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

INTERVENTION: Product Name: Waki X18 mg film‐coated tablets, Product Code:PRD3956063, Pharmaceutical Form: FILM‐COATED TABLET, Other descriptive name: , Strength: , Product Name: Matches test film‐coated tablets, Product Code:N/A, Pharmaceutical Form: N/A, Other descriptive name: N/A , Strength: , Pharmaceutical form of the placebo: N/A , Product Name: Waki X4.5 mg film‐coated tablets, Product Code:PRD3956062, Pharmaceutical Form: FILM‐COATED TABLET, Other descriptive name: , Strength: CONDITION: Narcolepsy ; MedDRA version: 20.0Level: LLTClassification code: 10028715Term: Narcolepsy with cataplexy Class: 10029205 Therapeutic area: Diseases [C] ‐ Nervous System Diseases [C10] INCLUSION CRITERIA: Male and female children from 6 to less than 18 years of age (at V8) suffering from narcolepsy with or without cataplexy ‐ meeting the International Classification of Sleep Disorders (ICSD‐3) criteria (Narcolepsy type 1 and 2). Diagnosis confirmed with polysomnography and Multiple Sleep Latency Test for patients without cataplexy (if these examinations were not performed within the last 12 months) (Italy) Male and female children from 6 to less than 18 years of age (at V8) suffering from narcolepsy with or without cataplexy ‐ meeting the International Classification of Sleep Disorders (ICSD‐3) criteria (Narcolepsy type 1 and 2). Diagnosis confirmed with polysomnography (to be performed if this examination was not performed within the last 12 months) and Multiple Sleep Latency Test for patients without cataplexy (France), PDSS score = 15 during baseline period (V1+V2) / 2., Patients should be free of non‐authorized medication, in particular psychostimulant treatments SECONDARY OUTCOME: Secondary end point(s):Changes between baseline and at each visit of the open‐label period in EDS Secondary end point(s):Changes between baseline and V6 will be compared for the TEA‐Ch test Secondary end point(s):Changes in EDS as measured by the Child and Adolescent Sleepiness Scale (CASS) between baseline and the end of treatment Secondary end point(s):Changes in EDS as measured by the maintenance of wakefulness test (MWT) between baseline and V7 Secondary end point(s):Changes in EDS measured by the Paediatric Daytime Sleepiness Scale (PDSS) between baseline: [V1 score (D‐14) + V2 score (D0)]/2 and the end of treatment: [V6 score (D49) + V7 score (D56)]/2 Secondary end point(s):Changes in the average number of cataplexy episodes per weeks: (recorded in sleep diary by patient and/or parent/teacher) between the 2 weeks of baseline and the 2 weeks of end study treatment period (V6, V7) Secondary end point(s):Comparison between placebo and pitolisant groups on Blood laboratory tests (haematology, blood chemistry) Secondary end point(s):Comparison between placebo and pitolisant groups on Changes in Physical examination and Vital signs Secondary end point(s):Comparison between placebo and pitolisant groups on ECG and calculation of Fridericia’s corrected QTc interval Secondary end point(s):Comparison between placebo and pitolisant groups on Mood appraisal adapted to children (CDI and C‐SSRS) Secondary end point(s):Comparison between placebo and pitolisant groups on Patients’ Global Opinion on treatment effect at the end of treatment if able to express himself. If not will be reported either by parents or teachers Secondary end point(s):Comparison between placebo and pitolisant groups on Tolerability as measured by Treatment Emergent Adverse Events Secondary end point(s):Comparison between placebo and pitolisant groups on withdrawal symptoms questionnaire (DSM IV) on D59 during a phone call (T1) and at the end‐of‐study visit on D63 (V8) after a one‐week period on placebo Secondary end point(s):Differences in weekly frequency of cataplexy episodes (recorded in sleep diary by patient and/or parent/teacher) between baseline and the 4 weeks of stable treatment period (V5 to V7) Secondary end point(s):Safety assessment will be done on monitoring of adverse events, physical examination, vital signs, ECG and Blood Laboratory tests modifications and the mood appraisal throughout the study. Secondary end point(s):Severity of cataplexy measured by the clinical Global Impression of severity and change. Changes between baseline and V6, V7 Secondary end point(s):Severity of EDS measured by the clinical Global Impression of severity and change. Changes between baseline and V6, V7