Narcolepsy with Cataplexy MedDRA version: 16.1 Level: LLT Classification code 10028715 Term: Narcolepsy with cataplexy System Organ Class: 100000004852
Conditions
Interventions
Product Name: Pitolisant
Product Code: BF2.649
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: pitolisant
CAS Number: 903576-44-3
Current Sponsor code: BF2.649
Concentration unit: mg milligram
Sponsors
Bioprojet
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Men or women, of 18 years of age and over. • Patients with a diagnosis of narcolepsy with cataplexy according to the International Classification of Sleep Disorders (ICSD-2) criteria • “De novo” patients, or patients treated by purported anticataplectic drugs, i.e. SSRIs and patients treated by sodium oxybate at stable dose, for a minimum period of one month, having shown an incidence of at least 3 weekly cataplexy attacks and with an ESS score = 12 • Patients should be free of prohibited treatments (see p.10) or have discontinued them for at least 7 days at the start of baseline period. • Women of child-bearing potential must use a medically accepted effective, method of birth control, estimated efficient enough by the investigator, and agree to continue this method for the duration of the study and the month following treatment discontinuation. Women must be negative to serum pregnancy test performed at the screening visit and should not be breast-feeding patients. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 137 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: • Patients with 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 = 10 per hour or and an Apnea/Hypopnea Index = 15 per hour, periodic limbs movement (PLM) disorders as defined by a PLM arousal index (PLMAI) = 10 per hour, shift work, chronic sleep deprivation, circadian sleep wake rhythm disorder or any other medical or neurological causes that could account for narcolepsy symptoms associated with EDS. • Psychiatric and neurological disorders, other than narcolepsy/cataplexy, such as moderate or severe psychosis or dementia, bipolar illness, severe anxiety, clinical severe depression (BDI = 16) with suicidal risk (item G BDI > 0), or depression treated for less than 8 weeks, history of seizure disorder or other problem that, in the investigator’s opinion, would preclude the patient’s participation and completion of this trial. • Patients unable or unwilling to temporarily suppress non-authorized drugs during the study. • Concurrent use of hypnotics, tranquilizers, sedating antihistamines, benzodiazepines, anticonvulsants, psychostimulants, (amphetamines, amphetamine-like, modafinil, methylphenidrate, or other CNS stimulants), tricyclic antidepressants (e.g. imipramine), clonidine will not be accepted since 3 weeks before randomisation and during study. • 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). • Other active clinically significant illness, including unstable cardiovascular, or neoplasic pathology 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 participation. • Patients with a known history of long QTc syndrome (e.g. syncope or arrhythmia) or presenting any significant serious abnormality of the ECG (e.g. recent myocardial infarction), or QTc Fridericia higher than 450 ms for male and 470ms for female (electrocardiogram Fridericia’s corrected QT interval =QT / 3 (cube root) RR) • Patients with Severe Hepatic Impairment or with Severe Renal Impairment, or with any other significant abnormality in the physical examination or clinical laboratory results. • Known hypersensitivity to the tested treatment including active substance and excipients. • Prior severe adverse reactions to CNS stimulants • Any patients presenting congenital galactosemia, glucose-galactose malabsorption or lactase deficiency due to the presence of lactose in investigational treatments • Patients participating in another study, or having participated in a study during the previous month
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The main objective is the measure of the anticataplectic efficacy of BF2.649 compared to placebo, assessed by the change in the average number of cataplexy attacks per week between the 2 weeks of baseline and the 4 weeks of stable treatment period. ;Secondary Objective: Change in the average number of cataplexy attacks per week (as recorded in patient cataplexy and sleep diaries) between the 2 weeks of baseline, and the 2 weeks of end treatment period Severity of cataplexy measured by the Clinical Global Impression of Severity . Difference between the mean values of baseline [(V1+V2) / 2] and end treatment period [(V5+V6) / 2] ESS score: difference between the mean value during baseline [(V1+V2) / 2] and the mean value during end treatment period [(V5+V6) / 2] Severity of EDS as measured by the Clinical Global Impression of Severity and of Change at V2 and V6 European Quality of life questionnaire (EQ-5D) at V2 and V6 Maintenance of Wakefulness Test (MWT) consisting of 4 sessions of 40-minute tests only at V2 and V6 Cataplexy and sleep diary: difference in weekly frequency of days with hallucinations between the 2 weeks of baseline and the 4 weeks of stable treatment period. Patient’s Global Opinion on the treatment effect (V6) ;Primary end point(s): The primary endpoint is the measure of anticataplectic efficacy assessed by the change in the average number of cataplexy attacks per week (as recorded in patient cataplexy and sleep diaries);Timepoint(s) of evaluation of this end point: endpoint is evaluated between the 2 weeks of baseline (Day-14 to Day 0) and the 4 weeks of stable treatment period (D 21 to D 49). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Change in the average number of cataplexy attacks per week (as recorded in patient cataplexy and sleep diaries) between the 2 weeks of baseline, and the 2 weeks of end treatment period. • Severity of cataplexy measured by the Clinical Global Impression of Severity and hange (CGI-S and CGI-C on cataplexy). Difference between the mean values of baseline [(V1+V2) / 2] and end treatment period [(V5+V6) / 2]. • ESS score: difference between the mean value during baseline [(V1+V2) / 2] and the mean value during end treatment period [(V5+V6) / 2]. • Severity of EDS as measured by the Clinical Global Impression of Severity and of Change (CGI-S and CGI-C on EDS) at V2 and V6. • European Quality of life questionnaire (EQ-5D) at V2 and V6. • Maintenance of Wakefulness Test (MWT) consisting of 4 sessions of 40-minute tests only at V2 and V6 • Cataplexy and sleep diary: difference in weekly frequency of days with hallucinations between the 2 weeks of baseline (D-14 to D0) and the 4 weeks of stable treatment period (D21 to D49). • Patient’s Global Opinion on the treatment effect (V6) ;Timepoint(s) of evaluation of this end point: described in section E.5.2 | — |
Countries
Bulgaria, Czech Republic, Hungary, Macedonia, the former Yugoslav Republic of, Poland, Russian Federation, Serbia, Turkey, Ukraine
Contacts
Public ContactBioprojet clinical department
Bioprojet
Outcome results
None listed