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    Summary
    EudraCT Number:2010-023804-28
    Sponsor's Protocol Code Number:P09-10/ BF2.649
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2011-06-10
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2010-023804-28
    A.3Full title of the trial
    An open-label naturalistic pragmatic study to assess the long term safety of BF2.649 (Pitolisant) in the treatment of Excessive Daytime Sleepiness (EDS) in narcolepsy (12 months follow-up).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to assess the safety of BF2.649 (Pitolisant) in the treatment of Excessive Daytime Sleepiness in narcolepsy
    A.3.2Name or abbreviated title of the trial where available
    Harmony III
    A.4.1Sponsor's protocol code numberP09-10/ BF2.649
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBioprojet
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportBioprojet
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationClinical Investigations Ltd
    B.5.2Functional name of contact pointProject Manager
    B.5.3 Address:
    B.5.3.1Street AddressImre u. 4.
    B.5.3.2Town/ cityBudapest
    B.5.3.3Post code1093
    B.5.3.4CountryHungary
    B.5.4Telephone number+3614620385
    B.5.5Fax number+3614620386
    B.5.6E-mailc.leka@clinical-investigations.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/07/459
    D.3 Description of the IMP
    D.3.1Product namePitolisant
    D.3.2Product code BF2.649
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPitolisant
    D.3.9.1CAS number BF2.649
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Narcolepsy
    E.1.1.1Medical condition in easily understood language
    Narcolepsy
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level LLT
    E.1.2Classification code 10028715
    E.1.2Term Narcolepsy with cataplexy
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 13.1
    E.1.2Level PT
    E.1.2Classification code 10028713
    E.1.2Term Narcolepsy
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 13.1
    E.1.2Level LLT
    E.1.2Classification code 10048323
    E.1.2Term Cataplexy aggravated
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 13.1
    E.1.2Level PT
    E.1.2Classification code 10007737
    E.1.2Term Cataplexy
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 13.1
    E.1.2Level LLT
    E.1.2Classification code 10048322
    E.1.2Term Narcolepsy aggravated
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • 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.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal 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).
    E.4Principal 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
    E.5 End points
    E.5.1Primary 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)
    E.5.1.1Timepoint(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.
    E.5.2Secondary 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
    E.5.2.1Timepoint(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.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Tha last visit of the last subject.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days1
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 15
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 100
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Neither specific treatment or care after the subject participation in the trial are expected. It is assumed that the subject will be treated according to the usual physician clinical practice.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2011-07-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-07-13
    P. End of Trial
    P.End of Trial StatusOngoing
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