Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period

Phase 3

Active, not recruiting

Brief Summary: The purpose of this multicenter double blind study is to assess efficacy and safety of Pitolisant versus placebo in paediatric Narcoleptic patients with or without cataplexy.

Recruitment & Eligibility

Inclusion Criteria

Male and female children from 6 to less than 18 years of age suffering from narcolepsy with or without cataplexy - ICSD-3 criteria (narcolepsy type 1 and 2).
PDSS
Patients should be free of non-authorized medication, in particular psychostimulant treatments as from the screening visit onwards.
Parents - and patients old enough to understand who have expressed a willingness to participate in the study, who have signed and dated the informed consent form prior to beginning protocol required procedures.
In the opinion of the investigator, the patient must have adequate support to comply with the entire study requirements as described in the protocol (e.g., transportation to and from trial site, self rating scales and diaries completion, drug compliance, scheduled visits, tests).

Exclusion Criteria

Any other conditions that can be considered the primary causes of EDS.
Cataplectic patients treated by anticataplectics which are not under a stable treatment at the time of inclusion.
Patients treated for cataplexy or any other pathology, by tricyclic antidepressants.
Any significant abnormality of the electrocardiogram and particularly Fridericia's QTc interval.
Patients with significant abnormality or clinical laboratory results.
Psychiatric and neurological disorders in the investigator's opinion, would preclude the patient's participation and completion of this trial or comprise reliable representation of subjective symptoms.
Active clinically significant illness, including unstable cardiovascular, endocrine, neoplastic, gastrointestinal, haematological, hepatic, immunologic, metabolic, neurological (other than narcolepsy/cataplexy), pulmonary, and/or renal disease which could interfere with the study conduct or counter-indicate the study treatments or place the patient at risk during the trial or compromise the study objectives.

Arm && Interventions

Group	Intervention	Description
placebo	Placebo	tablet, oral, once a day.
pitolisant	pitolisant	tablet, oral, once a day.

Primary Outcome Measures

Name	Time	Method
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.	8 weeks	Changes in EDS measured by the Ullanlinna Narcolepsy Scale Score (UNS) from baseline (mean of two pre-treatment measures) to the end of double-blind period (mean of the last two measures). The UNS is an 11-item scale used to measure the intensity and frequency of symptoms of narcolepsy (EDS and cataplexy). The total score varies from 0 to 44, with higher scores denoting greater narcoleptic tendencies.

Secondary Outcome Measures

Name	Time	Method
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.	8 weeks	Changes in EDS measured by the Pediatric Daytime Sleepiness Scale (PDSS) from baseline (mean of two pre-treatment measures) to the end of double-blind period (mean of the last two measures). The PDSS total score can range from 0 and 32 and a score \>13 is considered abnormal sleepiness.
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .	8 weeks	Changes in EDS measured by Ullanlinna Narcolepsy Scale-Cataplexy Subscore (UNS-CTP) in patients with type 1 narcolepsy from baseline (mean of two pre-treatment measures) to the end of double-blind period (mean of the last two measures). This subscore is defined as the sum of the first 4 items of the UNS, UNS-CTP subscore ranges from 0 to 16, with higher scores indicating more severe symptoms.

Locations (11): Hôpital Femme-Mère-Enfant
🇫🇷
Bron, France
Polikliniek Heemstede Neurologist-Somnologist
🇳🇱
Heemstede, Netherlands
Università di Bologna
🇮🇹
Bologna, Italy
Hôpital Robert Debré
🇫🇷
Paris, France
Terveystalo Helsinki Uniklinikka
🇫🇮
Helsinki, Finland
Hôpital Gui de Chauliac
🇫🇷
Montpellier, France
Scientific-Research Medical Complex "Your Health"
🇷🇺
Kazan, Russian Federation
I.M. Sechenov First Moscow State Medical University
🇷🇺
Moscow, Russian Federation
V.M. Behterev National Medical Research Psychiatry and Neurology Center
🇷🇺
Saint Petersburg, Russian Federation
Samara Regional Clinical Hospital
🇷🇺
Samara, Russian Federation
N.I. Balaban Crimea Repiblic Clinical Psychiatric Hospital No. 1
🇷🇺
Simferopol, Russian Federation