Active not recruitingPHASE3INTERVENTIONAL

EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway

The EMANATE study is investigating a new medication called Setmelanotide for individuals aged 6 to 65 living with obesity due to particular genetic changes. Specifically, it focuses on people who have certain variations in genes like POMC, PCSK1, LEPR, SRC1, or SH2B1, all of which are part of a system in the body called the MC4R pathway, known to affect appetite and weight. This is a Phase 3 trial, meaning the medicine has already been tested in smaller groups and is now being studied more widely. Participants will be randomly assigned to receive either Setmelanotide or a placebo (a dummy medicine) in a 'double-blind' way, so neither they nor their doctors will know which treatment they are getting. The main goal is to understand if Setmelanotide is safe and effective in helping people manage their weight and appetite when their obesity is caused by these specific genetic factors.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Rhythm Pharmaceuticals, Inc.

Enrolment target

296

Start

10 Dec 2021

Estimated completion

31 Dec 2026

Obesity Genetic Obesity

What is this study about?

This research study, called EMANATE, is exploring a new medicine named Setmelanotide. It's designed for people who have obesity that is linked to specific changes in their genes. Our bodies have a complex system, often called the MC4R pathway, which plays a big role in controlling our appetite and how our bodies use energy. Sometimes, changes or 'variants' in certain genes within this pathway can make it harder for the body to manage hunger, leading to obesity.

This study is focusing on individuals who have particular genetic variants, such as those in the POMC, PCSK1, LEPR, SRC1, or SH2B1 genes. These genetic differences can disrupt the normal signals that tell our brains when we're full. Setmelanotide aims to help restore some of these signals. By understanding how this medicine works for people with these specific genetic causes of obesity, researchers hope to find new ways to help improve their health and well-being.

The trial is structured into several 'sub-studies,' each looking at a different set of gene variants, but all studies share the same goals: to see if Setmelanotide is safe, and if it can help people manage their weight and reduce their appetite. Taking part in such a study contributes valuable knowledge that could one day benefit many others living with genetic forms of obesity.

Key takeaways

The study explores a new medicine, Setmelanotide, for specific genetic forms of obesity.
It's for people aged 6 to 65 with obesity linked to certain gene changes (POMC, PCSK1, LEPR, SRC1, or SH2B1).
Participants will receive either Setmelanotide or a placebo, without knowing which one.
Regular clinic visits and health monitoring are part of taking part.
Participation could offer access to a new treatment and contribute to medical knowledge.

Who may be eligible?

To be considered for this study, you would need to be between 6 and 65 years old and have obesity. Importantly, your obesity must be linked to specific genetic changes in genes like POMC, PCSK1, LEPR, SRC1, or SH2B1. You or your guardian should be able to understand what the study involves and be willing to participate.

There are also some things that would mean you couldn't take part. For example, if you've had recent significant weight loss, bariatric surgery, or certain serious health conditions such as significant heart, lung, or kidney disease. Also, if you have a history of melanoma (a type of skin cancer) or certain psychiatric conditions, you wouldn't be able to join.

The research team will carry out a thorough check to make sure the study is right for you and that taking part would be safe. They will also confirm your genetic makeup to ensure it matches the study's requirements.

Quick self-check

Are you between 6 and 65 years old?
Have you been diagnosed with obesity?
Do you know if your obesity is linked to a specific genetic change (e.g., in POMC, LEPR, SH2B1 genes)?
Have you experienced obesity since childhood?
Are you able to attend regular clinic visits and follow study instructions?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you are eligible and choose to take part, you will be assigned by chance (like flipping a coin) to receive either the study medicine, Setmelanotide, or a placebo (a dummy medicine with no active ingredients). Neither you nor your study doctor will know which one you are receiving – this is called a 'double-blind' approach and helps ensure the results are fair.

The study involves regular visits to the clinic for check-ups, blood tests, and other assessments to monitor your health and how you are responding to the treatment. You'll be given the study medication to take as instructed. The total duration of your participation in the study, including all follow-up, will be explained in full detail by the study team, but it will involve several months of regular engagement.

Potential risks and benefits

Participating in a clinical trial may offer potential benefits, such as access to a new medicine before it's widely available and receiving close medical monitoring. However, there are also potential risks, including side effects from the medication, which will be fully explained to you. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (56)

Phoenix Children's Hospital
Phoenix, United States
HonorHealth Bariatric Center
Scottsdale, United States
Children's Hospital Los Angeles
Los Angeles, United States
Children's Hospital Colorado
Aurora, United States
Yale Center for Clinical Investigation
New Haven, United States
Advent Health
Altamonte Springs, United States
InQuest Medical Research
Suwanee, United States
UMASS Memorial Medical Center
Worcester, United States
University of Michigan
Ann Arbor, United States
Metro Detroit Endocrinology Center
Dearborn, United States
Mayo Clinic
Rochester, United States
St. Luke's Hospital
Chesterfield, United States

+44 more sites — see the official record for the full list.

Common questions

What is Setmelanotide?

Setmelanotide is a new medicine being tested to see if it can help people with obesity caused by specific gene changes affecting appetite control.

What does 'genetic obesity' mean?

Genetic obesity means that your obesity is linked to specific changes or 'faults' in your genes that affect how your body manages hunger and weight.

Will I know if I'm getting the real medicine or a dummy pill?

For this study, neither you nor your doctor will know if you are receiving Setmelanotide or a placebo (dummy medicine). This helps ensure unbiased results.

How long will the study last?

The study involves regular clinic visits over several months. The exact duration for you will be discussed in detail by the study team if you are eligible.

Can children take part in this study?

Yes, children aged 6 and above can take part, provided they meet all other study requirements and their parent or guardian gives permission.