Recruiting

Phase 3

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

Sponsor:

UCB Biopharma SRL

Code:

NCT05077904

Conditions

Stereotypical Prolonged Seizures

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

Interventions

Staccato alprazolam

Placebo

Study Details

Brief summary:

The purpose of the study is to assess the success of a single administration of Staccato alprazolam compared with placebo both in rapidly terminating a seizure episode within 90 seconds and with no recurrence of seizure(s) up to 2 hours after investigational medicinal product (IMP) administration.

Conditions

Stereotypical Prolonged Seizures

Study ID

NCT05077904

Start date

Dec 7, 2021

Status verified date

Aug, 2026

Completion date

Sep 30, 2026

Anticipated

Primary completion date

Sep 30, 2026

Anticipated

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

Inclusion Criteria:

Participant must be ≥12 years of age at the Baseline/Randomization Visit
Participant must have a study caregiver ≥18 years of age at the Screening Visit; the study caregiver(s) must be a relative, partner, friend, or legally authorized representative (LAR) of the participant, or a person who provides daily care to the participant and has a significant personal relationship with the participant; 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 has experienced ≥4 stereotypical episodes of prolonged seizures in the past 6 months, and the last 2 stereotypical episodes of prolonged seizures must have occurred within the 3 months prior to the Screening Visit
Participant has had a documented brain computerized tomography or magnetic resonance imaging review, performed after diagnosis of epilepsy and within the 5 years prior to the Screening Visit, that confirms the absence of a progressive neurological disorder
Participant is receiving a regimen of antiseizure medications (ASMs) that has been stable (ie, no addition or removal of ASM\[s\]; dose adjustments are permitted to ASM\[s\]; dose adjustments are not permitted for benzodiazepines) for 30 days prior to the Screening Visit
Male and female participants:

1. A male participant must agree to use contraception during the Outpatient Treatment Period and for at least 7 days after IMP administration and refrain from donating sperm during this period
2. A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least 1 of the following conditions applies:

i) Not a woman of childbearing potential (WOCBP) OR ii) A WOCBP who agrees to follow the contraceptive guidance during the Outpatient Treatment Period and for at least 30 days after IMP administration

Participant is capable of giving signed informed consent (or giving assent, where required), which includes compliance with the requirements and restrictions listed in the informed consent form (ICF), the protocol, and the individualized participant management plan (iPMP). The ICF or a specific assent form, where required, will be signed and dated by minors
The participant's study caregiver(s) must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF, the protocol, and the iPMP

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 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 diagnosis of atrial fibrillation or mitral stenosis
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 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 upper respiratory tract infection within 4 weeks or 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
Chronic use of benzodiazepines for more than 3 days within a period of 7 days will be allowed for approximately 30 % of study participants
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
Participant is taking pharmacotherapy for an active major psychiatric disorder where major changes in regimen are needed or anticipated during the study
Participant has been treated with vagal nerve stimulation (VNS) for less than 6 months or VNS settings have changed within 30 days before the Screening Visit
Participant has a clinically significant laboratory abnormality that may increase the risk associated with study participation or may interfere with the interpretation of study results, according to the judgment of the Investigator
Participant has an oxygen saturation <95 % (or less than normal in regions of altitude >2500 meters) for greater than 30 seconds during the Screening Visit. In case of an out-of-range result, 1 repeat will be allowed. If the readings are out of range again, the study participant will be excluded
Participant has >2.0x upper limit of normal (ULN) of any of the following: alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP), or >1.0xULN total bilirubin (≥1.5xULN total bilirubin if known Gilbert's syndrome or >2.0xULN total bilirubin for liver impairment)
Participant has current unstable liver or biliary disease per Investigator assessment defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis
Participant has a QT interval corrected for heart rate (QTc) >450 msec (males), QTc interval >470 msec (females), or QTc interval >480 msec (participants with bundle branch block), PR interval ≥220 msec, or any other clinically significant electrocardiogram (ECG) abnormality according to the Investigator i) The QTc is the QT interval corrected for heart rate according to Fridericia's formula (QTcF). It is either machine-read or manually over-read
Participant has a positive urine screen for drugs of abuse at the Screening Visit
Participant has a blood pressure (BP) or heart rate (HR) outside the following range after 5 minutes rest: systolic BP: 90 mmHg to 150 mmHg; diastolic BP: 4 0mmHg to 95 mmHg; HR: 50 bpm to 100 bpm. In case of an out-of-range result, 1 repeat will be allowed. If the readings are out of range again, the study participant will be excluded

Study Design

Enrollment

350 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Staccato alprazolam Arm

Participants randomized to this arm will receive a single dose of Staccato alprazolam by inhalation.

placebo comparator: Placebo Arm

Participants randomized to this arm will receive a single dose of placebo by inhalation.

