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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03478982




Registration number
NCT03478982
Ethics application status
Date submitted
7/03/2018
Date registered
27/03/2018

Titles & IDs
Public title
Inpatient, Dose-Ranging Study of Staccato Alprazolam in Epilepsy With Predictable Seizure Pattern
Scientific title
A Double-Blind, Placebo-Controlled, Inpatient, Dose-Ranging Efficacy Study of Staccato Alprazolam (STAP-001) in Subjects With Epilepsy With a Predictable Seizure Pattern
Secondary ID [1] 0 0
ENGAGE-E-001
Universal Trial Number (UTN)
Trial acronym
StATES
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Epilepsy 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Staccato Alprazolam
Treatment: Drugs - Placebos

Experimental: Staccato Alprazolam 1.0 mg - single dose for inhalation

Experimental: Staccato Alprazolam 2.0 mg - single dose for inhalation

Placebo comparator: Placebo - single dose for inhalation


Treatment: Drugs: Staccato Alprazolam
single dose for inhalation

Treatment: Drugs: Placebos
single dose for inhalation
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants in Each Treatment Group Achieving Seizure Activity Cessation Within 2 Minutes and no Recurrent Seizure Within 2 Hours
Timepoint [1] 0 0
2 hours post-dosing on dosing day
Secondary outcome [1] 0 0
Percentage of Participants With Seizure Episode Severity Assessed by Seizure Episode Severity Scale
Timepoint [1] 0 0
6 hours post-dosing on dosing day
Secondary outcome [2] 0 0
Percentage of Participants With Use of Rescue Medication
Timepoint [2] 0 0
2 hours post-dosing on dosing day
Secondary outcome [3] 0 0
Percentage of Participants With Secondary Generalization (Evolution to a Complex Partial Seizure and/or a Generalized Tonic-Clonic Seizure)
Timepoint [3] 0 0
24 hours post-dosing on dosing day

Eligibility
Key inclusion criteria
1. 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.
2. Male or female = 18 years of age.
3. 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
4. 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.
5. 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.
6. Subject is able to comply by the requirements of the protocol, particularly the requirements and specific Institution policies during the in-clinic stay.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. History or diagnosis of non-epileptic seizures (e.g. metabolic or pseudo-seizures).
2. History of status epilepticus in the 6 months prior to Screening
3. 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
4. Use of strong CYP 3A4 inhibitors; including azole antifungal agents (e.g., etoconazole, itraconazole), nefazodone, fluvoxamine, cimetidine, HIV protease inhibitors (e.g., ritonavir)
5. Has severe chronic cardio-respiratory disease
6. History of HIV-positivity.
7. Pregnant or breast-feeding.
8. Clinically significant renal or hepatic insufficiency (hepatic transaminases >2 times the upper limit of normal (ULN) or creatinine = 1.5 x ULN).
9. History of acute narrow angle glaucoma, Parkinson's disease, hydrocephalus, or history of significant head trauma.
10. Subjects who use medications to treat airways disease, such as asthma or COPD or have any acute respiratory signs/symptoms (e.g., wheezing).
11. 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
12. A history within the past 1 year of drug or alcohol dependence or abuse.
13. 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).
14. Known allergy or hypersensitivity to alprazolam.
15. History of glaucoma.
16. Subjects who currently have an active major psychiatric disorder where changes in pharmacotherapy are needed or anticipated during the study.
17. Hypotension (systolic blood pressure =90 mm Hg, diastolic blood pressure =50 mm Hg), or hypertension (systolic blood pressure =140 mm Hg, diastolic blood pressure =100 mm Hg) measured while seated at screening or baseline.
18. Significant hepatic, renal, gastroenterologic, cardiovascular (including ischemic heart disease and congestive heart failure), endocrine, neurologic or hematologic disease.
19. Subjects who, in the opinion of the Investigator, should not participate in the study for any reason, including if there is a question about the stability or capability of the subject to comply with the trial requirements.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/03/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
4/01/2020
Sample size
Target
Accrual to date
Final
156
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Austin Hospital - Heidelberg
Recruitment hospital [2] 0 0
The Alfred - Melbourne
Recruitment hospital [3] 0 0
The Royal Melbourne Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
3084 - Heidelberg
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment postcode(s) [3] 0 0
3050 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
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United States of America
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Arizona
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United States of America
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Arkansas
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United States of America
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California
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Colorado
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United States of America
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Connecticut
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United States of America
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District of Columbia
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Florida
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United States of America
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Georgia
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United States of America
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Hawaii
Country [11] 0 0
United States of America
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Illinois
Country [12] 0 0
United States of America
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Kansas
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United States of America
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Louisiana
Country [14] 0 0
United States of America
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Maine
Country [15] 0 0
United States of America
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Maryland
Country [16] 0 0
United States of America
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Massachusetts
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Michigan
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Missouri
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Nevada
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New Jersey
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New York
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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Texas
Country [27] 0 0
United States of America
State/province [27] 0 0
Utah
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United States of America
State/province [28] 0 0
Virginia
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United States of America
State/province [29] 0 0
Washington
Country [30] 0 0
Jamaica
State/province [30] 0 0
Kingston 5

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Engage Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
J. Isojarvi, MD, PhD
Address 0 0
Engage Therapeutics, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
When will data be available (start and end dates)?
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.
Available to whom?
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: http://www.Vivli.org


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03478982