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Study · Non-randomized trial · n = 6 · 2022

8 results

Quality of life outcomes in two phase 3 trials of setmelanotide in patients with obesity due to LEPR or POMC deficiency.

Kühnen P, Wabitsch M, von Schnurbein J, Chirila C, Mallya UG, Callahan P, Gnanasakthy A, Poitou C, Krabusch PM, Stewart M, Clément K

Orphanet journal of rare diseases5 Feb 2022Cited by 23 (Europe PMC)

PMID35123544 (opens in a new tab)PMCIDPMC8817523 (opens in a new tab)DOI10.1186/s13023-022-02186-z (opens in a new tab)PepBankPUB-35123544
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Summary

IntroductionIndividuals with proopiomelanocortin (POMC) or leptin receptor (LEPR) deficiency are young and experience severe obesity, hyperphagia, and comorbidities, which can impair quality of life (QOL).MethodsTwo pivotal Phase 3 trials explored the effect of setmelanotide on body weight and hunger in individuals with obesity due to POMC (NCT02896192) or LEPR (NCT03287960) deficiency. QOL and depression were investigated in parallel using the disease-specific, age-appropriate Impact of Weight on Quality of Life-Lite (IWQOL-Lite), Pediatric Quality of Life Inventory (PedsQL), and Patient Health Questionnaire-9 (PHQ-9).ResultsIn total, the POMC and LEPR trials enrolled 21 patients. Adults (≥ 18 years old; n = 7) had moderate-to-severe impairment in QOL at baseline, with mean (standard deviation [SD]) IWQOL-Lite total score 60.3 (13.2; maximum IWQOL-Lite total score = 100). The effect of setmelanotide on IWQOL-Lite total score was observed as soon as Week 5. Among those with scores at Week 52, 5 of 6 adults experienced a clinically meaningful improvement, with mean (SD) total scores increased from baseline by 24.2 (12.1) points. Children (6-12 years old; n = 2) and adolescents (13-17 years old; n = 4) had impaired QOL at baseline, with mean (SD) self-reported PedsQL total scores 53.3 (6.2) and 63.3 (29.1), respectively (maximum PedsQL total score = 100). Three of 5 patients experienced clinically meaningful improvement in PedsQL, with 2 children whose PedsQL total score increased by 28.3 and 3.3 points and 3 adolescents whose mean (SD) total score increased from baseline by 5.8 (18.3) points. Baseline mean (SD) PHQ-9 score (in those ≥ 12 years old) was 5.3 (3.8) and was generally maintained through Week 52.ConclusionsPatients with POMC or LEPR deficiency had impaired, and in some cases severely impaired, QOL before setmelanotide treatment. Setmelanotide improved QOL in patients as early as Week 5, with some patients no longer experiencing impaired QOL at Week 52. Improvements in QOL may be related to a reduction in hunger and body weight associated with setmelanotide. Because of the highly complex psychological consequences of rare genetic diseases of obesity, some patients may require a long period of treatment to improve QOL and benefit from interdisciplinary care.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study8

8 results from studies that gave the peptide. 8 results from people.

  1. 01HumanNon-randomized trialEfficacy outcome

    Among adults with POMC or LEPR deficiency assessed at Week 52, 5 of 6 had meaningful quality-of-life improvement during setmelanotide.

    In plain wordsAmong adults assessed at Week 52, 5 of 6 had meaningful quality-of-life improvement from baseline during setmelanotide.

    Peptide
    Setmelanotide
    Outcome
    IncreaseMeaningful adult quality-of-life improvement
    Study

    n = 6

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated

    Measurements

    Adults with clinically meaningful IWQOL-Lite improvement
    5 adults
    of 6 adults with Week 52 scores

    Source sentences

    Summary
    Among those with scores at Week 52, 5 of 6 adults experienced a clinically meaningful improvement, with mean (SD) total scores increased from baseline by 24.2 (12.1) points.
    2 context sentences
    Summary · context
    Two pivotal Phase 3 trials explored the effect of setmelanotide on body weight and hunger in individuals with obesity due to POMC (NCT02896192) or LEPR (NCT03287960) deficiency.
    Summary · context
    QOL and depression were investigated in parallel using the disease-specific, age-appropriate Impact of Weight on Quality of Life-Lite (IWQOL-Lite), Pediatric Quality of Life Inventory (PedsQL), and Patient Health Questionnaire-9 (PHQ-9).
  2. 02HumanNon-randomized trialEfficacy outcome

    In adults with POMC or LEPR deficiency assessed at Week 52, IWQOL-Lite increased by a mean 24.2 points (SD 12.1).

