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    Summary
    EudraCT Number:2021-002637-42
    Sponsor's Protocol Code Number:EP0165
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Not Authorised
    Date on which this record was first entered in the EudraCT database:2021-10-07
    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 ConcernedFrance - ANSM
    A.2EudraCT number2021-002637-42
    A.3Full title of the trial
    An Open-Label, Multicenter, Outpatient Extension Study to Evaluate the Safety and Tolerability of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures
    Étude d’extension en ouvert, multicentrique, en ambulatoire, visant à évaluer la sécurité d’emploi et la tolérabilité de Staccato alprazolam chez des participants à l’étude âgés de 12 ans et plus présentant des crises stéréotypées prolongées
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to test the safety and tolerability of Staccato alprazolam in study participants 12 years of age and older with epilepsy
    Étude visant à évaluer la sécurité d’emploi et la tolérabilité de Staccato alprazolam chez des participants à l’étude âgés de ≥ 12 ans atteints d'épilepsie
    A.4.1Sponsor's protocol code numberEP0165
    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 SponsorUCB Biopharma SRL
    B.1.3.4CountryBelgium
    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 supportUCB Biopharma SRL
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUCB BIOSCIENCES GmbH
    B.5.2Functional name of contact pointClin Trial Reg & Results Disclosure
    B.5.3 Address:
    B.5.3.1Street AddressAlfred-Nobel-Strasse 10
    B.5.3.2Town/ cityMonheim
    B.5.3.3Post code40789
    B.5.3.4CountryGermany
    B.5.6E-mailclinicaltrials@ucb.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameStaccato alprazolam
    D.3.2Product code UCB7538
    D.3.4Pharmaceutical form Inhalation powder
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPInhalation use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNalprazolam
    D.3.9.3Other descriptive nameALPRAZOLAM
    D.3.9.4EV Substance CodeSUB05370MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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 Yes
    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
    Treatment of stereotypical prolonged seizure
    Traitement de la crise stéréotypée prolongée
    E.1.1.1Medical condition in easily understood language
    Epilepsy
    Épilepsie
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10076333
    E.1.2Term Prolonged seizure
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluation of the long-term safety and tolerability of Staccato alprazolam
    Évaluer la sécurité d’emploi et la tolérabilité à long terme de Staccato alprazolam
    E.2.2Secondary objectives of the trial
    - Evaluation of the probability of success of repeated treatment with Staccato alprazolam (for seizures occurring within the first 6 months [up to a maximum of 10 treated seizures])
    - Evaluation of the probability of success of repeated treatment with Staccato alprazolam with no recurrence of seizure(s) up to 2 hours (for seizures occurring within the first 6 months [up to a maximum of 10 treated seizures])
    - Evaluation of the long-term pulmonary safety of Staccato alprazolam
    - Évaluer la probabilité de succès d’un traitement répété par Staccato alprazolam (pour des crises survenant au cours des 6 premiers mois [jusqu’à un maximum de 10 crises traitées])
    - Évaluer la probabilité de succès d’un traitement répété par Staccato alprazolam sans récidive de la(des) crise(s) jusqu’à 2 heures (pour des crises survenant au cours des 6 premiers mois [jusqu’à un maximum de 10 crises traitées])
    - Évaluer la sécurité d’emploi à long terme de Staccato alprazolam sur le plan pulmonaire
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Participant must be ≥12 years of age at the time of signing informed consent (or giving assent, where required)
    - Participant must have a caregiver ≥18 years of age at the time of signing the informed consent; the caregiver(s) must be able to recognize and observe the participant
    - Participants with an established diagnosis of focal or generalized epilepsy or combined focal and generalized epilepsy with a documented history of stereotypical episodes of prolonged seizures that includes at least 1 of the following:
    a) Generalized seizure episodes starting with a flurry of absence seizures or myoclonic seizures with a minimum total duration of 5 minutes
    b) Episodes of a focal seizure with a minimum duration of 3 minutes
    c) Episodes of a focal seizure or myoclonic seizure for at least 90 seconds followed by a generalized/bilateral tonic-clonic seizure with a minimum total duration of 3 minutes
    - Prior to the Screening Visit, participant completed a study using Staccato alprazolam
    - Male and female participants:
    a) A male participant must agree to use contraception during the Treatment Period and for at least 7 days after investigational medicinal product (IMP) administration
    b) A female participant is eligible to participate if she is not pregnant, not breastfeeding, and:
    i) Not a woman of childbearing potential (WOCBP)
    OR
    ii) A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 30 days after IMP administration
    - Participant is capable of giving signed informed consent (or giving assent, where required).
