EpilepsyPhase 3NCT01999777
Study to Evaluate the Safety and Efficacy of USL261 in Patients With Increased Bouts of Seizure Activity in the EMU
A Phase 3 study testing USL261 in people with Epilepsy, run by UCB Biopharma S.P.R.L. at 51 sites in 8 countries.
Trial summary
This study is designed to evaluate the efficacy, safety, and tolerability of USL261 compared with that of intranasal (IN) placebo for the treatment of intermittent bouts of increased seizure activity. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Epilepsy
- What is being tested?
- USL261
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 62 participants
- Where
- 51 sitesin 8 countries
- Study dates
- Nov 2013to Aug 2015
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject has been admitted to the institution's EMU for seizure characterization or pre-surgical evaluation, or such admission is planned within 28 days
- Subject body weight is ≥ 40 kg to ≤ 125 kg (inclusive)
- Subject has an established diagnosis of partial or generalized epilepsy
Who cannot join
- Subject has history of status epilepticus in the 6 months prior to Screening
- Subject 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 12 months
- Subject has respiratory failure (or is at risk for respiratory failure) or other severe cardiorespiratory disease with New York Heart Association Class III or IV functional status, or requires supplemental oxygen
- Subject has acute narrow-angle glaucoma
- Subject is receiving chronic benzodiazepine treatment (defined as an average of ≥ 4 administrations per week) and cannot safely withdraw from such treatment within the washout period prior to treatment
Trial locations and contacts
51 sites in 8 countries.
51 sites in 8 countries
United States37 sites
Study site
Ann Arbor, Michigan
Study site
Augusta, Georgia
Study site
Boston, Massachusetts
Study site
Brooklyn, New York
Study site
Chapel Hill, North Carolina
Study site
Chicago, Illinois
Study site
Columbus, Ohio
Study site
Dallas, Texas
Study site
Denver, Colorado
Study site
Durham, North Carolina
Study site
Edison, New Jersey
Study site
Englewood, Colorado
Study site
Grand Rapids, Michigan
Study site
Hershey, Pennsylvania
Study site
Jackson, Mississippi
Study site
La Jolla, California
Study site
Lebanon, New Hampshire
Study site
Madison, Wisconsin
Study site
Memphis, Tennessee
Study site
Nashville, Tennessee
Study site
New Haven, Connecticut
Study site
New Orleans, Louisiana
Study site
Oklahoma City, Oklahoma
Study site
Orange, California
Study site
Philadelphia, Pennsylvania
Study site
Phoenix, Arizona
Study site
Portland, Oregon
Study site
Rochester, New York
Study site
San Antonio, Texas
Study site
San Francisco, California
Study site
St Louis, Missouri
Study site
Tampa, Florida
Study site
Temple, Texas
Study site
Tucson, Arizona
Study site
Wichita, Kansas
Study site
Winfield, Illinois
Study site
Winston-Salem, North Carolina
About this study
Official title: A Randomized, Double-Blind, Placebo Controlled Trial Examining the Safety and Efficacy of Midazolam Intranasal Spray (USL261) for the Treatment of Intermittent Bouts of Increased Seizure Activity in the Epilepsy Monitoring Unit (EMU)
This study is designed to evaluate the efficacy, safety, and tolerability of USL261 compared with that of intranasal (IN) placebo for the treatment of intermittent bouts of increased seizure activity.
- Conditions
- Epilepsy
- Being tested
- USL261, Placebo
- Keywords
- Epilepsy, seizure clusters, acute repetitive seizures, rescue treatment
- Medical subject headings
- Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
USL261Drug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| USL261 | Experimental | 5 mg intranasal midazolam Receives: Drug: USL261 |
| Placebo | Placebo comparator | intranasal placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants That Were Seizure-free
A participant was considered "seizure-free" if he or she completed the 6-hour Treatment Phase without seizures recorded, premature discontinuation of study drug, rescue intervention for acute central respiratory depression adverse event (AE), and alterations to background anti-epileptic drug (AED) therapy. Otherwise, the participant was included in the analysis for seizure-free events with the outcome of "seizure."
Measured: 6 hours
Secondary outcomes (1)
Time to First Seizure Following Treatment (TFSFT)
Time to first seizure following treatment was defined as time from treatment with study drug to the onset of the next seizure, rescue intervention (for acute central respiratory depression AE) to maintain subject safety, alterations to background AED therapy, early termination, or 6 hours, whichever came first.
