Clinical trial NCT05076617: A Study to Test the Safety and Tolerability of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures
Official 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
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Stereotypical Prolonged Seizures
Keywords: Stereotypical prolonged seizures
Phase 3
Staccato alprazolam
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2021-10-13
Enrollment: 300
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to evaluate the long-term safety and tolerability of Staccato alprazolam.


Eligibility 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))

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

Primary outcomes:
measure: Frequency of treatment-emergent adverse events (TEAEs)
timeFrame: From Baseline up to the End of Study Visit (up to 48 months)
measure: Frequency of TEAEs leading to withdrawal from study
timeFrame: From Baseline up to the End of Study Visit (up to 48 months)
measure: Frequency of serious TEAEs
timeFrame: From Baseline up to the End of Study Visit (up to 48 months)
Secondary outcomes:
measure: Treatment success after investigational medicinal product (IMP) administration for seizures occurring within the first 12 months
timeFrame: From start of IMP treatment up to 12 months
measure: Treatment success after IMP administration with no recurrence after 2 hours for seizures during the first 12 months
timeFrame: From start of IMP treatment up to 12 months
measure: Frequency of respiratory TEAEs
timeFrame: From Baseline up to the End of Study Visit (up to 48 months)
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Ep0165 50506
state: Arizona
city: Little Rock
country: United States
facility: Ep0165 50494
state: Arkansas
city: Downey
country: United States
facility: Ep0165 50118
state: California
city: Long Beach
country: United States
facility: Ep0165 50702
state: California
city: Los Angeles
country: United States
facility: Ep0165 50505
state: California
city: Orange
country: United States
facility: Ep0165 50492
state: California
city: New Haven
country: United States
facility: Ep0165 50367
state: Connecticut
city: Washington
country: United States
facility: Ep0165 50088
state: District of Columbia
city: Gulf Breeze
country: United States
facility: Ep0165 50515
state: Florida
city: Jacksonville
country: United States
facility: Ep0165 50508
state: Florida
city: Jacksonville
country: United States
facility: Ep0165 50342
state: Florida
city: Miami
country: United States
facility: Ep0165 50199
state: Florida
city: Miami
country: United States
facility: Ep0165 50676
state: Florida
city: Orlando
country: United States
facility: Ep0165 50509
state: Florida
city: Rockledge
country: United States
facility: Ep0165 50703
state: Florida
city: Tampa
country: United States
facility: Ep0165 50308
state: Florida
city: Honolulu
country: United States
facility: Ep0165 50323
state: Hawaii
city: Boise
country: United States
facility: Ep0165 50512
state: Idaho
city: Chicago
country: United States
facility: Ep0165 50493
state: Illinois
city: Springfield
country: United States
facility: Ep0165 50375
state: Illinois
city: Fort Wayne
country: United States
facility: Ep0165 50504
state: Indiana
city: Lexington
country: United States
facility: Ep0165 50561
state: Kansas
city: New Orleans
country: United States
facility: Ep0165 50517
state: Louisiana
city: Boston
country: United States
facility: Ep0165 50047
state: Massachusetts
city: Saint Louis
country: United States
facility: Ep0165 50507
state: Missouri
city: Neptune
country: United States
facility: Ep0165 50626
state: New Jersey
city: New Brunswick
country: United States
facility: Ep0165 50299
state: New Jersey
city: Amherst
country: United States
facility: Ep0165 50497
state: New York
city: New York
country: United States
facility: Ep0165 50298
state: New York
city: New York
country: United States
facility: Ep0165 50490
state: New York
city: New York
country: United States
facility: Ep0165 50518
state: New York
city: Rochester
country: United States
facility: Ep0165 50034
state: New York
city: Charlotte
country: United States
facility: Ep0165 50514
state: North Carolina
city: Akron
country: United States
facility: Ep0165 50371
state: Ohio
city: Cleveland
country: United States
facility: Ep0165 50528
state: Ohio
city: Philadelphia
country: United States
facility: Ep0165 50096
state: Pennsylvania
city: Philadelphia
country: United States
facility: Ep0165 50364
state: Pennsylvania
city: Philadelphia
country: United States
facility: Ep0165 50089
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Ep0165 50511
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Ep0165 50491
state: Pennsylvania
city: Memphis
country: United States
facility: Ep0165 50513
state: Tennessee
city: Nashville
country: United States
facility: Ep0165 50103
state: Tennessee
city: Houston
country: United States
facility: Ep0165 50525
state: Texas
city: Salt Lake City
country: United States
facility: Ep0165 50473
state: Utah
city: Fitzroy
country: Australia
facility: Ep0165 30016
city: Herston
country: Australia
facility: Ep0165 30030
city: Melbourne