    The informed consent form (ICF) or a specific assent form, where required, will be signed and dated by minors
    - The participant’s caregiver(s) must be capable of giving signed informed, which includes compliance with the requirements and restrictions listed in the ICF, the protocol, and the individualized participant management plan (iPMP)
    - Le(la) participant(e) doit être âgé(e) de ≥ 12 ans au moment de la signature du formulaire de consentement éclairé (FCE) (ou de l’assentiment, lorsque cela est requis)
    - Le(la) participant(e) doit être accompagné(e) d’un aidant âgé de ≥18 ans au moment de la signature du FCE; l’aidant ou les aidants doi(ven)t être capables de reconnaître et d’observer les crises du(de la) participant(e)
    - Participants ayant un diagnostic établi d’épilepsie focale ou généralisée ou d’une épilepsie associant des crises focales et généralisées avec antécédents documentés d’épisodes stéréotypés de crises prolongées incluant au moins 1 des éléments suivants :
    a) Épisodes de crises généralisées commençant par une série de crises de type absence ou crises myocloniques d’une durée totale minimum de 5 minutes
    b) Épisodes de crise focale d’une durée minimum de 3 minutes
    c) Épisodes de crise focale ou de crise myoclonique pendant au moins 90 secondes, suivis d’une crise généralisée/tonico-clonique bilatérale d’une durée totale minimum de 3 minutes
    -Avant la visite de sélection, le(la) participant(e) a terminé une étude utilisant Staccato alprazolam
    -Participants hommes et femmes :
    a)Un homme participant à l’étude doit accepter d’utiliser les moyens de contraception recommandés pendant la période de traitement et pendant au moins 7 jours après l’administration finale du ME et ne pas donner de sperme pendant cette période.
    b) Une femme susceptible de participer à l’étude est éligible pour participer si elle n’est pas enceinte, n’allaite pas et qu’au moins une des conditions suivantes est applicable :
    i) La participante n’est pas une femme en âge de procréer (FAP)
    OU
    ii)La participante est une FAP qui accepte de suivre les recommandations relatives à la contraception pendant la période de traitement et pendant au moins 30 jours après l’administration finale du ME.
    -Le(la) participant(e) a la capacité de donner son FCE signé (ou son assentiment, lorsque cela est requis). Le FCE ou un formulaire spécifique pour les mineurs (assentiment), le cas échéant, sera signé et daté par les mineurs.
    - Le(s) aidant(s) doi(ven)t avoir la capacité de donner leur FCE signé, ce qui inclut le respect des exigences et restrictions indiquées dans le FCE, le protocole et l’iPMP.
    E.4Principal exclusion criteria
    - Participant has a history of convulsive status epilepticus in the 8 weeks prior to the Screening Visit
    - Participant has a history or presence of known nonepileptic seizures which cannot be distinguished from qualifying epileptic seizures
    - Participant has a clinically significant known airway hypersensitivity (eg, bronchospasm to known allergens, such as pollen, animals, or food) and/or acute respiratory signs/symptoms (eg, shortness of breath, wheezing on lung auscultation)
    - Participant has a clinically significant chronic pulmonary disorder (eg, asthma, chronic obstructive pulmonary disease, restrictive lung diseases [including idiopathic pulmonary fibrosis]) and/or recent history or presence of hemoptysis or pneumothorax
    - Participant has a condition for which oral alprazolam is contraindicated (eg, myasthenia gravis, severe respiratory insufficiency, and sleep apnea syndrome)
    - Participant is taking any drug that is a strong CYP3A4 inhibitor, including azole antifungal agents (ketoconazole and itraconazole) and nefazodone
    - Participant is taking any opioids (eg, fentanyl, oxycodone, morphine) or sedative hypnotics on a chronic basis
    - Participant is taking nonselective beta blockers (eg, propranolol, nadolol, and timolol) on a chronic basis
    - Participant has an FEV1 <80 % of predicted forced expiratory volume in 1 second (FEV1) as measured via spirometry at the Screening Visit
    - Participant has an oxygen saturation <95 %
    - Le(la) participant(e) a des antécédents d’état de mal épileptique au cours des 8 semaines ayant précédé la visite de sélection.
