Clinical trial NCT02611687: Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Narcolepsy With Cataplexy
Narcolepsy Without Cataplexy
Lead sponsor: Bioprojet
Sponsor class: OTHER
First posted: 2015-11-23
Enrollment: 110
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.
timeFrame: 8 weeks
Secondary outcomes:
measure: To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.
timeFrame: 8 weeks
measure: To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .
timeFrame: 8 weeks
Other outcomes:
Locations:
city: Helsinki
country: Finland
facility: Terveystalo Helsinki Uniklinikka
city: Bron
country: France
facility: Hôpital Femme-Mère-Enfant
city: Montpellier
country: France
facility: Hôpital Gui de Chauliac
city: Paris
country: France
facility: Hôpital Robert Debré
city: Bologna
country: Italy
facility: Università di Bologna
city: Heemstede
country: Netherlands
facility: Polikliniek Heemstede Neurologist-Somnologist
city: Kazan
country: Russian Federation
facility: Scientific-Research Medical Complex "Your Health"
city: Moscow
country: Russian Federation
facility: I.M. Sechenov First Moscow State Medical University
city: Saint Petersburg
country: Russian Federation
facility: V.M. Behterev National Medical Research Psychiatry and Neurology Center
city: Samara
country: Russian Federation
facility: Samara Regional Clinical Hospital
city: Simferopol
country: Russian Federation
facility: N.I. Balaban Crimea Repiblic Clinical Psychiatric Hospital No. 1
Study officials:
affiliation: Dipartimento di Scienze Biomediche e Neuromotorie Alma Mater Studiorum - Università di Bologna
name: Giuseppe Plazzi, MD
role: STUDY_CHAIR
References: