A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)
This research study is for children and adults aged 4 and above who have a specific health condition called congenital Hypothalamic Obesity (cHO). This type of obesity is linked to issues with the hypothalamus, a part of the brain that controls hunger and hormones. We want to find out if a new medicine called Setmelanotide can help patients manage their weight, reduce feelings of hunger, and improve their overall quality of life. Participants will receive daily injections of either Setmelanotide or a placebo (a dummy medicine) for up to 26 weeks. It’s important to understand how effective and safe this new treatment is.
At a glance
What is this study about?
This study is particularly for individuals who have a condition called congenital Hypothalamic Obesity (cHO). This isn't typical obesity and is caused by problems with a small but very important part of the brain called the hypothalamus, which helps control basic functions like hunger and hormone levels. These problems might stem from conditions present from birth, such as Septo-Optic Dysplasia, Optic Nerve Hypoplasia, or issues with the pituitary gland, which is closely linked to the hypothalamus.
The main aim of this research is to test a new medicine called Setmelanotide. We want to see if it can help people with cHO lose weight, feel less hungry, and ultimately improve their day-to-day lives. People with cHO often struggle significantly with weight management and constant hunger, which can have a big impact on their health and well-being. By taking part, you could help us understand if Setmelanotide can make a real difference for those affected.
The study involves comparing Setmelanotide to a 'placebo'. A placebo looks exactly like the actual medicine but doesn't contain any active drug. This is a standard and important way to test new medicines, as it helps researchers figure out if any changes experienced by participants are genuinely due to the medicine or other factors. Both the dose and safety of Setmelanotide will be carefully monitored throughout the study.
Key takeaways
- The study investigates a new medicine, Setmelanotide, for congenital Hypothalamic Obesity (cHO).
- It aims to improve weight loss, reduce hunger, and enhance quality of life.
- Participants aged 4 and over will receive daily injections of Setmelanotide or a placebo.
- The study lasts up to 26 weeks with regular health checks.
- Specific criteria determine who can participate, including having a diagnosis linked to cHO and a certain BMI.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
This study is looking for children and adults, aged 4 and above, who have been diagnosed with certain conditions like Septo-Optic Dysplasia, Optic Nerve Hypoplasia, or other specific pituitary hormone issues from birth, and also have a high Body Mass Index (BMI). For adults (18 and over), this means a BMI of 30 or higher. For children aged 4 to under 18, it means a BMI that is at or above the 95th percentile for their age and sex. Essentially, we are looking for individuals whose significant weight gain is linked to these specific brain-related conditions.
There are some reasons why you might not be able to join. For example, if you have certain other genetic conditions like Prader-Willi syndrome, or if you've had significant weight loss recently. You also can't have had weight-loss surgery in the last two years. Certain significant long-term health problems (like severe heart, lung, or mental health conditions) or a history of skin cancer would also mean you couldn't take part. It's also important that you haven't been in another drug trial recently or previously taken Setmelanotide.
Women who are pregnant or breastfeeding cannot participate. If you're able to get pregnant, you'll need to agree to use effective contraception during the entire study and for three months afterwards. If you’re on hormone replacement therapy, your doses need to have been stable for at least 2 months before you start the study.
- Are you 4 years old or older?
- Have you been diagnosed with a condition like Septo-Optic Dysplasia or other specific pituitary problems from birth, along with significant weight gain?
- For adults (18+), is your BMI 30 or higher? For children (4 to <18), is your BMI at or above the 95th percentile for your age and sex?
- Are you able to manage daily injections?
- Are you not pregnant or breastfeeding, and if able to become pregnant, willing to use effective contraception?
- Have you not had weight-loss surgery in the last two years and do not have certain other serious health conditions or a history of skin cancer?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you will be asked to have daily injections. You will either receive the study medicine, Setmelanotide, or a placebo (a dummy medicine without any active drug). You won't know which one you are receiving, nor will the study team. You will be asked to continue these daily injections and take part in study assessments for up to 26 weeks, which is about six months.
During this time, you'll have regular visits to the clinic so the study team can monitor your health, check your weight, assess your hunger levels, and discuss your overall quality of life. These visits will also involve various tests, as needed, to make sure the medicine is safe and that you are responding well. There will also be a follow-up period after your 26 weeks on the treatment.
Potential risks and benefits
Locations (11)
- University of AlabamaBirmingham, United States· Recruiting
- Children's Hospital ColoradoAurora, United States· Recruiting
- Lurie Children's HospitalChicago, United States· Recruiting
- Boston Children's HospitalBoston, United States· Recruiting
- Children's MinnesotaSaint Paul, United States· Recruiting
- Columbia University Irving Medical CenterNew York, United States· Recruiting
- Children's Hospital of PhiladelphiaPhiladelphia, United States· Recruiting
- Seattle Children's Research InstituteSeattle, United States· Recruiting
- UCL Great Ormond Street Institute of Child HealthLondon, United Kingdom· Recruiting
- Birmingham Women and Children's Hospital NHS TrustBirmingham, United Kingdom· Recruiting
- Hull Royal InfirmaryHull, United Kingdom· Recruiting
Common questions
What is Hypothalamic Obesity (cHO)?
It's a type of obesity linked to problems with the hypothalamus, a part of the brain that controls hunger and hormones, often due to conditions present from birth.
What is Setmelanotide?
It's a new medicine being tested in this study to see if it can help people with cHO manage their weight and reduce hunger.
What is a placebo?
A placebo is a 'dummy' medicine that looks like the active drug but contains no active ingredients. It helps researchers fairly test the effects of the actual medicine.
How long does the study last?
Participants will take the daily injections and have assessments for up to 26 weeks, which is about six months.
Will I know if I'm getting the actual medicine or the placebo?
Neither you nor the study team will know whether you are receiving Setmelanotide or the placebo during the study.
How to find out more
Rhythm Clinical Trials
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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