En prøvelse av Setmelanotid i ervervet hypotalamisk fedme
16. juli 2026 oppdatert av: Rhythm Pharmaceuticals, Inc.
En fase 3, dobbeltblind, randomisert, placebokontrollert studie for å evaluere effekten og sikkerheten til setmelanotid hos pasienter med ervervet hypotalamisk fedme
Målet med denne studien er å lære hvor godt Setmelanotide virker for å forbedre vektreduksjon, sult og livskvalitet hos pasienter 4 år og eldre med ervervet hypotalamisk fedme (HO).
For å bestemme hvor godt setmelanotide virker og hvor trygt det er, vil pasienter med HO ta en daglig injeksjon med enten setmelanotid eller placebo og fullføre prøvevurderinger i opptil 60 uker.
Studieoversikt
Status
Aktiv, ikke rekrutterende
Forhold
Intervensjon / Behandling
Studietype
Intervensjonell
Registrering (Faktiske)
143
Fase
- Fase 3
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiesteder
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Ontario
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Toronto, Ontario, Canada, M5G 1X8
- Hospital for Sick Children
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Alabama
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Birmingham, Alabama, Forente stater, 35233
- UAN Pediatric Endocrinology
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California
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San Diego, California, Forente stater, 92123
- Rady Children's Hospital
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Colorado
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Aurora, Colorado, Forente stater, 80045
- Children's Hospital Colorado
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Florida
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Gainesville, Florida, Forente stater, 32610-0296
- University of Florida
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Illinois
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Chicago, Illinois, Forente stater, 60611
- Ann and Robert H. Lurie Children's Hospital
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Iowa
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Iowa City, Iowa, Forente stater, 52242
- University of Iowa Stead Family Department of Pediatrics
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Massachusetts
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Boston, Massachusetts, Forente stater, 02115
- Brigham and Women's Hospital
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Boston, Massachusetts, Forente stater, 02115
- Boston Children's Hospital
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Minnesota
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Saint Paul, Minnesota, Forente stater, 55102
- Children's Minnesota
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New York
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New York, New York, Forente stater, 10032
- Columbia University Irving Medical Center
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New York, New York, Forente stater, 100029
- Icahn School of Medicine at Mount Sinai
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Ohio
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Columbus, Ohio, Forente stater, 43203
- Ohio State Wexner Medical Center
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Oklahoma
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Oklahoma City, Oklahoma, Forente stater, 73122
- Lynn Health Science Institute
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Pennsylvania
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Philadelphia, Pennsylvania, Forente stater, 19104
- Children's Hospital of Philadelphia
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Tennessee
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Nashville, Tennessee, Forente stater, 37232
- Vanderbilt University School of Medicine
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Washington
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Seattle, Washington, Forente stater, 98101
- Seattle Children's Hospital, Research and Foundation - Center for Integrative Brain Research
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Aichi-ken
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Nagoya, Aichi-ken, Japan, 467-8602
- Nagoya City University Hospital
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Nagano
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Azumino, Nagano, Japan, 399-8288
- Nagano Children's Hospital
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Tokyo
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Minato, Tokyo, Japan, 105-8470
- Toranomon Hospital
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Utrecht, Nederland, 3584CS
- Prinses Maxima Center for Pediatric Oncology
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Birmingham, Storbritannia, B46NH
- Birmingham Women and Children's Hospital NHS Trust
