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    Summary
    EudraCT Number:2012-003076-39
    Sponsor's Protocol Code Number:P11-05
    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:2012-08-13
    Trial 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 number2012-003076-39
    A.3Full title of the trial
    A randomized, double blind study comparing BF2.649 (Pitolisant) to placebo in two parallel groups on the weekly frequency of cataplexy attacks and Excessive Daytime Sleepiness in narcoleptic patients with cataplexy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    NA
    A.3.2Name or abbreviated title of the trial where available
    NA
    A.4.1Sponsor's protocol code numberP11-05
    A.5.4Other Identifiers
    Name:abbreviated nameNumber: HARMONY CTP
    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 organisationBioprojet
    B.5.2Functional name of contact pointBioprojet clinical department
    B.5.3 Address:
    B.5.3.1Street Address9 rue Rameau
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75002
    B.5.3.4CountryFrance
    B.5.4Telephone number0033147036633
    B.5.5Fax number033147036630
    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 Capsule, hard
    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 903576-44-3
    D.3.9.2Current sponsor codeBF2.649
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNpitolisant
    D.3.9.1CAS number 903576-44-3
    D.3.9.2Current sponsor codeBF2.649
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNpitolisant
    D.3.9.1CAS number 903576-44-3
    D.3.9.2Current sponsor codeBF2.649
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Narcolepsy with Cataplexy
    E.1.1.1Medical condition in easily understood language
    Narcolepsy with cataplexy
    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 14.1
    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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    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.
    E.2.2Secondary objectives of the trial
    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)
    E.2.3Trial contains a sub-study No
    E.3Principal 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.
    E.4Principal 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
    E.5 End points
    E.5.1Primary 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)
    E.5.1.1Timepoint(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).
    E.5.2Secondary 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)
    E.5.2.1Timepoint(s) of evaluation of this end point
    described in section E.5.2
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA9
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Bulgaria
    Czech Republic
    Hungary
    Macedonia, the former Yugoslav Republic of
    Poland
    Russian Federation
    Serbia
    Turkey
    Ukraine
    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
    LVLS
    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 months0
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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: 110
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 120
    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)
    None
    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 Decision2012-10-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-09-13
    P. End of Trial
    P.End of Trial StatusOngoing
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