A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)
Recruiting · NCT06760546 · Interventional (participants receive a specific treatment) · Lead sponsor: Rhythm Pharmaceuticals, Inc.
View the official record on ClinicalTrials.gov →Interventions studied
PlaceboSetmelanotide
What this trial is about
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.
Who can take part
Age range
4 Years and older
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 3
Study type
Interventional (participants receive a specific treatment)
Inclusion criteria
- 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
- Key
Exclusion criteria
- Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET)
- 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
- Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior
- Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease
- Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
- History or close family history of skin cancer or melanoma
- 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
- Inability to comply with once daily (QD) injection regimen
- If female, pregnant and/or breastfeeding.
- If receiving hormone replacement therapy, dose has remained stable for at least 2 months before Screening
- ther protocol defined Inclusion/Exclusion criteria may apply.
Where it is running
11 locations listed across 8 US states.
- 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
- Hull Royal Infirmary - Hull, United Kingdom
- 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