A Double-blind, Placebo-controlled, Single and Multiple Ascending Dose Phase 1 Study of CMP-CPS-001 in Healthy Volunteers and Participants with Abnormal Heterozygous Ornithine Transcarbamylase (OTC) Genotype
This is a very early study looking at a new potential medicine called CMP-CPS-001. It's designed for people with urea cycle disorders, which are rare genetic conditions where the body has trouble getting rid of waste products properly. The main goal of this study is to check the safety of CMP-CPS-001 and see how it behaves in the body. Researchers will give different doses to healthy volunteers and people who have a specific genetic change linked to urea cycle disorder, even if they don't have symptoms. This helps them understand if the medicine is safe and how it might work before more advanced studies can happen. It's the first time this medicine is being given to humans.
At a glance
What is this study about?
This study is about a new medicine, CMP-CPS-001, for a group of rare genetic conditions called urea cycle disorders (UCDs). Our bodies naturally produce waste products, and UCDs mean the body struggles to break down a specific waste product called ammonia. Too much ammonia can be harmful. This new medicine aims to help the body process these waste products more effectively.
This is a 'Phase 1' study, which means it's one of the very first times this medicine is being tested in people. The main focus is to make sure the medicine is safe and to understand how it acts in the human body. Researchers will check for any side effects and how the medicine is absorbed, used, and cleared from the body. Because it's an early study, participants include healthy volunteers and people who have inherited a genetic change that can cause urea cycle disorder, even if they don't currently have symptoms.
The study involves giving participants either the new medicine or a dummy medicine (called a placebo) without them knowing which they receive. This helps researchers compare the effects accurately. Understanding the safety and initial effects of CMP-CPS-001 in this early stage is a crucial step towards potentially developing a new treatment for urea cycle disorders in the future.
Key takeaways
- This is an early-stage study checking safety and how a new medicine (CMP-CPS-001) works.
- It's for people with urea cycle disorders or specific genetic changes linked to them.
- Participants will receive either the study medicine or a dummy medicine (placebo).
- Close medical checks and monitoring will happen throughout the study.
- Your participation helps advance research for rare conditions.
- You can leave the study at any time if you change your mind.
Who may be eligible?
To join this study, you need to be an adult, aged 18 or older. Both men and women can take part.
The study is looking for two groups of people: healthy volunteers and people who have a specific genetic change related to urea cycle disorders, even if they feel well and don't have symptoms right now. This genetic change is found when doctors check your genes.
There might be other health checks or requirements that the study doctors will discuss with you to make sure it's safe for you to participate.
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 18 years old or older?
- Are you male or female?
- Have you been told you have a specific inherited genetic change related to urea cycle disorders (even if you're healthy)? OR Are you a healthy volunteer?
- Are you able to attend appointments at the study clinic?
What does participation involve?
If you decide to take part, you will be given either the study medicine (CMP-CPS-001) or a placebo (a dummy medicine) through a drip into your arm. You won't know which one you're getting because it's a 'double-blind' study. You will have several clinic visits over a period of time. These visits will involve various checks to monitor your health and how your body reacts to the medicine.
These checks typically include keeping an eye on your vital signs (like heart rate and blood pressure), doing electrical heart tests (ECGs), taking blood and urine samples for lab tests, and having physical examinations. You may also be asked to undergo specific tests to see how your body processes certain substances. The total time you'd be involved in the study, including follow-up, will be explained by the study team.
Potential risks and benefits
Locations (1)
- —UnverifiedNetherlands
Common questions
What are urea cycle disorders (UCDs)?
UCDs are rare conditions where the body can't remove harmful waste products (like ammonia) properly, which can be bad for your health.
What does 'Phase 1 study' mean?
It's an early study, mainly focused on checking if a new medicine is safe for people and how it works in the body for the first time.
Will I know if I'm getting the real medicine or a dummy one?
No, this is a 'double-blind' study, meaning neither you nor the study team will know which you receive until after the study is over.
Why include healthy people and those with a genetic change?
Comparing how the medicine works in both groups helps researchers understand its effects better and prepare for future studies in people with symptoms.
How long will I be involved if I join?
The total duration of your participation, including visits and follow-up, will be clearly explained by the study team before you decide.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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