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Hull, Storbritannia, HU32RW
- Hull University Teaching Hospital
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London, Storbritannia, WC1N 1EH
- UCL Great Ormond Street Institute of Child Health
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Hamburg, Tyskland, 20246
- Universitaetsklinikum Hamburg-Eppendorf (UKE) - Ambulanzzentrum des UKE GmbH
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München, Tyskland, 81667
- Medicover Neuroendokrinologie
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Oldenburg, Tyskland, 26133
- University Children's Hospital, Klinikum Oldenburg
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Ulm, Tyskland, 89075
- Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
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Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
4 år og eldre (Barn, Voksen, Eldre voksen)
Tar imot friske frivillige
Nei
Beskrivelse
Viktige inkluderingskriterier:
- Dokumentert bevis på ervervet hypotalamisk fedme (HO)
- Alder 4 år og eldre
- Vektøkning assosiert med hypotalamisk skade og en BMI på ≥30 kg/m2 for pasienter ≥18 år eller BMI ≥95. persentil for alder og kjønn for pasienter 4 til <18 år
- Godta å bruke en svært effektiv form for prevensjon gjennom hele studien og i 90 dager etter studien
Nøkkelekskluderingskriterier:
- Diagnostisering av Prader-Willi syndrom (PWS) eller raskt innsettende fedme med hypoventilasjon, hypotalamus, autonom dysregulering, nevroendokrint tumorsyndrom (ROHHADNET)
- Vekttap >2 % de siste 3 månedene for pasienter i alderen ≥18 år eller >2 % reduksjon i BMI for pasienter i alderen 4 til <18 år
- Fedmekirurgi eller prosedyre innen de siste 2 årene
- Diagnose av alvorlige psykiatriske lidelser; noen selvmordstanker, forsøk eller oppførsel
- Nåværende, klinisk signifikant lunge-, hjerte-, metabolsk eller onkologisk sykdom
- Signifikante dermatologiske funn relatert til melanom eller pre-melanom hudlesjoner (unntatt ikke-invasiv basal eller plateepitel lesjon)
- Historie eller nær familiehistorie med hudkreft eller melanom
- Deltakelse i enhver klinisk utprøving med et forsøkslegemiddel/-enhet innen 3 måneder før første prøvedose
- Tidligere registrert i en klinisk studie som involverer setmelanotid eller tidligere eksponering for setmelanotid
- Manglende evne til å overholde en gang daglig (QD) injeksjonsregime
- Hvis kvinne, gravid og/eller ammer
- Pasienter med fedme som kan tilskrives andre genetiske eller syndromiske tilstander (f.eks. PPL [POMC, PCSK1, LEPR, samlet], BBS) før hypotalamusskaden.
- Hvis du mottar hormonbehandling, har dosen holdt seg stabil i minst 2 måneder før screening
Andre protokolldefinerte Inkluderings-/eksklusjonskriterier kan gjelde.
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Randomisert
- Intervensjonsmodell: Parallell tildeling
- Masking: Trippel
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
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Eksperimentell: Setemelanotid
Randomisert 2:1 (Setmelanotide: Placebo)
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Løsning for daglig subkutan injeksjon
Andre navn:
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Placebo komparator: Placebo
Randomisert 2:1 (Setmelanotide: Placebo)
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Placebo matchet med setmelanotid for daglig subkutan injeksjon
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Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Pivotal Cohort: Mean Percent Change From Baseline in Body Mass Index (BMI) After 52 Weeks on a Therapeutic Regimen
Tidsramme: 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.
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Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
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)
Tidsramme: 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)
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Pivotal Cohort: Percentage of Participants With ≥5% Reduction From Baseline in BMI
Tidsramme: 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.
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After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
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Pivotal Cohort: Change From Baseline in Weekly Average Daily Most Hunger Score in Participants ≥12 Years of Age
Tidsramme: 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.
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Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
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Pivotal Cohort: Percentage of Participants (≥12 Years of Age) With a ≥2-point Reduction From Baseline in the Weekly Average Daily Most Hunger Score
Tidsramme: 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.
