Dravet Syndrome
Conditions
Keywords
seizure, tonic clonic, epilepsy, myoclonic, encephalopathy
Brief summary
This is an exploratory sub-study to ZX008-1503 \[NCT02823145\]. Subjects will be fitted with an Embrace seizure detection watch and seizures detected by the watch will be compared to those entered into an electronic seizure diary.
Detailed description
This sub study will include up to 20 participants who meet the entry criteria for the main Study ZX008-1503 and who are willing to wear the Embrace watch and use the Embrace Alert app for 12 consecutive weeks. Those invited to participate will undergo all procedures included in the main study during their participation in this sub-study,
Interventions
ZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will be titrated to an effective dose beginning with 0.2 mg/kg/day (maximum: 30 mg/day).
Sponsors
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
ZX008-1503 Fenfluramine, open-label
Eligibility
Sex/Gender
ALL
Age
2 Years to 35 Years
Healthy volunteers
No
Inclusion criteria
* Meeting all of the main study ZX008-1503 \[NCT02823145\] inclusion criteria * Subject is willing to wear the Embrace watch on the wrist (alternatively ankle, if needed for younger children). Subjects are asked to wear the watch for as many hours of the day as possible and for the entire night, if possible, for the duration of the sub-study. * Subject's parent/caregiver is willing to use the Alert App. * Subject/subject's caregiver is willing to ensure that the Embrace watch remains within close proximity of the paired iPod Touch running the Empatica Alert app.
Exclusion criteria
* Subject has a known hypersensitivity to any of the Embrace device materials. * Subject has a clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to Visit 1, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Usability of the Empatica Embrace Seizure Detection Watch System (Embrace) in Outpatients With Dravet Syndrome | Approximately 12 weeks | The assessment of user experience with Embrace watch system included Likert Ease of Use Questionnaire. The questionnaire had 3 items related to use of the Embrace watch, Mate App and Alert App. All the questions were responded on 5-point Likert scale (1-Very difficult, 2-difficult, 3-Neutral, 4-Easy and 5-Very easy). Response to each item on the questionnaire was reported. Responses across all items on the questionnaire were summed to create an overall index with total score of 0 to 15, where higher scores represents the better usability of the device. |
Countries
United States
Participant flow
Recruitment details
This exploratory sub-study started to enroll participants in July 2017 and concluded in July 2018. Investigating a rare disease in pediatrics, this exploratory sub-study was terminated due to low enrolment. Of the 20 participants the Sponsor attempted to enroll in the study, only 5 participants enrolled and terminated early from the study.
Participants by arm
| Arm | Count |
|---|---|
| ZX008 Participants received ZX008 (fenfluramine hydrochloride) as an oral solution in a concentration of 2.5 milligrams per milliliter (mg/mL). Participants were titrated to an effective dose beginning with 0.2 milligrams per kilogram per day (mg/kg/day) to maximum dose of 30 mg/day. Study medication was administered twice a day (BID) in equally divided doses with food for 12 consecutive weeks, as per main study protocol, ZX008-1503 \[NCT02823145\]. For this substudy, ZX008-1503-SS01, all participants were required to wear the noninvasive Empatica Embrace watch system (EMBRACE) wrist-worn monitoring device. | 0 |
| Total | 0 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Caregiver withdrew consent | 2 |
| Overall Study | Child would not keep the watch on | 3 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 0 |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
Overall Usability of the Empatica Embrace Seizure Detection Watch System (Embrace) in Outpatients With Dravet Syndrome
The assessment of user experience with Embrace watch system included Likert Ease of Use Questionnaire. The questionnaire had 3 items related to use of the Embrace watch, Mate App and Alert App. All the questions were responded on 5-point Likert scale (1-Very difficult, 2-difficult, 3-Neutral, 4-Easy and 5-Very easy). Response to each item on the questionnaire was reported. Responses across all items on the questionnaire were summed to create an overall index with total score of 0 to 15, where higher scores represents the better usability of the device.
Time frame: Approximately 12 weeks
Population: No data will be reported, as the study was investigating a rare disease in pediatrics with a high risk of participant identification due to low enrolment (participant privacy/confidentiality issue).