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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Narcolepsy Without Cataplexy; Narcolepsy With Cataplexy
• Intervention / Treatment: Drug: Placebo; Drug: pitolisant

• Study Type: Interventional
• Enrollment (Actual): 110
• Phase: Phase 3

Contacts and Locations

Study Locations

• Finland
  • Helsinki, Finland
    • Terveystalo Helsinki Uniklinikka
• France
  • Bron, France
    • Hôpital Femme-Mère-Enfant
  • Montpellier, France
    • Hopital Gui de Chauliac
  • Paris, France
    • Hopital Robert Debre
• Italy
  • Bologna, Italy
    • Universita di Bologna
• Netherlands
  • Heemstede, Netherlands
    • Polikliniek Heemstede Neurologist-Somnologist
• Russian Federation
  • Kazan, Russian Federation
    • Scientific-Research Medical Complex "Your Health"
  • Moscow, Russian Federation
    • I.M. Sechenov First Moscow State Medical University
  • Saint Petersburg, Russian Federation
    • V.M. Behterev National Medical Research Psychiatry and Neurology Center
  • Samara, Russian Federation
    • Samara Regional Clinical Hospital
  • Simferopol, Russian Federation
    • N.I. Balaban Crimea Repiblic Clinical Psychiatric Hospital No. 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: pitolisant; tablet, oral, once a day.	Drug: pitolisant; Tablet; Other Names: BF2.649
Placebo Comparator: placebo; tablet, oral, once a day.	Drug: Placebo; Tablet

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Bioprojet
• Investigators: Study Chair: Giuseppe Plazzi, MD, Dipartimento di Scienze Biomediche e Neuromotorie Alma Mater Studiorum - Università di Bologna

Study record dates

Study Major Dates

• Study Start (Actual): June 6, 2016
• Primary Completion (Actual): April 3, 2021
• Study Completion (Anticipated): December 1, 2023

Study Registration Dates

• First Submitted: November 16, 2015
• First Submitted That Met QC Criteria: November 20, 2015
• First Posted (Estimate): November 23, 2015

Study Record Updates

• Last Update Posted (Estimate): February 22, 2023
• Last Update Submitted That Met QC Criteria: February 21, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Nervous System Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Disorders of Excessive Somnolence; Narcolepsy; Cataplexy
• Other Study ID Numbers: P11-06/BF2.649; 2013-001506-29 (EudraCT Number)