Clinical trial NCT01067222: Efficacy and Safety Study of BF2.649 in the Treatment of Excessive Daytime Sleepiness in Narcolepsy
Official title: Prospective, Randomized, Double-blind Study, Placebo-controlled, Parallel-group, Multi-center Trial Assessing the Effects of BF2.649 in Treatment of Excessive Daytime Sleepiness in Narcolepsy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Narcolepsy
Excessive Daytime Sleepiness
Cataplexy
Sleep Disorders
Keywords: Narcolepsy
Excessive daytime Sleepiness
Cataplexy
Sleep Disorders
Sleep attacks
Orphan Drug
Pitolisant
Lead sponsor: Bioprojet
Sponsor class: OTHER
First posted: 2010-02-11
Enrollment: 110
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The objective of this study is to evaluate the efficacy and safety of BF2.649 administered by individual titration in narcoleptic patients with excessive daytime sleepiness (EDS)

Detailed description: BF 2.649, a new experimental drug, significantly decreases, in patient with narcolepsy, the excessive daytime sleepiness (EDS) evaluated by Epworth Sleepiness Scale (ESS), according the results of two previous clinical studies.

The objective of this study is to determine the efficacy and safety of BF2.649 administered by escalating dose (10, 20 or 40 mg/d) in narcoleptic patients with excessive daytime sleepiness versus placebo and Modafinil as assessed by both of objective and subjective measures including ESS, MWT, patients sleep diary.

60 patients with narcolepsy with or without cataplexy will be included.

Eligibility criteria: Inclusion Criteria:

* diagnosis of narcolepsy with or without cataplexy
* patients need to free of or discontinue psychostimulant medications for at least 14 days,
* patients with severe cataplexy are permitted to remain on their anticataplectic medications at stable doses
* patients must have adequate support to comply with the entire study requirements

Exclusion Criteria:

* Other conditions than Narcolepsy that can be considered as the primary causes of excessive daytime sleepiness
* Patients who are unable or unwilling to temporarily discontinue any no-authorized drugs or substances
* Psychiatric and neurological disorders such as psychosis or dementia, bipolar illness, severe anxiety, clinical depression, history of seizure disorder or other problem that in the investigator's opinion would preclude the patient's participation
* Current or recent history of a substance abuse or dependence disorder including alcohol abuse

Primary outcomes:
measure: Epworth Sleepiness Scale (ESS)
timeFrame: between baseline and at the end of 8 week DB phase
Secondary outcomes:
measure: Sleep Diary: Number and duration of diurnal sleep and sleepiness episodes, number of cataplexy attacks
timeFrame: 14 days before randomization and 7 days before each visit
measure: Maintenance of Wakefulness Test (MWT), Test of Sustained Attention to Response Task (SART).
timeFrame: at inclusion and after 8-week treatment
Other outcomes:
Locations:
city: Lugano
country: Switzerland
facility: Neurocenter (EOC) of Southern Switzerland
Study officials:
affiliation: Neurocenter (EOC) of Southern Switzerland, Lugano, Switzerland
name: Claudio Bassetti
role: PRINCIPAL_INVESTIGATOR
References:
citation: Meskill GJ, Davis CW, Zarycranski D, Doliba M, Schwartz JC, Dayno JM. Clinical Impact of Pitolisant on Excessive Daytime Sleepiness and Cataplexy in Adults With Narcolepsy: An Analysis of Randomized Placebo-Controlled Trials. CNS Drugs. 2022 Jan;36(1):61-69. doi: 10.1007/s40263-021-00886-x. Epub 2021 Dec 21.
citation: Watson NF, Davis CW, Zarycranski D, Vaughn B, Dayno JM, Dauvilliers Y, Schwartz JC. Time to Onset of Response to Pitolisant for the Treatment of Excessive Daytime Sleepiness and Cataplexy in Patients With Narcolepsy: An Analysis of Randomized, Placebo-Controlled Trials. CNS Drugs. 2021 Dec;35(12):1303-1315. doi: 10.1007/s40263-021-00866-1. Epub 2021 Nov 25.
citation: Dauvilliers Y, Bassetti C, Lammers GJ, Arnulf I, Mayer G, Rodenbeck A, Lehert P, Ding CL, Lecomte JM, Schwartz JC; HARMONY I study group. Pitolisant versus placebo or modafinil in patients with narcolepsy: a double-blind, randomised trial. Lancet Neurol. 2013 Nov;12(11):1068-75. doi: 10.1016/S1474-4422(13)70225-4. Epub 2013 Oct 7.