Setmelanotide: Solution for daily subcutaneous injection
Placebo: Placebo matched to setmelanotide for daily subcutaneous injection
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.
Eligibility
Sex
ALL
Min age
4 Years
Max age
—
Healthy volunteers
No
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.
Primary outcome measure(s)
Mean % change in BMI — From Baseline after 26 weeks on a therapeutic regimen
Trial sites (11)
Facility
City
Region
Status
University of Alabama
Birmingham
Alabama
Recruiting
Children's Hospital Colorado
Aurora
Colorado
Recruiting
Lurie Children's Hospital
Chicago
Illinois
Recruiting
Boston Children's Hospital
Boston
Massachusetts
Recruiting
Children's Minnesota
Saint Paul
Minnesota
Recruiting
Columbia University Irving Medical Center
New York
New York
Recruiting
Children's Hospital of Philadelphia
Philadelphia
Pennsylvania
Recruiting
Seattle Children's Research Institute
Seattle
Washington
Recruiting
UCL Great Ormond Street Institute of Child Health
London
Greater London
Recruiting
Birmingham Women and Children's Hospital NHS Trust
Birmingham
West Midlands
Recruiting
Hull Royal Infirmary
Hull
United Kingdom
Recruiting
More Rhythm Pharmaceuticals, Inc. trials in the UK
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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