    - Le(la) participant(e) a des antécédents ou présence de convulsions non épileptiques connues ne pouvant pas être distinguées des crises épileptiques qualifiantes.
    - Le(la) participant(e) a hypersensibilité connue cliniquement significative des voies aériennes (par exemple, bronchospasme en réaction à des allergènes connus, tels que le pollen, les animaux ou les aliments) et/ou signes/symptômes respiratoires aigus (par exemple, essoufflement, sifflements respiratoires à l’auscultation pulmonaire).
    - Le(la) participant(e) a pneumopathie chronique cliniquement significative (par exemple, asthme, bronchopneumopathie chronique obstructive, pneumopathie restrictive [y compris fibrose pulmonaire idiopathique]) et/ou antécédents récents ou présence d’une hémoptysie ou d'un pneumothorax.
    - Le(la) participant(e) a une pathologie constituant une contre-indication à l’administration d’alprazolam oral (par exemple, myasthénie grave, insuffisance respiratoire sévère, et syndrome d’apnées du sommeil).
    - Le(la) participant(e) n'importe quel médicament constituant un inhibiteur puissant du cytochrome P450 (CYP) 3A4, y compris les agents antifongiques azolés (kétoconazole et itraconazole) et néfazodone
    - Le(la) participant(e) prend d’opiacés (par exemple, fentanyl, oxycodone, morphine) ou de sédatifs hypnotiques de manière chronique
    - Le(la) participant(e) prend de bêta-bloquants non sélectifs (par exemple, propranolol, nadolol et timolol) de manière chronique.
    - Le(la) participant(e) a un volume expiratoire maximum seconde (VEMS) < 80 % du VEMS prédit, mesuré par spirométrie à la visite de sélection.
    - Le(la) participant(e) a la saturation en oxygène < 95 %
    E.5 End points
    E.5.1Primary end point(s)
    1. Frequency of treatment-emergent adverse events (TEAEs)
    2. Frequency of TEAEs leading to withdrawal from study
    3. Frequency of serious TEAEs
    1. Fréquence des EIAT
    2. Fréquence EIAT conduisant au retrait de l’étude
    3. Fréquence des EIAT graves
    E.5.1.1Timepoint(s) of evaluation of this end point
    1&3: From Baseline up to the End of Study Visit (up to 48 months)
    1&3: De l'inclusion jusqu'à la visite de fin de l'étude (jusqu'à 48 mois)
    E.5.2Secondary end point(s)
    1. Treatment success after investigational medicinal product (IMP) administration for seizures occurring within the first 6 months
    2. Treatment success after IMP administration with no recurrence after 2 hours for seizures occurring within first 6 months
    3. Frequency of respiratory TEAEs
    1. Succès thérapeutique après l’administration du ME pour des crises survenant au cours des 6 premiers mois.
    2. Succès thérapeutique après l’administration du ME et absence de récidive de la(des) crise(s) jusqu’à 2 heures après l’administration du ME survenant au cours des 6 premiers mois.
    3. Fréquence des EIAT respiratoires
    E.5.2.1Timepoint(s) of evaluation of this end point
    1&2: From start of IMP treatment up to 6 months
    3: From Baseline up to the End of Study Visit (up to 48 months)
    1&2: Du début du traitement avec ME jusqu'à 6 mois
    3: De l'inclusion jusqu'à la visite de fin de l'étude (jusqu'à 48 mois)
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    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 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.8.2.4Number of treatment arms in the trial1
    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 concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA100
    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
    Australia
    Japan
    Ukraine
    United States
    Bulgaria
    France
    Germany
    Hungary
    Italy
    Poland
    Spain
    United Kingdom
    Czechia
    E.8.7Trial has a data monitoring committee Yes
    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
    la dernière visite du dernier patient
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days10
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days20
    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: 49
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 49
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 236
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 15
    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 200
    F.4.2.2In the whole clinical trial 300
    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
    aucun
    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 Decision2022-01-06
    N.Ethics Committee Opinion of the trial applicationNot-Favourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-04-05
    P. End of Trial
    P.End of Trial StatusNot Authorised
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