    In plain wordsIn adults with POMC or LEPR deficiency, quality-of-life scores increased from baseline during setmelanotide.

    Peptide
    Setmelanotide
    Outcome
    IncreaseAdult IWQOL-Lite score change
    Study

    n = 6

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated

    Measurements

    IWQOL-Lite total score mean change from baseline
    24.2 points
    adults, Week 52
    IWQOL-Lite total score change SD
    12.1 points
    adults, Week 52

    Source sentences

    Summary
    Among those with scores at Week 52, 5 of 6 adults experienced a clinically meaningful improvement, with mean (SD) total scores increased from baseline by 24.2 (12.1) points.
    2 context sentences
    Summary · context
    Two pivotal Phase 3 trials explored the effect of setmelanotide on body weight and hunger in individuals with obesity due to POMC (NCT02896192) or LEPR (NCT03287960) deficiency.
    Summary · context
    QOL and depression were investigated in parallel using the disease-specific, age-appropriate Impact of Weight on Quality of Life-Lite (IWQOL-Lite), Pediatric Quality of Life Inventory (PedsQL), and Patient Health Questionnaire-9 (PHQ-9).
  3. 03HumanNon-randomized trialEfficacy outcome

    During setmelanotide in POMC or LEPR deficiency, 3 of 5 pediatric patients experienced meaningful PedsQL improvement.

    In plain wordsDuring setmelanotide, 3 of 5 children and adolescents had meaningful quality-of-life improvement from baseline.

    Peptide
    Setmelanotide
    Outcome
    IncreaseMeaningful pediatric quality-of-life improvement
    Study

    n = 5

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated

    Measurements

    Pediatric patients with clinically meaningful PedsQL improvement
    3 patients
    of 5 children and adolescents

    Source sentences

    Summary
    Three of 5 patients experienced clinically meaningful improvement in PedsQL, with 2 children whose PedsQL total score increased by 28.3 and 3.3 points and 3 adolescents whose mean (SD) total score increased from baseline by 5.8 (18.3) points.
    2 context sentences
    Summary · context
    Two pivotal Phase 3 trials explored the effect of setmelanotide on body weight and hunger in individuals with obesity due to POMC (NCT02896192) or LEPR (NCT03287960) deficiency.
    Summary · context
    QOL and depression were investigated in parallel using the disease-specific, age-appropriate Impact of Weight on Quality of Life-Lite (IWQOL-Lite), Pediatric Quality of Life Inventory (PedsQL), and Patient Health Questionnaire-9 (PHQ-9).
  4. 04HumanNon-randomized trialEfficacy outcome

    During setmelanotide in POMC or LEPR deficiency, adolescent PedsQL scores increased by a mean 5.8 points (SD 18.3).

    In plain wordsDuring setmelanotide, adolescents with POMC or LEPR deficiency had higher quality-of-life scores than at baseline.

    Peptide
    Setmelanotide
    Outcome
    IncreaseAdolescent PedsQL score change
    Study

    n = 3

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated

    Measurements

    PedsQL total score mean increase from baseline
    5.8 points
    adolescents (n=3)
    PedsQL total score increase SD
    18.3 points
    adolescents (n=3)

    Source sentences

    Summary
    Three of 5 patients experienced clinically meaningful improvement in PedsQL, with 2 children whose PedsQL total score increased by 28.3 and 3.3 points and 3 adolescents whose mean (SD) total score increased from baseline by 5.8 (18.3) points.
    2 context sentences
    Summary · context
    Two pivotal Phase 3 trials explored the effect of setmelanotide on body weight and hunger in individuals with obesity due to POMC (NCT02896192) or LEPR (NCT03287960) deficiency.
    Summary · context
    QOL and depression were investigated in parallel using the disease-specific, age-appropriate Impact of Weight on Quality of Life-Lite (IWQOL-Lite), Pediatric Quality of Life Inventory (PedsQL), and Patient Health Questionnaire-9 (PHQ-9).
  5. 05HumanNon-randomized trialEfficacy outcome

    During setmelanotide in POMC or LEPR deficiency, one child had a PedsQL increase of 28.3 points.

    In plain wordsDuring setmelanotide, one child with POMC or LEPR deficiency had a quality-of-life score increase of 28.3 points.

