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

Narcolepsy With Cataplexy Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02611687
• Other study ID #: P11-06/BF2.649
• Secondary ID: 2013-001506-29
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: June 6, 2016
• Est. completion date: December 2023

Study information

• Verified date: February 2023
• Source: Bioprojet
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 110
• Est. completion date: December 2023
• Est. primary completion date: April 3, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cataplexy
• Narcolepsy
• Narcolepsy With Cataplexy
• Narcolepsy Without Cataplexy

Intervention

Drug:
• pitolisant
Tablet
• Placebo
Tablet

Locations

Country	Name	City	State
Finland	Terveystalo Helsinki Uniklinikka	Helsinki
France	Hôpital Femme-Mère-Enfant	Bron
France	Hôpital Gui de Chauliac	Montpellier
France	Hôpital Robert Debré	Paris
Italy	Università di Bologna	Bologna
Netherlands	Polikliniek Heemstede Neurologist-Somnologist	Heemstede
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

Sponsors (1)

Lead Sponsor	Collaborator
Bioprojet

Countries where clinical trial is conducted

Finland,  France,  Italy,  Netherlands,  Russian Federation, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate the efficacy of pitolisant in reducing residual Excessive Daytime Sleepiness (EDS).	Changes in EDS measured by the Ullanlinna Narcolepsy Scale Score (UNS).	8 weeks
Primary	To evaluate the efficacy of pitolisant in reducing the number of cataplectic episodes (for patients with cataplexy)	Changes in the average number of cataplexy episodes per weeks (recorded in sleep diary).	8 weeks
Primary	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	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 .	8 weeks