Active not recruitingPHASE3INTERVENTIONAL

A Trial of Setmelanotide in Acquired Hypothalamic Obesity

This research study is looking at a new medicine, Setmelanotide, for children and adults aged 4 and older who have a specific type of weight problem called acquired hypothalamic obesity. This condition can cause significant weight gain. The study wants to find out how well Setmelanotide works to help people lose weight, feel less hungry, and improve their general well-being. Participants will receive daily injections of either Setmelanotide or a dummy medicine (placebo) for one year. Throughout this time, researchers will closely monitor their health and progress to understand both the benefits and any potential side effects of the treatment.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Rhythm Pharmaceuticals, Inc.

Enrolment target

143

Start

26 Apr 2023

Estimated completion

16 Apr 2027

Hypothalamic Obesity

Results

Results from this study

Posted May 2026

Results have been published for this study.

Primary outcome

Pivotal Cohort: Mean Percent Change From Baseline in Body Mass Index (BMI) After 52 Weeks on a Therapeutic Regimen

BMI was calculated as weight (kg)/height (m\^2). Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.

Full results on the registry

What is this study about?

This study is investigating a condition called acquired hypothalamic obesity. This is a specific type of weight gain that happens because of an injury or problem with a part of the brain called the hypothalamus. The hypothalamus plays a very important role in controlling things like hunger, thirst, energy levels, and body weight. When it's not working properly, it can lead to significant and often hard-to-manage weight gain.

The main goal of this particular trial is to see if a new medicine, Setmelanotide, can help people with this condition. Researchers want to understand if taking Setmelanotide can lead to weight loss, reduce feelings of hunger, and generally improve a person's quality of life. This is really important because finding effective treatments could make a big difference for those living with hypothalamic obesity.

To find this out, some people in the study will receive Setmelanotide, while others will receive a 'placebo'. A placebo looks just like the real medicine but contains no active drug. This allows researchers to compare the effects of the actual medicine with what happens when someone doesn't receive the active treatment, helping them to truly understand if Setmelanotide is making a difference. The study will last for 52 weeks, which is about one year.

Key takeaways

The study aims to test a new medicine, Setmelanotide, for hypothalamic obesity.
It will investigate effects on weight, hunger, and quality of life.
Participants will take daily injections for 52 weeks.
Some will receive the medicine, others a dummy treatment (placebo).
Both children (aged 4+) and adults can potentially join.
Regular health checks and assessments will be part of the study.

Who may be eligible?

To join this study, you would generally need to be at least 4 years old and have been diagnosed with acquired hypothalamic obesity. This means your weight gain is clearly linked to an injury to your hypothalamus, and your Body Mass Index (BMI) meets certain criteria—either 30 or higher for adults, or in the top 5% for your age and gender if you're under 18.

There are also some reasons why you might not be able to join. For example, if you have certain genetic conditions like Prader-Willi syndrome, or have had major weight loss surgery in the past two years, you wouldn't be suitable. The study also can't include people with severe mental health issues, a history of skin cancer or melanoma, or significant heart, lung, or other serious illnesses.

Also, if you're pregnant or breastfeeding, or if you've recently taken part in another drug trial, you wouldn't be able to participate. It's really important that you can give yourself a daily injection if you join the study. If you're using hormone replacement therapy, your dose must have been stable for at least two months.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 4 years old or older?
Do you have a diagnosis of acquired hypothalamic obesity?
Is your BMI at least 30 (if an adult) or in the top 5% for your age/sex (if under 18)?
Are you able to give yourself a daily injection?
Are you able to use reliable contraception throughout the study if you're a woman who could become pregnant?
Have you *not* had major weight loss surgery in the past two years?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be asked to give yourself an injection once a day with either the study medicine (Setmelanotide) or a dummy medicine (placebo). This will happen over a period of 52 weeks, which is about one year.

Throughout the study, you'll have regular visits to the clinic. During these visits, the research team will carry out various assessments. This includes checking your weight, how hungry you feel, and your general health. They will also collect blood samples and ask you questions about your quality of life to see how you are feeling overall.

Before you start, and during the study, you'll need to use reliable contraception if you're a woman of childbearing age, and continue to do so for 90 days after the study ends. The research team will explain all the procedures in detail and make sure you understand what's involved at each step.

Potential risks and benefits

Taking part in any medical study has potential advantages and disadvantages. The potential benefit of this study is that Setmelanotide might help reduce your weight and hunger, and improve your overall well-being if you have hypothalamic obesity. However, as it's a new medicine, there's also a chance it might not work for everyone, or you might experience side effects. These could be anything from minor discomfort from injections to more serious reactions, which the research team will discuss with you fully. It's very important to remember that you are free to stop participating in the study at any time, for any reason, without it affecting your medical care.

Locations (28)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

UAN Pediatric Endocrinology
Verified postcode
Birmingham, United States
Rady Children's Hospital
Verified postcode
San Diego, United States
Children's Hospital Colorado
Verified postcode
Aurora, United States
University of Florida
Verified postcode
Gainesville, United States
Ann and Robert H. Lurie Children's Hospital
Verified postcode
Chicago, United States
University of Iowa Stead Family Department of Pediatrics
Verified postcode
Iowa City, United States
Boston Children's Hospital
Verified postcode
Boston, United States
Brigham and Women's Hospital
Verified postcode
Boston, United States
Children's Minnesota
Verified postcode
Saint Paul, United States
Icahn School of Medicine at Mount Sinai
Verified postcode
New York, United States
Columbia University Irving Medical Center
Verified postcode
New York, United States
Ohio State Wexner Medical Center
Verified postcode
Columbus, United States

Common questions

What is hypothalamic obesity?

It's a type of weight gain caused by a problem in a part of the brain called the hypothalamus, which controls hunger and weight.

What is Setmelanotide?

It's a new medicine being tested to see if it can help people with hypothalamic obesity manage their weight and hunger.

Will I definitely get the new medicine?

No, you'll either receive the study medicine or a 'placebo' (a dummy medicine) through a daily injection.

How long does the study last?

The main treatment part of the study lasts for 52 weeks, which is about one year.

Can children join the study?

Yes, individuals aged 4 years and older can participate if they meet all the other criteria.