Current partner codePEPTIDESDE
NCT06760546·Phase 3·INTERVENTIONAL

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

Status

Recruiting

Phase

Phase 3

Enrollment

39

Locations

11

Results

Not posted

Publications

0

Study summary

What the protocol is testing.

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

Treatment arms and agents.

DRUG

Setmelanotide

Solution for daily subcutaneous injection

DRUG

Placebo

Placebo matched to setmelanotide for daily subcutaneous injection

Timeline

From registration to results.

  1. First posted

    Jan 7, 2025

  2. Study start

    Sep 23, 2025

  3. Primary completion

    Mar 13, 2027

  4. Study completion

    Mar 13, 2028

  5. Results posted

    Not reported

  6. Registry updated

    Feb 12, 2026

Outcomes

What the study measures.

Primary outcomes

Mean % change in BMI

Time frame · From Baseline after 26 weeks on a therapeutic regimen

Secondary outcomes

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

Who can take part.

Minimum age
4 Years
Maximum age
Not reported
Sex
ALL
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.

Study locations

11 registered sites.

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

Results and literature.

No PMID-linked publications were present in this registry snapshot.

Primary links

Continue at the source.

Related trials

More studies on Setmelanotide.

Related PeptideStat pages

Put the record in context.

Research pages describe evidence. Vendor pages, where available, describe independently tracked research-product listings and are not clinical recommendations.