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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05076617




Registration number
NCT05076617
Ethics application status
Date submitted
30/09/2021
Date registered
13/10/2021
Date last updated
15/10/2024

Titles & IDs
Public title
A Study to Test the Safety and Tolerability of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures
Scientific title
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
Secondary ID [1] 0 0
2021-002637-42
Secondary ID [2] 0 0
EP0165
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Stereotypical Prolonged Seizures 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Staccato alprazolam

Experimental: Staccato alprazolam - Participants will receive Staccato alprazolam by inhalation.


Treatment: Drugs: Staccato alprazolam
* Pharmaceutical form: Inhalation powder
* Route of administration: Inhalation

Participants will receive Staccato alprazolam during the Treatment Period.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Frequency of treatment-emergent adverse events (TEAEs)
Timepoint [1] 0 0
From Baseline up to the End of Study Visit (up to 48 months)
Primary outcome [2] 0 0
Frequency of TEAEs leading to withdrawal from study
Timepoint [2] 0 0
From Baseline up to the End of Study Visit (up to 48 months)
Primary outcome [3] 0 0
Frequency of serious TEAEs
Timepoint [3] 0 0
From Baseline up to the End of Study Visit (up to 48 months)
Secondary outcome [1] 0 0
Treatment success after investigational medicinal product (IMP) administration for seizures occurring within the first 12 months
Timepoint [1] 0 0
From start of IMP treatment up to 12 months
Secondary outcome [2] 0 0
Treatment success after IMP administration with no recurrence after 2 hours for seizures during the first 12 months
Timepoint [2] 0 0
From start of IMP treatment up to 12 months
Secondary outcome [3] 0 0
Frequency of respiratory TEAEs
Timepoint [3] 0 0
From Baseline up to the End of Study Visit (up to 48 months)

Eligibility
Key inclusion criteria
Inclusion criteria:

* Participant must be =12 years of age at the time of signing informed consent
* 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's seizures
* 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:

1. Generalized seizure episodes starting with a flurry of absence seizures or myoclonic seizures with a minimum total duration of 5 minutes
2. Episodes of a focal seizure with a minimum duration of 3 minutes
3. 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 (such as EP0162 (NCT05077904), ENGAGE-E-001 (NCT03478982), or UP0100 (NCT04857307))
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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). NOTE: Participants with mild asthma who qualify for inclusion in the are allowed to be enrolled even though they have known airway hypersensitivity
* 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 severe acute respiratory syndrome coronavirus 2 (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
* 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 or sedative hypnotics on a chronic basis
* Participant is taking nonselective beta blockers on a chronic basis

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/02/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/11/2028
Actual
Sample size
Target
300
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Ep0165 30016 - Fitzroy
Recruitment hospital [2] 0 0
Ep0165 30030 - Herston
Recruitment hospital [3] 0 0
Ep0165 30027 - Melbourne
Recruitment hospital [4] 0 0
Ep0165 30031 - South Brisbane
Recruitment postcode(s) [1] 0 0
- Fitzroy
Recruitment postcode(s) [2] 0 0
- Herston
Recruitment postcode(s) [3] 0 0
- Melbourne
Recruitment postcode(s) [4] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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Arkansas
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California
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Connecticut
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District of Columbia
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Florida
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Hawaii
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Idaho
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Illinois
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Indiana
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Kentucky
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Massachusetts
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Missouri
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New Jersey
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New York
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North Carolina
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Ohio
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Pennsylvania
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Tennessee
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Texas
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Utah
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Bulgaria
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Blagoevgrad
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Bulgaria
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Pazardzhik
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Bulgaria
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Pleven
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Bulgaria
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Sofia
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China
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Beijing
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China
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Changchun
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China
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Chengdu
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China
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Fuzhou
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China
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Guangzhou
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Hangzhou
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China
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Kunming
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China
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Lanzhou
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China
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Nanjing
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China
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Shijiazhuang
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Suzhou
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Tianjin
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Wenzhou
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Yinchuan
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Zhanjiang
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Zhengzhou
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Czechia
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Brno
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Czechia
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Ostrava - Poruba
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Czechia
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Praha 4
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Czechia
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Praha 5
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Czechia
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Praha 6
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Germany
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Berlin
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Germany
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Bielefeld
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Erlangen
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Frankfurt
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Kehl
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München
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Hungary
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Budapest
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Debrecen
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Genova
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Milano
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Italy
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Pavia
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Italy
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Roma
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Japan
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Fukuoka
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Hamamatsu
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Hofu
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Itami
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Kodaira
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Koshi
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Nagakute
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Niigata
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Omura
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Osaka
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Sapporo
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Shinjuku-ku
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Shizuoka
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Suita
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Toon
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Yamagata
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Poland
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Bydgoszcz
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Gdansk
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Krakow
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Lublin
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Nowa Sol
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Swidnik
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Spain
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Barcelona
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Hospitalet de Llobregat
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Madrid
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Spain
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Pamplona
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Sevilla
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Spain
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Terrassa
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Valencia
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Spain
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Valladolid
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United Kingdom
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Birmingham
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Cardiff
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United Kingdom
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Oxford
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United Kingdom
State/province [96] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
UCB Biopharma SRL
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of the study is to evaluate the long-term safety and tolerability of Staccato alprazolam.
Trial website
https://clinicaltrials.gov/study/NCT05076617
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
UCB Cares
Address 0 0
001 844 599 2273 (UCB)
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05076617