Inpatient, Dose-Ranging Study of Staccato Alprazolam in Epilepsy With Predictable Seizure Pattern
StATES
A Double-Blind, Placebo-Controlled, Inpatient, Dose-Ranging Efficacy Study of Staccato Alprazolam (STAP-001) in Subjects With Epilepsy With a Predictable Seizure Pattern
1 other identifier
interventional
156
3 countries
67
Brief Summary
This is a multi-center, double-blind, randomized, parallel group, dose-ranging study to investigate the efficacy and clinical usability of STAP-001 in adult (18 years of age and older) subjects with epilepsy with a predictable seizure pattern. These subjects have an established diagnosis of focal or generalized epilepsy with a documented history of predictable seizure episodes. This is an in-patient study. The subjects will be admitted to a Clinical Research Unit (CRU) or Epilepsy Monitoring Unit (EMU) for study participation. The duration of the stay in the in-patient unit will be 2-8 days. One seizure event per subject will be treated with study medication. The duration and timing of the seizure event and occurrence of subsequent seizures will be assessed by the Staff Caregiver(s)1 through clinical observation and confirmed with video electroencephalogram (EEG).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Mar 2018
67 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 7, 2018
CompletedStudy Start
First participant enrolled
March 16, 2018
CompletedFirst Posted
Study publicly available on registry
March 27, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 22, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
January 4, 2020
CompletedResults Posted
Study results publicly available
February 3, 2021
CompletedFebruary 3, 2021
February 1, 2021
1.8 years
March 7, 2018
December 16, 2020
February 2, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Percentage of Participants in Each Treatment Group Achieving Seizure Activity Cessation Within 2 Minutes and no Recurrent Seizure Within 2 Hours
Percentage of participants with onset of a predictable seizure through 2 minutes post dosing with study drug and no recurrence of seizure activity within 2 hours were reported for each treatment group based on clinical observation.
2 hours post-dosing on dosing day
Secondary Outcomes (3)
Percentage of Participants With Seizure Episode Severity Assessed by Seizure Episode Severity Scale
6 hours post-dosing on dosing day
Percentage of Participants With Use of Rescue Medication
2 hours post-dosing on dosing day
Percentage of Participants With Secondary Generalization (Evolution to a Complex Partial Seizure and/or a Generalized Tonic-Clonic Seizure)
24 hours post-dosing on dosing day
Study Arms (3)
Staccato Alprazolam 1.0 mg
EXPERIMENTALsingle dose for inhalation
Staccato Alprazolam 2.0 mg
EXPERIMENTALsingle dose for inhalation
Placebo
PLACEBO COMPARATORsingle dose for inhalation
Interventions
single dose for inhalation
Eligibility Criteria
You may qualify if:
- Subject is able to provide, personally signed, and dated informed consent to participate in the study or will have a legally authorized representative sign the informed consent on his or her behalf before completing any study related procedures.
- Male or female ≥ 18 years of age.
- Has an established diagnosis of focal or generalized epilepsy or focal and generalized epilepsy with a documented history of predictable seizure episodes that includes at least one of the following:
- Generalized seizure episodes starting with a flurry of absence seizures or myoclonic seizures with a minimum duration of 5 minutes
- Episodes of a prolonged focal seizure with a minimum duration of 3 minutes
- Episodes of multiple (≥2) seizures within a 2-hour time period
- Prior to randomization, has experienced ≥4 seizure episodes with predictable pattern during the last 4 weeks (qualification period) and no more than one week without a predictable seizure episode before entry into the in-patient unit.
- Female participants (if of child-bearing potential and sexually active) and male participants (if sexually active with a partner of child-bearing potential) who agree to use a medically acceptable and effective birth control method throughout the study and for 1 week following the end of the study. Medically acceptable methods of contraception that may be used by the participant and/or his/her partner include abstinence, birth control pills or patches, diaphragm with spermicide,intrauterine device (IUD), surgical sterilization, and progestin implant or injection. Prohibited methods include: the rhythm method, withdrawal, condoms alone, or diaphragm alone.
- Subject is able to comply by the requirements of the protocol, particularly the requirements and specific Institution policies during the in-clinic stay.
You may not qualify if:
- History or diagnosis of non-epileptic seizures (e.g. metabolic or pseudo-seizures).
- History of status epilepticus in the 6 months prior to Screening
- Has a progressive neurological disorder such as brain tumor, demyelinating disease, or degenerative central nervous system (CNS) disease that is likely to progress in the next 3 months
- Use of strong CYP 3A4 inhibitors; including azole antifungal agents (e.g., etoconazole, itraconazole), nefazodone, fluvoxamine, cimetidine, HIV protease inhibitors (e.g., ritonavir)
- Has severe chronic cardio-respiratory disease
- History of HIV-positivity.
- Pregnant or breast-feeding.
- Clinically significant renal or hepatic insufficiency (hepatic transaminases \>2 times the upper limit of normal (ULN) or creatinine ≥ 1.5 x ULN).
- History of acute narrow angle glaucoma, Parkinson's disease, hydrocephalus, or history of significant head trauma.
- Subjects who use medications to treat airways disease, such as asthma or COPD or have any acute respiratory signs/symptoms (e.g., wheezing).
- Use of any investigational drug within 30 days or 5 half-lives of the investigational drug prior to administration of study medication, whichever is longer
- A history within the past 1 year of drug or alcohol dependence or abuse.
- Positive urine screen for drugs of abuse at Screening.(positive Cannabis/Cannabinol results are acceptable if there is a documented history of stable use for medical purposes).
- Known allergy or hypersensitivity to alprazolam.
- History of glaucoma.
