A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

Phase 3

Completed

• Conditions: Dravet Syndrome; Lennox Gastaut Syndrome; Epileptic Encephalopathy
• Interventions: Drug: ZX008 (Fenfluramine Hydrochloride)

• Registration Number: NCT03936777
• Lead Sponsor: Zogenix, Inc.

Brief Summary

This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy

Detailed Description

This is an international, multicenter, open-label, long-term safety study of ZX008 in patients with epileptic encephalopathy, including Dravet syndrome or Lennox-Gastaut syndrome. Subjects eligible for participation are those with Dravet syndrome who are currently enrolled in Study ZX008-1503, or those with Lennox-Gastaut syndrome who have successfully completed Study ZX008-1601-Part 2, and are candidates for continued treatment with ZX008 for an extended period of time, or those with Dravet syndrome, Lennox-Gastaut syndrome, or another epileptic encephalopathy who have completed participation in another Zogenix-sponsored study and have been invited to participate in this study.

Study Timeline

• Study First Submitted: 2019-04-12
• Study Start: 2019-04-22
• Study First Submitted QC: 2019-04-30
• Study First Posted: 2019-05-03
• Primary Completion: 2025-05-08
• Study Completion: 2025-05-08
• Status Verified: 2025-06
• Last Update Submitted: 2025-06-06
• Last Update Posted: 2025-06-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 412

Inclusion Criteria

• Male or nonpregnant, nonlactating female
• Satisfactory completion of a core study
• Has a rare seizure disorder, such as epileptic encephalopathy and has successfully completed another Zogenix-sponsored clinical trials with ZX008
• Subject's caregiver is willing and able to be compliant with study procedures, visit schedule and study drug accountability

Exclusion Criteria

• Current cardiac valvulopathy or pulmonary hypertension that is clinically significant
• Moderate or severe hepatic impairment
• Receiving monoamine oxidase inhibitors, serotonin agonists, serotonin antagonists, and serotonin reuptake inhibitors within 14 days of receiving ZX008

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ZX008 (Fenfluramine Hydrochloride)	ZX008 (Fenfluramine Hydrochloride)	ZX008 is supplied as an open-label oral solution.Doses will include up to 0.8 mg/kg/day divided into 2 daily doses, up to a maximum of 30 mg/day (subjects taking concomitant STP will receive up to 0.5 mg/kg/day, up to a maximum of 20 mg/day) in a concentration of 2.5 mg/mL.

Primary Outcome Measures

Name	Time	Method
Change in body weight	Up to 36 months open-label	The analysis will include change in body weight and height by report of BMI in kg/m\^2
Changes in laboratory test results	Up to 36 months open-label	The analysis will include abnormal hematology and chemistry laboratory results outside of reference ranges
Changes in heart rate	Up to 36 months open-label	The analysis will include change in heart rate using standard measure
Changes in heart valve function	Up to 36 months open-label	The analysis will include change in heart valves as measured with standard echocardiogram
Changes in respiratory rate	Up to 36 months open-label	The analysis will include change in resting respiratory rate using standard measure
Changes in heart rhythm	Up to 36 months open-label	The analysis will include changes in heart beat as measured with 12-lead electrocardiogram
Change in Adverse Events and Serious Adverse Events leading to study withdrawal in subjects receiving open label ZX008	Up to 36 months open-label	Evaluate the long term safety and tolerability of oral dose administration of ZX008
Changes in blood pressure	Up to 36 months open-label	The analysis will include change in resting blood pressure using standard measure

Secondary Outcome Measures

Name	Time	Method
Change in Behavioral CGI by Investigator	Up to 36 months open-label	The rating by investigator of the patient's change in behavioral symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".
Change in Motor CGI by Investigator	Up to 36 months open-label	The rating by investigator of the patient's change in motor symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".
Change in convulsive seizure response	Up to 36 months open-label	The analysis will include percent improvement per investigator rating
Change in Cognitive CGI by Parent/Caregiver	Up to 36 months open-label	The rating by parent/caregiver of the patient's change in cognitive symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".
Change in Clinical Global Impression by Parent/Caregiver	Up to 36 months open-label	The rating by parent/caregiver of the patient's overall change in symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse"
Change in Clinical Global Impression by Investigator	Up to 36 months open-label	The rating by investigator of the patient's overall change in symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse"
Change in Behavioral CGI by Parent/Caregiver	Up to 36 months open-label	The rating by parent/caregiver of the patient's change in behavioral symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".
Change in Motor CGI by Parent/Caregiver	Up to 36 months open-label	The rating by parent/caregiver of the patient's change in motor symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".
Change in Cognitive CGI by Investigator	Up to 36 months open-label	The rating by investigator of the patient's change in cognitive symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".

Trial Locations

Locations (71)

Ep0215 107; 🇺🇸 Tucson, Arizona, United States

Ep0215 144; 🇺🇸 Los Angeles, California, United States

Ep0215 108; 🇺🇸 San Diego, California, United States

Ep0215 101; 🇺🇸 San Francisco, California, United States

Ep0215 103; 🇺🇸 Aurora, Colorado, United States

Ep0215 115; 🇺🇸 Gulf Breeze, Florida, United States

Ep0215 104; 🇺🇸 Miami, Florida, United States

Ep0215 141; 🇺🇸 Orlando, Florida, United States

Ep0215 121; 🇺🇸 Winter Park, Florida, United States

Ep0215 117; 🇺🇸 Atlanta, Georgia, United States

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