Study to Evaluate the Long-term Safety and Tolerability of USL261 in Patients With Seizure Clusters

January 19, 2023 updated by: UCB Biopharma S.P.R.L.

An Open-Label Safety Study of USL261 in the Outpatient Treatment of Subjects With Seizure Clusters

The purpose of this study is to examine the long-term safety and tolerability of USL261 in the treatment of seizure clusters.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Participants who completed study P261-401 (NCT01390220), a randomized double-blind study of USL261 (intranasal midazolam) versus placebo to acutely treat a seizure cluster episode, were eligible to to enroll in this open-label extension study (P261-402). The participant's caregiver administered a USL261 5 milligram (mg) dose for a seizure episode meeting study criteria. A second USL261 5 mg dose could be administered after 10 minutes and up to 6 hours after the first dose for persistent or recurrent seizures, unless the participant met exclusions to administration of the second dose. A participant could have more than 1 seizure cluster episode treated during his/her study participation.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
175

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Randwick, New South Wales, Australia
        • Australia, New South Wales
    • Victoria
      • Heidelberg west, Victoria, Australia
        • Australia, Victoria
      • Parkville, Victoria, Australia
        • Australia, Victoria
  • Canada
    • Ontario
      • Montreal, Ontario, Canada
        • CANADA
      • Toronto, Ontario, Canada
        • Canada, Toronto
    • Quebec
      • Toronto, Quebec, Canada
        • CANADA
  • Germany
    • Bayern
      • Munchen, Bayern, Germany
        • Germany
    • Hessen
      • Marberg, Hessen, Germany
        • Germany
    • Nordrhein-Westfalen
      • Bonn, Nordrhein-Westfalen, Germany
        • Germany
    • Westfalen-Lippe
      • Bielefeld, Westfalen-Lippe, Germany
        • Germany
  • Hungary
      • Budapest, Hungary
        • Hungary
  • Israel
      • Haifa, Israel
        • Israel
      • Petah Tikvah, Israel
        • Israel
      • Ramat Gan, Israel
        • Israel
  • New Zealand
    • Canterbury
      • Christchurch, Canterbury, New Zealand
        • New Zealand
  • Poland
      • Gdansk, Poland
        • Poland
      • Katowice, Poland
        • Poland
      • Lublin, Poland
        • Poland
  • Spain
      • Madrid, Spain
        • Spain
    • Andalucia
      • Sevilla, Andalucia, Spain
        • Spain
    • Cataluyna
      • Gerona, Cataluyna, Spain
        • Spain
  • Ukraine
      • Ivano-Frankivsk, Ukraine
        • Ukraine
      • Kharkiv, Ukraine
        • Ukraine
      • Odessa, Ukraine
        • Ukraine
      • Poltava, Ukraine
        • Ukraine
      • Ternopil, Ukraine
        • Ukraine
      • Vinnytsa, Ukraine
        • Ukraine
  • United States
    • Arizona
      • Phoenix, Arizona, United States
        • United States, Arizona
      • Scottsdale, Arizona, United States
        • United States, Arizona
      • Tucson, Arizona, United States
        • United States, Arizona
    • Arkansas
      • Little Rock, Arkansas, United States
        • United States, Arkansas
    • California
      • Fresno, California, United States
        • United States, California
      • Sacramento, California, United States
        • United States, California
      • Ventura, California, United States
        • United States, California
    • Colorado
      • Aurora, Colorado, United States
        • United States, Colorado
    • Connecticut
      • New Haven, Connecticut, United States
        • United States, Connecticut
    • Florida
      • Port Charlotte, Florida, United States
        • United States, Florida
      • Wellington, Florida, United States
        • United States, Florida
    • Idaho
      • Boise, Idaho, United States
        • United States, Idaho
    • Illinois
      • Chicago, Illinois, United States
        • United States, Illinois
    • Kentucky
      • Lexington, Kentucky, United States
        • United States, Kentucky
    • Maryland
      • Baltimore, Maryland, United States
        • United States, Maryland
    • Michigan
      • Detroit, Michigan, United States
        • United States, Michigan
    • Minnesota
      • Saint Paul, Minnesota, United States
        • United States, Minnesota
    • Missouri
      • Saint Louis, Missouri, United States
        • United States, Missouri
    • New Hampshire
      • Lebanon, New Hampshire, United States
        • United States, New Hampshire
    • New Jersey
      • Hackensack, New Jersey, United States
        • United States, New Jersey
    • New York
      • Bronx, New York, United States
        • United States, New York
      • New York, New York, United States
        • United States, New York
      • Stony Brook, New York, United States
        • United States, New York
    • North Carolina
      • Durham, North Carolina, United States
        • United States, North Carolina
      • Winston-Salem, North Carolina, United States
        • United States, North Carolina
    • Oklahoma
      • Oklahoma City, Oklahoma, United States
        • United States, Oklahoma
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States
        • United States, Pennsylvania
    • Tennessee
      • Memphis, Tennessee, United States
        • United States, Tennessee
      • Nashville, Tennessee, United States
        • United States, Tennessee
    • Texas
      • Dallas, Texas, United States
        • United States, Texas
      • Greenville, Texas, United States
        • United States, Texas

