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Um ensaio de setmelanotida na obesidade hipotalâmica adquirida

16 de julho de 2026 atualizado por: Rhythm Pharmaceuticals, Inc.

Um estudo de fase 3, duplo-cego, randomizado e controlado por placebo para avaliar a eficácia e a segurança da setmelanotida em pacientes com obesidade hipotalâmica adquirida

O objetivo deste estudo é saber o quão bem Setmelanotida funciona para melhorar a redução de peso, fome e qualidade de vida em pacientes de 4 anos de idade ou mais com obesidade hipotalâmica adquirida (HO). Para determinar o quão bem a setmelanotida funciona e quão segura ela é, os pacientes com HO receberão uma injeção diária de setmelanotida ou placebo e completarão as avaliações do estudo por até 60 semanas.

Visão geral do estudo

Status

Ativo, não recrutando

Condições

Intervenção / Tratamento

Tipo de estudo

Intervencional

Inscrição (Real)

143

Estágio

  • Fase 3

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

  • Alemanha
      • Hamburg, Alemanha, 20246
        • Universitaetsklinikum Hamburg-Eppendorf (UKE) - Ambulanzzentrum des UKE GmbH
      • München, Alemanha, 81667
        • Medicover Neuroendokrinologie
      • Oldenburg, Alemanha, 26133
        • University Children's Hospital, Klinikum Oldenburg
      • Ulm, Alemanha, 89075
        • Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
  • Canadá
    • Ontario
      • Toronto, Ontario, Canadá, M5G 1X8
        • Hospital for Sick Children
  • Estados Unidos
    • Alabama
      • Birmingham, Alabama, Estados Unidos, 35233
        • UAN Pediatric Endocrinology
    • California
      • San Diego, California, Estados Unidos, 92123
        • Rady Children's Hospital
    • Colorado
      • Aurora, Colorado, Estados Unidos, 80045
        • Children's Hospital Colorado
    • Florida
      • Gainesville, Florida, Estados Unidos, 32610-0296
        • University of Florida
    • Illinois
      • Chicago, Illinois, Estados Unidos, 60611
        • Ann and Robert H. Lurie Children's Hospital
    • Iowa
      • Iowa City, Iowa, Estados Unidos, 52242
        • University of Iowa Stead Family Department of Pediatrics
    • Massachusetts
      • Boston, Massachusetts, Estados Unidos, 02115
        • Brigham and Women's Hospital
      • Boston, Massachusetts, Estados Unidos, 02115
        • Boston Children's Hospital
    • Minnesota
      • Saint Paul, Minnesota, Estados Unidos, 55102
        • Children's Minnesota
    • New York
      • New York, New York, Estados Unidos, 10032
        • Columbia University Irving Medical Center
      • New York, New York, Estados Unidos, 100029
        • Icahn School of Medicine at Mount Sinai
    • Ohio
      • Columbus, Ohio, Estados Unidos, 43203
        • Ohio State Wexner Medical Center
    • Oklahoma
      • Oklahoma City, Oklahoma, Estados Unidos, 73122
        • Lynn Health Science Institute
    • Pennsylvania
      • Philadelphia, Pennsylvania, Estados Unidos, 19104
        • Children's Hospital of Philadelphia
    • Tennessee
      • Nashville, Tennessee, Estados Unidos, 37232
        • Vanderbilt University School of Medicine
    • Washington
      • Seattle, Washington, Estados Unidos, 98101
        • Seattle Children's Hospital, Research and Foundation - Center for Integrative Brain Research
  • Holanda
      • Utrecht, Holanda, 3584CS
        • Prinses Maxima Center for Pediatric Oncology
  • Japão
    • Aichi-ken
      • Nagoya, Aichi-ken, Japão, 467-8602
        • Nagoya City University Hospital
    • Nagano
      • Azumino, Nagano, Japão, 399-8288
        • Nagano Children's Hospital
    • Tokyo
      • Minato, Tokyo, Japão, 105-8470
        • Toranomon Hospital
  • Reino Unido
      • Birmingham, Reino Unido, B46NH
        • Birmingham Women and Children's Hospital NHS Trust
      • Hull, Reino Unido, HU32RW
        • Hull University Teaching Hospital
      • London, Reino Unido, WC1N 1EH
        • UCL Great Ormond Street Institute of Child Health

