Narcolepsy MedDRA version: 14.0 Level: LLT Classification code 10028715 Term: Narcolepsy with cataplexy System Organ Class: 10029205 - Nervous system disorders MedDRA version: 13.1 Level: PT Classification code 10028713 Term: Narcolepsy System Organ Class: 10029205 - Nervous system disorders MedDRA version: 13.1 Level: LLT Classification code 10048323 Term: Cataplexy aggravated System Organ Class: 10029205 - Nervous system disorders MedDRA version: 13.1 Level: PT Classification code 10007737
Conditions
Interventions
Product Name: Pitolisant
Product Code: BF2.649
Pharmaceutical Form: Tablet
INN or Proposed INN: Pitolisant
CAS Number: BF2.649
Concentration unit: mg milligram(s)
Concentration type: equal
Concentrati
Sponsors
Bioprojet
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Males or females, aged 18 years old and over. 2. Patients with a diagnosis of narcolepsy according to the International Classification of Sleep Disorders (ICSD-2) criteria. 3. Patients should have complained of EDS with an ESS score ? 12 (historical assessment). 4. Patients having previously participated in and completed a Bioprojet narcolepsy study assessing BF2.649 efficacy (P05-03, P06-06, P07-03 HARMONY I or P07-07 HARMONY II, P09-15 HARMONY I bis) or narcoleptic patients complaining with EDS which in the opinion of the investigator would not be able to participate in a double blind study against placebo but who could benefit from testing a new therapy such as the BF2.649 in an open label study. or patient receiving BF2.649(Pitolisant) under condition of“ATU nominative” according to the French law (called named temporary authorization of use approved by the Afssaps) for Excessive Daytime Sleepiness associated with narcolepsy. 5. Patient must have voluntarily expressed a willingness to participate in this 12-month open label study, signed and dated a new informed consent prior to beginning this protocol required procedures. 6. Females of child-bearing potential must use a medically accepted effective method of birth control, agree to continue this method for the duration of the study and be negative to serum pregnancy test performed at the screening visit. Females should not be breast-feeding patient. 7. 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, drug compliance, scheduled visits, etc). Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 15 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: 1. Patients who have discontinued study treatment during the previous studies due to adverse events related to BF2.649. 2. Patients with an untreated sleep apnoea syndrome or who have any other cause of daytime sleepiness 3. Patients working in an occupation requiring variable shift work or routine night shifts. 4. 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 or comprise reliable representation of subjective symptoms. 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. 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. 7. Patient 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 interval strictly higher than 450 ms (electrocardiogram Bazett’s corrected QT interval (QT /? [60/HR]). 8. Patients with Severe Hepatic Impairment or with Severe Renal Impairment, or with any other significant abnormality in the physical examination or clinical laboratory results. 9. Known hypersensitivity to the tested treatment including active substance and excipients. 10. Patients participating in an other study - in the 30 days prior to the entry in this study - except patient coming from the ongoing study HARMONY I bis sponsorised by bioprojet testing BF2.649 in narcolepsy. 11. Women of childbearing potential who intend to be pregnant during the next year. 12. Patient without any medical care insurance
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •To assess the long-term safety of BF2.649 in the treatment of EDS in narcoleptic patients with or without cataplexy. •To assess the drug-drug interactions with possible concomitant therapies. •To assess the efficacy of long-term therapy with BF2.649 on EDS after one year treatment. ;Secondary Objective: Not applicable;Primary end point(s): The main endpoint of the study will be to estimate the occurrence of Adverse Events and to assess the incidence of the most frequent AEs. -Adverse events are collected throughout the study -Vital sign checking at each visit including blood pressure, heart rate and body weight. -The physical and neurological examination, 12-lead electrocardiograms and clinical laboratory tests (haematology and blood chemistry including renal and hepatic functional panel) are repeated at inclusion visit, 6-month, and 12-month, and at the time of the last on-study visit for any subject who withdraws prior to the completion of the 12-month treatment phase. -BDI (16-item Beck Depression Inventory);Timepoint(s) of evaluation of this end point: These evaluations will be administrated at visits (0, 1 month, 3, 6, 9 and 12 months) based on protocol or at final evaluation in case of drop out for any reason. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Daytime subjective somnolence assessed using the Epworth Sleepiness Scale (ESS). - Physician-evaluated Clinical Global Impression of Severity and of Change (CGI-C and CGI-S on EDS) - European Quality of life questionnaire (EQ-5D) - Information reported by patients daily in sleep diaries - completed 7 days before each visit- including: - Mean number and duration of diurnal involuntary sleep attacks and episodes of severe sleepiness - Frequency of cataplexy attacks by reporting the number of cataplexy attacks. - Frequency of hallucinations, incidence of sleep paralysis. - Number and duration of nocturnal awakening and total duration of nocturnal sleep time. - Patient’s Global Opinion on Effect of treatment - Compliance with study medication;Timepoint(s) of evaluation of this end point: These evaluations will be administrated at each visit (0, 1 month, 3, 6, 9 and 12 months) or final evaluation in case of drop out for any reason. | — |
Countries
Hungary
Contacts
Public ContactProject Manager
Clinical Investigations Ltd
Outcome results
None listed