country: Australia
facility: Ep0165 30027
city: South Brisbane
country: Australia
facility: Ep0165 30031
city: Blagoevgrad
country: Bulgaria
facility: Ep0165 40650
city: Pazardzhik
country: Bulgaria
facility: Ep0165 40708
city: Pleven
country: Bulgaria
facility: Ep0165 40665
city: Pleven
country: Bulgaria
facility: Ep0165 40709
city: Sofia
country: Bulgaria
facility: Ep0165 40651
city: Beijing
country: China
facility: Ep0165 20128
city: Beijing
country: China
facility: Ep0165 20246
city: Beijing
country: China
facility: Ep0165 20268
city: Beijing
country: China
facility: Ep0165 20299
city: Changchun
country: China
facility: Ep0165 20261
city: Chengdu
country: China
facility: Ep0165 20133
city: Chengdu
country: China
facility: Ep0165 20137
city: Chongqing
country: China
facility: Ep0165 20334
city: Fuzhou
country: China
facility: Ep0165 20179
city: Guangzhou
country: China
facility: Ep0165 20124
city: Guangzhou
country: China
facility: Ep0165 20260
city: Guangzhou
country: China
facility: Ep0165 20264
city: Guangzhou
country: China
facility: Ep0165 20269
city: Guangzhou
country: China
facility: Ep0165 20300
city: Hangzhou
country: China
facility: Ep0165 20022
city: Kunming
country: China
facility: Ep0165 20320
city: Lanzhou
country: China
facility: Ep0165 20258
city: Nanjing
country: China
facility: Ep0165 20267
city: Nanning
country: China
facility: Ep0165 20331
city: Qingdao
country: China
facility: Ep0165 20333
city: Shanghai
country: China
facility: Ep0165 20292
city: Shenzhen
country: China
facility: Ep0165 20332
city: Shijiazhuang
country: China
facility: Ep0165 20289
city: Suzhou
country: China
facility: Ep0165 20119
city: Tianjin
country: China
facility: Ep0165 20257
city: Wenzhou
country: China
facility: Ep0165 20025
city: Yinchuan
country: China
facility: Ep0165 20255
city: Zhanjiang
country: China
facility: Ep0165 20262
city: Zhengzhou
country: China
facility: Ep0165 20251
city: Brno
country: Czechia
facility: Ep0165 40670
city: Ostrava - Poruba
country: Czechia
facility: Ep0165 40672
city: Praha 4
country: Czechia
facility: Ep0165 40714
city: Praha 5
country: Czechia
facility: Ep0165 40063
city: Praha 6
country: Czechia
facility: Ep0165 40671
city: Berlin
country: Germany
facility: Ep0165 40683
city: Bielefeld
country: Germany
facility: Ep0165 40685
city: Erlangen
country: Germany
facility: Ep0165 40023
city: Frankfurt
country: Germany
facility: Ep0165 40645
city: Kehl
country: Germany
facility: Ep0165 40689
city: Marburg
country: Germany
facility: Ep0165 40529
city: München
country: Germany
facility: Ep0165 40724
city: Balassagyarmat
country: Hungary
facility: Ep0165 40666
city: Budapest
country: Hungary
facility: Ep0165 40673
city: Budapest
country: Hungary
facility: Ep0165 40704
city: Debrecen
country: Hungary
facility: Ep0165 40653
city: Genova
country: Italy
facility: Ep0165 40674
city: Milano
country: Italy
facility: Ep0165 40144
city: Pavia
country: Italy
facility: Ep0165 40477
city: Roma
country: Italy
facility: Ep0165 40257
city: Roma
country: Italy
facility: Ep0165 40675
city: Fukuoka
country: Japan
facility: Ep0165 20248
city: Hamamatsu
country: Japan
facility: Ep0165 20237
city: Hiroshima
country: Japan
facility: Ep0165 20249
city: Hofu
country: Japan
facility: Ep0165 20236
city: Itami
country: Japan
facility: Ep0165 20239
city: Kodaira
country: Japan
facility: Ep0165 20143
city: Koshi
country: Japan
facility: Ep0165 20315
city: Kyoto
country: Japan
facility: Ep0165 20147
city: Nagakute
country: Japan
facility: Ep0165 20243
city: Niigata
country: Japan
facility: Ep0165 20238
city: Omura
country: Japan
facility: Ep0165 20241
city: Osaka
country: Japan
facility: Ep0165 20302
city: Sapporo
country: Japan
facility: Ep0165 20316
city: Shinjuku-ku
country: Japan
facility: Ep0165 20070
city: Shinjuku-ku
country: Japan
facility: Ep0165 20297
city: Shizuoka
country: Japan
facility: Ep0165 20240
city: Suita
country: Japan
facility: Ep0165 20242
city: Toon
country: Japan
facility: Ep0165 20266
city: Yamagata
country: Japan
facility: Ep0165 20244
city: Bydgoszcz
country: Poland
facility: Ep0165 40707
city: Gdansk
country: Poland
facility: Ep0165 40677
city: Krakow
country: Poland
facility: Ep0165 40502
city: Lublin
country: Poland
facility: Ep0165 40676
city: Nowa Sól
country: Poland
facility: Ep0165 40091
city: Swidnik
country: Poland
facility: Ep0165 40678
city: Barcelona
country: Spain
facility: Ep0165 40160
city: Hospitalet de Llobregat
country: Spain
facility: Ep0165 40157
city: Madrid
country: Spain
facility: Ep0165 40540
city: Pamplona
country: Spain
facility: Ep0165 40352
city: Sevilla
country: Spain
facility: Ep0165 40668
city: Terrassa
country: Spain
facility: Ep0165 40453
city: Valencia
country: Spain
facility: Ep0165 40230
city: Valladolid
country: Spain
facility: Ep0165 40667
city: Birmingham
country: United Kingdom
facility: Ep0165 40686
city: Cardiff
country: United Kingdom
facility: Ep0165 40300
city: Oxford
country: United Kingdom
facility: Ep0165 40163
city: Salford
country: United Kingdom
facility: Ep0165 40108
Study officials:
affiliation: 001 844 599 2273 (UCB)
name: UCB Cares
role: STUDY_DIRECTOR
References: