Hope for hypothalamic obesity: daily shot targets weight and hunger
NCT ID NCT06760546
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial tests whether a daily injection of setmelanotide can help people aged 4 and older with hypothalamic obesity lose weight and feel less hungry. Participants will receive either the drug or a placebo for 26 weeks. The study aims to see if the treatment leads to meaningful reductions in body mass index (BMI) and improves quality of life.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Setmelanotide (also known as Imcivree), a daily injection
- What this could lead to
- If it works, this could offer a new treatment option to help control weight and reduce hunger in people with hypothalamic obesity.
- What could go wrong
- This is a small, early-phase 3 trial with only 39 participants. The drug may not work better than placebo, and side effects from daily injections are possible.
Why investors are watching
Rhythm Pharmaceuticals is running a small phase 3 trial of setmelanotide, a daily injection, in patients with acquired hypothalamic obesity, a condition that causes severe weight gain after brain damage. For a small company, this readout matters because setmelanotide is already approved for other rare obesity conditions, and a positive result here could expand its use to a new patient group. The trial has only 39 planned participants, so the outcome will hinge on whether the drug clearly beats placebo on weight, hunger, and quality of life over 26 weeks.
If it works: If the trial succeeds, Rhythm could seek approval for setmelanotide in acquired hypothalamic obesity, adding a new revenue source and broadening the drug's market beyond its current rare-disease uses. A positive result would also validate the drug's mechanism in a different form of obesity, which could strengthen the company's overall pipeline story.
If it fails: If the trial fails or is delayed, Rhythm would lose the investment in this study and face a setback in expanding setmelanotide's approved uses. Clinical trials often fail, and with only 39 patients, a negative or unclear result would leave the company without a new indication and could hurt investor confidence in its growth plans.
AI-written from the trial record. Speculative, and not investment advice.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 39 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Sep 2025
- Expected to finish
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Mar 2028
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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4 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
11 sites in 2 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Birmingham Women and Children's Hospital NHS Trust
RECRUITINGBirmingham, West Midlands, B46NH, United Kingdom
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Children's Minnesota
RECRUITINGSaint Paul, Minnesota, 55102, United States
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Columbia University Irving Medical Center
RECRUITINGNew York, New York, 10032, United States
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Hull Royal Infirmary
RECRUITINGHull, HU32RW, United Kingdom
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Lurie Children's Hospital
RECRUITINGChicago, Illinois, 60611, United States
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Seattle Children's Research Institute
RECRUITINGSeattle, Washington, 98105, United States
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UCL Great Ormond Street Institute of Child Health
RECRUITINGLondon, Greater London, WC1N 1EH, United Kingdom
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University of Alabama
RECRUITINGBirmingham, Alabama, 35233, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Promising drug targets obesity caused by brain damage
- Can a diabetes drug tame obesity caused by brain tumor treatment?
- New pill targets hard-to-treat obesity after brain injury
- Could a diabetes drug help teens with rare obesity syndromes shed pounds?
- New weekly shot aims to curb obesity in rare genetic disorders
- New hope for hypothalamic obesity: Long-Term drug trial launches