A Study to Assess the Pharmacokinetics (PK) and Safety of Staccato Alprazolam in Adolescent Study Participants With Epilepsy

NCT04857307 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: UP0100
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 8

Brief Summary

The purpose of the study is to assess the pharmacokinetics (PK), tolerability, and safety of Staccato alprazolam in adolescent study participants with epilepsy following single-dose administration.

Conditions

Epilepsy

Keywords

Epilepsy; Staccato alprazolam; Phase 1; Adolescents; STAP-001; Alprazolam

Outcome Measures

Primary Outcomes (6)

• Maximum plasma concentration (Cmax) following single inhaled dose of Staccato alprazolam

  Cmax = Maximum plasma concentration.

  Plasma samples will be taken on Day 1 of the Treatment Period, predose and then following administration of Investigational Medicinal Product (IMP) at 2 minutes, 10 minutes, 30 minutes, 1 hour, 2 hours, 6 hours, 24 hours, and 36 hours postdose.

• Area under the plasma concentration-time curve from zero to the last quantifiable concentration (AUC(0-t)) following single inhaled dose of Staccato alprazolam

  AUC(0-t) = Area under the plasma concentration-time curve from zero to the last quantifiable concentration.

  Plasma samples will be taken on Day 1 of the Treatment Period, predose and then following administration of Investigational Medicinal Product (IMP) at 2 minutes, 10 minutes, 30 minutes, 1 hour, 2 hours, 6 hours, 24 hours, and 36 hours postdose.

• Area under the plasma concentration-time curve from time 0 to infinity (AUC) following single inhaled dose of Staccato alprazolam

  AUC = Area under the plasma concentration-time curve from time 0 to infinity.

  Plasma samples will be taken on Day 1 of the Treatment Period, predose and then following administration of Investigational Medicinal Product (IMP) at 2 minutes, 10 minutes, 30 minutes, 1 hour, 2 hours, 6 hours, 24 hours, and 36 hours postdose.

• Apparent total body clearance (CL/F) following single inhaled dose of Staccato alprazolam

  CL/F = Apparent total body clearance.

  Plasma samples will be taken on Day 1 of the Treatment Period, predose and then following administration of Investigational Medicinal Product (IMP) at 2 minutes, 10 minutes, 30 minutes, 1 hour, 2 hours, 6 hours, 24 hours, and 36 hours postdose.

• Percentage of participants with treatment-emergent adverse event (TEAEs)

  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.

  From baseline (Day 1) till end of Safety Follow-up (up to Day 9)

• Percentage of participants with serious treatment-emergent adverse event (serious TEAEs)

  A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: a. Results in death c. Requires inpatient hospitalization or prolongation of existing hospitalization d. Results in persistent disability/incapacity e. Is a congenital anomaly/birth defect f. Important medical events.

  From baseline (Day 1) till end of Safety Follow-up (up to Day 9)

Study Arms (1)

Staccato alprazolam

EXPERIMENTAL

The study participants will receive a single dose of Staccato alprazolam.

Drug: Alprazolam

Interventions

Alprazolam DRUG

* Pharmaceutical form: Inhalation powder. * Study participants will receive Staccato alprazolam at prespecified time-points.

Also known as: UCB7538

Staccato alprazolam

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant must be 12 to 17 years of age inclusive, at the time of signing the Informed Consent form (ICF) and the Assent form
• Participant has an established diagnosis of focal, generalized, or focal and generalized epilepsy
• Participant is in good general health as determined by medical evaluation including medical history and physical examination
• Participants with a body weight ≥29 kg and body mass index (BMI) within the range 14 to 32 kg/m\^2 (inclusive)
• A male participant must agree to use contraception
• A female participant is eligible to participate if she is not pregnant
• Participant is capable of and provides assent, and the study participant's parent/legal representative provides signed informed consent for minor study participants, which includes compliance with the requirements and restrictions listed in the ICF, Assent form, and in this protocol
• Participant has a lifetime history of never smoking \>5 cigarettes/day, and a current history (for at least 6 months prior to Screening Visit) of not smoking at all (including e-cigarette and vaping products)
• Participant has forced expiratory volume in 1 second (FEV1) and forced vital capacity (FVC) \>80% predicted at Screening. In case of an out-of-range result, 1 repeat test will be allowed. If the readings are out-of-range again, the study participant will be excluded
• Participant is willing and able to be confined to a clinical research facility for up to 36 hours (including 1 overnight stay) and comply with the study schedule and study requirements.
• Note: If there are no clinical contraindications, as per Investigator's judgment, study participants may leave the clinical research facility after the 6-hour postdose assessments and return to the clinic on Day 2 for the 24-hour and 36-hour postdose assessments
• Participant is currently taking at least 1 background antiepileptic drug (AED)
• Participant is able to actuate the training device during Screening, according to Instructions for Use

You may not qualify if:

• Participant has history or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, neurological, hematological, cerebrovascular, or other major disorders capable of significantly altering the absorption, metabolism, or elimination of Investigational Medicinal Product (IMP); constituting a risk when taking the study intervention; or interfering with the interpretation of data in the opinion of the Investigator
• Participant has a known hypersensitivity to any components of the IMP or comparative drugs (and/or an investigational device) as stated in the protocol
• Participant has severe chronic cardio-respiratory disease
• Participant has history of acute narrow angle glaucoma, hydrocephalus, or Myasthenia Gravis
• Participant has history or has current airway disease such asthma, cystic fibrosis, or chronic obstructive pulmonary disease
• Participant has any acute respiratory signs/symptoms (ie, wheezing) and active acute respiratory infection (or within 1 week of dosing) with exception of symptoms of mild rhinitis
• Participant has a known hypersensitivity to albuterol or similar short-acting beta2-agonist (SABA) that may be used as rescue medication administered in response to potential bronchospasm
• Participant is taking strong liver inducing agents (eg, phenytoin, phenobarbital, carbamazepine, and primidone) or strong Cytochrome P450 3A4 (CYP3A4) inhibitors
• Participant has a SpO2 measured by pulse oximetry \<95% for \>30 seconds during the Screening Visit

Sponsors & Collaborators

• UCB Biopharma SRL: lead

Study Sites (8)

Up0100 102

Little Rock, Arkansas, 72205, United States

Location

Up0100 110

Orlando, Florida, 32806, United States

Location

Up0100 103

Honolulu, Hawaii, 96817, United States

Location

Up0100 101

Bethesda, Maryland, 20817, United States

Location

Up0100 108

Rochester, New York, 14642, United States

Location

Up0100 106

Cincinnati, Ohio, 45229, United States

Location

Up0100 105

Memphis, Tennessee, 38103, United States

Location

Up0100 107

Tacoma, Washington, 98405, United States

Location

MeSH Terms

Conditions

Epilepsy

Interventions

Alprazolam

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Benzodiazepines; Benzazepines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• UCB Cares

  001 844 599 2273 (UCB)

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 22, 2021
• First Posted: April 23, 2021
• Study Start: April 28, 2021
• Primary Completion: April 5, 2022
• Study Completion: April 5, 2022
• Last Updated: June 30, 2022

Record last verified: 2022-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)