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

NCT05077904 | Recruiting Phase 3 DMC FDA Drug

• 4 other identifiers: EP01622021-002686-18U1111-1303-25882024-510950-29
• Study Type: interventional
• Target: 350
• Locations: 13 countries
• Sites: 183

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

Keywords

Stereotypical prolonged seizures; Phase 3; Staccato alprazolam

Outcome Measures

Primary Outcomes (2)

• Treatment success for the treated seizure within 90 seconds after investigational medicinal product (IMP) administration

  Treatment success for the treated seizure is defined as termination of the seizure within 90 seconds after IMP administration.

  From start of IMP treatment through 6 hours

• Treatment success for the treated seizure with no recurrence up to 2 hours

  Treatment success for the treated seizure is defined as termination of the treated 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.

  From start of IMP treatment through 2 hours

Secondary Outcomes (6)

• Treatment success for treated seizure with no recurrence after 4 hours

  From start of IMP treatment through 4 hours

• Treatment success for treated seizure with no recurrence after 6 hours

  From start of IMP treatment through 6 hours

• Time from IMP administration to cessation of the treated seizure

  From start of IMP treatment through 6 hours

• Frequency of respiratory treatment emergent adverse events (TEAEs)

  From start of IMP treatment up to the Safety Follow-up Visit (Week 19)

• Number of subsequent seizure(s) up to 2 hours after IMP administration

  From start of IMP treatment through 2 hours

Study Arms (2)

Staccato alprazolam Arm

EXPERIMENTAL

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

Drug: Staccato alprazolam

Placebo Arm

PLACEBO COMPARATOR

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

Other: Placebo

Interventions

Staccato alprazolam DRUG

Route of administration: Inhalation Participants will receive one dose of Staccato alprazolam during the Treatment Period.

Also known as: UCB7538

Staccato alprazolam Arm

Placebo OTHER

Route of administration: Inhalation Participants will receive one dose of placebo during the Treatment Period.

Placebo Arm

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Generalized seizure episodes starting with a flurry of absence seizures or myoclonic seizures with a minimum total duration of 5 minutes
• Episodes of a focal seizure with a minimum duration of 3 minutes
• 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:
• 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
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• UCB Biopharma SRL: lead

Study Sites (183)