Interventions

Staccato alprazolam

Route of administration: Inhalation

Participants will receive one dose of Staccato alprazolam during the Treatment Period.

Placebo

Route of administration: Inhalation

Participants will receive one dose of placebo during the Treatment Period.

Primary outcome measure

Treatment success for the treated seizure within 90 seconds after investigational medicinal product (IMP) administration [ Time Frame: From start of IMP treatment through 6 hours ]
Treatment success for the treated seizure with no recurrence up to 2 hours [ Time Frame: From start of IMP treatment through 2 hours ]

Central Contacts and Locations

Central contacts

UCB Cares

1-844-599-2273 (USA)UCBCares@ucb.com

UCB Cares

0018445992273 UCBCares@ucb.com

Locations

Ep0162 50506

Recruiting

Phoenix, Arizona, United States, 85004

Ep0162 50720

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Scottsdale, Arizona, United States, 85253

Ep0162 50118

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Downey, California, United States, 90242

Ep0162 50416

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La Jolla, California, United States, 92037

Ep0162 50702

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Long Beach, California, United States, 90806

Ep0162 50716

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Sacramento, California, United States, 95817

Ep0162 50088

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Washington D.C., District of Columbia, United States, 20037

Ep0162 50508

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Jacksonville, Florida, United States, 32209-6533

Ep0162 50342

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Jacksonville, Florida, United States, 32224-1865

Ep0162 50199

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Miami, Florida, United States, 33136

Ep0162 50676

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Miami, Florida, United States, 33176

Ep0162 50703

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Rockledge, Florida, United States, 32955

Ep0162 50308

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Tampa, Florida, United States, 33606

Ep0162 50323

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Honolulu, Hawaii, United States, 96817

Ep0162 50512

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Boise, Idaho, United States, 83702

Ep0162 50493

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Chicago, Illinois, United States, 60611

Ep0162 50504

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Fort Wayne, Indiana, United States, 46804

Ep0162 50517

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New Orleans, Louisiana, United States, 70122

Ep0162 50615

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Boston, Massachusetts, United States, 02118

Ep0162 50507

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St Louis, Missouri, United States, 63110

Ep0162 50299

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New Brunswick, New Jersey, United States, 08901

Ep0162 50497

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Buffalo, New York, United States, 14221

Ep0162 50625

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New Hyde Park, New York, United States, 11042

Ep0162 50298

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New York, New York, United States, 10016

Ep0162 50490

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New York, New York, United States, 10029

Ep0162 50518

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New York, New York, United States, 10075

Ep0162 50034

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Rochester, New York, United States, 14642

Ep0162 50514

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Charlotte, North Carolina, United States, 28204

Ep0162 50715

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Durham, North Carolina, United States, 27705

Ep0162 50701

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Portland, Oregon, United States, 97239

Ep0162 50096

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Philadelphia, Pennsylvania, United States, 19104

Ep0162 50364

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Philadelphia, Pennsylvania, United States, 19107

Ep0162 50089

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Philadelphia, Pennsylvania, United States, 19140

Ep0162 50511

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Pittsburgh, Pennsylvania, United States, 15212

Ep0162 50491

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Pittsburgh, Pennsylvania, United States, 15213

Ep0162 50700

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Chattanooga, Tennessee, United States, 37403

Ep0162 50513

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Memphis, Tennessee, United States, 38103-2800

Ep0162 50103

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Nashville, Tennessee, United States, 37232

Ep0162 50731

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Austin, Texas, United States, 78749

Ep0162 50714

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Houston, Texas, United States, 77030

Ep0162 50525

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Houston, Texas, United States, 77459

Ep0162 50473

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Salt Lake City, Utah, United States, 84132

Ep0162 50726

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Winchester, Virginia, United States, 22601

More Information

Sponsor

UCB Biopharma SRL

Last update posted

Aug 27, 2026

Last verified

Aug, 2026

Keywords

Stereotypical prolonged seizures
Phase 3
Staccato alprazolam

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by UCB Biopharma SRL on 2026-08-27.