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    Summary
    EudraCT Number:2021-002637-42
    Sponsor's Protocol Code Number:EP0165
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-10-04
    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 ConcernedSpain - AEMPS
    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
    Estudio de extensión, abierto, multicéntrico y en pacientes ambulatorios, para evaluar la seguridad y la tolerabilidad de Staccato® Alprazolam en participantes en el estudio de 12 o más años de edad con estereotipias prolongadas
    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
    Estudio para evaluar la seguridad y la tolerabilidad de Staccato® alprazolam en participantes en el estudio >=12 años de edad con epilepsia
    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
    Tratamiento de episodios de estereotipia prolongada
    E.1.1.1Medical condition in easily understood language
    Epilepsy
    Epilepsia
    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
    Evaluar la seguridad y tolerabilidad a largo plazo 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
    - Evaluar la probabilidad de éxito del tratamiento repetido con Staccato® alprazolam (en los episodios producidos en los 6 primeros meses [con un máximo de 10 episodios tratados])
    - Evaluar la probabilidad de éxito del tratamiento repetido con Staccato® alprazolam sin recurrencia del episodio(s) en el plazo de 2 horas (en los episodios producidos en los 6 primeros meses [con un máximo de 10 episodios tratados])
    - Evaluar la seguridad pulmonar a largo plazo de Staccato® alprazolam
    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)
    - El participante debe tener >=12 años de edad en el momento de firmar el consentimiento informado (o de dar su asentimiento, cuando proceda).
    - El participante debe tener un cuidador >=18 años de edad en el momento de firmar el ICF; el cuidador(es) debe ser capaz de reconocer y observar las crisis del participante.
    - Participante con diagnóstico establecido de epilepsia focal o generalizada o de epilepsia focal y generalizada combinada, con historia documentada de episodios de estereotipia prolongados que incluyen como mínimo 1 de lo siguiente:
    a) Episodios de crisis generalizadas que comienzan con un racimo de crisis de ausencia o de crisis mioclónicas con una duración total mínima de 5 minutos.
    b) Episodios de una crisis focal con una duración mínima de 3 minutos.
    c) Episodios de una crisis focal o mioclónica de como mínimo 90 segundos seguida por crisis tónico-clónica generalizada/bilateral con una duración total mínima de 3 minutos.
    - Antes de la visita de Selección, el participante ha completado un estudio con Staccato® alprazolam
    - Participantes de ambos sexos
    a) Si se trata de un participante masculino, deberá estar de acuerdo en seguir las recomendaciones acerca de anticoncepción durante el Periodo de Tratamiento y como mínimo los 7 días siguientes a la última administración del IMP.
    b) Las mujeres serán elegibles para participar si no están embarazadas o practicando la lactancia natural y:
    i) No se trata de una mujer potencialmente fértil (woman of childbearing potential, WOCBP)
    O
    ii) Se trata de una WOCBP que está de acuerdo en seguir las recomendaciones acerca de anticoncepción durante el Periodo de Tratamiento y como mínimo los 30 días siguientes a la última administración del IMP.
    - Participante que es capaz de firmar el ICF (o de dar su asentimiento, según proceda). Los menores firmarán y fecharán el ICF o el documento específico de asentimiento, según proceda.
    - Cuidador(es) del participante que es capaz de firmar el ICF, lo que incluye el cumplimiento de los requisitos y restricciones que se señalan en el ICF, el protocolo y el 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 %
    - Participante con antecedente de status epilepticus convulsivo en las 8 semanas previas a la Visita de Selección.
    - Participante con historia o presencia de crisis no epilépticas conocidas que no pueden diferenciarse de las crisis epilépticas cualificadoras.
    - Participante con hipersensibilidad conocida de las vías aéreas clínicamente importante (por ejemplo, broncoespasmo a alérgenos conocidos, como polen, animales o alimentos) y/o signos/síntomas respiratorios agudos (por ejemplo, disnea, ruidos torácicos en la auscultación pulmonar).
    - Participante que tiene un trastorno pulmonar crónico clínicamente importante (por ejemplo, asma, enfermedad pulmonar obstructiva crónica, enfermedad pulmonar restrictiva [incluida la fibrosis pulmonar idiopática]) y/o historia reciente o presencia de hemoptisis o neumotórax.
    - Participante que presenta un proceso para el que está contraindicado el alprazolam oral (por ejemplo, miastenia gravis, insuficiencia respiratoria severa y síndrome de apnea del sueño).
    - Participante que está en tratamiento con un medicamento que es un inhibidor potente de la isoenzima 3A4 del citocromo P450 (CYP), incluidos los azoles antifúngicos (ketoconazol e itraconazol) y la nefazodona
    - Participante que está en tratamiento crónico con opioides (por ejemplo, fentanilo, oxicodona, morfina) o hipnóticos sedantes.
    - Participante que está en tratamiento crónico con betabloqueantes no selectivos (por ejemplo, propranolol, nadolol y timolol).
    - Participante que presenta un volumen espiratorio forzado en un 1 segundo (forced expiratory volume in 1 second, FEV1) <80% del teórico en la espirometría de la Visita de Selección.
    - Participante con una saturación de oxígeno <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. Frecuencia de acontecimientos adversos emergentes durante el tratamiento (treatment-emergent adverse events, TEAEs)
    2. Frecuencia de TEAEs que resulten en el abandono del tratamiento
    3. Frecuencia de TEAEs 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 y 3: desde la visita basal hasta la visita de fin del estudio (hasta 48 meses)
    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. Éxito del tratamiento tras la administración del IMP en los episodios producidos en los 6 primeros meses
    2. Éxito del tratamiento tras la administración del IMP sin recurrencia del episodio(s) en el plazo de 2 horas en los episodios producidos en los 6 primeros meses
    3. Frecuencia de TEAEs respiratorios
    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 y 2: desde el inicio del tratamiento con IMP hasta los 6 meses
    3: desde la visita basal hasta la visita de fin del estudio (hasta 48 meses)
    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
    Tolerabilidad
    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 concerned6
    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
    última visita del último paciente
    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 days21
    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 state20
    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
    Ninguno
    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 Decision2022-02-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-19
    P. End of Trial
    P.End of Trial StatusOngoing
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