A Long-term Safety and Tolerability Study of USL261 in Patients With Seizure Clusters

An Open-Label Safety Study of USL261 in the Outpatient Treatment of Adolescent and Adult 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: Terminated
• Conditions: Epilepsy
• Intervention / Treatment: Drug: USL261

• Study Type: Interventional
• Enrollment (Actual): 7
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Tucson, Arizona, United States
  • California
    • Fresno, California, United States
    • Ventura, California, United States
  • Colorado
    • Aurora, Colorado, United States
  • Florida
    • Port Charlotte, Florida, United States
    • Tampa, Florida, United States
  • Idaho
    • Boise, Idaho, United States
  • Illinois
    • Chicago, Illinois, United States
  • Kentucky
    • Lexington, Kentucky, United States
  • Minnesota
    • Saint Paul, Minnesota, United States
  • Nevada
    • Reno, Nevada, United States
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States
  • Tennessee
    • Nashville, Tennessee, United States
  • Texas
    • Dallas, Texas, United States
    • Houston, Texas, United States
    • San Antonio, Texas, United States

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 65 years (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Has a competent, adult caregiver(s) who is able to recognize and observe the subject's seizure cluster episodes

2. Has an established diagnosis of partial or generalized epilepsy that includes all the following:

   • A documented history of seizure clusters lasting a minimum of 10 minutes, seizure cluster pattern is observable, stereotyped, and recognizably different from the subject's other non-cluster seizure activity (if any)
   • A second seizure in the seizure cluster typically occurs within 6 hours from the time of recognition
   • A seizure cluster pattern composed of multiple (≥ 2) partial or generalized seizures
   • A seizure cluster pattern established >3 months before Visit 1
   • A frequency of ≥ 3 stereotyped seizure clusters during the year before Visit 1
   • At least 1 stereotyped seizure cluster occuring ≤ 4 months before Visit 1
3. Currently on a stable regimen of AED(s) that includes a benzodiazepine
4. Weight is 40 kg to 125 kg, inclusive

Exclusion Criteria:

1. Has a neurological disorder that is likely to progress in the next year
2. Has a severe chronic cardio-respiratory disease
3. Has a psychogenic, non-epileptic seizure(s) within the 5 years before Visit 1
4. Has a history of their stereotypical seizure cluster progressing to status epilepticus within 2 years before Visit 1
5. Has a history of acute narrow-angle glaucoma
6. Has had active suicidal plan/intent or active suicidal thoughts in the 6 months before Visit 1 or a suicide attempt in the past 5 years

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
OTHER: USL261	Drug: USL261; 5 mg, intranasal dose for seizure cluster, may repeat as indicated by protocol; Other Names: Intranasal Midazolam

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Death, Serious Adverse Events, Treatment Emergent Adverse Events (TEAEs) Leading to Discontinuation	Number of Participants with Death, Serious Adverse Events, Treatment emergent adverse events (TEAEs) leading to subject discontinuation from study	Duration of individual subject participation was open-ended. Study ended early. Longest subject participation approximately 6 months.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment Success	Termination of seizure(s) within 10 minutes and no recurrence within 6 hours after study drug administration. Due to early termination of the study, this data was not analyzed.	Duration of individual subject participation was open-ended. Study ended early. Longest subject participation approximately 6 months.

Collaborators and Investigators

• Sponsor: UCB Biopharma S.P.R.L.
• Investigators: Study Director: TzeChiang Meng, MD, Proximagen, LLC

Study record dates

Study Major Dates

• Study Start: May 1, 2014
• Primary Completion (ACTUAL): May 1, 2015
• Study Completion (ACTUAL): May 1, 2015

Study Registration Dates

• First Submitted: June 9, 2014
• First Submitted That Met QC Criteria: June 10, 2014
• First Posted (ESTIMATE): June 11, 2014

Study Record Updates

• Last Update Posted (ACTUAL): October 10, 2019
• Last Update Submitted That Met QC Criteria: October 8, 2019
• Last Verified: October 1, 2019

More Information

Terms related to this study

• Keywords: Epilepsy; open-label; acute repetitive seizures; seizure cluster; intranasal Midazolam; USL261; Upsher-Smith
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Epilepsy; Seizures; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Central Nervous System Depressants; Anesthetics, Intravenous; Anesthetics, General; Anesthetics; Tranquilizing Agents; Psychotropic Drugs; Hypnotics and Sedatives; Adjuvants, Anesthesia; Anti-Anxiety Agents; GABA Modulators; GABA Agents; Midazolam
• Other Study ID Numbers: P261-408; 2014-001604-22 (EUDRACT_NUMBER)