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

NCT02611687 | Completed Phase 3 Results DMC

• 2 other identifiers: P11-06/BF2.6492013-001506-29
• Study Type: interventional
• Target: 110
• Locations: 5 countries
• Sites: 11

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.

Conditions

Narcolepsy With Cataplexy; Narcolepsy Without Cataplexy

Outcome Measures

Primary Outcomes (1)

• To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.

  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 Outcomes (2)

• To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.

  8 weeks

• To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .

  8 weeks

Study Arms (2)

pitolisant

EXPERIMENTAL

tablet, oral, once a day.

Drug: pitolisant

placebo

PLACEBO COMPARATOR

tablet, oral, once a day.

Drug: Placebo

Interventions

pitolisant DRUG

Tablet

Also known as: BF2.649

pitolisant

Placebo DRUG

Tablet

placebo

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• Any other conditions that can be considered the primary causes of EDS.
• 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.

Sponsors & Collaborators

• Bioprojet: lead

Study Sites (11)

Terveystalo Helsinki Uniklinikka

Helsinki, Finland

Location

Hôpital Femme-Mère-Enfant

Bron, France

Location

Hôpital Gui de Chauliac

Montpellier, France

Location

Hôpital Robert Debré

Paris, France

Location

Università di Bologna

Bologna, Italy

Location

Polikliniek Heemstede Neurologist-Somnologist

Heemstede, Netherlands

Location

Scientific-Research Medical Complex "Your Health"

Kazan', Russia

Location

I.M. Sechenov First Moscow State Medical University

Moscow, Russia

Location

V.M. Behterev National Medical Research Psychiatry and Neurology Center

Saint Petersburg, Russia

Location

Samara Regional Clinical Hospital

Samara, Russia

Location

N.I. Balaban Crimea Repiblic Clinical Psychiatric Hospital No. 1

Simferopol, Russia

Location

Related Publications (1)

• Dauvilliers Y, Lecendreux M, Lammers GJ, Franco P, Poluektov M, Causse C, Lecomte I, Lecomte JM, Lehert P, Schwartz JC, Plazzi G. Safety and efficacy of pitolisant in children aged 6 years or older with narcolepsy with or without cataplexy: a double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2023 Apr;22(4):303-311. doi: 10.1016/S1474-4422(23)00036-4.

  PMID: 36931805 DERIVED

MeSH Terms

Conditions

Narcolepsy

Interventions

pitolisant

Condition Hierarchy (Ancestors)

Disorders of Excessive Somnolence; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Nervous System Diseases; Mental Disorders

Results Point of Contact

• Title: Clinical Project Manager
• Organization: Bioprojet Pharma

a.viatte@bioprojet.com

+33 (0)1 47 03 66 23

Study Officials

• Giuseppe Plazzi, MD

  Dipartimento di Scienze Biomediche e Neuromotorie Alma Mater Studiorum - Università di Bologna

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2015
• First Posted: November 23, 2015
• Study Start: June 6, 2016
• Primary Completion: April 3, 2021
• Study Completion: June 5, 2025
• Last Updated: June 26, 2025
• Results First Posted: September 19, 2024

Record last verified: 2025-06

Locations

RU (5); IT (1); NL (1); FI (1); FR (3)