) Deficiency Obesity

Rhythm Pharmaceuticals, Inc.n = 1514 Feb 2017 to 25 May 2020

Pro-opiomelanocortin (POMC) Deficiency Obesity

Trial pageClinicalTrials.govUpdated 21 Sept 2023

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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