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Un ensayo de setmelanotida en la obesidad hipotalámica adquirida

16 de julio de 2026 actualizado por: Rhythm Pharmaceuticals, Inc.

Un ensayo de fase 3, doble ciego, aleatorizado, controlado con placebo para evaluar la eficacia y la seguridad de setmelanotida en pacientes con obesidad hipotalámica adquirida

El objetivo de este ensayo es conocer qué tan bien funciona Setmelanotide para mejorar la reducción de peso, el hambre y la calidad de vida en pacientes de 4 años de edad y mayores con obesidad hipotalámica (HO) adquirida. Para determinar qué tan bien funciona setmelanotide y qué tan seguro es, los pacientes con HO recibirán una inyección diaria de setmelanotide o placebo y completarán las evaluaciones de prueba por hasta 60 semanas.

Descripción general del estudio

Estado

Activo, no reclutando

Condiciones

Intervención / Tratamiento

Tipo de estudio

Intervencionista

Inscripción (Actual)

143

Fase

  • Fase 3

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

  • Alemania
      • Hamburg, Alemania, 20246
        • Universitaetsklinikum Hamburg-Eppendorf (UKE) - Ambulanzzentrum des UKE GmbH
      • München, Alemania, 81667
        • Medicover Neuroendokrinologie
      • Oldenburg, Alemania, 26133
        • University Children's Hospital, Klinikum Oldenburg
      • Ulm, Alemania, 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
  • Japón
    • Aichi-ken
      • Nagoya, Aichi-ken, Japón, 467-8602
        • Nagoya City University Hospital
    • Nagano
      • Azumino, Nagano, Japón, 399-8288
        • Nagano Children's Hospital
    • Tokyo
      • Minato, Tokyo, Japón, 105-8470
        • Toranomon Hospital
  • Países Bajos
      • Utrecht, Países Bajos, 3584CS
        • Prinses Maxima Center for Pediatric Oncology
  • 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

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

4 años y mayores (Niño, Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Descripción

Criterios clave de inclusión:

  1. Evidencia documentada de obesidad hipotalámica adquirida (HO)
  2. Edad 4 años y más
  3. Aumento de peso asociado a la lesión hipotalámica e IMC ≥30 kg/m2 para pacientes ≥18 años o IMC ≥percentil 95 para edad y sexo para pacientes de 4 a <18 años
  4. Aceptar usar un método anticonceptivo altamente eficaz durante todo el estudio y durante los 90 días posteriores al estudio

Criterios clave de exclusión:

  1. Diagnóstico de síndrome de Prader-Willi (PWS) u obesidad de inicio rápido con hipoventilación, desregulación autonómica hipotalámica, síndrome de tumor neuroendocrino (ROHHADNET)
  2. Pérdida de peso >2% en los 3 meses previos para pacientes ≥18 años o reducción >2% del IMC para pacientes de 4 a <18 años
  3. Cirugía o procedimiento bariátrico en los últimos 2 años
  4. Diagnóstico de trastornos psiquiátricos graves; cualquier ideación, intento o comportamiento suicida
  5. Enfermedad pulmonar, cardíaca, metabólica u oncológica clínicamente significativa actual
  6. Hallazgos dermatológicos significativos relacionados con lesiones cutáneas de melanoma o premelanoma (excluyendo lesiones de células escamosas o basales no invasivas)
  7. Antecedentes o antecedentes familiares cercanos de cáncer de piel o melanoma
  8. Participación en cualquier ensayo clínico con un fármaco/dispositivo en investigación dentro de los 3 meses anteriores a la primera dosis del ensayo
  9. Inscrito previamente en un ensayo clínico que involucre setmelanotide o cualquier exposición previa a setmelanotide
  10. Incapacidad para cumplir con el régimen de inyección una vez al día (QD)
  11. Si es mujer, embarazada y/o amamantando
  12. Pacientes con obesidad atribuible a otras condiciones genéticas o sindrómicas (p. ej., PPL [POMC, PCSK1, LEPR, colectivamente], BBS) antes de la lesión hipotalámica.
  13. Si recibe terapia de reemplazo hormonal, la dosis se ha mantenido estable durante al menos 2 meses antes de la selección

Pueden aplicarse otros criterios de inclusión/exclusión definidos en el protocolo.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: Aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Triple

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Setemelanotida
Aleatorizado 2:1 (Setmelanotide: Placebo)
Droga: Setmelanotida
Solución para inyección subcutánea diaria
Otros nombres:
  • RM-493
  • Imcivree
Comparador de placebos: Placebo
Aleatorizado 2:1 (Setmelanotide: Placebo)
Droga: Placebo
Placebo emparejado con setmelanotida para inyección subcutánea diaria

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Pivotal Cohort: Mean Percent Change From Baseline in Body Mass Index (BMI) After 52 Weeks on a Therapeutic Regimen
Periodo de tiempo: 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 resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
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)
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Rhythm Pharmaceuticals, Inc.

Investigadores

  • Silla de estudio: David Meeker, MD, Rhythm Pharmaceuticals, Inc.

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Publicaciones Generales

Enlaces Útiles

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

26 de abril de 2023

Finalización primaria (Actual)

18 de marzo de 2025

Finalización del estudio (Estimado)

16 de abril de 2027

Fechas de registro del estudio

Enviado por primera vez

1 de marzo de 2023

Primero enviado que cumplió con los criterios de control de calidad

16 de marzo de 2023

Publicado por primera vez (Actual)

20 de marzo de 2023

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

10 de agosto de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

16 de julio de 2026

Última verificación

1 de julio de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • RM-493-040

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

NO

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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