Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

Randomized, double-blind, placebo-controlled study assessing the safety, tolerability, pharmacokinetics and pharmacodynamics of pitolisant after repeated oral ascending doses for 14 days, including the pitolisant effect on cardiac repolarization in healthy volunteers.

This research study is testing a new medicine called pitolisant. This medicine is designed to help people with narcolepsy, a condition that causes extreme daytime sleepiness and sometimes sudden muscle weakness (cataplexy). The main goals of this study are to check if pitolisant is safe and how well healthy male volunteers tolerate taking it repeatedly for 14 days. We are also looking at how the medicine works in the body and how it affects the heart. This is an early-stage study, meaning it's one of the first times this medicine is being tested in humans to gather basic information before it can be studied in people with narcolepsy.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Bioprojet Pharma

Enrolment target

Start

13 Jun 2023

Narcolepsy with cataplexy Narcolepsy

What is this study about?

This study is about a new medicine called pitolisant. It's hoped that this medicine could help people who have narcolepsy. Narcolepsy is a condition that makes you feel extremely sleepy during the day, even after a full night's sleep. Some people with narcolepsy also experience something called cataplexy, which is when strong emotions (like laughing or excitement) can suddenly make their muscles weak.

In this particular study, we're testing pitolisant in healthy volunteers, not yet in people with narcolepsy. This is a very early step in developing new medicines. The main things we want to find out are how safe the medicine is when taken for 14 days, whether people can easily tolerate it, how much of the medicine gets into the body, and how the body uses and gets rid of it. We're also making sure it doesn't cause any problems with the heart's electrical activity.

By carefully studying these things in healthy people first, researchers can gather important information about pitolisant. This helps them understand if it's worth continuing to test the medicine in larger studies with people who actually have narcolepsy. The results will help guide future research to see if pitolisant could become a helpful treatment option.

Key takeaways

This is an early study of a new medicine for narcolepsy.
It aims to check the safety and how the medicine works in healthy men.
Participants will take the medicine or a dummy pill for 14 days.
It's a 'double-blind' study, meaning no one knows who gets the real medicine.
Your involvement helps research into future narcolepsy treatments.

Who may be eligible?

To be able to take part in this study, there are some specific requirements. One important rule is that you must be at least 18 years old. There isn't an upper age limit mentioned, so older adults might be able to join as well, as long as they meet the other criteria.

Another key requirement for this particular study is that only men can participate. This means that women will not be able to join this study. All participants must also be in generally good health.

Before you can officially join, you'll have a health check-up to make sure you're suitable and that taking part would be safe for you. This often includes reviewing your medical history and having some tests.

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 a man?
Are you generally in good health?
Are you able to commit to 14 days of taking medicine and follow-up visits?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be taking a medicine called pitolisant, or a dummy pill (placebo), every day for 14 days. You wouldn't know which one you're taking, which helps to make the results more reliable. You would likely have regular visits to the study clinic during this time to check your health, measure the medicine in your body, and see how you're feeling. This would involve giving blood, having heart checks (like an ECG), and answering questions about your health. After the 14 days of taking the medicine, there would be follow-up appointments to make sure you are well and to complete all necessary assessments. The total time you would be involved in the study, including all visits and follow-ups, would be explained to you in detail.

Potential risks and benefits

Participating in research studies has potential benefits and risks. While there is no direct benefit to your health expected from taking part in this specific study (as you are healthy volunteers), you would be contributing valuable information that could help develop new treatments for people with narcolepsy. Potential risks include side effects from the study medicine, which could range from mild discomforts to more serious reactions. There's also the inconvenience of clinic visits and tests like blood draws. 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
Belgium

Common questions

What is narcolepsy?

Narcolepsy is a long-term condition that causes a person to feel extremely sleepy during the day and can sometimes lead to sudden muscle weakness called cataplexy.

Why are you studying healthy people first?

We study healthy people first to safely learn how the new medicine works in the body, how much ends up in the bloodstream, and if it causes any unexpected side effects, before testing it in patients.

What is a 'placebo'?

A placebo is a 'dummy' pill that looks like the study medicine but contains no active drug. Some participants receive it to help us see the real effects of the actual medicine.

Will I know if I'm taking the real medicine or the placebo?

No, neither you nor the study team will know if you're taking the real medicine or the placebo. This is called 'double-blind' and makes the study results more accurate.

Do I have to pay to join this study?

No, you do not have to pay to be in a clinical study. All study-related care, tests, and medicine are provided at no cost, and you may receive some payment for your time and travel.