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

4 anos e mais velhos (Filho, Adulto, Adulto mais velho)

Aceita Voluntários Saudáveis

Não

Descrição

Principais Critérios de Inclusão:

  1. Evidência documentada de obesidade hipotalâmica adquirida (HO)
  2. Idade 4 anos ou mais
  3. Ganho de peso associado à lesão hipotalâmica e IMC ≥30 kg/m2 para pacientes ≥18 anos de idade ou IMC ≥95º percentil para idade e sexo para pacientes de 4 a <18 anos de idade
  4. Concordar em usar uma forma altamente eficaz de contracepção durante todo o estudo e por 90 dias após o estudo

Principais Critérios de Exclusão:

  1. Diagnóstico de síndrome de Prader-Willi (PWS) ou obesidade de início rápido com hipoventilação, hipotálamo, desregulação autonômica, síndrome de tumor neuroendócrino (ROHHADNET)
  2. Perda de peso >2% nos últimos 3 meses para pacientes com idade ≥18 anos ou redução >2% no IMC para pacientes com idade de 4 a <18 anos
  3. Cirurgia ou procedimento bariátrico nos últimos 2 anos
  4. Diagnóstico de transtornos psiquiátricos graves; qualquer ideação, tentativa ou comportamento suicida
  5. Doença pulmonar, cardíaca, metabólica ou oncológica atual e clinicamente significativa
  6. Achados dermatológicos significativos relacionados a lesões cutâneas de melanoma ou pré-melanoma (excluindo lesão de células escamosas ou basais não invasivas)
  7. História ou história familiar próxima de câncer de pele ou melanoma
  8. Participação em qualquer ensaio clínico com um medicamento/dispositivo experimental dentro de 3 meses antes da primeira dose do ensaio
  9. Anteriormente inscrito em um ensaio clínico envolvendo setmelanotida ou qualquer exposição anterior a setmelanotida
  10. Incapacidade de cumprir o regime de injeção uma vez ao dia (QD)
  11. Se mulher, grávida e/ou amamentando
  12. Pacientes com obesidade atribuível a outras condições genéticas ou sindrômicas (por exemplo, PPL [POMC, PCSK1, LEPR, coletivamente], BBS) antes da lesão hipotalâmica.
  13. Se estiver recebendo terapia de reposição hormonal, a dose permaneceu estável por pelo menos 2 meses antes da triagem

Outros critérios de inclusão/exclusão definidos pelo protocolo podem ser aplicados.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Triplo

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Setemelanotide
Randomizado 2:1 (Setmelanotide: Placebo)
Medicamento: Setmelanotida
Solução para injeção subcutânea diária
Outros nomes:
  • RM-493
  • Imcivree
Comparador de Placebo: Placebo
Randomizado 2:1 (Setmelanotide: Placebo)
Medicamento: Placebo
Placebo combinado com setmelanotida para injeção subcutânea diária

