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

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ClinicalTrials.gov Identifier: NCT05077904
Recruitment Status : Recruiting
First Posted : October 14, 2021
Last Update Posted : April 12, 2024
Sponsor:
Information provided by (Responsible Party):
UCB Pharma ( UCB Biopharma SRL )

Study Description
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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.

Condition or disease Intervention/treatment Phase
Stereotypical Prolonged Seizures Drug: Staccato alprazolam Other: Placebo Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 250 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : December 7, 2021
Estimated Primary Completion Date : April 17, 2024
Estimated Study Completion Date : April 17, 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Seizures
Drug Information available for: Alprazolam

Arms and Interventions
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Arm Intervention/treatment
Experimental: Staccato alprazolam Arm
Participants randomized to this arm will receive a single dose of Staccato alprazolam by inhalation.
 Drug: Staccato alprazolam

Route of administration: Inhalation

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

Other Name: UCB7538
Placebo Comparator: Placebo Arm
Participants randomized to this arm will receive a single dose of placebo by inhalation.
 Other: Placebo

Route of administration: Inhalation

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


Outcome Measures
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Primary Outcome Measures :
  1. 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 is defined as termination of the seizure within 90 seconds after IMP administration.

  2. Treatment success for the treated seizure with no recurrence up to 2 hours [ Time Frame: From start of IMP treatment through 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.


Secondary Outcome Measures :
  1. Treatment success for treated seizure with no recurrence after 4 hours [ Time Frame: From start of IMP treatment through 4 hours ]
    Treatment success defined as termination of the treated seizure within 90 seconds after IMP administration, with no recurrence of seizure(s) up to 4 hours after IMP administration and no initiation of seizure rescue treatment from 90 seconds to 4 hours after IMP administration.

  2. Treatment success for treated seizure with no recurrence after 6 hours [ Time Frame: From start of IMP treatment through 6 hours ]
    Treatment success defined as termination of the treated seizure within 90 seconds after IMP administration, with no recurrence of seizure(s) up to 6 hours after IMP administration and no initiation of seizure rescue treatment from 90 seconds to 6 hours after IMP administration.

  3. Time from IMP administration to cessation of the treated seizure [ Time Frame: From start of IMP treatment through 6 hours ]
    The time will be assessed from IMP administration to cessation of the treated seizure (taking administration of seizure rescue treatment as censoring point).

  4. Frequency of respiratory treatment emergent adverse events (TEAEs) [ Time Frame: From start of IMP treatment up to the Safety Follow-up Visit (Week 19) ]
    An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of IMP, whether or not considered related to the IMP.

  5. Number of subsequent seizure(s) up to 2 hours after IMP administration [ Time Frame: From start of IMP treatment through 2 hours ]
    The number of subsequent seizure(s) will be assessed up to 2 hours after IMP administration.

  6. Time to first subsequent seizure up to 2 hours after IMP administration [ Time Frame: From start of IMP treatment through 2 hours ]
    The time to first subsequent seizure will be assessed up to 2 hours after IMP administration and is defined as time from end of IMP treated seizure to start of first subsequent seizure.


Eligibility Criteria
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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05077904


Contacts
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Contact: UCB Cares 1-844-599-2273 (USA) UCBCares@ucb.com
Contact: UCB Cares 0018445992273 UCBCares@ucb.com

Locations
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Sponsors and Collaborators
UCB Biopharma SRL
Investigators
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Study Director: UCB Cares 001 844 599 2273 (UCB)
More Information
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Responsible Party: UCB Biopharma SRL
ClinicalTrials.gov Identifier: NCT05077904    
Other Study ID Numbers: EP0162
2021-002686-18 ( EudraCT Number )
First Posted: October 14, 2021    Key Record Dates
Last Update Posted: April 12, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: 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. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, 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 prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of reidentifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (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.
URL: http://www.Vivli.org

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by UCB Pharma ( UCB Biopharma SRL ):
Stereotypical prolonged seizures
Phase 3
Staccato alprazolam
Additional relevant MeSH terms:
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Seizures
Status Epilepticus
Neurologic Manifestations
Nervous System Diseases
Alprazolam
Hypnotics and Sedatives
Central Nervous System Depressants
Physiological Effects of Drugs
 Anti-Anxiety Agents
Tranquilizing Agents
Psychotropic Drugs
GABA Modulators
GABA Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action