Study on the Effects of Pitolisant Hydrochloride for Patients with Prader-Willi Syndrome

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What is this study about?

This clinical trial is focused on studying Prader-Willi syndrome, a genetic condition that affects many parts of the body. The study will evaluate the effects of a medication called pitolisant hydrochloride, which is also known by the code name HBS-101 and is marketed under the name WAKIX. The purpose of the study is to assess how this medication impacts the severity of excessive daytime sleepiness in patients with Prader-Willi syndrome.

The study will be conducted in two main phases. Initially, participants will be randomly assigned to receive either the medication or a placebo, which looks like the medication but does not contain the active ingredient. This phase is double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication. After this phase, all participants will have the opportunity to receive the medication in an open-label extension, where everyone knows they are receiving the active treatment. The study will last for several weeks, with regular check-ins to monitor the participants’ health and any changes in their symptoms.

Throughout the study, the effects of pitolisant hydrochloride on various symptoms of Prader-Willi syndrome will be closely monitored. This includes not only the primary focus on excessive daytime sleepiness but also other related symptoms such as irritable and disruptive behaviors. The safety of the medication will also be assessed by tracking any side effects experienced by participants. The study aims to provide valuable information on the potential benefits and safety of using pitolisant hydrochloride for managing symptoms in individuals with Prader-Willi syndrome.

1 double-blind treatment period

During this phase, the patient will receive either the study medication, pitolisant hydrochloride, or a placebo. The medication is administered in the form of a tablet taken orally.

The dosage of the medication will be determined by the study team and will be adjusted as necessary based on the patient’s response and any side effects experienced.

This phase lasts for 77 days, during which the patient will be monitored for changes in the severity of excessive daytime sleepiness (EDS) and other symptoms associated with Prader-Willi syndrome.

2 open-label extension

After completing the double-blind treatment period, the patient will enter the open-label extension phase.

In this phase, all patients will receive pitolisant hydrochloride regardless of their previous group assignment. The medication will continue to be administered orally in tablet form.

The duration of this phase will be determined by the study team, and the patient’s response to the medication will be closely monitored.

3 monitoring and assessments

Throughout the study, the patient will undergo regular assessments to evaluate the effectiveness and safety of the treatment.

These assessments will include measuring changes in the severity of EDS, irritable and disruptive behaviors, and other symptoms using various standardized questionnaires and scales.

The patient’s health and any side effects experienced will be closely monitored to ensure safety and well-being.

Who Can Join the Study?

Patients must be male or female and at least 6 years old at the time of screening.
Patients with a history of seizures must have a stable seizure history, meaning the frequency and severity of seizures have not changed, for at least 6 months before screening.
Female patients who can become pregnant must have a negative pregnancy test at screening and agree to use a reliable nonhormonal birth control method during the study and for 21 days after the last dose of the study drug. If using hormonal birth control, they must also use a nonhormonal method during the study and for 21 days after stopping the study drug. Male patients who can father children must agree to use a barrier method of birth control during the study and for 21 days after the last dose of the study drug.
Patients must have a consistent parent or caregiver, preferably the same person throughout the study, who is willing and able to complete the required study assessments.
The patient, along with their parent(s), caregiver(s), or legal guardian(s), must be able to understand and follow the study requirements and take the oral study drug as instructed.
The patient or their parent(s), caregiver(s), or legal guardian(s) must be able to provide written consent to participate in the study. If applicable, the patient must also provide written agreement to participate.
The patient must have a confirmed diagnosis of Prader-Willi syndrome (PWS) through genetic testing or medical records. If not confirmed, genetic testing will be provided by the study sponsor.
The patient must meet criteria for excessive daytime sleepiness (EDS) based on specific questionnaires.
The patient must get an appropriate amount of sleep each night based on their age.
If the patient is taking any long-term medications or supplements that are not prohibited, they must be on a stable dose for at least 30 days before screening and agree to continue the stable dose during the study or stop taking them for a certain period before screening.
If the patient is taking hormone treatments like growth hormone, testosterone, or estrogen, they must be on a stable dose for 30 days before screening and during the study, with a small variation allowed.
If the patient is using cannabidiol (CBD) or tetrahydrocannabinol (THC), they must be on a stable dose for 30 days before screening and agree to continue the stable dose during the study or stop using them for a certain period before screening.
If the patient is taking a strong CYP2D6 inhibitor, a type of medication that affects how the body processes drugs, they must be on a stable dose for at least 30 days before screening and during the study or stop taking it for a certain period before screening.

