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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02611687
Recruitment Status : Active, not recruiting
First Posted : November 23, 2015
Last Update Posted : January 14, 2022
Information provided by (Responsible Party):

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.

Condition or disease Intervention/treatment Phase
Narcolepsy With Cataplexy Narcolepsy Without Cataplexy Drug: pitolisant Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 110 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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
Actual Study Start Date : June 6, 2016
Actual Primary Completion Date : April 3, 2021
Estimated Study Completion Date : September 2022

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: pitolisant
tablet, oral, once a day.
Drug: pitolisant
Other Name: BF2.649

Placebo Comparator: placebo
tablet, oral, once a day.
Drug: Placebo

Primary Outcome Measures :
  1. To evaluate the efficacy of pitolisant in reducing residual Excessive Daytime Sleepiness (EDS). [ Time Frame: 8 weeks ]
    Changes in EDS measured by the Ullanlinna Narcolepsy Scale Score (UNS).

  2. To evaluate the efficacy of pitolisant in reducing the number of cataplectic episodes (for patients with cataplexy) [ Time Frame: 8 weeks ]
    Changes in the average number of cataplexy episodes per weeks (recorded in sleep diary).

  3. 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 [ Time Frame: 8 weeks ]
    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 .

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02611687

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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
Russian Federation
Scientific-Research Medical Complex "Your Health"
Kazan, Russian Federation
I.M. Sechenov First Moscow State Medical University
Moscow, Russian Federation
V.M. Behterev National Medical Research Psychiatry and Neurology Center
Saint Petersburg, Russian Federation
Samara Regional Clinical Hospital
Samara, Russian Federation
N.I. Balaban Crimea Repiblic Clinical Psychiatric Hospital No. 1
Simferopol, Russian Federation
Sponsors and Collaborators
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Study Chair: Giuseppe Plazzi, MD Dipartimento di Scienze Biomediche e Neuromotorie Alma Mater Studiorum - Università di Bologna
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Responsible Party: Bioprojet
ClinicalTrials.gov Identifier: NCT02611687    
Other Study ID Numbers: P11-06/BF2.649
2013-001506-29 ( EudraCT Number )
First Posted: November 23, 2015    Key Record Dates
Last Update Posted: January 14, 2022
Last Verified: December 2021
Additional relevant MeSH terms:
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Disorders of Excessive Somnolence
Sleep Disorders, Intrinsic
Sleep Wake Disorders
Nervous System Diseases
Mental Disorders