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Pivotal Cohort: Mean Percent Change From Baseline in Body Mass Index (BMI) After 52 Weeks on a Therapeutic Regimen
Prazo: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
BMI was calculated as weight (kg)/height (m^2). Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Pivotal Cohort: Percentage of Participants With ≥5% Reduction From Baseline in BMI (≥18 Years of Age) or ≥0.2-point Reduction From Baseline in BMI Z-score (<18 Years of Age)
Prazo: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
BMI was calculated as weight (kg)/height (m^2). BMI Z-Score calculated for participants <18 years old only is a measure of relative weight adjusted for child's age, sex and height at the time of data collection. The Z-Scores were calculated using the World Health Organization's WHO 2007 BMI SAS Macro Package. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease in BMI Z-score (< 0) indicates a reduction in BMI from Baseline whereas an increase in BMI-Z score (> 0) indicated an increase in BMI from Baseline. Baseline was defined as the most recent measurement prior to the first administration of study drug. Results below represent the percentage of estimated participants with either ≥5% BMI reduction or ≥0.2 point reduction in BMI Z-score by age in each treatment arm as calculated through the MIANALYZE SAS procedure.
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
Pivotal Cohort: Percentage of Participants With ≥5% Reduction From Baseline in BMI
Prazo: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
BMI was calculated as weight (kg)/height (m^2). Results below represent the percentage of estimated participants with ≥5% BMI reduction in each treatment arm as calculated through the MIANALYZE SAS procedure.
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
Pivotal Cohort: Change From Baseline in Weekly Average Daily Most Hunger Score in Participants ≥12 Years of Age
Prazo: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Change from baseline in hunger scores for participants ≥12 years of age with acquired hypothalamic obesity was evaluated. Hunger score ranged from 0= "not hungry at all" to 10= "hungriest possible" on an 11-point numeric rating scale. On Daily Hunger Questionnaire, each of the 2 items (average hunger and most hunger) was scored separately and averaged on weekly basis. LSM and SE were calculated using ANCOVA model.
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Pivotal Cohort: Percentage of Participants (≥12 Years of Age) With a ≥2-point Reduction From Baseline in the Weekly Average Daily Most Hunger Score
Prazo: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
Hunger score ranged from 0= "not hungry at all" to 10= "hungriest possible" on an 11-point numeric rating scale. On Daily Hunger Questionnaire, each of the 2 items (average hunger and most hunger) was scored separately and averaged on weekly basis. Results below represent the percentage of estimated participants ≥12 years of age with ≥2-point reduction in each treatment arm as calculated through the MIANALYZE SAS procedure.
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
Pivotal Cohort: Mean Change From Baseline in the Weekly Average of the Symptoms of Hyperphagia Composite Score in Participants ≥12 Years of Age
Prazo: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Participants ≥12 years of age who were able to self-report were administered the Symptoms of Hyperphagia: Patient (Version 1.0). The questionnaire consisted of 4 items related to the frequency of a participant's hunger symptoms on a scale (Never, 1 or 2 times, 3 or more times) over the past 24 hours. The scores on this scale range from 0 to 2 with higher scores indicating more hyperphagia. Daily composite score was derived as the sum of daily answered questions divided by the number of answered questions per day. The weekly average of the daily composite scores equals to the sum of daily composite scores divided by the number of days with a composite score within the 7 identified days prior to the visit. LSM and SE were calculated using ANCOVA model.
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Pivotal Cohort: Percentage of Participants With a ≥10% Reduction From Baseline in BMI
Prazo: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
BMI was calculated as weight (kg)/height (m^2). Results below represent the percentage of estimated participants with ≥10% BMI reduction in each treatment arm as calculated through the MIANALYZE SAS procedure.
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
Pivotal Cohort: Percentage of Participants With a ≥10% Reduction From Baseline in Body Weight
Prazo: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
Body weight was captured for analysis in kilograms. Results below represent the percentage of estimated participants with ≥10% reduction in body weight in each treatment arm as calculated through the MIANALYZE SAS procedure.
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
Pivotal Cohort: Mean Percent Change From Baseline in Body Weight in Participants ≥18 Years
Prazo: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Body weight was captured for analysis in kilograms. LSM and SE were calculated using ANCOVA model.
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Pivotal Cohort: Mean Change From Baseline in BMI Z-Score in Participants <18 Years of Age
Prazo: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
BMI was calculated as weight (kg)/height (m^2). BMI Z-Score calculated for participants <18 years old only is a measure of relative weight adjusted for child's age, sex and height at the time of data collection. The Z-Scores were calculated using the World Health Organization's WHO 2007 BMI SAS Macro Package. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease in BMI Z-score (< 0) indicates a reduction in BMI from Baseline whereas an increase in BMI-Z score (> 0) indicates an increase in BMI from Baseline. Baseline was defined as the most recent measurement prior to the first administration of study drug. LSM and SE were calculated using ANCOVA model.
