Study on the Safety and Effectiveness of Pitolisant for Children Aged 6-17 with Narcolepsy, with or without Cataplexy

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What is this study about?

This clinical trial is focused on studying narcolepsy, a sleep disorder that causes excessive daytime sleepiness and sudden loss of muscle control, known as cataplexy. The trial is testing a medication called pitolisant, which is available in film-coated tablets. The purpose of the study is to evaluate the safety and effectiveness of pitolisant in children aged 6 to less than 18 years who have narcolepsy, with or without cataplexy.

Participants in the study will be randomly assigned to receive either pitolisant or a placebo. The study will begin with a double-blind period, where neither the participants nor the researchers know who is receiving the actual medication or the placebo. This will be followed by an open-label period, where all participants will receive pitolisant. The study will last for a total of eight weeks, during which the effects of the medication on symptoms like excessive daytime sleepiness and the frequency of cataplexy episodes will be monitored.

The trial aims to observe any improvements in the symptoms of narcolepsy and to ensure the medication is well-tolerated by the participants. The study will also monitor any side effects and changes in health indicators such as heart rate and blood tests. This research is important for understanding how pitolisant can help manage narcolepsy in children and improve their quality of life.

1 initial visit and assessment

Upon joining the clinical trial, the first step involves an initial visit where a detailed assessment is conducted. This includes confirming the diagnosis of narcolepsy with or without cataplexy using specific tests if they have not been performed in the last 12 months. These tests include polysomnography and the Multiple Sleep Latency Test.

During this visit, the patient and their guardians will review and sign the informed consent form, indicating their understanding and willingness to participate in the study.

2 baseline period

The baseline period involves two visits, referred to as V1 and V2. During this time, the patient’s symptoms are measured using the Ullanlinna Narcolepsy Scale (UNS) and the Paediatric Daytime Sleepiness Scale (PDSS). These scales help to establish a baseline for the patient’s symptoms before starting the trial medication.

The average score from these two visits will be used to compare changes in symptoms throughout the trial.

3 double-blind treatment phase

In this phase, the patient will receive either the trial medication, pitolisant, or a placebo. The medication is administered orally in the form of film-coated tablets. The dosage of pitolisant can vary between 5 mg and 40 mg per day.

This phase is designed to evaluate the effectiveness of pitolisant in reducing symptoms of narcolepsy, such as excessive daytime sleepiness and cataplexy episodes. The patient’s progress will be monitored through regular visits and assessments.

4 end of double-blind phase

At the end of the double-blind phase, the patient’s symptoms will be reassessed using the same scales as during the baseline period. This includes the UNS and PDSS, among others.

The results will be compared to the baseline to determine any changes in the patient’s condition.

5 open-label treatment phase

Following the double-blind phase, the patient will enter an open-label treatment phase where all participants receive pitolisant. The dosage during this phase can range from 5 mg to 40 mg per day, adjusted based on the patient’s response and tolerance.

This phase allows for further evaluation of the medication’s long-term safety and effectiveness.

6 final assessment and follow-up

At the conclusion of the open-label phase, a final assessment will be conducted. This includes a comprehensive evaluation of the patient’s symptoms, any side effects experienced, and overall health.

The results from this assessment will contribute to the overall findings of the clinical trial, helping to determine the safety and efficacy of pitolisant for treating narcolepsy in children.

Who Can Join the Study?

Children aged 6 to less than 18 years old who have been diagnosed with narcolepsy, with or without cataplexy. Narcolepsy is a sleep disorder that causes excessive daytime sleepiness. Cataplexy is a sudden loss of muscle strength.
The diagnosis must be confirmed with tests called polysomnography and Multiple Sleep Latency Test. Polysomnography is a sleep study that records brain waves, oxygen levels, heart rate, and breathing. The Multiple Sleep Latency Test measures how quickly a person falls asleep in a quiet environment during the day.
A PDSS score of 15 or higher during the initial assessment period. PDSS stands for Pediatric Daytime Sleepiness Scale, which measures sleepiness in children.
Participants must not be taking any non-approved medications, especially psychostimulant treatments, from the start of the study. Psychostimulants are drugs that increase activity in the brain and are often used to treat attention deficit hyperactivity disorder (ADHD).
Both the child and their parents must agree to participate in the study and sign a consent form before starting any study procedures.
The child must have enough support to follow all study requirements, such as attending visits, completing questionnaires, and taking the study medication as instructed.
Female participants who have started puberty must use an effective birth control method during the study and for one month after stopping the treatment.
Participants should have access to a suitable health insurance system, if required by local regulations.

Who Cannot Join the Study?

Patients with any other sleep disorders besides narcolepsy cannot participate.
Patients who have a history of severe allergic reactions to medications cannot participate.
Patients who are currently taking medications that might interfere with the study drug cannot participate.
Patients with significant heart problems cannot participate.
Patients with uncontrolled high blood pressure cannot participate.
Patients with severe liver or kidney disease cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients with a history of drug or alcohol abuse cannot participate.
Patients who have participated in another clinical trial recently cannot participate.
Patients with any condition that the study doctors believe would make it unsafe for them to participate cannot participate.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Robert Debre University Hospital	Paris	France
Awomfew Uoilk Sskwqndpt Lkqbnn Dw Bhjvmes	Bologna	Italy

Trial status

Country	Status	Recruitment Start
France	Not recruiting	06.06.2016
Italy	Not recruiting	06.06.2016

Trial locations

Investigated drugs:

Pitolisant

Pitolisant is a medication being tested in this clinical trial to see if it can help children and teenagers who have narcolepsy, a condition that makes them very sleepy during the day. Some people with narcolepsy also have cataplexy, which is when they suddenly lose muscle strength. This trial is looking at whether pitolisant can reduce daytime sleepiness and the number of cataplexy episodes. The study will also check how well children tolerate the medication, meaning they will see if it causes any side effects or problems.

Narcolepsy – Narcolepsy is a chronic sleep disorder characterized by overwhelming daytime drowsiness and sudden attacks of sleep. People with narcolepsy often find it difficult to stay awake for long periods, regardless of the circumstances. The condition can cause serious disruptions in daily routines. Narcolepsy is often accompanied by cataplexy, which is a sudden loss of muscle tone triggered by strong emotions. The disorder is linked to a deficiency of a brain chemical called hypocretin, which helps regulate wakefulness. Symptoms typically begin in childhood or adolescence and can persist throughout life.

Trial ID:

2024-515568-30-00

Protocol code:

P11-06

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Safety and Effectiveness of Pitolisant for Children Aged 6-17 with Narcolepsy, with or without Cataplexy

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