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

Phase 3

Completed

• Conditions: Hypersomnia; Sleep disorder; 10040998

• Registration Number: NL-OMON53057
• Lead Sponsor: Bioprojet

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 10

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).

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 β-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)., 11) Patients with severe hepatic Impairment (Child Pugh C) or with any
other significant abnormality in the physical examination or clinical
laboratory results., 12) Psychiatric and neurological disorders, such as
moderate or severe psychosis or dementia, depression, history of seizure
disorder or other problem that, in the investigator*s opinion, would preclude
the patient*s participation and completion of this trial or comprise reliable
representation of subjective symptoms., 13) 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.,
14) Prior severe adverse reactions to CNS stimulants., 15) Known
hypersensitivity to the tested treatment including active substance and
excipients., 16) The inability to continue daily activities safely, without the
use of treatment against EDS., 17) Any patient presenting congenital
galactosemia, glucose-galactose malabsorption or lactase deficiency due to the
presence of lactose in investigational treatments., 18) Patients participating
in another study or being in a follow-up period for another study., 19) Cannot
be contacted in case of emergency.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Change in the intensity and frequency of symptoms of narcolepsy (EDS and<br /><br>cataplexy) as measured by the Ullanlinna Narcolepsy Scale (UNS) between<br /><br>baseline: [V1 score (D-14) + V2 score (D0)]/2 and the end of treatment: [V6<br /><br>score (D49) + V7 score (D56)]/2. We will compare the results between pitolisant<br /><br>and placebo groups.</p><br>