Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43873   clinical trials with a EudraCT protocol, of which   7293   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2013-001505-93
    Sponsor's Protocol Code Number:P11-11
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2015-09-25
    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 ConcernedFrance - ANSM
    A.2EudraCT number2013-001505-93
    A.3Full title of the trial
    A MULTI-CENTRE, SINGLE DOSE TRIAL TO EVALUATE PHARMACOKINETICS OF PITOLISANT (BF2.649) IN CHILDREN FROM 6 TO LESS THAN 18 YEARS WITH NARCOLEPSY
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to determinate the fate of the drug pitolisant in the body of children from 6 to less than18 years who are suffering from sleepiness and sleep during the day without or with sudden loss of muscle tone without loss of consciousness
    A.3.2Name or abbreviated title of the trial where available
    PITOLISANT PHARMACOKINETICS SINGLE DOSE TRIAL IN CHILDREN FROM 6 TO LESS THAN 18 YEARS OLD
    A.4.1Sponsor's protocol code numberP11-11
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/194/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBioprojet Pharma
    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 Pharma
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBioprojet Pharma
    B.5.2Functional name of contact pointBioprojet clinical department
    B.5.3 Address:
    B.5.3.1Street Address09, rue Rameau
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75002
    B.5.3.4CountryFrance
    B.5.4Telephone number33147036633
    B.5.5Fax number33147036630
    B.5.6E-mailcontact@bioprojet.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 Film-coated 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 903576-44-3
    D.3.9.2Current sponsor codeBF2.649
    D.3.9.4EV Substance CodeSUB29188
    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 18.0
    E.1.2Level PT
    E.1.2Classification code 10028713
    E.1.2Term Narcolepsy
    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 determine the pharmacokinetic parameters of Pitolisant in children, including at least Cmax, Tmax, AUC 0-t and AUC 0-∞, t1/2
    E.2.2Secondary objectives of the trial
    Safety and tolerability assessments:
    - Collection of observed or reported adverse events
    - Physical examination including vital signs to be repeated at each sampling
    - Biological work-up (haematology, biochemistry) at the pre-study screening visit (V0), on D1 before study drug administration for all patients, after treatment at H+10 for children and at H+24 for adolescents, then at V2 follow-up visit for all patients.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    * Male and female patients from 6 to less than 18 years of age
    * Both, de novo or previously diagnosed narcoleptic patients with or without cataplexy could be included. All patients should meet the International Classification of Sleep Disorders (ICSD-2) criteria.
    * Patients who are free of drugs for 3 days prior to study onset (with the exception of a contraceptive, if required for female patients).
    * Patients with a complete physical examination including height (cm), weight (kg), a body mass index between 22 and 30 kg/m², an ECG within the normal range, and laboratory parameters strictly below the upper laboratory norm.
    * Parents – and patients old enough to understand - have expressed a willingness to participate in and complete the study, signed and dated the informed consent form after getting acquainted with the patient’s information sheet, prior to beginning protocol required procedures.
    * In the opinion of the investigator, the patient must have adequate support to comply with the entire study requirements as described in the protocol.
    * Patients with no other morbid pathology than narcolepsy associated or not to cataplexy.
    * Female patients likely to be pregnant shall have a hormonal contraception, and agree to continue this birth control method throughout the study and during the month following treatment discontinuation. They will be negative to the serum pregnancy test performed at the screening visit and on D1.
    * Patients (adolescents) should be non-smokers or smoking less than 10 cigarettes per day.
    * Patients should have normal eating habits
    * Patients should be assured by appropriate health insurance system (only applicable where mandatory e.g. in France).
    * Patients must not participate in another study or be in a follow–up period for another study.
    E.4Principal exclusion criteria
    * The use of BF2.649 within a 30-day period prior to initial screening visit (V0) for this trial.
