Setmelanotide for congenital hypothalamic obesity

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3 sub-study enrolls people aged 4 years and older with congenital hypothalamic obesity secondary to conditions such as multiple pituitary hormone deficiency, septo-optic dysplasia, optic nerve hypoplasia, childhood-onset combined pituitary hormone deficiency, or pituitary stalk interruption syndrome. Participants will receive a daily injection of either setmelanotide or placebo and complete scheduled assessments for up to 26 weeks to measure weight change, hunger, and quality of life. Safety and tolerability will be monitored throughout the treatment period. The trial is run as a sub-study of a larger setmelanotide program and is conducted at three U.S. pediatric/adult endocrine centers.

Why it matters

Who can join: the full rules

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.

Where it's running

• University of Alabama
  Birmingham, Alabama, United States Recruiting
• Children's Hospital Colorado
  Aurora, Colorado, United States Recruiting
• Lurie Children's Hospital
  Chicago, Illinois, United States Recruiting
• Boston Children's Hospital
  Boston, Massachusetts, United States Recruiting
• Children's Minnesota
  Saint Paul, Minnesota, United States Recruiting
• Columbia University Irving Medical Center
  New York, New York, United States Recruiting

Study contacts

• Study coordinator: Rhythm Clinical Trials
• Email: clinicaltrials@rhythmtx.com
• Phone: (857) 264-4280

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Using Tirzepatide to treat obesity in young adults with Prader-Willi Syndrome and hypothalamic obesity

  This study is testing if a weight loss medication called Tirzepatide can help young adults with Prader-Willi Syndrome and hypothalamic obesity lose weight and improve their health.

  Recruiting at 3 sites Phase 4 Seattle Children's Hospital · Saint Paul, Minnesota and 2 other locations · NCT06901245

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.