DRUG
Setmelanotide
Solution for daily subcutaneous injection
Status
Recruiting
Phase
Phase 3
Enrollment
39
Locations
11
Results
Not posted
Publications
0
Study summary
This is a sub-study of Study RM-493-040 (NCT05774756). The goal of this sub-study is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with congenital Hypothalamic Obesity (cHO). To determine how well setmelanotide works and how safe it is, patients with cHO will take a daily injection of either setmelanotide or placebo and complete trial assessments for up to 26 weeks on a therapeutic regimen.
Interventions
DRUG
Solution for daily subcutaneous injection
DRUG
Placebo matched to setmelanotide for daily subcutaneous injection
Timeline
First posted
Jan 7, 2025
Study start
Sep 23, 2025
Primary completion
Mar 13, 2027
Study completion
Mar 13, 2028
Results posted
Not reported
Registry updated
Feb 12, 2026
Outcomes
Mean % change in BMI
Time frame · From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients with ≥5% reduction in BMI in adult patients (≥18 years of age) or a BMI Z-score reduction of ≥0.2 points in pediatric patients (<18 years of age)
Time frame · From Baseline after 26 weeks on a therapeutic regimen
Mean change in the weekly average of the daily most hunger score in patients ≥12 years old
Time frame · From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients with a ≥2 point reduction in the weekly average of the daily most hunger score
Time frame · From Baseline after 26 weeks on a therapeutic regimen
Mean change in Symptoms of Hyperphagia total score
Time frame · From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients with a ≥10% reduction in BMI
Time frame · From Baseline after 26 weeks on a therapeutic regimen
Mean percent change in weight in patients ≥18 years
Time frame · From Baseline after 26 weeks on a therapeutic regimen
Mean BMI-z score and BMI percentile reduction in patients <18 (using combined height and weight to report BMI in kg/m2)
Time frame · From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients aged ≥4 to <18 years with ≥0.2-point reduction of BMI Z-score
Time frame · From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients with BMI <30 kg/m2 (patients aged ≥18 years) or <95th percentile (patients aged <18 years)
Time frame · From Baseline after 26 weeks on a therapeutic regimen
Mean change in physical functioning score and total score for the Impact of Weight on Quality of Life-Lite (IWQOL)
Time frame · From Baseline after 26 weeks on a therapeutic regimen
Eligibility
Key Inclusion Criteria: 1. Diagnosis of multiple pituitary hormone deficiency (MPHD), or septo-optic dysplasia (SOD), or optic nerve hypoplasia (ONH), or Childhood-onset combined pituitary hormone deficiency (CPHD), or Pituitary Stalk Interruption Syndrome (PSIS) with at least one pituitary deficiency AND a body mass index (BMI) of ≥30 kg/m2 for patients ≥18 years of age, or BMI ≥95th percentile for age and sex for patients 4 to \<18 years 2. Age 4 years and older 3. Weight gain associated with the hypothalamic injury and a BMI of ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and sex for patients 4 to \<18 years of age 4. Agree to use a highly effective form of contraception throughout the study and for 90 days after the study Key Exclusion Criteria: 1. Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET) 2. Weight loss \>2% in the previous 3 months for patients aged ≥18 years or \>2% reduction in BMI for patients aged 4 to \<18 years 3. Bariatric surgery or procedure within last 2 years 4. Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior 5. Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease 6. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion) 7. History or close family history of skin cancer or melanoma 8. Participation in any clinical trial with an investigational drug/device within 3 months prior to the first trial dose 9. Previously enrolled in a clinical trial involving setmelanotide or any previous exposure to setmelanotide 10. Inability to comply with once daily (QD) injection regimen 11. If female, pregnant and/or breastfeeding. 12. If receiving hormone replacement therapy, dose has remained stable for at least 2 months before Screening Other protocol defined Inclusion/Exclusion criteria may apply.
Study locations
United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
University of Alabama
Birmingham, Alabama, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Lurie Children's Hospital
Chicago, Illinois, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Children's Minnesota
Saint Paul, Minnesota, United States
Columbia University Irving Medical Center
New York, New York, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Seattle Children's Research Institute
Seattle, Washington, United States
UCL Great Ormond Street Institute of Child Health
London, Greater London, United Kingdom
Birmingham Women and Children's Hospital NHS Trust
Birmingham, West Midlands, United Kingdom
Hull Royal Infirmary
Hull, United Kingdom
Publications
No PMID-linked publications were present in this registry snapshot.
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