- +4 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (67)
UAB Hospital
Birmingham, Alabama, 35294, United States
University of Arizona
Phoenix, Arizona, 85006, United States
St. Joseph's Hospital and Medical Center - Barrow Neurological Institute
Phoenix, Arizona, 85013, United States
Clinical Trials Inc
Little Rock, Arkansas, 72205, United States
Rancho Research Institute Inc.
Downey, California, 90242, United States
UCLA
Los Angeles, California, 90095, United States
Hoag Hospital
Newport Beach, California, 92663, United States
Stanford Neuroscience Health Center
Palo Alto, California, 94304, United States
Havana Research Institute LLC.
Pasadena, California, 91105, United States
University of Colorado
Aurora, Colorado, 80045, United States
Yale University
New Haven, Connecticut, 06511, United States
VA Connecticut Healthcare System
West Haven, Connecticut, 06516, United States
Georgetown University Hospital
Washington D.C., District of Columbia, 20007, United States
GW Medical Faculty Associates
Washington D.C., District of Columbia, 20037, United States
NW FL Clinical Research Group LLC
Gulf Breeze, Florida, 32561, United States
University of Florida Health Science Center Jacksonville
Jacksonville, Florida, 32209, United States
Mayo Clinic Florida
Jacksonville, Florida, 32224, United States
Clinical Translational Research Site
Miami, Florida, 33136, United States
Advanced Pharma Cr, LLC
Miami, Florida, 33147, United States
Nicklaus Children's Hospital
Miami, Florida, 33155, United States
AdventHealth Orlando
Orlando, Florida, 32803, United States
Research Institute of Orlando, LLC
Orlando, Florida, 32806, United States
NeuroMedical Research Institute
Panama City, Florida, 33607, United States
Center for Rare Neurological Diseases
Norcross, Georgia, 30093, United States
Clinical Research Institute
Stockbridge, Georgia, 30281, United States
Hawaii Pacific Neuroscience
Honolulu, Hawaii, 96817, United States
Northwestern Memorial Hospital
Chicago, Illinois, 60611, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Ochsner Health System
New Orleans, Louisiana, 70121, United States
Maine Medical Center
Scarborough, Maine, 04074, United States
Mid-Atlantic Epilepsy And Sleep Center, LLC
Bethesda, Maryland, 20817, United States
Harvard Medical School - Brigham and Women's Hospital
Boston, Massachusetts, 02115, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
Bronson Methodist Hospital
Kalamazoo, Michigan, 49007, United States
SRI International
West Bloomfield, Michigan, 48322, United States
Washington University School of Medicine
St Louis, Missouri, 63110, United States
Impact Clinical Trials Las Vegas
Las Vegas, Nevada, 89106, United States
JFK Medical Center
Edison, New Jersey, 08820, United States
Institute of Neurology & Neurosurgery at St. Barnabas
Livingston, New Jersey, 07039, United States
Rutgers University
New Brunswick, New Jersey, 08901, United States
Dent Neurologic Institute
Amherst, New York, 14226, United States
SUNY Downstate Medical Center - Comprehensive Epilepsy Center
Brooklyn, New York, 11203, United States
Kaleida Health Oishei Children's Hospital
Buffalo, New York, 14203, United States
NYU Comprehensive Epilepsy Center
New York, New York, 10016, United States
Mount Sinai Health System
New York, New York, 10029, United States
Lenox Hill Hospital
New York, New York, 10075, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
Carolinas Neurosciences Institute
Charlotte, North Carolina, 28207, United States
OnSite Clinical Solutions, LLC
Concord, North Carolina, 28025, United States
The Promedica-University of Toledo Neuroscience Center
Toledo, Ohio, 43606, United States
Oregon Health & Science University - Brain Institute - Comprehensive Epilepsy Center
Portland, Oregon, 97239, United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107, United States
Lewis Katz School of Medicine at Template University
Philadelphia, Pennsylvania, 19140, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213, United States
University of Texas Southwestern Medical Center - Neurology Clinic
Dallas, Texas, 75390, United States
UT Houston
Houston, Texas, 77030, United States
Clinical Trial Network
Houston, Texas, 77074, United States
University of Utah Hospital
Salt Lake City, Utah, 84132, United States
Centra Medical Group Neurology Center
Lynchburg, Virginia, 24502, United States
Carilion Roanoke Memorial Hospital
Roanoke, Virginia, 24014, United States
Multi-Care Institute for Research and Innovation
Tacoma, Washington, 98405, United States
Austin Hospital
Heidelberg, Victoria, 3084, Australia
The Alfred
Melbourne, Victoria, 3004, Australia
The Royal Melbourne Hospital
Melbourne, Victoria, 3050, Australia
The Tower
Kingston, Jamaica
Related Publications (1)
French J, Biton V, Dave H, Detyniecki K, Gelfand MA, Gong H, Liow K, O'Brien TJ, Sadek A, DiVentura B, Reich B, Isojarvi J. A randomized phase 2b efficacy study in patients with seizure episodes with a predictable pattern using Staccato(R) alprazolam for rapid seizure termination. Epilepsia. 2023 Feb;64(2):374-385. doi: 10.1111/epi.17441. Epub 2022 Dec 7.
PMID: 36268811DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- UCB
- Organization
- Cares
Study Officials
- STUDY CHAIR
J. Isojarvi, MD, PhD
Engage Therapeutics, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 7, 2018
First Posted
March 27, 2018
Study Start
March 16, 2018
Primary Completion
December 22, 2019
Study Completion
January 4, 2020
Last Updated
February 3, 2021
Results First Posted
February 3, 2021
Record last verified: 2021-02
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, 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.
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 re-identifying 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.