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

June 18, 2025 updated by: Bioprojet

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

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

110

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Finland
      • Helsinki, Finland
        • Terveystalo Helsinki Uniklinikka
  • France
      • Bron, France
        • Hôpital Femme-Mère-Enfant
      • Montpellier, France
        • Hôpital Gui de Chauliac
      • Paris, France
        • Hopital Robert Debre
  • Italy
      • Bologna, Italy
        • Università 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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

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), Ullanlinna Narcolepsy Scale Score.
Time Frame: 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.
8 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.
Time Frame: 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.
8 weeks
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .
Time Frame: 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.
8 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bioprojet

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 6, 2016

Primary Completion (Actual)

April 3, 2021

Study Completion (Actual)

June 5, 2025

Study Registration Dates

First Submitted

November 16, 2015

First Submitted That Met QC Criteria

November 20, 2015

First Posted (Estimated)

November 23, 2015

Study Record Updates

Last Update Posted (Actual)

June 26, 2025

Last Update Submitted That Met QC Criteria

June 18, 2025

Last Verified

June 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • P11-06/BF2.649
  • 2013-001506-29 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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