Cataplexy in patients with narcolepsy MedDRA version: 9.1 Level: LLT Classification code 10028713 Term: Narcolepsy MedDRA version: 9.1 Level: LLT Classification code 10048322 Term: Narcolepsy aggravated MedDRA version: 9.1 Level: LLT Classification code 10021235 Term: Idiopathic narcolepsy MedDRA version: 9.1 Level: LLT Classification code 10007737 Term: Cataplexy MedDRA version: 9.1 Level: LLT Classification code 10048323 Term: Cataplexy aggravated
Conditions
Sponsors
Bioprojet
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Males and females of any ethnic origin, 18 years old and over. 2. De novo patients i.e. with newly diagnosed narcolepsy and cataplexy and not taking any treatment for EDS and cataplexy. Patients with previously diagnosed narcolepsy and cataplexy and not taking any treatment for EDS and cataplexy for more than 3 months 3. Partial or total cataplexy attacks with a frequency of at least 5 per week during a 14-day baseline period and Epworth Sleepiness Scale (ESS) score = 14/24 at the end of baseline period (V2). 4. The patient has expressed a willingness to participate in and complete the study, and signed and dated informed consent prior to beginning protocol required procedures. 5. Women must be surgically sterile or 2 years postmenopausal. Females of child-bearing potential must use a medically accepted effective method of birth control. These women must use oral contraceptives containing at least 0.05 mg Ethinyl estradiol since the effectiveness of steroidal contraceptives may be reduced when used with Modafinil. The steroidal contraceptives at micro and mini doses should be excluded. Patients should agree to continue this method for the duration of the study and for one month after thediscontinuation of Modafinil treatment. Women should be negative to serum pregnancy test performed at the screening visit. Females should not be breast-feeding patient. 6. 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). 7. Patients should be warned that change in tratment as a consequence of inclusion in this study may reduce their ability to drive as well as his / her impaired ability to operate machinery. If indicated by investigator, the patient must be willing to not operate a car or heavy machinery for the duration of the trial or as long as the investigator deems clinically indicated. In addition, the patient should be willing to maintain during the study their usual behaviours which could affect their diurnal sleepiness (e.g., circadian rhythm, caffeine consumption, nocturnal sleep duration). 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 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. The use of BF2.649 or any previous investigational drugs within 30-day period prior to initial screening visit (V1) for this trial. 2. Patients who are unable or unwilling to temporarily discontinue non authorized drugs or substances. 3. 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). 4. Any significant abnormality in the physical examination or clinical laboratory results (e.g. liver or kidney function deficiency). 5. Patients with severe depression (BDI=16) or with suicidal risk (item G BDI > 0). 6. Any significant serious abnormality of the cardiovascular illness e.g. recent myocardial infarction, angina, hypertension or dysrhythmias (within the prior 6 months), Electrocardiogram Bazett’s corrected QT interval (QT x root from [HR/60]) strickly higher than 450 ms, history of left ventricular hypertrophy or mitral valve prolapse. 7. Patients with Severe hepatic Impairment (e.g. prothrombin ratio < 50% or factor V < 50% for patients receiving anti-vitamin K medication) or with Severe Renal Impairment (e.g. serum creatine greater than 2.0 mg/dl), or with any other significant abnormality in the physical examination or clinical laboratory results. 8. Psychiatric and neurological disorders, such as moderate or severe 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 and completion of this trial or comprise reliable representation of subjective symptoms. 9. Prior severe adverse reactions to CNS stimulants. 10. Known hypersensitivity to the tested treatment including active substance and inactive excipients. 11. Other active clinically significant illness, including unstable cardiovascular, endocrine, neoplastic, gastrointestinal, hematologic, 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. 12. Any patients presenting congenital galactosemia, glucose-galactose malabsorption or lactase deficiency due to the presence of lactose in investigational treatments.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluate and compare the efficacy and safety of escalating doses of BF2.649 and BF2.649 add on Modafinil on cataplexy attacks.;Secondary Objective: Evaluate the additive/synergistic effect of the combination of BF2.649 and Modafinil on excessive daytime sleepiness (EDS), on vigilance/attention, and on changes in disease severity assessed by investigators in patients with narcolepsy.;Primary end point(s): The primary measure of efficacy is the change from baseline in weekly cataplexy attacks. Frequency of cataplexy attacks is collected daily by reporting the number of total and partial cataplexy attacks in patient’s daily sleep diary. The number of weekly cataplexy attacks will be evaluated at baseline and at endpoint corresponding to the end of 8-week double-blind phase or to the time of the last on-study visit for any subject who withdraws prior to study completion. | — |
Countries
France, Germany, Hungary, Netherlands
Outcome results
None listed