A Phase 2 interventional study of Staccato Alprazolam and Placebos in Epilepsy, sponsored by Engage Therapeutics, Inc.. Completed at 67 sites in 3 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-02-03.
Sponsored by Engage Therapeutics, Inc. (part of UCB S.A.) · Phase 2, Interventional, and Treatment
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).
1,848 studies on the registry are indexed under Epilepsy; 422 are open to participants now.
This study's enrollment of 156 is above the median of 50 across 1,239 interventional studies indexed under Epilepsy.
Browse Epilepsy studies →Engage Therapeutics, Inc. is the lead sponsor of 2 studies on the registry; none are open to participants now.
First submitted before Engage Therapeutics, Inc. became part of UCB S.A..
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Chronic obstructive pulmonary disease: long-term lung disease that makes it hard to breathe out, including emphysema and chronic bronchitis.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
COPD
Chronic obstructive pulmonary disease: long-term lung disease that makes it hard to breathe out, including emphysema and chronic bronchitis.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
single dose for inhalation
Drug: Staccato Alprazolam
single dose for inhalation
Drug: Staccato Alprazolam
single dose for inhalation
Drug: Placebos
single dose for inhalation
Also known as: STAP-001
single dose for inhalation
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.
Time frame: 2 hours post-dosing on dosing day
Percentage of Participants With Seizure Episode Severity Assessed by Seizure Episode Severity Scale
Severity of on study seizure episode compared to previously experienced seizures was assessed with Seizure Episode Severity Scale. It is a 5-point scale with range from 1 to 5, where 1 indicates much worse than and 5 indicates much better than.
Time frame: 6 hours post-dosing on dosing day
Percentage of Participants With Use of Rescue Medication
Percentage of participants with use of rescue medication to stop a seizure episode at the discretion of the principal investigator were reported.
Time frame: 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)
Percentage of participants who had seizures that evolved to a complex partial seizure and/or a generalized tonic-clonic seizure were reported.
Time frame: 24 hours post-dosing on dosing day
The study started to enroll participants in March 2018 and concluded in January 2020.
| Milestone | Open-label: Staccato Alprazolam (STAP-001) 1.0 Milligram (mg) | Double-blind: Placebo | Double-blind: Staccato Alprazolam 1.0 mg | Double-blind: Staccato Alprazolam 2.0 mg |
|---|---|---|---|---|
| Started | 11 | 50 | 47 | 48 |
| Enrolled participants but not treated | 3 | 10 | 9 | 10 |
| Intent-to-treat (itt) population | 8 | 40 | 38 | 38 |
| Completed | 11 | 50 | 47 | 48 |
| Not completed | 0 | 0 | 0 | 0 |
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.
| percentage of participants | Open-label: Staccato Alprazolam (STAP-001) 1.0 Milligram (mg) | Double-blind: Placebo | Double-blind: Staccato Alprazolam 1.0 mg | Double-blind: Staccato Alprazolam 2.0 mg |
|---|---|---|---|---|
| Percentage of Participants in Each Treatment Group Achieving Seizure Activity Cessation Within 2 Minutes and no Recurrent Seizure Within 2 Hours | 62.5 | 42.5 | 65.8 | 65.8 |
Severity of on study seizure episode compared to previously experienced seizures was assessed with Seizure Episode Severity Scale. It is a 5-point scale with range from 1 to 5, where 1 indicates much worse than and 5 indicates much better than.
| percentage of participants | Open-label: Staccato Alprazolam (STAP-001) 1.0 Milligram (mg) | Double-blind: Placebo | Double-blind: Staccato Alprazolam 1.0 mg | Double-blind: Staccato Alprazolam 2.0 mg |
|---|---|---|---|---|
| Much worse than | 0 | 2.5 | 0 | 0 |
| Worse than | 0 | 5.0 | 2.6 | 5.3 |
| Same as | 12.5 | 42.5 | 57.9 | 55.3 |
| Better than | 50.0 | 35.0 | 18.4 | 28.9 |
| Much better than | 25.0 | 7.5 | 15.8 | 5.3 |
| Not done | 12.5 | 7.5 | 5.3 | 5.3 |
Percentage of participants with use of rescue medication to stop a seizure episode at the discretion of the principal investigator were reported.
| percentage of participants | Open-label: Staccato Alprazolam (STAP-001) 1.0 Milligram (mg) | Double-blind: Placebo | Double-blind: Staccato Alprazolam 1.0 mg | Double-blind: Staccato Alprazolam 2.0 mg |
|---|---|---|---|---|
| Percentage of Participants With Use of Rescue Medication | 0 | 7.5 | 15.8 | 7.9 |
Percentage of participants who had seizures that evolved to a complex partial seizure and/or a generalized tonic-clonic seizure were reported.