Ep0162 50506

Phoenix, Arizona, 85004, United States

RECRUITING

Ep0162 50720

Scottsdale, Arizona, 85254, United States

RECRUITING

Ep0162 50494

Little Rock, Arkansas, 72205, United States

RECRUITING

Ep0162 50118

Downey, California, 90242, United States

RECRUITING

Ep0162 50416

La Jolla, California, 92037, United States

RECRUITING

Ep0162 50702

Long Beach, California, 90806, United States

RECRUITING

Ep0162 50505

Los Angeles, California, 90095, United States

WITHDRAWN

Ep0162 50492

Orange, California, 92868, United States

COMPLETED

Ep0162 50722

Poway, California, 92064, United States

RECRUITING

Ep0162 50716

Sacramento, California, 95817, United States

RECRUITING

Ep0162 50367

New Haven, Connecticut, 06519, United States

COMPLETED

Ep0162 50088

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

RECRUITING

Ep0162 50721

Boca Raton, Florida, 33486, United States

RECRUITING

Ep0162 50515

Gulf Breeze, Florida, 32561, United States

COMPLETED

Ep0162 50508

Jacksonville, Florida, 32209-6533, United States

RECRUITING

Ep0162 50342

Jacksonville, Florida, 32224-1865, United States

RECRUITING

Ep0162 50199

Miami, Florida, 33136, United States

RECRUITING

Ep0162 50676

Miami, Florida, 33176, United States

RECRUITING

Ep0162 50509

Orlando, Florida, 32806, United States

COMPLETED

Ep0162 50703

Rockledge, Florida, 32955, United States

RECRUITING

Ep0162 50308

Tampa, Florida, 33606, United States

RECRUITING

Ep0162 50323

Honolulu, Hawaii, 96817, United States

RECRUITING

Ep0162 50512

Boise, Idaho, 83702, United States

RECRUITING

Ep0162 50493

Chicago, Illinois, 60611, United States

RECRUITING

Ep0162 50375

Springfield, Illinois, 62702, United States

RECRUITING

Ep0162 50504

Fort Wayne, Indiana, 46804, United States

RECRUITING

Ep0162 50561

Lexington, Kentucky, 40536, United States

WITHDRAWN

Ep0162 50395

New Orleans, Louisiana, 70121, United States

WITHDRAWN

Ep0162 50517

New Orleans, Louisiana, 70122, United States

RECRUITING

Ep0162 50093

Baltimore, Maryland, 21287, United States

WITHDRAWN

Ep0162 50488

Bethesda, Maryland, 20817, United States

WITHDRAWN

Ep0162 50615

Boston, Massachusetts, 02118, United States

RECRUITING

Ep0162 50047

Boston, Massachusetts, 02215, United States

COMPLETED

Ep0162 50110

Ann Arbor, Michigan, 48109, United States

COMPLETED

Ep0162 50507

St Louis, Missouri, 63110, United States

RECRUITING

Ep0162 50499

Las Vegas, Nevada, 89128, United States

WITHDRAWN

Ep0162 50626

Neptune City, New Jersey, 07753, United States

RECRUITING

Ep0162 50299

New Brunswick, New Jersey, 08901, United States

RECRUITING

Ep0162 50497

Buffalo, New York, 14221, United States

RECRUITING

Ep0162 50625

New Hyde Park, New York, 11042, United States

RECRUITING

Ep0162 50298

New York, New York, 10016, United States

RECRUITING

Ep0162 50490

New York, New York, 10029, United States

RECRUITING

Ep0162 50518

New York, New York, 10075, United States

RECRUITING

Ep0162 50034

Rochester, New York, 14642, United States

RECRUITING

Ep0162 50514

Charlotte, North Carolina, 28204, United States

RECRUITING

Ep0162 50487

Charlotte, North Carolina, 28207, United States

WITHDRAWN

Ep0162 50715

Durham, North Carolina, 27705, United States

RECRUITING

Ep0162 50371

Akron, Ohio, 44308, United States

WITHDRAWN

Ep0162 50528

Cleveland, Ohio, 44195, United States

WITHDRAWN

Ep0162 50724

Portland, Oregon, 97227, United States

