Study of KM-023 in Healthy Volunteers and Patients With Olmsted Syndrome.
This study is looking at a new medicine called KM-023. It aims to understand how safe it is, how the body uses and gets rid of it, and what effects it has. The study will happen in two main parts. First, healthy volunteers will take KM-023 to check its safety. Then, patients with Olmsted Syndrome will receive the medicine to see if it helps their condition and how safe it is for them. The researchers will also check if the medicine reaches the skin. Some participants will receive KM-023, while others will get a placebo (a dummy medicine with no active ingredients). This trial is important for developing new treatments for Olmsted Syndrome.
At a glance
What is this study about?
This research study is about a new medicine called KM-023, which is being investigated for a skin condition called Olmsted Syndrome. The main goal of the study is to find out if KM-023 is safe for people to take and to understand how the body handles it. This includes looking at how the medicine is absorbed, what happens to it inside the body, and how the body gets rid of it. Researchers also want to see if KM-023 has any helpful effects or any unwanted side effects.
The study will happen in stages. First, healthy volunteers will take the medicine. This helps researchers understand the basic safety of KM-023 before it is given to patients who are unwell. Once they have this information, the study will move on to people with Olmsted Syndrome to see if the medicine can help their condition. The study will also specifically look at whether KM-023 reaches the different layers of the skin, which is important for a skin medicine.
Some people in the study will receive KM-023, while others will be given a 'placebo'. A placebo looks exactly like the real medicine but contains no active ingredients. This is a standard way to test new medicines, as it helps researchers compare the effects of the active drug against no drug at all. The study will be conducted in both France and the United Kingdom, involving hospital stays for some parts and home treatment for others.
Key takeaways
- Tests a new medicine, KM-023, for Olmsted Syndrome.
- First checks safety in healthy adults aged 18-55.
- Then examines effects and safety in Olmsted Syndrome patients.
- Involves hospital stays and home treatment for some.
- Some participants receive a placebo (dummy medicine).
- Participation can last up to 18 weeks for patients.
Who may be eligible?
This study is open to both men and women. For the first part, healthy volunteers aged 18 to 55 are needed. You must not smoke or use nicotine products, and generally shouldn't be taking other medications, especially those that might affect your liver or kidneys. Your weight needs to be within a certain range (BMI of 18-32 and weighing between 50kg and 100kg).
For the second part, which involves patients, you must have a clear diagnosis of Olmsted Syndrome that has been confirmed by genetic testing and show visible signs of the skin condition on your palms and soles. Your health should have been stable for at least a month before joining. Everyone joining must be able to understand and sign a consent form, follow the study instructions, and meet specific contraception requirements. There are also some conditions that would prevent you from taking part, such as being a staff member directly involved in the study.
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 between 18 and 55 years old (for healthy volunteers)?
- Do you have a confirmed diagnosis of Olmsted Syndrome (for patients)?
- Do you not smoke or use nicotine products?
- Are you generally healthy and not taking most other medicines?
- Are you able to attend appointments and follow study instructions?
- Do you meet the weight requirements (50-100 kg and BMI 18-32)?
What does participation involve?
If you take part, your involvement will vary depending on which part of the study you are in. Healthy volunteers will have either short hospital stays of about 2 days for single doses or around 8 days for repeated doses. During these stays, you'll receive the study medicine (KM-023) or a placebo. Researchers will take blood samples regularly to check medicine levels and monitor your health carefully with various tests. Some healthy volunteers will take the medicine twice, once on an empty stomach and once with food, to see how food affects it.
Patients with Olmsted Syndrome will start with a hospital day where you'll get your first dose and learn how to take the medicine at home. You'll then take the medicine twice daily for 12 weeks. During this time, you'll have five follow-up visits, two of which will involve a hospital stay. Between visits, the study team will call you at home to check how you’re feeling. Your blood will be monitored, and safety and effectiveness tests will be performed throughout. The total time you'd be involved can range from about 36 days for a single dose in healthy volunteers to up to 18 weeks for patients with Olmsted Syndrome, including initial checks and a final follow-up.
Potential risks and benefits
Locations (4)
- Northwestern University, Department of DermatologyVerified postcodeChicago, United States· Recruiting
- Paris Descartes Necker Cochin, Hôpital Necker-Enfants Malades Bat Imagine RDCVerified postcodeParis, France· Recruiting
- BiotrialVerified postcodeRennes, France· Recruiting
- Royal London HospitalVerified postcodeLondon, United Kingdom· Recruiting
Common questions
What is Olmsted Syndrome?
Olmsted Syndrome is a rare genetic skin condition that mainly affects the palms of the hands and soles of the feet, causing them to thicken. It can also affect other parts of the body.
What is a 'placebo'?
A placebo is a 'dummy' medicine that looks like the real thing but doesn't contain any active ingredients. It's used so researchers can compare the effects of the actual medicine against not having any medicine.
Will I have to stay in hospital?
Depending on which part of the study you join, you might have short hospital stays (a few days for healthy volunteers) or regular hospital visits and home treatment (for Olmsted Syndrome patients).
Who can take part in this study?
The study is for healthy volunteers (adults aged 18-55) first, and then for patients with a confirmed diagnosis of Olmsted Syndrome. Specific health checks and conditions apply to both groups.
What happens after the study ends?
After your planned participation period, there will be a follow-up period to ensure your health is fully monitored.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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