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
An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy for Seizures in Patients With Rare Seizure Disorders Such as Epileptic Encephalopathies Including Dravet Syndrome and Lennox-Gastaut Syndrome
4 other identifiers
interventional
412
14 countries
71
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Apr 2019
Longer than P75 for phase_3
71 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 12, 2019
CompletedStudy Start
First participant enrolled
April 22, 2019
CompletedFirst Posted
Study publicly available on registry
May 3, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 8, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
May 8, 2025
CompletedResults Posted
Study results publicly available
November 17, 2025
CompletedNovember 17, 2025
November 1, 2025
6 years
April 12, 2019
November 3, 2025
November 3, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. An AE can, therefore, be any unfavorable and unintended sign (including an abnormal, clinically significant laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the medicinal (investigational) product. TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment-emergent. The percentage of participants was rounded to one decimal place.
From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
Secondary Outcomes (9)
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)
- +4 more secondary outcomes
Study Arms (1)
ZX008 (Fenfluramine Hydrochloride)
EXPERIMENTALZX008 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.
Interventions
Fenfluramine hydrochloride provided in a concentration of 2.5 mg/mL.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (71)
Ep0215 107
Tucson, Arizona, 85718, United States
Ep0215 144
Los Angeles, California, 90095, United States
Ep0215 108
San Diego, California, 92123, United States
Ep0215 101
San Francisco, California, 94158, United States
Ep0215 103
Aurora, Colorado, 80045, United States
Ep0215 115
Gulf Breeze, Florida, 32561, United States
Ep0215 104
Miami, Florida, 33155, United States
Ep0215 141
Orlando, Florida, 32803, United States
Ep0215 121
Winter Park, Florida, 32789, United States
Ep0215 117
Atlanta, Georgia, 30328, United States
Ep0215 110
Chicago, Illinois, 60611-2605, United States
Ep0215 140
Bethesda, Maryland, 20817, United States
Ep0215 112
Boston, Massachusetts, 02114, United States
Ep0215 109
Rochester, Minnesota, 55905, United States
Ep0215 132
Roseville, Minnesota, 55113, United States
Ep0215 105
Hackensack, New Jersey, 07601, United States
Ep0215 118
Livingston, New Jersey, 07039, United States
Ep0215 150
Hawthorne, New York, 10532, United States
Ep0215 142
New York, New York, 10016, United States
Ep0215 131
Cleveland, Ohio, 44106, United States
Ep0215 124
Memphis, Tennessee, 38103, United States
Ep0215 146
Dallas, Texas, 75207, United States
Ep0215 126
Fort Worth, Texas, 76104, United States
Ep0215 106
Salt Lake City, Utah, 84108, United States
Ep0215 119
Seattle, Washington, 98105, United States
Ep0215 125
Tacoma, Washington, 98405, United States
Ep0215 301
Heidelberg, Australia
Ep0215 302
South Brisbane, Australia
Ep0215 303
Westmead, Australia
Ep0215 803
Brussels, Belgium
Ep0215 801
Edegem, Belgium
Ep0215 802
Jette, Belgium
Ep0215 202
Montreal, Canada
Ep0215 204
Toronto, Canada
Ep0215 201
Vancouver, Canada
Ep0215 701
Dianalund, Denmark
Ep0215 1004
Bordeaux, France
Ep0215 1005
Lille, France
Ep0215 1007
Marseille, France
Ep0215 1001
Paris, France
Ep0215 1002
Paris, France
Ep0215 1008
Salouël, France
Ep0215 902
Bielefeld, Germany
Ep0215 906
Freiburg im Breisgau, Germany
Ep0215 905
Jena, Germany
Ep0215 908
Kiel, Germany
Ep0215 903
Radeberg, Germany
Ep0215 901
Vogtareuth, Germany
Ep0215 1201
Florence, Italy
Ep0215 1204
Genova, Italy
Ep0215 1205
Mantova, Italy
Ep0215 1207
Milan, Italy
Ep0215 1206
Roma, Italy
Ep0215 1208
Roma, Italy
Ep0215 1202
Verona, Italy
Ep0215 1604
Guadalajara, Mexico
Ep0215 1402
Heeze, Netherlands
Ep0215 1401
Zwolle, Netherlands
Ep0215 1702
Bydgoszcz, Poland
Ep0215 1701
Krakow, Poland
Ep0215 1105
Barcelona, Spain
Ep0215 1107
Barcelona, Spain
Ep0215 1103
Esplugues de Llobregat, Spain
Ep0215 1101
Madrid, Spain
Ep0215 1102
Pamplona, Spain
Ep0215 502
Gothenburg, Sweden
Ep0215 605
Birmingham, United Kingdom
Ep0215 601
Glasgow, United Kingdom
Ep0215 603
Liverpool, United Kingdom
Ep0215 602
London, United Kingdom
Ep0215 606
London, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- UCB
- Organization
- Cares
Study Officials
- STUDY DIRECTOR
UCB Cares
001 844 599 2273
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Masking Details
- None (open label)
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 12, 2019
First Posted
May 3, 2019
Study Start
April 22, 2019
Primary Completion
May 8, 2025
Study Completion
May 8, 2025
Last Updated
November 17, 2025
Results First Posted
November 17, 2025
Record last verified: 2025-11
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, CSR
- Time Frame
- Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
- Access Criteria
- Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.