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
- Conditions
- Dravet SyndromeLennox Gastaut SyndromeEpileptic 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 412
- 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
- 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 108
🇺🇸San Diego, California, United States
Ep0215 101
🇺🇸San Francisco, California, United States
Ep0215 106
🇺🇸Salt Lake City, Utah, United States
Ep0215 124
🇺🇸Memphis, Tennessee, United States
Ep0215 1402
🇳🇱Heeze, Netherlands
Ep0215 802
🇧🇪Jette, Belgium
Ep0215 1206
🇮🇹Roma, Italy
Ep0215 107
🇺🇸Tucson, Arizona, United States
Ep0215 104
🇺🇸Miami, Florida, United States
Ep0215 144
🇺🇸Los Angeles, California, United States
Ep0215 115
🇺🇸Gulf Breeze, Florida, United States
Ep0215 103
🇺🇸Aurora, Colorado, United States
Ep0215 117
🇺🇸Atlanta, Georgia, United States
Ep0215 141
🇺🇸Orlando, Florida, United States
Ep0215 121
🇺🇸Winter Park, Florida, United States
Ep0215 110
🇺🇸Chicago, Illinois, United States
Ep0215 112
🇺🇸Boston, Massachusetts, United States
Ep0215 140
🇺🇸Bethesda, Maryland, United States
Ep0215 132
🇺🇸Roseville, Minnesota, United States
Ep0215 109
🇺🇸Rochester, Minnesota, United States
Ep0215 105
🇺🇸Hackensack, New Jersey, United States
Ep0215 118
🇺🇸Livingston, New Jersey, United States
Ep0215 150
🇺🇸Hawthorne, New York, United States
Ep0215 131
🇺🇸Cleveland, Ohio, United States
Ep0215 146
🇺🇸Dallas, Texas, United States
Ep0215 126
🇺🇸Fort Worth, Texas, United States
Ep0215 125
🇺🇸Tacoma, Washington, United States
Ep0215 301
🇦🇺Heidelberg, Australia
Ep0215 302
🇦🇺South Brisbane, Australia
Ep0215 204
🇨🇦Toronto, Canada
Ep0215 202
🇨🇦Montreal, Canada
Ep0215 803
🇧🇪Bruxelles, Belgium
Ep0215 801
🇧🇪Edegem, Belgium
Ep0215 1004
🇫🇷Bordeaux Cedex, France
Ep0215 701
🇩🇰Dianalund, Denmark
Ep0215 1005
🇫🇷Lille, France
Ep0215 201
🇨🇦Vancouver, Canada
Ep0215 1002
🇫🇷Paris, France
Ep0215 1007
🇫🇷Marseille Cedex 05, France
Ep0215 902
🇩🇪Bielefeld, Germany
Ep0215 1001
🇫🇷Paris, France
Ep0215 1008
🇫🇷Salouel, France
Ep0215 908
🇩🇪Kiel, Germany
Ep0215 906
🇩🇪Freiburg Im Breisgau, Germany
Ep0215 905
🇩🇪Jena, Germany
Ep0215 903
🇩🇪Radeberg, Germany
Ep0215 1205
🇮🇹Mantova, Italy
Ep0215 1207
🇮🇹Milano, Italy
Ep0215 1204
🇮🇹Genova, Italy
Ep0215 901
🇩🇪Vogtareuth, Germany
Ep0215 1201
🇮🇹Firenze, Italy
Ep0215 1208
🇮🇹Roma, Italy
Ep0215 1604
🇲🇽Guadalajara, Mexico
Ep0215 1202
🇮🇹Verona, Italy
Ep0215 1702
🇵🇱Bydgoszcz, Poland
Ep0215 1105
🇪🇸Barcelona, Spain
Ep0215 1401
🇳🇱Zwolle, Netherlands
Ep0215 1701
🇵🇱Krakow, Poland
Ep0215 1101
🇪🇸Madrid, Spain
Ep0215 1107
🇪🇸Barcelona, Spain
Ep0215 1102
🇪🇸Pamplona, Spain
Ep0215 502
🇸🇪Göteborg, Sweden
Ep0215 603
🇬🇧Liverpool, United Kingdom
Ep0215 601
🇬🇧Glasgow, United Kingdom
Ep0215 602
🇬🇧London, United Kingdom
Ep0215 606
🇬🇧London, United Kingdom
Ep0215 303
🇦🇺Westmead, Australia
Ep0215 142
🇺🇸New York, New York, United States
Ep0215 119
🇺🇸Seattle, Washington, United States
Ep0215 1103
🇪🇸Esplugues de Llobregat, Spain
Ep0215 605
🇬🇧Birmingham, United Kingdom