Who Cannot Join the Study?

Patients who do not have Prader-Willi syndrome cannot participate. Prader-Willi syndrome is a genetic condition that affects many parts of the body.
Patients who are not within the specified age ranges cannot participate. The age ranges include children, adolescents, and adults.
Patients who are not part of the specified clinical trial groups cannot participate. Clinical trial groups refer to specific categories of patients that the study is focusing on.
Both male and female patients are eligible, so exclusion is not based on gender.
Patients who are not considered part of a vulnerable population cannot participate. Vulnerable populations include groups that may need special protection or consideration in research.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Uniwersytecki Szpital Kliniczny Im Jana Mikulicza Radeckiego We Wroclawiu	Wroclaw	Poland
Pomeranian Medical University	Szczecin	Poland

Other Sites

Site Name	City	Country
Hopital Des Enfants	Toulouse	France
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Azienda Ospedaliero Universitaria Di Modena	Modena	Italy
Azienda Ospedaliera Universitaria Meyer IRCCS	Florence	Italy
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Hospital Universitario 12 De Octubre	Madrid	Spain
National Institute Of Endocrinology C.I. Parhon	Bucharest	Romania
Centrul National Clinic De Recuperare Neuropsihomotorie Copii Dr. Nicolae Robanescu	Bucharest	Romania
Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo	Trieste	Italy
Azienda Ospedaliera di Padova	Padua	Italy
Spitalul Clinic De Urgenta Pentru Copii Louis Turcanu Timisoara	Timisoara	Romania
Ospedale San Raffaele S.r.l.	Milan	Italy
Karolinska University Hospital	Solna	Sweden
Region Midtjylland	Aarhus	Denmark
Vseobecna Fakultni Nemocnice V Praze	Prague	Czechia
Rigshospitalet	Copenhagen	Denmark
Vrije Universiteit Brussel	Jette	Belgium
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Hospital Universitario Virgen De La Victoria	Malaga	Spain
Uvimsuyttnzdzuaeopunf Eucbo Auq	Essen	Germany
Pesc Tyejx Hswnqtac Uhbrybfqfanq	Sabadell	Spain
Cjposz Hikjxmjsssb Rpzlqwgh Dhigbdmatbsknp	Angers	France

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	19.11.2024
Czechia	Not recruiting	19.11.2024
Denmark	Recruiting	19.11.2024
France	Recruiting	19.11.2024
Germany	Recruiting	19.11.2024
Italy	Recruiting	19.11.2024
Poland	Recruiting	19.11.2024
Romania	Recruiting	19.11.2024
Spain	Recruiting	19.11.2024
Sweden	Recruiting	19.11.2024

Trial locations

Pitolisant is a medication being studied for its effectiveness in reducing excessive daytime sleepiness (EDS) in patients with Prader-Willi Syndrome (PWS). It works by increasing the levels of certain chemicals in the brain that help promote wakefulness and alertness. This study aims to determine how well pitolisant can improve the symptoms of EDS in individuals with PWS.

Investigated diseases:

Prader-Willi syndrome

Prader-Willi syndrome – This is a rare genetic disorder that affects many parts of the body. It is characterized by a constant sense of hunger, leading to overeating and obesity. Individuals with this syndrome often have low muscle tone, short stature, and cognitive disabilities. Behavioral problems such as temper outbursts and stubbornness are common. Sleep disturbances, including excessive daytime sleepiness, are also frequently observed. The condition is caused by a loss of function of specific genes on chromosome 15.

Trial ID:

2023-508307-21-00

Protocol code:

HBS-101-CL-312

NCT ID:

NCT06366464

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Effects of Pitolisant Hydrochloride for Patients with Prader-Willi Syndrome

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