Measured: 6 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2013 (estimated)
- Study startNov 2013
- Primary completionAug 2015
- Study completionAug 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for EpilepsyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 62 participants (actual)
Sponsor and investigators
- Lead sponsor
- UCB Biopharma S.P.R.L.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01999777
- Sponsor's protocol ID
- USL261-301
- Status last verified
- Oct 2019
- First submitted
- 25 Nov 2013
- Last updated
- 10 Oct 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Spencer DC, Sinha SR, Choi EJ, Cleveland JM, King A, Meng TC, Pullman WE, Sequeira DJ, Van Ess PJ, Wheless JW. Safety and efficacy of midazolam nasal spray for the treatment of intermittent bouts of increased seizure activity in the epilepsy monitoring unit: A double-blind, randomized, placebo-controlled trial. Epilepsia. 2020 Nov;61(11):2415-2425. doi: 10.1111/epi.16704. Epub 2020 Nov 2.
PubMed 33140403
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo Controlled Trial Examining the Safety and Efficacy of Midazolam Intranasal Spray (USL261) for the Treatment of Intermittent Bouts of Increased Seizure Activity in the Epilepsy Monitoring Unit (EMU)
- ClinicalTrials.gov ID
- NCT01999777
- Acronym / sponsor ID
- USL261-301
- Conditions
- Epilepsy
- Keywords
- Epilepsy, seizure clusters, acute repetitive seizures, rescue treatment
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 62 participants (actual)
- Lead sponsor
- UCB Biopharma S.P.R.L.Industry (pharma, biotech, devices)
- Study start
- Nov 2013
- Primary completion
- Aug 2015 (actual)
- Study completion
- Aug 2015 (actual)
- First posted
- 3 Dec 2013 (estimated)
- Results
- Posted 27 Nov 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is designed to evaluate the efficacy, safety, and tolerability of USL261 compared with that of intranasal (IN) placebo for the treatment of intermittent bouts of increased seizure activity.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| USL261 | Experimental | Drug: USL2615 mg intranasal midazolam |
| Placebo | Placebo comparator | Drug: Placebointranasal placebo |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Subject has been admitted to the institution's EMU for seizure characterization or pre-surgical evaluation, or such admission is planned within 28 days
- Subject body weight is ≥ 40 kg to ≤ 125 kg (inclusive)
- Subject has an established diagnosis of partial or generalized epilepsy
Key exclusion criteria (5 listed)
- Subject has history of status epilepticus in the 6 months prior to Screening
- Subject 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 12 months
- Subject has respiratory failure (or is at risk for respiratory failure) or other severe cardiorespiratory disease with New York Heart Association Class III or IV functional status, or requires supplemental oxygen
- Subject has acute narrow-angle glaucoma
- Subject is receiving chronic benzodiazepine treatment (defined as an average of ≥ 4 administrations per week) and cannot safely withdraw from such treatment within the washout period prior to treatment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| USL261 | Drug | USL261 |
| Placebo | Drug | Placebo |
Primary outcome
Number of Participants That Were Seizure-free
Time frame: 6 hours
A participant was considered "seizure-free" if he or she completed the 6-hour Treatment Phase without seizures recorded, premature discontinuation of study drug, rescue intervention for acute central respiratory depression adverse event (AE), and alterations to background anti-epileptic drug (AED) therapy. Otherwise, the participant was included in the analysis for seizure-free events with the outcome of "seizure."
1 secondary outcome
Time to First Seizure Following Treatment (TFSFT)
Time frame: 6 hours
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33140403Spencer DC, Sinha SR, Choi EJ, Cleveland JM, King A, Meng TC, Pullman WE, Sequeira DJ, Van Ess PJ, Wheless JW. Safety and efficacy of midazolam nasal spray for the treatment of intermittent bouts of increased seizure activity in the epilepsy monitoring unit: A double-blind, randomized, placebo-controlled trial. Epilepsia. 2020 Nov;61(11):2415-2425. doi: 10.1111/epi.16704. Epub 2020 Nov 2.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
51 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing USL261 in people with Epilepsy, run by UCB Biopharma S.P.R.L. at 51 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 51 sites in United States, Belgium, Germany, Spain, Czechia and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Nov 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants That Were Seizure-free (time frame: 6 hours). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01999777, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Oct 2019.