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NCT06760546PHASE3Recruiting

A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)

Rhythm Pharmaceuticals, Inc.

Start Date

9/23/2025

Completion Date

3/13/2028

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 Criteria

Age Range: 4 years to No maximum

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.

Interventions

DRUG

Setmelanotide

DRUG

Placebo

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Conditions

Hypothalamic ObesityMultiple Pituitary Hormone Deficiency Genetic FormSepto-Optic DysplasiaOptic Nerve HypoplasiaChildhood-onset Combined Pituitary Hormone DeficiencyPituitary Stalk Interruption Syndrome

Locations

University of Alabama

Birmingham, Alabama 35233

United States

Children's Hospital Colorado

Aurora, Colorado 80045

United States

Lurie Children's Hospital

Chicago, Illinois 60611

United States

Boston Children's Hospital

Boston, Massachusetts 02115

United States

Children's Minnesota

Saint Paul, Minnesota 55102

United States

Columbia University Irving Medical Center

New York, New York 10032

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

Seattle Children's Research Institute

Seattle, Washington 98105

United States

UCL Great Ormond Street Institute of Child Health

London, Greater London WC1N 1EH

United Kingdom

Birmingham Women and Children's Hospital NHS Trust

Birmingham, West Midlands B46NH

United Kingdom

Hull Royal Infirmary

Hull, HU32RW

United Kingdom