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Een proef met setmelanotide bij verworven hypothalamische obesitas

16 juli 2026 bijgewerkt door: Rhythm Pharmaceuticals, Inc.

Een dubbelblinde, gerandomiseerde, placebogecontroleerde fase 3-studie om de werkzaamheid en veiligheid van setmelanotide te evalueren bij patiënten met verworven hypothalamusobesitas

Het doel van deze proef is om te leren hoe goed Setmelanotide werkt om gewichtsvermindering, honger en kwaliteit van leven te verbeteren bij patiënten van 4 jaar en ouder met verworven hypothalamusobesitas (HO). Om te bepalen hoe goed setmelanotide werkt en hoe veilig het is, zullen patiënten met HO een dagelijkse injectie van setmelanotide of placebo krijgen en de proefbeoordelingen gedurende maximaal 60 weken voltooien.

Studie Overzicht

Toestand

Actief, niet wervend

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Werkelijk)

143

Fase

  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

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

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

4 jaar en ouder (Kind, Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Beschrijving

Belangrijkste opnamecriteria:

  1. Gedocumenteerd bewijs van verworven hypothalamische obesitas (HO)
  2. Leeftijd 4 jaar en ouder
  3. Gewichtstoename geassocieerd met hypothalamusletsel en een BMI van ≥ 30 kg/m2 voor patiënten van ≥ 18 jaar of BMI ≥ 95e percentiel voor leeftijd en geslacht voor patiënten van 4 tot < 18 jaar
  4. Stem ermee in om tijdens het onderzoek en gedurende 90 dagen na het onderzoek een zeer effectieve vorm van anticonceptie te gebruiken

Belangrijkste uitsluitingscriteria:

  1. Diagnose van Prader-Willi-syndroom (PWS) of snel optredende obesitas met hypoventilatie, hypothalamus, autonome ontregeling, neuro-endocrien tumorsyndroom (ROHHADNET)
  2. Gewichtsverlies >2% in de voorgaande 3 maanden voor patiënten van ≥18 jaar of >2% verlaging van de BMI voor patiënten van 4 tot <18 jaar
  3. Bariatrische chirurgie of ingreep in de afgelopen 2 jaar
  4. Diagnose van ernstige psychiatrische stoornissen; elke zelfmoordgedachte, -poging of -gedrag
  5. Huidige, klinisch significante long-, hart-, metabolische of oncologische ziekte
  6. Significante dermatologische bevindingen met betrekking tot melanoom of pre-melanoom huidlaesies (exclusief niet-invasieve basale of plaveiselcellaesie)
  7. Geschiedenis of naaste familiegeschiedenis van huidkanker of melanoom
  8. Deelname aan een klinische studie met een onderzoeksgeneesmiddel/apparaat binnen 3 maanden voorafgaand aan de eerste proefdosis
  9. Eerder deelnamen aan een klinisch onderzoek met setmelanotide of enige eerdere blootstelling aan setmelanotide
  10. Onvermogen om te voldoen aan het eenmaal daagse (QD) injectieregime
  11. Indien vrouwelijk, zwanger en/of borstvoeding gevend
  12. Patiënten met obesitas toe te schrijven aan andere genetische of syndromale aandoeningen (bijv. PPL [POMC, PCSK1, LEPR, collectief], BBS) voorafgaand aan de hypothalamische verwonding.
  13. Als u hormoonvervangingstherapie krijgt, is de dosis gedurende ten minste 2 maanden vóór de screening stabiel gebleven

Andere in het protocol gedefinieerde opname-/uitsluitingscriteria kunnen van toepassing zijn.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Verdrievoudigen

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Setemelanotide
Gerandomiseerd 2:1 (Setmelanotide: Placebo)
Geneesmiddel: Melanotide instellen
Oplossing voor dagelijkse subcutane injectie
Andere namen:
  • RM-493
  • Imcivree
Placebo-vergelijker: Placebo
Gerandomiseerd 2:1 (Setmelanotide: Placebo)
Geneesmiddel: Placebo
Placebo afgestemd op setmelanotide voor dagelijkse subcutane injectie

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Pivotal Cohort: Mean Percent Change From Baseline in Body Mass Index (BMI) After 52 Weeks on a Therapeutic Regimen
Tijdsspanne: 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)

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
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)
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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)

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Rhythm Pharmaceuticals, Inc.

Onderzoekers

  • Studie stoel: David Meeker, MD, Rhythm Pharmaceuticals, Inc.

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Algemene publicaties

Nuttige links

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

26 april 2023

Primaire voltooiing (Werkelijk)

18 maart 2025

Studie voltooiing (Geschat)

16 april 2027

Studieregistratiedata

Eerst ingediend

1 maart 2023

Eerst ingediend dat voldeed aan de QC-criteria

16 maart 2023

Eerst geplaatst (Werkelijk)

20 maart 2023

Updates van studierecords

Laatste update geplaatst (Werkelijk)

10 augustus 2026

Laatste update ingediend die voldeed aan QC-criteria

16 juli 2026

Laatst geverifieerd

1 juli 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • RM-493-040

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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