    * Narcoleptic patients should not have 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 ≥ 5 per hour or/and an Apnea/Hypopnea Index ≥ 5 per hour, periodic limbs movement (PLM) disorders as defined by a PLM arousal index (PLMAI) ≥ 10 per hour, chronic sleep deprivation, circadian sleep wake rhythm disorder or any other medical or neurological causes that could account for narcolepsy symptoms associated with EDS.
    * Patients who are unable or unwilling to temporarily discontinue other drugs or substances.
    * Current or recent (within one year) history of a substance abuse or dependence disorder including alcohol abuse (more than 3 glasses of wine per day) as defined in Diagnostic and Statistical Manual of Mental Disorders (DSM-IV) for adolescents from 12 to less than 18 years of age.
    * Current of recent (within one year) history of drinking excessive amounts of tea, coffee, chocolate and/or beverages containing caffeine (more than 6 cups per day) or nicotine (more than 10 cigarettes/day) for adolescents from 12 to less than 18 years of age.
    * History of severe allergy, asthma, allergic skin rash, or sensitivity to any drug.
    * Severe headaches and/or migraine, recurrent nausea and/or vomiting (more than twice a month).
    * Positive laboratory test for urine drug screening (opiates, barbiturates, amphetamine, cannabis, benzodiazepines, cocaine), and positive alcohol breath test for the group of patients from 12 to less than 18 years.
    * Consumption of citrus fruits and their juices within 3 days before study drug administration
    * Having a requirement of a restricted or special diet which would be non compliant with the study diet
    * If patients of the female sex, pregnancy (defined as positive β-HCG blood test), breast-feeding, or unwilling to have a birth control method assessed to be efficient by the investigator
    * Surgery or blood donation within the one month prior to the start of study
    * Any medication within 3 days before hospitalization, or within 5 times the elimination half-life of that drug, whichever the longest, (with the exception of hormonal contraception).
    * Any clinical condition or prior therapy which, in the investigator’s opinion, makes the patient unsuitable for the study.
    * Any significant serious abnormality of the cardiovascular system, Electrocardiogram Fridericia’s corrected QTc interval (QTcF = QT / 3 60/HR) higher than 450 ms.
    * Patients with any significant abnormality in the physical, psychological, or neurological examinations, or clinical laboratory results.
    * Psychiatric and neurological disorders, such as moderate or severe psychosis or dementia, 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.
    * Active clinically significant illness, including unstable cardiovascular, endocrine, neoplastic, gastrointestinal, haematological, 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.
    * Prior severe adverse reaction to CNS stimulants.
    * Any patient presenting congenital galactosemia, glucose-galactose malabsorption or lactase deficiency due to the presence of lactose in investigational treatments.
    * Any patient having previously participated in a study, the non-inclusion period of which is not over.
    * Cannot be contacted in case of emergency.
    E.5 End points
    E.5.1Primary end point(s)
    To determine the pharmacokinetic parameters of Pitolisant in children, including at least Cmax, Tmax, AUC0-t and AUC0-∞, t1/2
    E.5.1.1Timepoint(s) of evaluation of this end point
    endpoint is evaluated between the H-1 and H+10 for patients from 6 to less than 12 years of -endpoint is evaluated between the H-1 and H+24 for patients from 12 to less than 18 years of age
    E.5.2Secondary end point(s)
    Safety and tolerability assessments:
    - Collection of observed or reported adverse events
    - Physical examination including vital signs to be repeated at each sampling
    - Biological work-up (haematology, biochemistry) at the pre-study screening visit (V0), on D1 before study drug administration for all patients, after treatment at H+10 for children and at H+24 for adolescents, then at V2 follow-up visit for all patients.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary endpoint is evaluated between Day-14 to Day +6.
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic Yes
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    First trial in children and adolescent
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    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.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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
    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 months
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 24
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 12
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 12
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state24
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 24
    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)
    Patients will be included in the next clinical study if they desire.
    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 Decision2013-08-05
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Tue May 07 10:25:55 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA