A Study to Test the Safety and Tolerability of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures

Phase 3

• Conditions: Stereotypical Prolonged Seizures
• Interventions: Drug: Staccato alprazolam

• Registration Number: NCT05076617
• Lead Sponsor: UCB Biopharma SRL

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-09-30
• Study First Submitted QC: 2021-09-30
• Study First Posted: 2021-10-13
• Study Start: 2022-02-03
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-31
• Last Update Posted: 2025-08-01
• Primary Completion: 2028-11-30
• Study Completion: 2028-11-30

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 300

Inclusion Criteria

Not provided

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

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Staccato alprazolam	Staccato alprazolam	Participants will receive Staccato alprazolam by inhalation.

Primary Outcome Measures

Name	Time	Method
Frequency of treatment-emergent adverse events (TEAEs)	From Baseline up to the End of Study Visit (up to 48 months)	An Adverse event (AE) is defined as any untoward medical occurrence, unintended disease or injury, or untoward clinical signs (including abnormal laboratory finding) in study participants, users, or other persons, whether or not related to the investigational medical product (IMP) and whether anticipated or unanticipated. A treatment-emergent adverse event (TEAE) is defined as any AE with a start date/time on or after the first IMP administration.
Frequency of serious TEAEs	From Baseline up to the End of Study Visit (up to 48 months)	A serious adverse event (SAE) is any untoward medical occurrence that at any dose: * Results in death; * Is life-threatening; * Requires inpatient hospitalisation or prolongation of existing hospitalisation; * Results in persistent or significant disability/incapacity, or; * Is a congenital anomaly/birth defect; * Other important medical events which based on medical or scientific judgement may jeopardize the patients, or may require medical or surgical intervention to prevent any of the above
Frequency of TEAEs leading to withdrawal from study	From Baseline up to the End of Study Visit (up to 48 months)	An Adverse event (AE) is defined as any untoward medical occurrence, unintended disease or injury, or untoward clinical signs (including abnormal laboratory finding) in study participants, users, or other persons, whether or not related to the investigational medical product (IMP) and whether anticipated or unanticipated. A treatment-emergent adverse event (TEAE) is defined as any AE with a start date/time on or after the first IMP administration.

Secondary Outcome Measures

Name	Time	Method
Treatment success after IMP administration with no recurrence after 2 hours for seizures during the first 12 months	From start of IMP treatment up to 12 months	A responder after up to a maximum of 10 treated seizures will be defined as termination of seizure within 90 seconds after IMP administration and no recurrence of seizure(s) from IMP administration to 2 hours after IMP administration and no initiation of seizure rescue treatment from 90 seconds to 2 hours after IMP administration.
Treatment success after investigational medicinal product (IMP) administration for seizures occurring within the first 12 months	From start of IMP treatment up to 12 months	A responder after up to a maximum of 10 treated seizures will be defined as having a termination of seizure within 90 seconds after IMP dministration.
Frequency of respiratory TEAEs	From Baseline up to the End of Study Visit (up to 48 months)	An Adverse event (AE) is defined as any untoward medical occurrence, unintended disease or injury, or untoward clinical signs (including abnormal laboratory finding) in study participants, users, or other persons, whether or not related to the investigational medical product (IMP) and whether anticipated or unanticipated. A treatment-emergent adverse event (TEAE) is defined as any AE with a start date/time on or after the first IMP administration.

Trial Locations

Locations (135)

Ep0165 50506; 🇺🇸 Phoenix, Arizona, United States

Ep0165 50494; 🇺🇸 Little Rock, Arkansas, United States

Ep0165 50118; 🇺🇸 Downey, California, United States

Ep0165 50702; 🇺🇸 Long Beach, California, United States

Ep0165 50492; 🇺🇸 Orange, California, United States

Ep0165 50367; 🇺🇸 New Haven, Connecticut, United States

Ep0165 50088; 🇺🇸 Washington, District of Columbia, United States

Ep0165 50515; 🇺🇸 Gulf Breeze, Florida, United States

Ep0165 50508; 🇺🇸 Jacksonville, Florida, United States

Ep0165 50342; 🇺🇸 Jacksonville, Florida, United States

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