Clinical trial NCT04857307: A Study to Assess the Pharmacokinetics (PK) and Safety of Staccato Alprazolam in Adolescent Study Participants With Epilepsy
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Epilepsy
Keywords: Epilepsy
Staccato alprazolam
Phase 1
Adolescents
STAP-001
Alprazolam
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2021-04-23
Enrollment: 14
Sex: ALL
Minimum age (years): 12
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: BASIC_SCIENCE

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.


Eligibility criteria: 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

Primary outcomes:
measure: Maximum plasma concentration (Cmax) following single inhaled dose of Staccato alprazolam
timeFrame: 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.
measure: Area under the plasma concentration-time curve from zero to the last quantifiable concentration (AUC(0-t)) following single inhaled dose of Staccato alprazolam
timeFrame: 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.
measure: Area under the plasma concentration-time curve from time 0 to infinity (AUC) following single inhaled dose of Staccato alprazolam
timeFrame: 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.
measure: Apparent total body clearance (CL/F) following single inhaled dose of Staccato alprazolam
timeFrame: 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.
measure: Percentage of participants with treatment-emergent adverse event (TEAEs)
timeFrame: From baseline (Day 1) till end of Safety Follow-up (up to Day 9)
measure: Percentage of participants with serious treatment-emergent adverse event (serious TEAEs)
timeFrame: From baseline (Day 1) till end of Safety Follow-up (up to Day 9)
Secondary outcomes:
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Up0100 102
state: Arkansas
city: Orlando
country: United States
facility: Up0100 110
state: Florida
city: Honolulu
country: United States
facility: Up0100 103
state: Hawaii
city: Bethesda
country: United States
facility: Up0100 101
state: Maryland
city: Rochester
country: United States
facility: Up0100 108
state: New York
city: Cincinnati
country: United States
facility: Up0100 106
state: Ohio
city: Memphis
country: United States
facility: Up0100 105
state: Tennessee
city: Tacoma
country: United States
facility: Up0100 107
state: Washington
Study officials:
affiliation: 001 844 599 2273 (UCB)
name: UCB Cares
role: STUDY_DIRECTOR
References: