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. - P11-06

BIOPROJET PHARMA0 sites108 target enrollmentJune 17, 2021

Conditionsarcolepsy MedDRA version: 20.0Level: LLTClassification code 10028715Term: Narcolepsy with cataplexySystem Organ Class: 10029205 - Nervous system disorders MedDRA version: 22.1Level: LLTClassification code 10048322Term: Narcolepsy aggravatedSystem Organ Class: 10029205 - Nervous system disorders MedDRA version: 20.0Level: PTClassification code 10028713Term: NarcolepsySystem Organ Class: 10029205 - Nervous system disorders MedDRA version: 21.1Level: LLTClassification code 10048323Term: Cataplexy aggravatedSystem Organ Class: 10029205 - Nervous system disorders MedDRA version: 20.0Level: PTClassification code 10007737Term: CataplexySystem Organ Class: 10029205 - Nervous system disorders Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: arcolepsy
Sponsor: BIOPROJET PHARMA
Enrollment: 108
Status: Active, not recruiting
Last Updated: 4 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

June 17, 2021

End Date

TBD

Last Updated

4 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

BIOPROJET PHARMA

Eligibility Criteria

Inclusion Criteria

•1\)Male and female children from 6 to less than 18 years of age (until V8\) suffering from narcolepsy with or without cataplexy \- meeting the International Classification of Sleep Disorders (ICSD\-3\) criteria (narcolepsy type 1 and 2\). Diagnosis confirmed with polysomnography and Multiple Sleep Latency Test for patients without cataplexy (if these examinations were not performed within the last 12 months)
•2\)PDSS score \= 15 during baseline period (V1\+V2\) / 2\.
•3\)Patients should be free of non\-authorized medication, in particular psychostimulant treatments as from the screening visit (V0\) onwards.
•4\)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.
•5\)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).
•6\)Female pubescent patients shall use a birth control method, judged efficient by the investigator, throughout the study and during the month following treatment discontinuation.
•7\)Patients should benefit from appropriate healthy insurance system (only applicable where mandatory e.g. in France).
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1\.2 Adults (18\-64 years) no

Exclusion Criteria

•1\)Any other conditions that can be considered the primary causes of EDS: such as sleep related breathing disorders as defined by a sleep Apnea Index \= 5 per hour or/and an Apnea/Hypopnea Index \= 10 per hour, chronic sleep deprivation, circadian sleep wake rhythm disorder or any other medical or neurological causes that could account for narcolepsy symptoms associated with EDS.
•2\)Cataplectic patients treated by anticataplectics (SNRI, SSRI, sodium oxybate) which are not under a stable treatment since at least 4 weeks at the time of inclusion (V2\).
•3\)Patients treated for cataplexy or any other pathology, by tricyclic antidepressants (clomipramine, imipramine, mirtazapine, desmethylimipramine and protriptyline) are not authorized because they display histamine H1 receptor antagonist activity.
•4\)The use of pitolisant within a 30\-day period prior to initial screening visit (V0\) for this trial.
•5\)Current or recent (within one year) history of a substance abuse or dependence disorder including alcohol abuse as defined in Diagnostic and Statistical Manual of Mental Disorders (DSM\-IV).
•6\)Any significant abnormality of the electrocardiogram and particularly Fridericia’s QTc interval (QTcF \= QT/3¿ 60/HR) higher than 450ms.
•7\)Patients with severe depression (CDI \= 16\)
•8\)Patient with suicidal risk (C\-SSRS)
•9\)Positive urinary drug testing (test applicable to patients from 12 years)
•10\)Pregnancy (defined as positive ß\-HCG blood test), breast\-feeding, or patients and unable to use an efficient method of birth control shall not be included in the study (for pubescent female only).