COMPLETED

Ep0162 50510

Portland, Oregon, 97239-3098, United States

WITHDRAWN

Ep0162 50701

Portland, Oregon, 97239, United States

RECRUITING

Ep0162 50096

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Ep0162 50364

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

Ep0162 50089

Philadelphia, Pennsylvania, 19140, United States

RECRUITING

Ep0162 50511

Pittsburgh, Pennsylvania, 15212, United States

RECRUITING

Ep0162 50491

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

Ep0162 50700

Chattanooga, Tennessee, 37403, United States

RECRUITING

Ep0162 50513

Memphis, Tennessee, 38103-2800, United States

RECRUITING

Ep0162 50103

Nashville, Tennessee, 37232, United States

RECRUITING

Ep0162 50731

Austin, Texas, 78749, United States

RECRUITING

Ep0162 50714

Houston, Texas, 77030, United States

RECRUITING

Ep0162 50525

Houston, Texas, 77459, United States

RECRUITING

Ep0162 50496

Round Rock, Texas, 78681, United States

WITHDRAWN

Ep0162 50473

Salt Lake City, Utah, 84132, United States

RECRUITING

Ep0162 50726

Winchester, Virginia, 22601, United States

RECRUITING

Ep0162 30016

Fitzroy, Australia

RECRUITING

Ep0162 30030

Herston, Australia

RECRUITING

Ep0162 30027

Melbourne, Australia

WITHDRAWN

Ep0162 30031

South Brisbane, Australia

ACTIVE NOT RECRUITING

Ep0162 40650

Blagoevgrad, Bulgaria

RECRUITING

Ep0162 40708

Pazardzhik, Bulgaria

RECRUITING

Ep0162 40665

Pleven, Bulgaria

COMPLETED

Ep0162 40709

Pleven, Bulgaria

RECRUITING

Ep0162 40651

Sofia, Bulgaria

COMPLETED

Ep0162 20128

Beijing, China

WITHDRAWN

Ep0162 20246

Beijing, China

RECRUITING

Ep0162 20268

Beijing, China

COMPLETED

Ep0162 20299

Beijing, China

RECRUITING

Ep0162 20261

Changchun, China

RECRUITING

Ep0162 20133

Chengdu, China

RECRUITING

Ep0162 20137

Chengdu, China

RECRUITING

Ep0162 20250

Chongqing, China

WITHDRAWN

Ep0162 20334

Chongqing, China

RECRUITING

Ep0162 20179

Fuzhou, China

RECRUITING

Ep0162 20124

Guangzhou, China

RECRUITING

Ep0162 20260

Guangzhou, China

COMPLETED

Ep0162 20264

Guangzhou, China

COMPLETED

Ep0162 20269

Guangzhou, China

RECRUITING

Ep0162 20300

Guangzhou, China

RECRUITING

Ep0162 20022

Hangzhou, China

RECRUITING

Ep0162 20320

Kunming, China

RECRUITING

Ep0162 20258

Lanzhou, China

RECRUITING

Ep0162 20253

Nanchang, China

WITHDRAWN

Ep0162 20267

Nanjing, China

RECRUITING

Ep0162 20331

Nanning, China

RECRUITING

Ep0162 20333

Qingdao, China

RECRUITING

Ep0162 20123

Shanghai, China

WITHDRAWN

Ep0162 20292

Shanghai, China

RECRUITING

Ep0162 20332

Shenzhen, China

WITHDRAWN

Ep0162 20289

Shijiazhuang, China

RECRUITING

Ep0162 20119

Suzhou, China

RECRUITING

Ep0162 20257

Tianjin, China

RECRUITING

Ep0162 20025

Wenzhou, China

RECRUITING

Ep0162 20252

Wuhan, China

WITHDRAWN

Ep0162 20255

Yinchuan, China

WITHDRAWN

Ep0162 20262

Zhanjiang, China

RECRUITING

Ep0162 20251

Zhengzhou, China

RECRUITING

Ep0162 40670

Brno, Czechia

RECRUITING

Ep0162 40672

Ostrava - Poruba, Czechia

RECRUITING

Ep0162 40063

Prague, Czechia

COMPLETED

Ep0162 40671

Prague, Czechia

RECRUITING

Ep0162 40714

Prague, Czechia

RECRUITING

Ep0162 40681

Bron, France

RECRUITING

Ep0162 40688

Bron, France

WITHDRAWN

Ep0162 40680

Dijon, France

RECRUITING

Ep0162 40130

Marseille, France

WITHDRAWN

Ep0162 40019

Paris, France

RECRUITING

Ep0162 40682

Paris, France

RECRUITING

Ep0162 40201

Rennes, France

RECRUITING

