RECRUITING

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

Conditions

Stereotypical Prolonged Seizures Phase 3 Staccato alprazolam

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Double-Blind, Randomized, Placebo-Controlled, Multicenter, Outpatient, Parallel-Group Study to Assess the Efficacy and Safety of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures

Quick Facts

Study Start:2021-12-07

Study Completion:2026-04-06

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05077904

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:12 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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: * 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	* 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

Inclusion Criteria

Exclusion 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:
* 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

* 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

Contacts and Locations

Study Contact

UCB Cares

CONTACT

1-844-599-2273 (USA)

UCBCares@ucb.com

UCB Cares

CONTACT

0018445992273

UCBCares@ucb.com

Principal Investigator

UCB Cares

STUDY_DIRECTOR

001 844 599 2273 (UCB)

Study Locations (Sites)

Ep0162 50506

Phoenix, Arizona, 85004

United States

Ep0162 50494

Little Rock, Arkansas, 72205

United States

Ep0162 50118

Downey, California, 90242

United States

Ep0162 50505

Los Angeles, California, 90095

United States

Ep0162 50492

Orange, California, 92868

United States

Ep0162 50367

New Haven, Connecticut, 06519

United States

Ep0162 50088

Washington, District of Columbia, 20037

United States

Ep0162 50515

Gulf Breeze, Florida, 32561

United States

Ep0162 50508

Jacksonville, Florida, 32209

United States

Ep0162 50342

Jacksonville, Florida, 32224-1865

United States

Ep0162 50199

Miami, Florida, 33136

United States

Ep0162 50509

Orlando, Florida, 32806

United States

Ep0162 50308

Tampa, Florida, 33606

United States

Ep0162 50323

Honolulu, Hawaii, 96817

United States

Ep0162 50512

Boise, Idaho, 83702

United States

Ep0162 50493

Chicago, Illinois, 60611

United States

Ep0162 50375

Springfield, Illinois, 62702

United States

Ep0162 50504

Fort Wayne, Indiana, 46804

United States

Ep0162 50561

Lexington, Kentucky, 40536-0284

United States

Ep0162 50395

New Orleans, Louisiana, 70121

United States

Ep0162 50517

New Orleans, Louisiana, 70122

United States

Ep0162 50093

Baltimore, Maryland, 21287

United States

Ep0162 50488

Bethesda, Maryland, 20817

United States

Ep0162 50047

Boston, Massachusetts, 02215

United States

Ep0162 50110

Ann Arbor, Michigan, 48109-0944

United States

Ep0162 50507

Saint Louis, Missouri, 63110

United States

Ep0162 50499

Las Vegas, Nevada, 89128

United States

Ep0162 50626

Neptune, New Jersey, 07753

United States

Ep0162 50299

New Brunswick, New Jersey, 08901

United States

Ep0162 50497

Buffalo, New York, 14221

United States

Ep0162 50298

New York, New York, 10016

United States

Ep0162 50490

New York, New York, 10029

United States

Ep0162 50518

New York, New York, 10075

United States

Ep0162 50034

Rochester, New York, 14642

United States

Ep0162 50514

Charlotte, North Carolina, 28204

United States

Ep0162 50487

Charlotte, North Carolina, 28207

United States

Ep0162 50371

Akron, Ohio, 44308

United States

Ep0162 50528

Cleveland, Ohio, 44195

United States

Ep0162 50510

Portland, Oregon, 97239-3098

United States

Ep0162 50096

Philadelphia, Pennsylvania, 19104

United States

Ep0162 50364

Philadelphia, Pennsylvania, 19107

United States

Ep0162 50089

Philadelphia, Pennsylvania, 19140

United States

Ep0162 50511

Pittsburgh, Pennsylvania, 15212

United States

Ep0162 50491

Pittsburgh, Pennsylvania, 15213

United States

Ep0162 50513

Memphis, Tennessee, 38103

United States

Ep0162 50103

Nashville, Tennessee, 37232

United States

Ep0162 50525

Houston, Texas, 77459

United States

Ep0162 50496

Round Rock, Texas, 78681

United States

Ep0162 50473

Salt Lake City, Utah, 84132

United States

Collaborators and Investigators

Sponsor: UCB Biopharma SRL

UCB Cares, STUDY_DIRECTOR, 001 844 599 2273 (UCB)

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-12-07

Study Completion Date2026-04-06

Study Record Updates

Study Start Date2021-12-07

Study Completion Date2026-04-06

Terms related to this study

Keywords Provided by Researchers

Stereotypical prolonged seizures
Phase 3
Staccato alprazolam

Additional Relevant MeSH Terms

Stereotypical Prolonged Seizures