Ongoing, recruitingHuman Pharmacology (Phase I)- First administration to humansInterventional

Safety and tolerability of CTH120, first-in-human phase I study encompassing three parts: Single and Multiple Ascending doses and potential Food Interaction

This is an important early-stage medical study, often called a 'Phase I' trial. It's the very first time a new medicine, known as CTH120, will be given to people. The main goal is to check if CTH120 is safe for individuals with Fragile X syndrome and to see what side effects might occur. Researchers will start with a small dose and gradually increase it, observing how people react. They also want to understand how the body handles the medicine – for example, if taking it with food makes a difference. This kind of study doesn't aim to cure, but rather to gather crucial safety information before potentially moving on to bigger studies that look at how well the medicine works.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- First administration to humans

Sponsor

Connecta Therapeutics S.L.

Enrolment target

Start

17 May 2023

Fragile X syndrome

What is this study about?

This study is an early but very important step in developing a new treatment for Fragile X syndrome. It's called a 'Phase I' study, which means it’s the first time the new medicine, CTH120, will be given to human volunteers. Think of it like taking a new car out for its very first test drive – the main thing is to make sure it's safe to be on the road.

The main purpose of this study is to carefully check the safety of CTH120. This involves giving different groups of people different doses of the medicine – starting very low and slowly increasing it – to see what side effects, if any, occur. The researchers will be looking closely at how participants react to the medicine and how comfortable they feel with the dose given. They'll also explore how the medicine behaves in the body, including whether eating food at the same time changes how the medicine is absorbed.

It's important to remember that this study isn't designed to find out if the medicine *treats* Fragile X syndrome. Instead, it's about gathering key information on safety and how the body handles the medicine. If CTH120 proves to be safe in this study, it may then be tested in future studies to see if it can help improve symptoms of Fragile X syndrome. This foundational work is crucial before any medicine can move closer to being a widely available treatment.

Key takeaways

This is an early-stage study (Phase I) of a new medicine for Fragile X syndrome.
The main aim is to check the medicine's safety and how the body processes it.
Participants must be 18 years or older and can be male or female.
You will receive the new medicine, CTH120, and have regular health checks.
There might not be direct health benefits to you, as this is a safety study.
Participation helps scientists gather vital information for future treatments.

Who may be eligible?

To join this study, people generally need to be adults. This means you must be 18 years old or older. The study is open to both men and women.

While the main requirements are being an adult and having Fragile X syndrome, there will be other specific health checks and tests. These checks are to make sure the study will be safe for you and that the results they get are clear and accurate. For example, they might check your overall health, any other medicines you're taking, or if you have certain medical conditions.

The research team will explain all the requirements in detail. They will assess if the study is a good fit for you and if you meet all the necessary health and safety criteria.

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?
Do you have a diagnosis of Fragile X syndrome?
Are you able to attend regular study visits and undergo tests?
Are you open to taking an experimental medicine?
Are you able to follow detailed study instructions?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will have several visits to the study centre, which might include hospital stays. You will receive the study medicine, CTH120, either as a single dose or multiple doses over a period, depending on which part of the study you are in. Researchers will take blood and urine samples regularly to check how your body is handling the medicine and to monitor for any side effects. You might also have physical examinations, heart scans (like an ECG), and other tests to keep a close eye on your health.

Some parts of the study will involve seeing how food affects the medicine, so you might be asked to take the medicine at specific times, either with or without a meal. The exact number of visits, tests, and the total length of your involvement will vary depending on your specific group within the study, but all details will be clearly explained before you agree to participate. You'll receive full instructions at each step.

Potential risks and benefits

As with any new medicine, there's always a chance of side effects or risks when joining a study like this. These can range from mild, like a headache, to more serious, which is why researchers monitor you so closely. There might not be any direct health benefits for you personally from taking CTH120 in this early stage, as the main goal is safety. However, by taking part, you'll be contributing important information that could help develop future treatments for Fragile X syndrome. Importantly, you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

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

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Unverified
Spain

Common questions

What is Fragile X syndrome?

Fragile X syndrome is a genetic condition that causes a range of developmental problems including learning disabilities and cognitive impairment.

What does 'Phase I' mean?

Phase I means this is the very first time the new medicine will be given to people. The main aim is to check its safety and how the body handles it, not yet to see if it treats the condition.

Will I get better if I join?

This study is focused on safety, not on treating the condition. There's no guarantee you'll feel better, but your participation helps scientists learn more about the medicine safely.

Do I have to stay in a hospital?

Some parts of the study may require you to stay at the study centre or hospital for a short period, especially when you first receive the medicine, so you can be monitored closely.

What is CTH120?

CTH120 is the new medicine being tested in this study. It's an experimental treatment, meaning it hasn't been approved for use outside of research yet.