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

Sponsor

UCB Biopharma SRL (Industry)

Overall Status

Completed

CT.gov ID

NCT04857307

Collaborator

(none)

Enrollment

Locations

Arm

11.2

Actual Duration (Months)

1.8

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Epilepsy	Drug: Alprazolam	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

14 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Basic Science

Official Title:

A Multicenter, Open-Label Study to Evaluate the Pharmacokinetics, Tolerability, and Safety of a Single Dose of Staccato Alprazolam in Adolescent Study Participants With Epilepsy

Actual Study Start Date :

Apr 28, 2021

Actual Primary Completion Date :

Apr 5, 2022

Actual Study Completion Date :

Apr 5, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Staccato alprazolam The study participants will receive a single dose of Staccato alprazolam.	Drug: Alprazolam Pharmaceutical form: Inhalation powder. Study participants will receive Staccato alprazolam at prespecified time-points. Other Names: UCB7538

Arm

Intervention/Treatment

Experimental: Staccato alprazolam

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

Drug: Alprazolam

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

Other Names:

UCB7538

Outcome Measures

Primary Outcome Measures

Maximum plasma concentration (Cmax) following single inhaled dose of Staccato alprazolam [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.]
Cmax = Maximum plasma concentration.
Area under the plasma concentration-time curve from zero to the last quantifiable concentration (AUC(0-t)) following single inhaled dose of Staccato alprazolam [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.]
AUC(0-t) = Area under the plasma concentration-time curve from zero to the last quantifiable concentration.
Area under the plasma concentration-time curve from time 0 to infinity (AUC) following single inhaled dose of Staccato alprazolam [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.]
AUC = Area under the plasma concentration-time curve from time 0 to infinity.
Apparent total body clearance (CL/F) following single inhaled dose of Staccato alprazolam [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.]
CL/F = Apparent total body clearance.
Percentage of participants with treatment-emergent adverse event (TEAEs) [From baseline (Day 1) till end of Safety Follow-up (up to Day 9)]
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.
Percentage of participants with serious treatment-emergent adverse event (serious TEAEs) [From baseline (Day 1) till end of Safety Follow-up (up to Day 9)]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years to 17 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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

Exclusion Criteria:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Up0100 102	Little Rock	Arkansas	United States	72205
2	Up0100 110	Orlando	Florida	United States	32806
3	Up0100 103	Honolulu	Hawaii	United States	96817
4	Up0100 101	Bethesda	Maryland	United States	20817
5	Up0100 108	Rochester	New York	United States	14642
6	Up0100 106	Cincinnati	Ohio	United States	45229
7	Up0100 105	Memphis	Tennessee	United States	38103
8	Up0100 107	Tacoma	Washington	United States	98405