    Peptide
    Setmelanotide
    Outcome
    IncreaseChild PedsQL score change
    Study

    n = 1

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated

    Measurements

    PedsQL total score increase from baseline
    28.3 points
    one child

    Source sentences

    Summary
    Three of 5 patients experienced clinically meaningful improvement in PedsQL, with 2 children whose PedsQL total score increased by 28.3 and 3.3 points and 3 adolescents whose mean (SD) total score increased from baseline by 5.8 (18.3) points.
    2 context sentences
    Summary · context
    Two pivotal Phase 3 trials explored the effect of setmelanotide on body weight and hunger in individuals with obesity due to POMC (NCT02896192) or LEPR (NCT03287960) deficiency.
    Summary · context
    QOL and depression were investigated in parallel using the disease-specific, age-appropriate Impact of Weight on Quality of Life-Lite (IWQOL-Lite), Pediatric Quality of Life Inventory (PedsQL), and Patient Health Questionnaire-9 (PHQ-9).
  6. 06HumanNon-randomized trialEfficacy outcome

    During setmelanotide in POMC or LEPR deficiency, another child had a PedsQL increase of 3.3 points.

    In plain wordsDuring setmelanotide, another child with POMC or LEPR deficiency had a quality-of-life score increase of 3.3 points.

    Peptide
    Setmelanotide
    Outcome
    IncreaseChild PedsQL score change
    Study

    n = 1

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated

    Measurements

    PedsQL total score increase from baseline
    3.3 points
    another child

    Source sentences

    Summary
    Three of 5 patients experienced clinically meaningful improvement in PedsQL, with 2 children whose PedsQL total score increased by 28.3 and 3.3 points and 3 adolescents whose mean (SD) total score increased from baseline by 5.8 (18.3) points.
    2 context sentences
    Summary · context
    Two pivotal Phase 3 trials explored the effect of setmelanotide on body weight and hunger in individuals with obesity due to POMC (NCT02896192) or LEPR (NCT03287960) deficiency.
    Summary · context
    QOL and depression were investigated in parallel using the disease-specific, age-appropriate Impact of Weight on Quality of Life-Lite (IWQOL-Lite), Pediatric Quality of Life Inventory (PedsQL), and Patient Health Questionnaire-9 (PHQ-9).
  7. 07HumanNon-randomized trialEfficacy outcome

    In adults with POMC or LEPR deficiency, an effect of setmelanotide on IWQOL-Lite appeared by Week 5.

    In plain wordsIn adults with POMC or LEPR deficiency, setmelanotide affected quality-of-life scores by Week 5.

    Peptide
    Setmelanotide
    Outcome
    Direction not reportedEarly IWQOL-Lite effect
    Study

    n: not reported in source

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    The effect of setmelanotide on IWQOL-Lite total score was observed as soon as Week 5.
    2 context sentences
    Summary · context
    Two pivotal Phase 3 trials explored the effect of setmelanotide on body weight and hunger in individuals with obesity due to POMC (NCT02896192) or LEPR (NCT03287960) deficiency.
    Summary · context
    QOL and depression were investigated in parallel using the disease-specific, age-appropriate Impact of Weight on Quality of Life-Lite (IWQOL-Lite), Pediatric Quality of Life Inventory (PedsQL), and Patient Health Questionnaire-9 (PHQ-9).
  8. 08HumanNon-randomized trialEfficacy outcome

    In patients with POMC or LEPR deficiency receiving setmelanotide, mean baseline PHQ-9 of 5.3 was generally maintained through Week 52.

    In plain wordsIn patients with POMC or LEPR deficiency, depression scores generally remained near baseline during setmelanotide through Week 52.

    Peptide
    Setmelanotide
    Outcome
    Direction not reportedPHQ-9 score maintenance
    Study

    n: not reported in source

    Outcome group
    Anxiety and mood · Mental health and mood
    Studied dose
    Dose not stated

    Measurements

    Baseline PHQ-9 mean
    5.3 points
    age ≥12 years
    Baseline PHQ-9 SD
    3.8 points
    age ≥12 years

    Source sentences

    Summary
    Baseline mean (SD) PHQ-9 score (in those ≥ 12 years old) was 5.3 (3.8) and was generally maintained through Week 52.
    2 context sentences
    Summary · context
    Two pivotal Phase 3 trials explored the effect of setmelanotide on body weight and hunger in individuals with obesity due to POMC (NCT02896192) or LEPR (NCT03287960) deficiency.
    Summary · context
    QOL and depression were investigated in parallel using the disease-specific, age-appropriate Impact of Weight on Quality of Life-Lite (IWQOL-Lite), Pediatric Quality of Life Inventory (PedsQL), and Patient Health Questionnaire-9 (PHQ-9).

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

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