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

NCT ID: NCT02611687

Last Updated: 2025-06-26

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 110 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-06-06
• Study Completion Date: 2025-06-05

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

pitolisant

tablet, oral, once a day.

Group Type: EXPERIMENTAL

pitolisant

Intervention Type: DRUG

Tablet

placebo

tablet, oral, once a day.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Tablet

Interventions

pitolisant

Tablet

Intervention Type: DRUG

Placebo

Tablet

Intervention Type: DRUG

Other Intervention Names

BF2.649

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.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Bioprojet

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Giuseppe Plazzi, MD

Role: STUDY_CHAIR

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

Locations

Terveystalo Helsinki Uniklinikka

Helsinki, , Finland

Hôpital Femme-Mère-Enfant

Bron, , France

Hôpital Gui de Chauliac

Montpellier, , France

Hôpital Robert Debré

Paris, , France

Università di Bologna

Bologna, , Italy

Polikliniek Heemstede Neurologist-Somnologist

Heemstede, , Netherlands

Scientific-Research Medical Complex "Your Health"

Kazan', , Russia

I.M. Sechenov First Moscow State Medical University

Moscow, , Russia

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

Saint Petersburg, , Russia

Samara Regional Clinical Hospital

Samara, , Russia

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

Simferopol, , Russia

Countries

Finland; France; Italy; Netherlands; Russia

References

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.

Reference Type: DERIVED

PMID: 36931805 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2013-001506-29

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

P11-06/BF2.649

Identifier Type: -

Identifier Source: org_study_id