Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
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
22/11/2024

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
Country [2] 0 0
United States of America
State/province [2] 0 0
Arkansas
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Connecticut
Country [5] 0 0
United States of America
State/province [5] 0 0
District of Columbia
Country [6] 0 0
United States of America
State/province [6] 0 0
Florida
Country [7] 0 0
United States of America
State/province [7] 0 0
Hawaii
Country [8] 0 0
United States of America
State/province [8] 0 0
Idaho
Country [9] 0 0
United States of America
State/province [9] 0 0
Illinois
Country [10] 0 0
United States of America
State/province [10] 0 0
Indiana
Country [11] 0 0
United States of America
State/province [11] 0 0
Kentucky
Country [12] 0 0
United States of America
State/province [12] 0 0
Louisiana
Country [13] 0 0
United States of America
State/province [13] 0 0
Massachusetts
Country [14] 0 0
United States of America
State/province [14] 0 0
Missouri
Country [15] 0 0
United States of America
State/province [15] 0 0
New Jersey
Country [16] 0 0
United States of America
State/province [16] 0 0
New York
Country [17] 0 0
United States of America
State/province [17] 0 0
North Carolina
Country [18] 0 0
United States of America
State/province [18] 0 0
Ohio
Country [19] 0 0
United States of America
State/province [19] 0 0
Pennsylvania
Country [20] 0 0
United States of America
State/province [20] 0 0
Tennessee
Country [21] 0 0
United States of America
State/province [21] 0 0
Texas
Country [22] 0 0
United States of America
State/province [22] 0 0
Utah
Country [23] 0 0
Bulgaria
State/province [23] 0 0
Blagoevgrad
Country [24] 0 0
Bulgaria
State/province [24] 0 0
Pazardzhik
Country [25] 0 0
Bulgaria
State/province [25] 0 0
Pleven
Country [26] 0 0
Bulgaria
State/province [26] 0 0
Sofia
Country [27] 0 0
China
State/province [27] 0 0
Beijing
Country [28] 0 0
China
State/province [28] 0 0
Changchun
Country [29] 0 0
China
State/province [29] 0 0
Chengdu
Country [30] 0 0
China
State/province [30] 0 0
Fuzhou
Country [31] 0 0
China
State/province [31] 0 0
Guangzhou
Country [32] 0 0
China
State/province [32] 0 0
Hangzhou
Country [33] 0 0
China
State/province [33] 0 0
Kunming
Country [34] 0 0
China
State/province [34] 0 0
Lanzhou
Country [35] 0 0
China
State/province [35] 0 0
Nanjing
Country [36] 0 0
China
State/province [36] 0 0
Shanghai
Country [37] 0 0
China
State/province [37] 0 0
Shijiazhuang
Country [38] 0 0
China
State/province [38] 0 0
Suzhou
Country [39] 0 0
China
State/province [39] 0 0
Tianjin
Country [40] 0 0
China
State/province [40] 0 0
Wenzhou
Country [41] 0 0
China
State/province [41] 0 0
Yinchuan
Country [42] 0 0
China
State/province [42] 0 0
Zhanjiang
Country [43] 0 0
China
State/province [43] 0 0
Zhengzhou
Country [44] 0 0
Czechia
State/province [44] 0 0
Brno
Country [45] 0 0
Czechia
State/province [45] 0 0
Ostrava - Poruba
Country [46] 0 0
Czechia
State/province [46] 0 0
Praha 4
Country [47] 0 0
Czechia
State/province [47] 0 0
Praha 5
Country [48] 0 0
Czechia
State/province [48] 0 0
Praha 6
Country [49] 0 0
Germany
State/province [49] 0 0
Berlin
Country [50] 0 0
Germany
State/province [50] 0 0
Bielefeld
Country [51] 0 0
Germany
State/province [51] 0 0
Erlangen
Country [52] 0 0
Germany
State/province [52] 0 0
Frankfurt
Country [53] 0 0
Germany
State/province [53] 0 0
Kehl
Country [54] 0 0
Germany
State/province [54] 0 0
Marburg
Country [55] 0 0
Germany
State/province [55] 0 0
München
Country [56] 0 0
Hungary
State/province [56] 0 0
Balassagyarmat
Country [57] 0 0
Hungary
State/province [57] 0 0
Budapest
Country [58] 0 0
Hungary
State/province [58] 0 0
Debrecen
Country [59] 0 0
Italy
State/province [59] 0 0
Genova
Country [60] 0 0
Italy
State/province [60] 0 0
Milano
Country [61] 0 0
Italy
State/province [61] 0 0
Pavia
Country [62] 0 0
Italy
State/province [62] 0 0
Roma
Country [63] 0 0
Japan
State/province [63] 0 0
Fukuoka
Country [64] 0 0
Japan
State/province [64] 0 0
Hamamatsu
Country [65] 0 0
Japan
State/province [65] 0 0
Hiroshima
Country [66] 0 0
Japan
State/province [66] 0 0
Hofu
Country [67] 0 0
Japan
State/province [67] 0 0
Itami
Country [68] 0 0
Japan
State/province [68] 0 0
Kodaira
Country [69] 0 0
Japan
State/province [69] 0 0
Koshi
Country [70] 0 0
Japan
State/province [70] 0 0
Nagakute
Country [71] 0 0
Japan
State/province [71] 0 0
Niigata
Country [72] 0 0
Japan
State/province [72] 0 0
Omura
Country [73] 0 0
Japan
State/province [73] 0 0
Osaka
Country [74] 0 0
Japan
State/province [74] 0 0
Sapporo
Country [75] 0 0
Japan
State/province [75] 0 0
Shinjuku-ku
Country [76] 0 0
Japan
State/province [76] 0 0
Shizuoka
Country [77] 0 0
Japan
State/province [77] 0 0
Suita
Country [78] 0 0
Japan
State/province [78] 0 0
Toon
Country [79] 0 0
Japan
State/province [79] 0 0
Yamagata
Country [80] 0 0
Poland
State/province [80] 0 0
Bydgoszcz
Country [81] 0 0
Poland
State/province [81] 0 0
Gdansk
Country [82] 0 0
Poland
State/province [82] 0 0
Krakow
Country [83] 0 0
Poland
State/province [83] 0 0
Lublin
Country [84] 0 0
Poland
State/province [84] 0 0
Nowa Sol
Country [85] 0 0
Poland
State/province [85] 0 0
Swidnik
Country [86] 0 0
Spain
State/province [86] 0 0
Barcelona
Country [87] 0 0
Spain
State/province [87] 0 0
Hospitalet de Llobregat
Country [88] 0 0
Spain
State/province [88] 0 0
Madrid
Country [89] 0 0
Spain
State/province [89] 0 0
Pamplona
Country [90] 0 0
Spain
State/province [90] 0 0
Sevilla
Country [91] 0 0
Spain
State/province [91] 0 0
Terrassa
Country [92] 0 0
Spain
State/province [92] 0 0
Valencia
Country [93] 0 0
Spain
State/province [93] 0 0
Valladolid
Country [94] 0 0
United Kingdom
State/province [94] 0 0
Birmingham
Country [95] 0 0
United Kingdom
State/province [95] 0 0
Cardiff
Country [96] 0 0
United Kingdom
State/province [96] 0 0
Oxford
Country [97] 0 0
United Kingdom
State/province [97] 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