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Una prova di Setmelanotide nell'obesità ipotalamica acquisita

4 maggio 2026 aggiornato da: Rhythm Pharmaceuticals, Inc.

Uno studio di fase 3, in doppio cieco, randomizzato, controllato con placebo per valutare l'efficacia e la sicurezza del setmelanotide nei pazienti con obesità ipotalamica acquisita

L'obiettivo di questo studio è scoprire quanto bene Setmelanotide funzioni per migliorare la riduzione del peso, la fame e la qualità della vita nei pazienti di età pari o superiore a 4 anni con obesità ipotalamica acquisita (HO). Per determinare quanto bene funziona setmelanotide e quanto è sicuro, i pazienti con HO prenderanno un'iniezione giornaliera di setmelanotide o placebo e completeranno le valutazioni di prova per un massimo di 60 settimane.

Panoramica dello studio

Stato

Attivo, non reclutante

Condizioni

Intervento / Trattamento

Tipo di studio

Interventistico

Iscrizione (Effettivo)

143

Fase

  • Fase 3

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

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

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

4 anni e precedenti (Bambino, Adulto, Adulto più anziano)

Accetta volontari sani

No

Descrizione

Criteri chiave di inclusione:

  1. Prove documentate di obesità ipotalamica acquisita (HO)
  2. Età 4 anni e oltre
  3. Aumento di peso associato al danno ipotalamico e un BMI ≥30 kg/m2 per i pazienti di età ≥18 anni o BMI ≥95° percentile per età e sesso per i pazienti di età compresa tra 4 e <18 anni
  4. Accetta di utilizzare una forma di contraccezione altamente efficace durante lo studio e per 90 giorni dopo lo studio

Criteri chiave di esclusione:

  1. Diagnosi di sindrome di Prader-Willi (PWS) o obesità a insorgenza rapida con ipoventilazione, disregolazione ipotalamica, autonomica, sindrome da tumore neuroendocrino (ROHHADNET)
  2. Perdita di peso >2% nei 3 mesi precedenti per i pazienti di età ≥18 anni o >2% di riduzione del BMI per i pazienti di età compresa tra 4 e <18 anni
  3. Chirurgia o procedura bariatrica negli ultimi 2 anni
  4. Diagnosi di gravi disturbi psichiatrici; qualsiasi idea, tentativo o comportamento suicidario
  5. Malattia polmonare, cardiaca, metabolica o oncologica in corso, clinicamente significativa
  6. Reperti dermatologici significativi relativi a lesioni cutanee di melanoma o pre-melanoma (escluse lesioni a cellule basali o squamose non invasive)
  7. Storia o storia familiare stretta di cancro della pelle o melanoma
  8. Partecipazione a qualsiasi sperimentazione clinica con un farmaco/dispositivo sperimentale entro 3 mesi prima della prima dose di prova
  9. Precedentemente arruolato in uno studio clinico che coinvolge setmelanotide o qualsiasi precedente esposizione a setmelanotide
  10. Incapacità di rispettare il regime di iniezioni una volta al giorno (QD).
  11. Se donna, gravidanza e/o allattamento
  12. Pazienti con obesità attribuibile ad altre condizioni genetiche o sindromiche (p. es., PPL [POMC, PCSK1, LEPR, collettivamente], BBS) prima della lesione ipotalamica.
  13. In caso di terapia ormonale sostitutiva, la dose è rimasta stabile per almeno 2 mesi prima dello screening

Potrebbero essere applicati altri criteri di inclusione/esclusione definiti dal protocollo.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Randomizzato
  • Modello interventistico: Assegnazione parallela
  • Mascheramento: Triplicare

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: Setemelanotide
Randomizzato 2:1 (Setmelanotide: Placebo)
Droga: Setmelanotide
Soluzione per iniezione sottocutanea giornaliera
Altri nomi:
  • RM-493
  • Imcivree
Comparatore placebo: Placebo
Randomizzato 2:1 (Setmelanotide: Placebo)
Droga: Placebo
Placebo abbinato a setmelanotide per iniezione sottocutanea giornaliera

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Pivotal Cohort: Mean Percent Change From Baseline in Body Mass Index (BMI) After 52 Weeks on a Therapeutic Regimen
Lasso di tempo: 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)

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Rhythm Pharmaceuticals, Inc.

Investigatori

  • Cattedra di studio: David Meeker, MD, Rhythm Pharmaceuticals, Inc.

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

26 aprile 2023

Completamento primario (Effettivo)

18 marzo 2025

Completamento dello studio (Stimato)

16 aprile 2027

Date di iscrizione allo studio

Primo inviato

1 marzo 2023

Primo inviato che soddisfa i criteri di controllo qualità

16 marzo 2023

Primo Inserito (Effettivo)

20 marzo 2023

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

29 maggio 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

4 maggio 2026

Ultimo verificato

1 maggio 2026

Maggiori informazioni

Termini relativi a questo studio

Parole chiave

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • RM-493-040

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

NO

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

Sì

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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