| percentage of participants | Open-label: Staccato Alprazolam (STAP-001) 1.0 Milligram (mg) | Double-blind: Placebo | Double-blind: Staccato Alprazolam 1.0 mg | Double-blind: Staccato Alprazolam 2.0 mg |
|---|---|---|---|---|
| Percentage of Participants With Secondary Generalization (Evolution to a Complex Partial Seizure and/or a Generalized Tonic-Clonic Seizure) | 12.5 | 0.0 | 0.0 | 0.0 |
Collected over From Screening up to 12 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Open-label: Staccato Alprazolam (STAP-001) 1.0 Milligram (mg) | 0/8 (0%) | 0/8 (0%) | 3/8 (37.5%) |
| Double-blind: Placebo | 0/40 (0%) | 2/40 (5%) | 10/40 (25%) |
| Double-blind: Staccato Alprazolam 1.0 mg | 0/38 (0%) | 1/38 (2.6%) | 14/38 (36.8%) |
| Double-blind: Staccato Alprazolam 2.0 mg | 0/38 (0%) | 0/38 (0%) | 19/38 (50%) |
| Event | Open-label: Staccato Alprazolam (STAP-001) 1.0 Milligram (mg) | Double-blind: Placebo | Double-blind: Staccato Alprazolam 1.0 mg | Double-blind: Staccato Alprazolam 2.0 mg |
|---|---|---|---|---|
| Generalised tonic-clonic seizureNervous system disorders | 0/8 | 0/40 | 1/38 | 0/38 |
| Seizure clusterNervous system disorders | 0/8 | 1/40 | 0/38 | 0/38 |
| Status epilepticusNervous system disorders | 0/8 | 1/40 | 0/38 | 0/38 |
| PneumoniaInfections and infestations | 0/8 | 1/40 | 0/38 | 0/38 |
| Event | Open-label: Staccato Alprazolam (STAP-001) 1.0 Milligram (mg) | Double-blind: Placebo | Double-blind: Staccato Alprazolam 1.0 mg | Double-blind: Staccato Alprazolam 2.0 mg |
|---|---|---|---|---|
| SomnolenceNervous system disorders | 0/8 | 1/40 | 7/38 | 4/38 |
| CoughRespiratory, thoracic and mediastinal disorders | 1/8 | 0/40 | 4/38 | 7/38 |
| DysgeusiaNervous system disorders | 0/8 | 1/40 | 6/38 | 4/38 |
| Throat irritationRespiratory, thoracic and mediastinal disorders | 1/8 | 0/40 | 0/38 | 2/38 |
| Musculoskeletal chest painMusculoskeletal and connective tissue disorders | 1/8 | 0/40 | 0/38 | 0/38 |
| Gastroenteritis viralInfections and infestations | 1/8 | 0/40 | 0/38 | 0/38 |
| DysmenorrhoeaReproductive system and breast disorders | 1/8 | 0/40 | 0/38 | 0/38 |
| DizzinessNervous system disorders | 0/8 | 2/40 | 2/38 | 2/38 |
| SedationNervous system disorders | 0/8 | 0/40 | 0/38 | 2/38 |
| NauseaGastrointestinal disorders | 0/8 | 1/40 | 1/38 | 2/38 |
Baseline Characteristics refers to Intent-to-treat (ITT) population. ITT population included all participants who had a seizure event and received study drug during the Treatment Period.
| Age, Continuous(years) | Open-label: Staccato Alprazolam (STAP-001) 1.0 Milligram (mg) | Double-blind: Placebo | Double-blind: Staccato Alprazolam 1.0 mg | Double-blind: Staccato Alprazolam 2.0 mg | Total Title |
|---|---|---|---|---|---|
| Mean | 48.1 ± 14.51 | 33.1 ± 9.80 | 34.8 ± 11.18 | 33.5 ± 11.74 | 34.7 ± 11.60 |
| Sex: Female, Male(Participants) | Open-label: Staccato Alprazolam (STAP-001) 1.0 Milligram (mg) | Double-blind: Placebo | Double-blind: Staccato Alprazolam 1.0 mg | Double-blind: Staccato Alprazolam 2.0 mg | Total Title |
|---|---|---|---|---|---|
| Female | 8 | 20 | 21 | 26 | 75 |
| Male | 0 | 20 | 17 | 12 | 49 |
| Race/Ethnicity, Customized(Participants) | Open-label: Staccato Alprazolam (STAP-001) 1.0 Milligram (mg) | Double-blind: Placebo | Double-blind: Staccato Alprazolam 1.0 mg | Double-blind: Staccato Alprazolam 2.0 mg | Total Title |
|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 1 | 0 | 0 | 1 |
| Asian | 0 | 2 | 0 | 1 | 3 |
| Black or African American | 0 | 8 | 2 | 2 | 12 |
| Native Hawaiian / Pacific Islander | 2 | 2 | 0 | 1 | 5 |
| White | 4 | 26 | 36 | 28 | 94 |
| Declined to Answer | 0 | 1 | 0 | 2 | 3 |
| Other | 2 | 0 | 0 | 4 | 6 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — 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 information: Study protocol, Statistical analysis plan, Clinical study report
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 sharing details ↗ · www.vivli.org
This study is completed, as verified in Feb 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Alprazolam