Tests setmelanotide against placebo in people with congenital hypothalamic obesity
Tests setmelanotide injections against placebo in patients aged 4 years and older.
Plain-language title generated from the registry record
Registry title: A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)
Key facts
- Where
- Birmingham, Hull, and London, United KingdomSee the 3 sites
- Phase
- Phase 3
Where in United Kingdom
- Birmingham Women and Children's Hospital NHS TrustBirmingham, United KingdomTaking people
- Hull Royal InfirmaryHull, United KingdomTaking people
- UCL Great Ormond Street Institute of Child HealthLondon, United KingdomTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Rhythm Pharmaceuticals, Inc.
- Trial code
- NCT06760546
- Record updated
- 12 February 2026
- Original description
This is a sub-study of Study RM-493-040 (NCT05774756).Read full original descriptionShow less
A Phase 3, Double Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Setmelanotide in Patients With Acquired Hypothalamic Obesity
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.
Inclusion: 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; Age 4 years and older; 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; Agree to use a highly effective form of contraception throughout the study and for 90 days after the study.
Exclusion: 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; Bariatric surgery or procedure within last 2 years; Participation in any clinical trial with an investigational drug/device within 3 months prior to the first trial dose; Previously enrolled in a clinical trial involving setmelanotide or any previous exposure to setmelanotide; If receiving hormone replacement therapy, dose has remained stable for at least 2 months before Screening; Other protocol defined Inclusion/Exclusion criteria may apply; Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET); Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior; Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease; History or close family history of skin cancer or melanoma; Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion); Inability to comply with once daily (QD) injection regimen; If female, pregnant and/or breastfeeding.