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Un essai de Setmelanotide dans l'obésité hypothalamique acquise

16 juillet 2026 mis à jour par: Rhythm Pharmaceuticals, Inc.

Un essai de phase 3, en double aveugle, randomisé et contrôlé par placebo pour évaluer l'efficacité et l'innocuité du setmélanotide chez les patients atteints d'obésité hypothalamique acquise

L'objectif de cet essai est d'apprendre dans quelle mesure Setmelanotide agit pour améliorer la réduction de poids, la faim et la qualité de vie chez les patients de 4 ans et plus atteints d'obésité hypothalamique (HO) acquise. Pour déterminer l'efficacité et l'innocuité du setmélanotide, les patients atteints d'HO recevront une injection quotidienne de setmélanotide ou de placebo et effectueront des évaluations d'essais jusqu'à 60 semaines.

Aperçu de l'étude

Statut

Actif, ne recrute pas

Les conditions

Intervention / Traitement

Type d'étude

Interventionnel

Inscription (Réel)

143

Phase

  • Phase 3

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

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

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

4 ans et plus (Enfant, Adulte, Adulte plus âgé)

Accepte les volontaires sains

Non

La description

Critères d'inclusion clés :

  1. Preuve documentée de l'obésité hypothalamique acquise (HO)
  2. Âge 4 ans et plus
  3. Gain de poids associé à la lésion hypothalamique et un IMC ≥ 30 kg/m2 pour les patients âgés de ≥ 18 ans ou un IMC ≥ 95e centile pour l'âge et le sexe pour les patients âgés de 4 à < 18 ans
  4. Accepter d'utiliser une forme de contraception hautement efficace tout au long de l'étude et pendant 90 jours après l'étude

Critères d'exclusion clés :

  1. Diagnostic du syndrome de Prader-Willi (PWS) ou de l'obésité d'apparition rapide avec hypoventilation, hypothalamique, dysrégulation autonome, syndrome de tumeur neuroendocrinienne (ROHHADNET)
  2. Perte de poids > 2 % au cours des 3 mois précédents pour les patients âgés de ≥ 18 ans ou réduction > 2 % de l'IMC pour les patients âgés de 4 à < 18 ans
  3. Chirurgie ou intervention bariatrique au cours des 2 dernières années
  4. Diagnostic des troubles psychiatriques graves ; toute idée, tentative ou comportement suicidaire
  5. Maladie pulmonaire, cardiaque, métabolique ou oncologique actuelle et cliniquement significative
  6. Résultats dermatologiques significatifs liés à des lésions cutanées de mélanome ou de pré-mélanome (à l'exclusion des lésions basales ou épidermoïdes non invasives)
  7. Antécédents ou antécédents familiaux proches de cancer de la peau ou de mélanome
  8. Participation à tout essai clinique avec un médicament/dispositif expérimental dans les 3 mois précédant la première dose d'essai
  9. Précédemment inscrit à un essai clinique impliquant le setmélanotide ou toute exposition antérieure au setmélanotide
  10. Incapacité à se conformer au régime d'injection une fois par jour (QD)
  11. Si femme, enceinte et/ou allaitante
  12. Patients souffrant d'obésité attribuable à d'autres conditions génétiques ou syndromiques (par exemple, PPL [POMC, PCSK1, LEPR, collectivement], BBS) avant la lésion hypothalamique.
  13. Si vous recevez un traitement hormonal substitutif, la dose est restée stable pendant au moins 2 mois avant le dépistage

D'autres critères d'inclusion/exclusion définis par le protocole peuvent s'appliquer.

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Randomisé
  • Modèle interventionnel: Affectation parallèle
  • Masquage: Tripler

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: Setemelanotide
Randomisé 2:1 (Setmelanotide : Placebo)
Médicament: Setmélanotide
Solution pour injection sous-cutanée quotidienne
Autres noms:
  • RM-493
  • Imcivrée
Comparateur placebo: Placebo
Randomisé 2:1 (Setmelanotide : Placebo)
Médicament: Placebo
Placebo apparié au setmélanotide pour une injection sous-cutanée quotidienne

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Pivotal Cohort: Mean Percent Change From Baseline in Body Mass Index (BMI) After 52 Weeks on a Therapeutic Regimen
Délai: 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)

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
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)
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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)
Délai: 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)
Délai: 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
Délai: 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
Délai: 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)

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Rhythm Pharmaceuticals, Inc.

Les enquêteurs

  • Chaise d'étude: David Meeker, MD, Rhythm Pharmaceuticals, Inc.

Publications et liens utiles

La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.

Publications générales

Liens utiles

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

26 avril 2023

Achèvement primaire (Réel)

18 mars 2025

Achèvement de l'étude (Estimé)

16 avril 2027

Dates d'inscription aux études

Première soumission

1 mars 2023

Première soumission répondant aux critères de contrôle qualité

16 mars 2023

Première publication (Réel)

20 mars 2023

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

10 août 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

16 juillet 2026

Dernière vérification

1 juillet 2026

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • RM-493-040

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

NON

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Oui

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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