Dravet SyndromePhase 3NCT03936777
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
A Phase 3 study testing ZX008 (Fenfluramine Hydrochloride) in people with Dravet Syndrome and Lennox Gastaut Syndrome, run by Zogenix, Inc. at 71 sites in 14 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Dravet Syndrome, Lennox Gastaut Syndrome, Epileptic Encephalopathy
- What is being tested?
- ZX008 (Fenfluramine Hydrochloride)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 412 participants
- Where
- 71 sitesin 14 countries
- Study dates
- 22 Apr 2019to 8 May 2025
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
71 sites in 14 countries.
71 sites in 14 countries
United States26 sites
Ep0215 117
Atlanta, Georgia, 30328
Ep0215 103
Aurora, Colorado, 80045
Ep0215 140
Bethesda, Maryland, 20817
Ep0215 112
Boston, Massachusetts, 02114
Ep0215 110
Chicago, Illinois, 60611-2605
Ep0215 131
Cleveland, Ohio, 44106
Ep0215 146
Dallas, Texas, 75207
Ep0215 126
Fort Worth, Texas, 76104
Ep0215 115
Gulf Breeze, Florida, 32561
Ep0215 105
Hackensack, New Jersey, 07601
Ep0215 150
Hawthorne, New York, 10532
Ep0215 118
Livingston, New Jersey, 07039
Ep0215 144
Los Angeles, California, 90095
Ep0215 124
Memphis, Tennessee, 38103
Ep0215 104
Miami, Florida, 33155
Ep0215 142
New York, New York, 10016
Ep0215 141
Orlando, Florida, 32803
Ep0215 109
Rochester, Minnesota, 55905
Ep0215 132
Roseville, Minnesota, 55113
Ep0215 106
Salt Lake City, Utah, 84108
Ep0215 108
San Diego, California, 92123
Ep0215 101
San Francisco, California, 94158
Ep0215 119
Seattle, Washington, 98105
Ep0215 125
Tacoma, Washington, 98405
Ep0215 107
Tucson, Arizona, 85718
Ep0215 121
Winter Park, Florida, 32789
Italy7 sites
Ep0215 1201
Florence
Ep0215 1204
Genova
Ep0215 1205
Mantua
Ep0215 1207
Milan
Ep0215 1206
Roma
Ep0215 1208
Roma
Ep0215 1202
Verona
France6 sites
Ep0215 1004
Bordeaux
Ep0215 1005
Lille
Ep0215 1007
Marseille
Ep0215 1001
Paris
Ep0215 1002
Paris
Ep0215 1008
Salouël
Germany6 sites
Ep0215 902
Bielefeld
Ep0215 906
Freiburg im Breisgau
Ep0215 905
Jena
Ep0215 908
Kiel
Ep0215 903
Radeberg
Ep0215 901
Vogtareuth
Spain5 sites
Ep0215 1105
Barcelona
Ep0215 1107
Barcelona
Ep0215 1103
Esplugues de Llobregat
Ep0215 1101
Madrid
Ep0215 1102
Pamplona
United Kingdom5 sites
Ep0215 605
Birmingham
Ep0215 601
Glasgow
Ep0215 603
Liverpool
Ep0215 602
London
Ep0215 606
London
Australia3 sites
Ep0215 301
Heidelberg
Ep0215 302
South Brisbane
Ep0215 303
Westmead
About this study
Official title: 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
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy
Read the 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.
- Being tested
- ZX008 (Fenfluramine Hydrochloride)
- Medical subject headings
- Epilepsies, Myoclonic, Lennox Gastaut Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZX008 (Fenfluramine Hydrochloride)Drug
Fenfluramine hydrochloride provided in a concentration of 2.5 mg/mL.
| Study group | Role | What they receive |
|---|---|---|
| ZX008 (Fenfluramine Hydrochloride) | Experimental | 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. Receives: Drug: ZX008 (Fenfluramine Hydrochloride) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
The Clinical Global Impression-Improvement (CGI-I) scale is used to assess the severity of illness and global improvement in patients. Global function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The global CGI-I assessment was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Measured: 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
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Global function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The global CGI-I assessment was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Measured: 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
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Cognitive function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of cognition was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Measured: 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
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Behavior is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of behavior was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Measured: 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
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Motor abilities function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of motor abilities was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Measured: 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 Investigator
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Cognitive function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of cognition was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Measured: 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 Investigator
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Behavior is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of behavior was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Measured: 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 Investigator
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Motor abilities is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of motor abilities was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Measured: Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
The change in seizure burden from the previous visit was assessed by the investigator using following categories: <25%, ≥25%, ≥50%, ≥75%, 100% (i.e., seizure-free) improvement. Improvement in seizure burden at Baseline was based on comparison with the participant's last visit in the feeder studies (ZX008-1601, ZX008-1503, and ZX008-1602). Number of participants in each of the 5 categories is reported for each visit as compared to last visit.
Measured: Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2019
- Study start22 Apr 2019
- Primary completion8 May 2025
- Study completion8 May 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Dravet SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single Group Assisgnment
- Masking
- None (open label)
- Enrolment
- 412 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zogenix, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
- Responsible party
- Sponsor
- Investigators
- UCB Cares, Study director, 001 844 599 2273
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03936777
- Sponsor's protocol ID
- ZX008-1900
- Other IDs
- 2019-001331-31, U1111-1309-4338, 2024-515680-61, 2019
- Status last verified
- Nov 2025
- First submitted
- 12 Apr 2019
- Last updated
- 17 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.