Epilepsy
Conditions
Keywords
Epilepsy, seizure cluster, acute repetitive seizures, open-label, intranasal Midazolam, USL261, Upsher-Smith
Brief summary
The purpose of this study is to examine the long-term safety and tolerability of USL261 in the treatment of seizure clusters.
Interventions
DRUGUSL261
5 mg, intranasal dose for seizure cluster, may repeat as indicated by protocol
Sponsors
UCB Biopharma S.P.R.L.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 65 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Death, Serious Adverse Events, Treatment Emergent Adverse Events (TEAEs) Leading to Discontinuation | Duration of individual subject participation was open-ended. Study ended early. Longest subject participation approximately 6 months. | Number of Participants with Death, Serious Adverse Events, Treatment emergent adverse events (TEAEs) leading to subject discontinuation from study |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Treatment Success | Duration of individual subject participation was open-ended. Study ended early. Longest subject participation approximately 6 months. | 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| USL261 USL261: 5 mg, intranasal dose for seizure cluster, may repeat as indicated by protocol | 7 |
| Total | 7 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Treatment Phase | Administrative/Other | 7 |
Baseline characteristics
| Characteristic | USL261 |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 7 Participants |
| Age, Continuous | 27.0 years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 7 Participants |
| Region of Enrollment United States | 7 participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 7 |
| other Total, other adverse events | 4 / 7 |
| serious Total, serious adverse events | 0 / 7 |
Outcome results
Primary
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
Time frame: Duration of individual subject participation was open-ended. Study ended early. Longest subject participation approximately 6 months.
Population: Participants who received at least one dose of study drug
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| USL261 | Number of Participants With Death, Serious Adverse Events, Treatment Emergent Adverse Events (TEAEs) Leading to Discontinuation | 0 Participants |
Secondary
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.
Time frame: Duration of individual subject participation was open-ended. Study ended early. Longest subject participation approximately 6 months.
Population: Not applicable. Only 6 subjects treated at least 1 seizure cluster; of the treated seizure clusters, the majority were in only 2 subjects. Analysis of this endpoint was not performed as the sponsor believed the results would not be interpretable.