Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

Double blind, multicentre, randomized, placebo-controlled trial to evaluate safety and efficacy of pitolisant in children from 6 to less than 18 years with narcolepsy with/without cataplexy, followed by a prolonged open-label period

This study is investigating a new medicine called pitolisant for children and teenagers between 6 and 17 years old who have narcolepsy. Narcolepsy can cause extreme daytime sleepiness and sometimes sudden temporary muscle weakness, known as cataplexy. The main goal of this study is to find out if pitolisant can help reduce these symptoms and improve how children and teenagers feel. Researchers will also be checking carefully to make sure the medicine is safe. Participants will be given either pitolisant or a dummy pill (placebo) without knowing which one they have, to fairly compare the effects. After this initial period, all participants will have the chance to receive pitolisant for a longer time.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Bioprojet Pharma

Enrolment target

Start

05 Dec 2024

Narcolepsy

What is this study about?

This research study is about a medicine called pitolisant, which is being tested for children and teenagers who have narcolepsy. Narcolepsy is a long-term condition that mainly affects how well you sleep and stay awake. People with narcolepsy often feel very sleepy during the day, even after a full night's rest. Some also experience 'cataplexy,' which is a sudden, brief loss of muscle control, often triggered by strong emotions like laughter or surprise.

The study aims to see if pitolisant can help children and teenagers aged 6 to 17 manage their narcolepsy symptoms, specifically reducing their excessive daytime sleepiness and cataplexy attacks. The researchers will also be very carefully monitoring for any side effects to make sure the medicine is safe for young people.

To get clear results, some participants will receive the active medicine (pitolisant) and others will receive a 'placebo,' which looks exactly like the medicine but contains no active drug. Neither the participant nor their doctor will know who is getting which. This helps researchers understand the true effects of the medicine. Later on, everyone in the study will have the chance to take the pitolisant medicine for a longer period.

Key takeaways

This study is testing a medicine called pitolisant for children and teenagers with narcolepsy.
It aims to see if the medicine helps with daytime sleepiness and muscle weakness (cataplexy).
Some participants will receive the active medicine, others a dummy pill, initially.
The study also carefully checks for side effects to ensure the medicine is safe.
Participation includes regular clinic visits, questionnaires, and possibly sleep tests.

Who may be eligible?

This study is looking for children and teenagers with narcolepsy.

If you have narcolepsy and are aged between 6 and 17 years old (up to, but not including your 18th birthday), you might be able to take part.

Both boys and girls can be included in this 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.

Do you have narcolepsy?
Are you between 6 and 17 years old?
Are you currently experiencing problems with daytime sleepiness or cataplexy?
Are you able to attend regular clinic appointments?

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve several visits to a clinic over a period of time. Initially, for about eight weeks, you would take either the study medicine (pitolisant) or a placebo (a dummy pill). You wouldn't know which one you were taking, and neither would your doctor. After this, if you're still suitable, you would have the option to continue taking the active study medicine for a much longer period, known as the open-label phase, which could last for many months.

Throughout the study, you would undergo various assessments. These include filling out questionnaires about your sleepiness and how often you experience cataplexy, as well as keeping a diary about your sleep and any cataplexy episodes. You might also have some tests to measure how alert you are during the day. Your parent or guardian, and possibly your teacher, might also be asked to help with some of these observations.

There will also be follow-up phone calls after the main treatment period to check on your well-being. The exact number of visits and their timing will be explained fully by the study team.

Potential risks and benefits

Participating in a clinical trial like this could offer potential benefits, such as gaining access to a new treatment that might help manage your narcolepsy symptoms. You will also receive careful medical monitoring throughout the study. However, there are also potential risks, including the possibility of experiencing side effects from the medication or the assessments. There's also a chance you might receive the placebo and not the active drug during the first part of the study, meaning your symptoms might not improve. Remember, taking part in any clinical trial is always voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

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

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Unverified
Italy
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Unverified
France

Common questions

What is narcolepsy?

Narcolepsy is a long-term brain condition that causes you to feel extremely sleepy during the day, even if you’ve had enough sleep at night. You might also have sudden attacks of muscle weakness called cataplexy.

What is pitolisant?

Pitolisant is a medicine being studied to see if it can help improve symptoms like daytime sleepiness and cataplexy in people with narcolepsy.

What does 'double-blind' mean?

Double-blind means that neither you (nor your parents) nor the doctors and nurses involved in the study will know whether you are receiving the active medicine or a dummy pill (placebo) during the first part of the study. This helps make the study fair and accurate.

Will I get the actual medicine?

In the first part of the study, you might get the active medicine or a placebo (a dummy pill). However, after this initial stage, if you're still suitable, you'll have the option to receive the active medicine (pitolisant) for a longer period.

How long will the study last?

The first part of the study where you might receive either the medicine or the placebo will last for about eight weeks. After that, there's an option for a longer period where everyone receives the active medicine, which could last for many months.