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    Summary
    EudraCT Number:2021-002637-42
    Sponsor's Protocol Code Number:EP0165
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-11-23
    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 ConcernedPoland - Office for Medicinal Products
    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
    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
    A.4.1Sponsor's protocol code numberEP0165
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/220/2022
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboInhalation powder
    D.8.4Route of administration of the placeboInhalation use
    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
    E.1.1.1Medical condition in easily understood language
    Epilepsy
    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 10029205 - Nervous system disorders
    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
    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 12 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 12 months [up to a maximum of 10 treated seizures])
    - Evaluation of the long-term pulmonary safety of 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 study caregiver ≥18 years of age at the time of signing the informed consent; the study 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 a flurry of myoclonic seizures 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)

    Additional inclusion criteria for treatment arms 2 to 5:
    Prior to the Screening Visit, the participant completed the Phase 3
    efficacy study with Staccato alprazolam (EP0162) at a clinical site
    located in the EU, UK, and UA, and experienced an IMP-treated seizure
    during the Outpatient Treatment Period.
    E.4Principal exclusion criteria
    - Participant has a current history of alcohol or drug use disorder, as
    defined in the Diagnostic and Statistical Manual of Mental Disorders 5,
    within the previous 1 year
    - Participant has a known hypersensitivity to any components of the
    investigational medicinal product (IMP) or comparable drugs (and/or an
    investigational device) as stated in this protocol or to albuterol (or
    similar bronchospasm rescue medication if needed to meet country-
    specific requirements)
    - Participant has a history of convulsive (generalized tonic-clonic) 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 other
    than mild asthma (eg, chronic obstructive pulmonary disease, restrictive
    lung diseases [including idiopathic pulmonary fibrosis]) and/or recent
    history or presence of hemoptysis or pneumothorax
    - Participant has had a positive antigen test for SARS-CoV-2 and
    experienced moderate to severe signs/symptoms of respiratory distress
    necessitating hospitalization or outpatient treatment such as ambulatory
    oxygen, extensive treatment with inhaler medications, and/or oral
    medications for a duration of 4 weeks or more, unless full resolution
    occurred at least 6 months prior to Screening
    - Participant has experienced a severe upper respiratory tract infection
    within 4 weeks or severe bronchitis/pneumonia within 3 months before
    the Screening Visit
    - Participant has a history or presence of acute narrow-angle glaucoma
    - Participant has a condition for which oral alprazolam is contraindicated
    (eg, myasthenia gravis, severe respiratory insufficiency, and sleep apnea
    syndrome)
    - Participant has a history or presence of long QT syndrome, a family
    history of sudden death due to long QT syndrome, or unexplained
    syncope
    - 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 on a chronic basis
    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
    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)
    E.5.2Secondary end point(s)
    1. Treatment success after investigational medicinal product (IMP) administration for seizures occurring within the first 12 months
    2. Treatment success after IMP administration with no recurrence after 2 hours for seizures occurring within first 12 months
    3. Frequency of respiratory TEAEs
    E.5.2.1Timepoint(s) of evaluation of this end point
    1&2: From start of IMP treatment up to 12 months
    3: From Baseline up to the End of Study Visit (up to 48 months)
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    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 trial5
    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 concerned9
    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
    Ukraine
    Australia
    China
    Japan
    United Kingdom
    United States
    Bulgaria
    Czechia
    Germany
    Hungary
    Italy
    Poland
    Spain
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days6
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days25
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Study population includes minors and cognitively impaired adults.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state
    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
    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 Opinion2021-10-26
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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