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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Narcolepsy With Cataplexy, Narcolepsy Without Cataplexy

Intervention / Treatment
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* Drug: pitolisant
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: pitolisant<br>tablet, oral, once a day. | Drug: pitolisant<br>* Tablet<br>* Other names: BF2.649;|
| Placebo Comparator: placebo<br>tablet, oral, once a day. | Drug: Placebo<br>* Tablet<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the efficacy of pitolisant in reducing residual Excessive Daytime Sleepiness (EDS). | Changes in EDS measured by the Ullanlinna Narcolepsy Scale Score (UNS). | 8 weeks | 
| To evaluate the efficacy of pitolisant in reducing the number of cataplectic episodes (for patients with cataplexy) | Changes in the average number of cataplexy episodes per weeks (recorded in sleep diary). | 8 weeks | 
| To determine safety in children and adolescents - Safety assessment will be done on monitoring of adverse events, physical examination, vital signs, ECG (QTc) and Blood Laboratory tests modifications and the mood appraisal | Safety assessment will be done on monitoring of adverse events (Incidence of Treatment-Emergent Adverse Events), physical examination, vital signs (blood pressure and heart rate), ECG (QTc) and Blood Laboratory tests modifications (Haematology and Biochemistry) and the mood appraisal (Childhood Depression Inventory [CDI] and Columbia-Suicide Severity Rating Scale [C-SSRS]) throughout the study . | 8 weeks |