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Pivotal Cohort: Mean Change From Baseline in Percent of BMI 95th Percentile in Participants <18 Years of Age
Prazo: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m^2. BMI percentile scores are measures of relative weight adjusted for child's age and gender. The percent of the BMI 95th percentile score expresses the participant's BMI as a percentage of the Centers for Disease Control (CDC) 95th percentile reference population. Baseline was defined as the most recent measurement prior to the first administration of study drug. LSM and SE were calculated using ANCOVA model.
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Pivotal Cohort: Percentage of Participants <18 Years of Age With ≥0.2-Point Reduction From Baseline in BMI Z-Score
Prazo: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
BMI was calculated as weight (kg)/height (m^2). BMI Z-Score calculated for participants <18 years old only is a measure of relative weight adjusted for child's age, sex and height at the time of data collection. The Z-Scores were calculated using the World Health Organization's WHO 2007 BMI SAS Macro Package. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). A decrease in BMI Z-score (< 0) indicates a reduction in BMI from Baseline whereas an increase in BMI-Z score (> 0) indicates an increase in BMI from Baseline. Baseline was defined as the most recent measurement prior to the first administration of study drug. Results below represent the percentage of estimated participants with ≥0.2 point reduction in BMI Z-score in each treatment arm and the difference between the treatment arms as calculated through the MIANALYZE SAS procedure.
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
Pivotal Cohort: Percentage of Participants With BMI <30 kg/m^2 (Aged ≥18 Years) or <95th Percentile (Aged <18 Years) From Baseline
Prazo: After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m^2. BMI Percentile scores are measures of relative weight adjusted for child's age and gender. The percent of the BMI 95th percentile score expresses the participant's BMI as a percentage of the CDC 95th percentile reference population. Baseline was defined as the most recent measurement prior to the first administration of study drug. Results below represent the percentage of estimated participants with either BMI <30 kg/m^2 (aged ≥18 years) or <95th percentile (aged <18 years) in each treatment arm and the difference between the treatment arms as calculated through the MIANALYZE SAS procedure.
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
Pivotal Cohort: Mean Change From Baseline in Physical Functioning Score and Total Score on the Impact of Weight on Quality of Life-Lite (IWQOL)
Prazo: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
The IWQOL-Lite-Clinical Trials (administered to participants ≥18 years of age) is a validated 20-item self-report measure of obesity-specific quality of life questionnaire. It assessed 2 primary domains of obesity-related health-related quality of life (HRQoL): physical (7 items) and psychosocial (13 items). Each item was rated on a scale from 0 (worst) to 100 (best), with higher scores indicating better levels of functioning. It provided composite scores for each domain, as well as a total score, all ranging from 0 (worst) to 100 (best). Higher scores reflect better levels of functioning and quality of life. LSM and SE were calculated using ANCOVA model.
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Pivotal Cohort: Mean Change From Baseline in Total Score on the Impact of Weight on Quality of Life-Kids (IWQOL-Kids)
Prazo: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
The IWQOL-Kids (administered to participants between the ages of 11 and <18 years) is a validated 27-item self-report measure of weight-related quality of life for youth. It provided a total score inclusive of 4 domains: physical comfort, body esteem, social life, and family relations. Results below represent the total score, which is rated on a scale from 0 (worst) to 100 (best), with higher scores indicating better levels of functioning. LSM and SE were calculated using ANCOVA model.
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Pivotal Cohort: Mean Change From Baseline in Waist Circumference
Prazo: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Waist circumference was captured for analysis in centimeters. LSM and SE were calculated using ANCOVA model.
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Pivotal Cohort: Change From Baseline in Systolic and Diastolic Blood Pressure
Prazo: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
Blood pressure was calculated in millimeters of mercury.
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Rhythm Pharmaceuticals, Inc.

Investigadores

  • Cadeira de estudo: David Meeker, MD, Rhythm Pharmaceuticals, Inc.

Publicações e links úteis

A pessoa responsável por inserir informações sobre o estudo fornece voluntariamente essas publicações. Estes podem ser sobre qualquer coisa relacionada ao estudo.

Publicações Gerais

Links úteis

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

26 de abril de 2023

Conclusão Primária (Real)

18 de março de 2025

Conclusão do estudo (Estimado)

16 de abril de 2027

Datas de inscrição no estudo

Enviado pela primeira vez

1 de março de 2023

Enviado pela primeira vez que atendeu aos critérios de CQ

16 de março de 2023

Primeira postagem (Real)

20 de março de 2023

Atualizações de registro de estudo

Última Atualização Postada (Real)

10 de agosto de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

16 de julho de 2026

Última verificação

1 de julho de 2026

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • RM-493-040

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

NÃO

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Sim

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

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