A Study to Assess the Usability of the Embrace Seizure Detection Watch in Children and Young Adults With Dravet Syndrome

• ClinicalTrials.gov Identifier: NCT03299842
• Recruitment Status: Terminated
• First Posted: October 3, 2017
• Results First Posted: November 13, 2023
• Last Update Posted: November 13, 2023
• Sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
• Information provided by (Responsible Party): UCB Pharma ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. )

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Dravet Syndrome
• Intervention: Drug: ZX008 (Fenfluramine Hydrochloride)
• Enrollment: 5

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.
Pre-assignment Details

Arm/Group Title	ZX008
Arm/Group Description	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.
Period Title: Overall Study
Started	5
Completed	0
Not Completed	5
Reason Not Completed
Caregiver withdrew consent	2
Child would not keep the watch on	3

Baseline Characteristics

Arm/Group Title		ZX008
Arm/Group Description		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.
Overall Number of Baseline Participants		0
Baseline Analysis Population Description		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).
Age, Categorical; Unit of measure: Participants
	Number Analyzed	0 participants
	<=18 years
	Between 18 and 65 years
	>=65 years
Age, Continuous; Unit of measure: Years	Number Analyzed	0 participants
Sex: Female, Male; Unit of measure: Participants
	Number Analyzed	0 participants
	Female
	Male
Race/Ethnicity, Customized; Unit of measure: Participants	Number Analyzed	0 participants
Race/Ethnicity, Customized; Unit of measure: Participants	Number Analyzed	0 participants

Outcome Measures

1. Primary Outcome

Title	Overall Usability of the Empatica Embrace Seizure Detection Watch System (Embrace) in Outpatients With Dravet Syndrome
Description	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

Outcome Measure Data

Analysis Population Description

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).

Arm/Group Title	ZX008
Arm/Group Description:	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.
Overall Number of Participants Analyzed	0

No data displayed because Outcome Measure has zero total analyzed.

Adverse Events

Time Frame	Approximately 12 weeks
Adverse Event Reporting Description	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).
Arm/Group Title	ZX008
Arm/Group Description	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.
All-Cause Mortality
	ZX008
	Affected / at Risk (%)
Total	0/0
Serious Adverse Events
	ZX008
	Affected / at Risk (%)
Total	0/0
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	ZX008
	Affected / at Risk (%)
Total	0/0

Limitations and Caveats

No data will be reported, as the exploratory sub-study was investigating a rare disease in pediatrics with a high risk of participant identification due to low enrolment (participant privacy/confidentiality issue). Safety Data for eligible participants in the sub-study was collected in the main study ZX008-1503 (NCT02823145) as pre-specified in the protocol. The data will be disclosed with the main study results posting.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: UCB
• Organization: Cares
• Phone: 001 844 599 2273
• EMail: UCBCares@ucb.com

• Responsible Party: UCB Pharma ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. )
• ClinicalTrials.gov Identifier: NCT03299842
• Other Study ID Numbers: ZX008-1503-SS01
• First Submitted: September 10, 2017
• First Posted: October 3, 2017
• Results First Submitted: September 18, 2023
• Results First Posted: November 13, 2023
• Last Update Posted: November 13, 2023