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Trial · Interventional

RecruitingPhase 3Registry data

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

NCTNCT06760546 (opens ClinicalTrials.gov in a new tab)Other idRM-493-040-A
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Recruiting
Phase
Phase 3
Enrollment
39 participants (estimated)
Sponsor
Rhythm Pharmaceuticals, Inc. · Industry
Design
Randomized · Triple masking
Countries
2 countries

United States, United Kingdom

Start
23 Sept 2025
Primary completion
13 Mar 2027 (estimated)
Completion
13 Mar 2028 (estimated)
First posted
7 Jan 2025
Last update posted
12 Feb 2026
Conditions
Hypothalamic Obesity · Multiple Pituitary Hormone Deficiency Genetic Form · Septo-Optic Dysplasia · Optic Nerve Hypoplasia · Childhood-onset Combined Pituitary Hormone Deficiency · Pituitary Stalk Interruption Syndrome

Timeline

1 trial · 1 ongoing

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20252026202720282029
  1. Ph 3

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

    NCT06760546

    23 Sept 2025 to est. 13 Mar 2028

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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

  • SetmelanotideDrug
  • PlaceboDrug

Arms2

  • Experimental

    Setemelanotide

    Randomized 2:1 (Setmelanotide: Placebo)

  • Placebo comparator

    Placebo

    Randomized 2:1 (Setmelanotide: Placebo)

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Mean % change in BMI

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

Secondary outcomes(11)

  1. 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

  2. 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

  3. 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

  4. Mean change in Symptoms of Hyperphagia total score

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

  5. Proportion of patients with a ≥10% reduction in BMI

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

  6. Mean percent change in weight in patients ≥18 years

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

  7. 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

  8. 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

  9. 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

  10. 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

  11. Safety and tolerability of setmelanotide compared to placebo assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)

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

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

A trial that is still running, or that stopped early, may post results later.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 4 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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.

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