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

• ClinicalTrials.gov Identifier: NCT02611687
• Recruitment Status: Active, not recruiting
• First Posted: November 23, 2015
• Last Update Posted: January 14, 2022
• Sponsor: Bioprojet
• Information provided by (Responsible Party): Bioprojet

Study Description

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

Study Design

• 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

Arms and Interventions

Arm	Intervention/treatment
Experimental: pitolisant; tablet, oral, once a day.	Drug: pitolisant; Tablet; Other Name: BF2.649
Placebo Comparator: placebo; tablet, oral, once a day.	Drug: Placebo; Tablet

Outcome Measures

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 .

Eligibility Criteria

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

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.

Contacts and Locations

Locations

Finland

Terveystalo Helsinki Uniklinikka

Helsinki, Finland

France

Hôpital Femme-Mère-Enfant

Bron, France

Hôpital Gui de Chauliac

Montpellier, France

Hôpital Robert Debré

Paris, France

Italy

Università di Bologna

Bologna, Italy

Netherlands

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

Bioprojet

Investigators

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

More Information

• 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
• Last Update Posted: January 14, 2022
• Last Verified: December 2021

Additional relevant MeSH terms:

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