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Has a competent, adult caregiver who can recognize and observe the subject's seizure cluster episodes
  • Has successfully completed study P261-401, and the subject and caregiver have demonstrated adequate compliance with P261-401 study procedures as determined by the investigator

Exclusion Criteria:

  • Has experienced status epilepticus during or since the P261-401 study
  • In the opinion of the investigator, is experiencing an ongoing, uncontrolled, clinically significant adverse event(s) from P261-401 at Visit 1 or did experience a clinically significant adverse event in study P261-401 that might prevent the subject from safely participating in the study
  • Has a neurological disorder that is likely to progress in the next year
  • Has a history of acute narrow-angle glaucoma
  • Has a medical condition including uncontrolled cardiac, pulmonary, renal, hepatic, or gastrointestinal disease that could interfere with the study, subject safety/safety monitoring, or is not stable despite current therapy
  • Subject has severe chronic cardio-respiratory disease or the need for ambulatory oxygen
  • Has had psychogenic, non-epileptic seizure(s) during or since the P261-401 study
  • Has active suicidal plan or intent as determined by the C-SSRS at Visit 1 or medical history
  • Subject has had vagus nerve stimulator (VNS) implanted since the completion of study P261-401

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: USL261
Intranasal midazolam 5 mg
Drug: USL261

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Duration of Safety Observation
Time Frame: From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Duration of participant study participation for collection of long term safety data
From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants Meeting Predefined Safety Criteria for Vital Signs
Time Frame: From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants meeting predefined safety criteria for vital signs (systolic blood pressure [SBP] <85 mm Hg, SBP change from baseline >/= 40 mm Hg, diastolic BP [DBP] <50 mm Hg, DBP change from baseline >/=30 mm Hg, pulse rate <50 beats per minute (bpm), pulse rate >120 bpm, pulse rate change >/= 40 bpm at any visit post baseline or for caregiver recorded participant respiration rate [RR] <8 breaths per minute (brpm) or >24 brpm) after any USL261 treated seizure cluster episode. Abnormal vital signs were assessed separately by investigator and recorded as adverse events if applicable.
From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants With Laboratory Abnormalities Meeting Predefined Criteria
Time Frame: From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants with abnormal laboratory finding, at any time post baseline, meeting predefined criteria. Abnormal laboratory findings were assessed separately by investigator and recorded as adverse events if applicable. Alanine aminotransferase (ALT); Alkaline phosphatase (ALP); Aspartate aminotransferase (AST); Gamma glutamyl transferase (GGT); upper limit of normal (ULN)
From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants With Clinically Significant Abnormalities Physical Examination
Time Frame: From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants with abnormal findings, at any time post baseline, on physical examination considered clinically significant by the investigator.
From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants With Clinically Significant Abnormalities on Neurologic Examination
Time Frame: From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants with abnormal findings, at any time post baseline, on neurologic examination considered clinically significant by the investigator
From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants With Clinically Significant Abnormalities on Nasal Examination
Time Frame: From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants with abnormal findings, at any time post baseline, on nasal examination considered clinically significant by the investigator
From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participant Change in B-SIT Score
Time Frame: From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Change in participant Brief Smell Identification Test (B-SIT) score from baseline to last visit with assessment. The B-SIT is a self-administered 12-item test; the score indicates odors correctly identified (0 to 12). The B-SIT was added while the study was already ongoing (Protocol Amendment 4, 20 May 2015) in response to a regulatory request. The test was only implemented at sites in the United States and included only participants considered by the investigator to have adequate cognitive ability to perform the test. Baseline was defined as the latest non-missing value prior to administration of USL261 in the Test Dose Phase of Study P261-401.
From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants With Suicidal Ideation
Time Frame: From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants with suicidal ideation reported on Columbia-Suicide Severity Rating Scale (C-SSRS) questionnaire at any post-baseline visit. Responses including: Wish to be Dead; Non-Specific Active Suicidal Thoughts; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; Active Suicidal Ideation with Specific Plan and Intent; and Any Suicidal Ideation Regardless of Type.
From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Emergency Room/Emergency Medical Service Visits
Time Frame: From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.
Participants requiring emergency room (ER)/emergency medical service (EMS) visit within 24 hours after any USL261 treated seizure cluster (including for continued seizures)
From Baseline/(Screening) to End of Safety-Follow-up (up to 56 months) as per assessment table of the study.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Number of Treated Seizure Clusters Meeting Criteria for Treatment Success
Time Frame: 6 hours after first dose of USL261 for each treated seizure cluster
Number of Treated Seizure Clusters Meeting Criteria for Treatment Success: Termination of seizure(s) within 10 minutes and no recurrence within 6 hours after administration of first dose of USL261 (intranasal midazolam 5 mg)
6 hours after first dose of USL261 for each treated seizure cluster

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
UCB Biopharma S.P.R.L.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 1, 2012

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 1, 2017

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 3, 2012

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 7, 2012

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 8, 2012

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 20, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 19, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • P261-402
  • 2011-004109-25 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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