Ep0162 40199

Strasbourg, France

RECRUITING

Ep0162 40577

Aachen, Germany

WITHDRAWN

Ep0162 40683

Berlin, Germany

RECRUITING

Ep0162 40685

Bielefeld, Germany

RECRUITING

Ep0162 40023

Erlangen, Germany

RECRUITING

Ep0162 40645

Frankfurt am Main, Germany

RECRUITING

Ep0162 40689

Kehl-kork, Germany

RECRUITING

Ep0162 40529

Marburg, Germany

RECRUITING

Ep0162 40724

München, Germany

RECRUITING

Ep0162 40666

Balassagyarmat, Hungary

RECRUITING

Ep0162 40673

Budapest, Hungary

RECRUITING

Ep0162 40704

Budapest, Hungary

RECRUITING

Ep0162 40653

Debrecen, Hungary

RECRUITING

Ep0162 40690

Catanzaro, Italy

WITHDRAWN

Ep0162 40674

Genova, Italy

RECRUITING

Ep0162 40144

Milan, Italy

RECRUITING

Ep0162 40477

Pavia, Italy

RECRUITING

Ep0162 40257

Roma, Italy

RECRUITING

Ep0162 40675

Roma, Italy

RECRUITING

Ep0162 20248

Fukuoka, Japan

RECRUITING

Ep0162 20070

Hachioji-shi, Japan

RECRUITING

Ep0162 20237

Hamamatsu, Japan

WITHDRAWN

Ep0162 20249

Hiroshima, Japan

RECRUITING

Ep0162 20236

Hōfu, Japan

RECRUITING

Ep0162 20239

Itami, Japan

RECRUITING

Ep0162 20143

Kodaira, Japan

RECRUITING

Ep0162 20315

Koshi-shi, Japan

RECRUITING

Ep0162 20147

Kyoto, Japan

RECRUITING

Ep0162 20243

Nagakute, Japan

RECRUITING

Ep0162 20235

Nagoya, Japan

RECRUITING

Ep0162 20238

Niigata, Japan

RECRUITING

Ep0162 20302

Osaka, Japan

RECRUITING

Ep0162 20241

Ōmura, Japan

RECRUITING

Ep0162 20316

Sapporo, Japan

RECRUITING

Ep0162 20337

Shimotsuga-gun, Japan

RECRUITING

Ep0162 20297

Shinjuku-ku, Japan

RECRUITING

Ep0162 20240

Shizuoka, Japan

RECRUITING

Ep0162 20242

Suita, Japan

RECRUITING

Ep0162 20266

Tōon, Japan

RECRUITING

Ep0162 20244

Yamagata, Japan

RECRUITING

Ep0162 40707

Bydgoszcz, Poland

RECRUITING

Ep0162 40677

Gdansk, Poland

RECRUITING

Ep0162 40219

Krakow, Poland

WITHDRAWN

Ep0162 40502

Krakow, Poland

COMPLETED

Ep0162 40842

Krakow, Poland

RECRUITING

Ep0162 40676

Lublin, Poland

RECRUITING

Ep0162 40091

Nowa Sól, Poland

RECRUITING

Ep0162 40153

Poznan, Poland

WITHDRAWN

Ep0162 40678

Świdnik, Poland

RECRUITING

Ep0162 40160

Barcelona, Spain

RECRUITING

Ep0162 40157

L'Hospitalet de Llobregat, Spain

RECRUITING

Ep0162 40540

Madrid, Spain

RECRUITING

Ep0162 40352

Pamplona, Spain

RECRUITING

Ep0162 40668

Seville, Spain

RECRUITING

Ep0162 40453

Terrassa, Spain

RECRUITING

Ep0162 40230

Valencia, Spain

RECRUITING

Ep0162 40667

Valladolid, Spain

COMPLETED

Ep0162 40686

Birmingham, United Kingdom

RECRUITING

Ep0162 40300

Cardiff, United Kingdom

WITHDRAWN

Ep0162 40735

Glasgow, United Kingdom

RECRUITING

Ep0162 40833

London, United Kingdom

RECRUITING

Ep0162 40163

Oxford, United Kingdom

RECRUITING

Ep0162 40108

Salford, United Kingdom

RECRUITING

Study Officials

• UCB Cares

  001 844 599 2273 (UCB)

  STUDY DIRECTOR

Central Study Contacts

UCB Cares

CONTACT

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

UCB Cares

CONTACT

0018445992273; UCBCares@ucb.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 30, 2021
• First Posted: October 14, 2021
• Study Start: December 7, 2021
• Primary Completion: April 1, 2026
• Study Completion: April 1, 2026
• Last Updated: April 27, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
• Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.

Locations

DE (8); US (66); FR (8); HU (4); CZ (5); IT (6); PL (9); BU (5); ES (8); CN (33); GB (6); JP (21); AU (4)