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After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
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Pivotal Cohort: Mean Change From Baseline in the Weekly Average of the Symptoms of Hyperphagia Composite Score in Participants ≥12 Years of Age
Tidsramme: 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)
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Pivotal Cohort: Percentage of Participants With a ≥10% Reduction From Baseline in BMI
Tidsramme: 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)
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Pivotal Cohort: Percentage of Participants With a ≥10% Reduction From Baseline in Body Weight
Tidsramme: 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)
|
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Pivotal Cohort: Mean Percent Change From Baseline in Body Weight in Participants ≥18 Years
Tidsramme: 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)
|
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Pivotal Cohort: Mean Change From Baseline in BMI Z-Score in Participants <18 Years of Age
Tidsramme: 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)
|
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Pivotal Cohort: Mean Change From Baseline in Percent of BMI 95th Percentile in Participants <18 Years of Age
Tidsramme: 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
Tidsramme: 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)
|
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Pivotal Cohort: Percentage of Participants With BMI <30 kg/m^2 (Aged ≥18 Years) or <95th Percentile (Aged <18 Years) From Baseline
Tidsramme: 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)
|
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Pivotal Cohort: Mean Change From Baseline in Physical Functioning Score and Total Score on the Impact of Weight on Quality of Life-Lite (IWQOL)
Tidsramme: 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)
|
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Pivotal Cohort: Mean Change From Baseline in Total Score on the Impact of Weight on Quality of Life-Kids (IWQOL-Kids)
Tidsramme: 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)
|
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Pivotal Cohort: Mean Change From Baseline in Waist Circumference
Tidsramme: 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)
|
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Pivotal Cohort: Change From Baseline in Systolic and Diastolic Blood Pressure
Tidsramme: 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)
|
Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Etterforskere
- Studiestol: David Meeker, MD, Rhythm Pharmaceuticals, Inc.
Publikasjoner og nyttige lenker
Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.
Generelle publikasjoner
- Miller JL, van Santen HM, Phillips SA, Hamilton J, Aberle J, Sathyapalan T, Mohamed Z, McCormack SE, Shoemaker AH, Kelsey MM, Ghalib L, Stalla G, Thaker VV, Habiby R, Ode KL, Wabitsch M, Dattani M, Abuzzahab MJ, Abdullatif H, Morgan R, Stefater-Richards M, Aroda VR, Friedrich C, Han JC, Ono H, Nagasaki K, Tanaka T, Isojima T, Arima H, Scimia C, Yuan G, Muller HL, Roth CL; TRANSCEND Trial Group. Setmelanotide for the Treatment of Acquired Hypothalamic Obesity. N Engl J Med. 2026 Jul 9;395(2):138-150. doi: 10.1056/NEJMoa2512275.
- Roth CL, Phillips SA, McCormack SE, Ode KL, Kelsey MM, Liu J, Ervin C, Mallya UG, Norcross L, Miller JL. Quality of life improvements with setmelanotide treatment in acquired hypothalamic obesity: TRANSCEND trial interview results from US participants. Front Behav Neurosci. 2026 Jul 15;20:1830070. doi: 10.3389/fnbeh.2026.1830070. eCollection 2026.
Studierekorddatoer
Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.
Studer hoveddatoer
Studiestart (Faktiske)
26. april 2023
Primær fullføring (Faktiske)
18. mars 2025
Studiet fullført (Antatt)
16. april 2027
Datoer for studieregistrering
Først innsendt
1. mars 2023
Først innsendt som oppfylte QC-kriteriene
16. mars 2023
Først lagt ut (Faktiske)
20. mars 2023
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
10. august 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
16. juli 2026
Sist bekreftet
1. juli 2026
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
- Urogenitale sykdommer
- Sykdommer i det endokrine systemet
- Mannlige urogenitale sykdommer
- Kvinnelige urogenitale sykdommer
- Kvinnelige urogenitale sykdommer og graviditetskomplikasjoner
- Gonadal lidelser
- Medfødte abnormiteter
- Forstyrrelser i kjønnsutvikling
- Urogenitale abnormiteter
- Gonadal dysgenese
- Hypogonadisme
- Medfødte, arvelige og neonatale sykdommer og abnormiteter
- Seksuell infantilisme
- setmelanotid
Andre studie-ID-numre
- RM-493-040
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
NEI
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Ja
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .