Dravet SyndromePhase 3NCT02823145
An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome
A Phase 3 study testing ZX008 (Fenfluramine Hydrochloride) in people with Dravet Syndrome, run by Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. at 67 sites in 12 countries.
Trial summary
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome. Read more about this study
- Who is it for?
- Ages 2 to 35 years
- Conditions the study is for
- Dravet Syndrome
- 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
- 375 participants
- Where
- 67 sitesin 12 countries
- Study dates
- 8 Jun 2016to 27 Jan 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or non-pregnant, non-lactating female, age 2 to 18 years, inclusive as of the day of the core study Screening Visit.
- Satisfactory completion of the core study in the opinion of the investigator and the sponsor.
- Subjects who are >18 to ≤35 years of age at the time of screening and did not participate in one of the core studies may be eligible for participation.
- A documented medical history to support a clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs.
- Parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability.
- Subject's parent/caregiver has been compliant with diary completion during the core study, in the opinion of the investigator (eg, at least 90% compliant).
Who cannot join
- Current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
- Current cardiac valvulopathy or pulmonary hypertension that is clinically significant and warrants discontinuation of study medication.
- Current or past history of glaucoma.
- Moderate or severe hepatic impairment.
- Receiving concomitant therapy with: centrally-acting anorectic agents; monoamineoxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, and/or cytochrome P450 (CYP) 2D6/3A4/2B6 inhibitors/substrates.
- Currently taking carbamazepine, oxcarbamazepine, eslicarbazepine, phenobarbital, or phenytoin, or has taken any of these within the past 30 days, as maintenance therapy.
- A clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness at Visit 1, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject.
Trial locations and contacts
67 sites in 12 countries.
67 sites in 12 countries
United States24 sites
Clinical Integrative Research Center of Atlanta, Panda Neurology
Atlanta, Georgia, 30328
The Children's Hospital Colorado
Aurora, Colorado, 80045
Massachusetts General Hospital
Boston, Massachusetts, 02114
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44103
Children's Hospital Of Michigan
Detroit, Michigan, 48201
Cook Children's Medical Center
Fort Worth, Texas, 76104
NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, 32561
Institute of Neurology and Neurosurgery at St. Barnabus
Livingston, New Jersey, 07039
Le Bonheur Children's Hospital
Memphis, Tennessee, 38103
Miami Children's Hospital Brain Institute
Miami, Florida, 33155
Children's Hospital of Orange County
Orange, California, 92868
Neurology and Epilepsy Research Center
Orlando, Florida, 32819
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Mayo Clinic
Rochester, Minnesota, 55905
Minnesota Epilepsy Group, P.A.
Saint Paul, Minnesota, 55102
University of Utah
Salt Lake City, Utah, 84113
Rady Children's Hospital San Diego
San Diego, California, 92123
University of California San Francisco
San Francisco, California, 94158
Seattle Children's Hospital
Seattle, Washington, 98105
MultiCare Institute for Research & Innovation
Tacoma, Washington, 98405
Center for Neurosciences - Tucson
Tucson, Arizona, 85718
Germany8 sites
DRK Kliniken Berlin - Westend Epilepsiezentrum / Neuropaediatrie
Berlin
Krankenhaus Mara Epilepsie-Zentrum Bethel
Bielefeld
Universitaetsklinikum Freiburg Zentrum fuer Kinder- und Jugendmedizin
Freiburg im Breisgau
Universitaetsklinikum Jena Klinik fuer Kinder- und Jugendmedizin Neuropaediatrie
Jena
Universitaetsklinikum Schleswig-Holstein Campus Kiel Klinik fuer Neuropaediatrie
Kiel
Kleinwachau Saechsisches Epilepsiezentrum Radeberg gemeinnuetzige GmbH
Radeberg
Universitaetsklinik fuer Kinder- und Jugendmedizin Abteilung III
Tübingen
Schoen Klinik Vogtareuth Neuropaediatrie und Neurologische Rehabilitation, Epilepsiezentrum fuer Kinder und Jugendlische, Tagesklinik fuer Neuropaediatrie
Vogtareuth
Italy8 sites
AOU Anna Meyer Clinica di Neurologia Pediatrica
Florence
Istituto Pediatrico Giannina Gaslini Dipartimento di Neurologia
Genova
A.O. Carlo Poma
Mantua
Istituto Neurologica Carlo Besta
Milan
Ospedale Fatebenefratelli e Oftalmico
Milan
Policlinico A. Gemelli
Roma
U.O. Neurologia Dipartimento di Neuroscienze Ospedale Pediatrico Bambino Gesù
Roma
Ospedale Civile Maggiore di Borgo Trento - Ospedale della Donna e del Bambino
Verona
France6 sites
CHU Amiens-Picardie Service De Neurologie Pediatrique
Amiens
CHU De Bordeaux Service De Pédiatrie Médicale
Bordeaux
CHRU Lille Antenne Pédiatrique Du CIC - Hôpital Jeanne De Flandre
Lille
HOPITAL DEL LA TIMONE - HOPITAL HENRI GASTAUT Hôpital De La Timone Neurologie Pédiatrique Pneumologie Pédiatrique Et Médecine Infantile
Marseille
Hôpital Robert Debré Pôle: Pédiatrie Médicale Service: Neurologie Et Maladies Métaboliques
Paris
Hôpital Universitaire Necker-Enfants Malades Service de neurologie pédiatrique Centre de référence épilepsies rares (CReER)
Paris
United Kingdom6 sites
Birmingham Children Hospital NHS Foundation Trust
Birmingham
Institute of Neurosciences Queen Elizabeth University Hospital
Glasgow
Alder Hey Hospital
Liverpool
Great Ormond Street Hospital for Children NHS Foundation Trust
London
St. Thomas Hospital
London
Sheffield Children's Hospital
Sheffield
Australia3 sites
Melbourne Brain Centre Austin Hospital
Heidelberg
Children's Health Queensland Hospital and Health Service at Lady Cilento Children's Hospital
South Brisbane
The Children's Hospital Westmead Dept. of Neurology and Neurosurgery
Westmead
Japan3 sites
Saitama Children's Medical Center
Saitama
National Epilepsy Center Shizuoka Institute of Epilepsy and Neurological Disorders
Shizuoka
Tokyo Women's Medical University Hospital
Tokyo
Spain3 sites
Hospital Sant Joan de Déu
Barcelona
Hospital Ruber Internacional Primera Planta Servicio de Neurologia
Madrid
Clinica Universitaria de Navarra Fase 4. Segunda planta, Consulta de Pediatria
Pamplona
Canada2 sites
Centre Hospitalier Universitaire Sainte-Justine
Montreal
British Columbia Children's Hospital
Vancouver
Netherlands2 sites
Kempenhaeghe
Heeze
Stichting Epilepsie Instellingen Nederland
Zwolle
About this study
Official title: An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome.
Read the detailed description
This is an international, multicenter, open-label, long-term safety study of ZX008 in pediatric and young adult subjects with Dravet syndrome who participated in one of the core studies (ZX008-1501 and ZX008-1502) and are candidates for continuous treatment for an extended period of time. This trial will consist of a 36-month Open-Label Extension (OLE) Treatment Period and a 2-week Post-Dosing Period.
- Conditions
- Dravet Syndrome
- Being tested
- ZX008 (Fenfluramine Hydrochloride)
- Keywords
- seizure, tonic clonic, epilepsy, myoclonic, encephalopathy
- Medical subject headings
- Epilepsies, Myoclonic, Seizures, Epilepsy, Brain Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZX008 (Fenfluramine Hydrochloride)Drug
| Study group | Role | What they receive |
|---|---|---|
| ZX008 | Experimental | ZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will be titrated to an effective dose beginning with 0.2 mg/kg/day (maximum: 30 mg/day). 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) During the Open-label Extension (OLE) Treatment Period
Treatment-emergent adverse events (TEAE) were defined as any AEs that based on start date information occurs after the first intake of study treatment.
Measured: From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42)
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From Investigational Medicinal Product (IMP) During the OLE Treatment Period
A TEAE was defined as any AE that based on start date information occurs after the first intake of study treatment. Percentage of participants with TEAEs leading to withdrawal from IMP during OLE Treatment Period were reported.
Measured: From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42)
Percentage of Participants With Serious Treatment-emergent Adverse Events (TEAEs) During the OLE Treatment Period
Serious Adverse event (SAE) was defined as any untoward medical occurrence that at any dose: • results in death, • is life-threatening threatening, • results in initial inpatient hospitalization or prolongation of hospitalization, •results in persistent or significant disability or incapacity, • results in a congenital anomaly/birth defect, • results in any medically significant event that did not meet any of the other 5 SAE criteria, but which was judged by a physician to potentially jeopardize the participant or require medical or surgical intervention to prevent one of the above outcomes listed as an SAE criterion.
Measured: From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42)
Secondary outcomes (5)
Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Day 1 to End of Study (EOS) Visit (Month 42) in the OLE Treatment Period
Baseline (Core) was defined as Baseline prior to double-blind treatment in the core studies (ZX008-1501/ZX008-1502, and ZX008-1504 Cohort 2). Participants in 1504- Cohort 1 and de novo participants (who entered 1503 without having been in any of the core studies) did not have a Baseline (Core), and were not included in the analysis of this outcome measure. The total number of convulsive seizures from Day 1 to EOS was divided by the total number of days from Day 1 to EOS with nonmissing diary data and the result was then multiplied by 28 to get a 28-day convulsive seizure frequency (CSF). The change from Baseline for any individual participant was calculated by subtracting the Baseline (Core) from the post-baseline value. Monthly (28 day) CSF was based on electronic diary data obtained for each participant.
Measured: From Day 1 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core)
Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Month 2 to EOS (Month 42) in the OLE Treatment Period
Baseline (Core) was defined as Baseline prior to double-blind treatment in the core studies. Participants in 1504-Cohort 1 and de novo participants did not have a Baseline (Core), and were not included in the analysis of this outcome measure. The total number of convulsive seizures from Month 2 to EOS was divided by the total number of days from Month 2 to EOS with nonmissing diary data and the result was then multiplied by 28 to get a 28-day CSF. The change from Baseline for any individual participant was calculated by subtracting the Baseline (Core) from the post-baseline value. Monthly (28 day) CSF was based on electronic diary data obtained for each participant.
Measured: From Month 2 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core)
Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36)
Monthly (28 day) CSF was based on electronic diary data obtained for each participant. The total number of convulsive seizures in the ith interval (CSF in OLE, where, i=1, 2, 3, … , 14 ) was divided by the total number of days in the ith interval with nonmissing diary data and the result was then multiplied by 28 to get a 28-day CSF of OLE.
Measured: At Month 1, Month 2, Month 3, Month 4-6, Month 7-9, Month 10-12, Month 13-15, Month 16-18, Month 19-21, Month 22-24, Month 25-27, Month 28-30, Month 31-33, and Month 34-36
Convulsive Seizure Frequency (CSF) by Mean Daily Dose During the Overall OLE Treatment Period
Convulsive seizure frequency over time, reported as per 28 days was analyzed by the actual dose administered. Participants were grouped into low (0.2 to <0.4 mg/kg), medium (0.4 to <0.6 mg/kg), and high dose (>0.6 mg/kg) groups depending on their mean daily doses of ZX008 during the OLE Treatment period. For each participant, the seizure frequency per 28 days was calculated as the number of seizures recorded during the period, divided by the number of days in the period and multiplied by 28. The convulsive seizure frequency was calculated from all available data collected.
Measured: From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42)
Percentage of Participants With Changes in Antiepileptic Drug (AED) Medications During First 6 Months of OLE Treatment Period
Participants in the study were required to be on stable background therapy for the first 6 months of treatment, after which background AEDs could be reduced or withdrawn so long as one background AED remained. The percentage of participants who had changes in dose or type of concomitant AED medications during the first, second, third, fourth, fifth, and sixth months were analyzed and reported.
Measured: At Month 1, 2, 3, 4 , 5, and 6 of OLE Treatment Period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jul 2016 (estimated)
- Study start8 Jun 2016
- Primary completion27 Jan 2023
- Study completion27 Jan 2023
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.
- Publications (2)Papers the sponsor has linked to this study.
- 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
- Masking
- None (open label)
- Enrolment
- 375 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- UCB Cares, Study director, 001 844 599 2273
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02823145
- Sponsor's protocol ID
- ZX008-1503
- Other IDs
- 2016-002804-14, 2016
- Status last verified
- Jul 2025
- First submitted
- 13 Jun 2016
- Last updated
- 14 Jul 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 prespecified 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.
Publications
Scheffer IE, Nabbout R, Lagae L, Devinsky O, Auvin S, Thiele EA, Wirrell EC, Polster T, Specchio N, Pringsheim M, Imai K, Lock MD, Langlois M, Roper RZ, Lothe A, Sullivan J. Long-term safety and effectiveness of fenfluramine in children and adults with Dravet syndrome. Epilepsia. 2025 Jun;66(6):1919-1932. doi: 10.1111/epi.18342. Epub 2025 Mar 12.
PubMed 40072476Cross JH, Galer BS, Gil-Nagel A, Devinsky O, Ceulemans B, Lagae L, Schoonjans AS, Donner E, Wirrell E, Kothare S, Agarwal A, Lock M, Gammaitoni AR. Impact of fenfluramine on the expected SUDEP mortality rates in patients with Dravet syndrome. Seizure. 2021 Dec;93:154-159. doi: 10.1016/j.seizure.2021.10.024. Epub 2021 Nov 2.
PubMed 34768178
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome
- ClinicalTrials.gov ID
- NCT02823145
- Acronym / sponsor ID
- ZX008-1503
- Conditions
- Dravet Syndrome
- Keywords
- seizure, tonic clonic, epilepsy, myoclonic, encephalopathy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 375 participants (actual)
- Lead sponsor
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.Industry (pharma, biotech, devices)
- Study start
- 8 Jun 2016 (actual)
- Primary completion
- 27 Jan 2023 (actual)
- Study completion
- 27 Jan 2023 (actual)
- First posted
- 6 Jul 2016 (estimated)
- Results
- Posted 25 Sep 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome.
Detailed description
This is an international, multicenter, open-label, long-term safety study of ZX008 in pediatric and young adult subjects with Dravet syndrome who participated in one of the core studies (ZX008-1501 and ZX008-1502) and are candidates for continuous treatment for an extended period of time. This trial will consist of a 36-month Open-Label Extension (OLE) Treatment Period and a 2-week Post-Dosing Period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ZX008 | Experimental | Drug: ZX008 (Fenfluramine Hydrochloride)ZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will be titrated to an effective dose beginning with 0.2 mg/kg/day (maximum: 30 mg/day). |
Who may qualify
- Ages
- 2 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Male or non-pregnant, non-lactating female, age 2 to 18 years, inclusive as of the day of the core study Screening Visit.
- Satisfactory completion of the core study in the opinion of the investigator and the sponsor.
- Subjects who are >18 to ≤35 years of age at the time of screening and did not participate in one of the core studies may be eligible for participation.
- A documented medical history to support a clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs.
- Parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability.
Key exclusion criteria (7 listed)
- Current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
- Current cardiac valvulopathy or pulmonary hypertension that is clinically significant and warrants discontinuation of study medication.
- Current or past history of glaucoma.
- Moderate or severe hepatic impairment.
- Receiving concomitant therapy with: centrally-acting anorectic agents; monoamineoxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, and/or cytochrome P450 (CYP) 2D6/3A4/2B6 inhibitors/substrates.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZX008 (Fenfluramine Hydrochloride) | Drug | ZX008 |
Primary outcome
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Open-label Extension (OLE) Treatment Period
Time frame: From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42)
Treatment-emergent adverse events (TEAE) were defined as any AEs that based on start date information occurs after the first intake of study treatment.
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From Investigational Medicinal Product (IMP) During the OLE Treatment Period
Time frame: From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42)
A TEAE was defined as any AE that based on start date information occurs after the first intake of study treatment. Percentage of participants with TEAEs leading to withdrawal from IMP during OLE Treatment Period were reported.
Percentage of Participants With Serious Treatment-emergent Adverse Events (TEAEs) During the OLE Treatment Period
Time frame: From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42)
Serious Adverse event (SAE) was defined as any untoward medical occurrence that at any dose: • results in death, • is life-threatening threatening, • results in initial inpatient hospitalization or prolongation of hospitalization, •results in persistent or significant disability or incapacity, • results in a congenital anomaly/birth defect, • results in any medically significant event that did not meet any of the other 5 SAE criteria, but which was judged by a physician to potentially jeopardize the participant or require medical or surgical intervention to prevent one of the above outcomes listed as an SAE criterion.
5 secondary outcomes
Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Day 1 to End of Study (EOS) Visit (Month 42) in the OLE Treatment Period
Time frame: From Day 1 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core)
Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Month 2 to EOS (Month 42) in the OLE Treatment Period
Time frame: From Month 2 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core)
Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36)
Time frame: At Month 1, Month 2, Month 3, Month 4-6, Month 7-9, Month 10-12, Month 13-15, Month 16-18, Month 19-21, Month 22-24, Month 25-27, Month 28-30, Month 31-33, and Month 34-36
Convulsive Seizure Frequency (CSF) by Mean Daily Dose During the Overall OLE Treatment Period
Time frame: From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42)
Percentage of Participants With Changes in Antiepileptic Drug (AED) Medications During First 6 Months of OLE Treatment Period
Time frame: At Month 1, 2, 3, 4 , 5, and 6 of OLE Treatment Period
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 40072476Scheffer IE, Nabbout R, Lagae L, Devinsky O, Auvin S, Thiele EA, Wirrell EC, Polster T, Specchio N, Pringsheim M, Imai K, Lock MD, Langlois M, Roper RZ, Lothe A, Sullivan J. Long-term safety and effectiveness of fenfluramine in children and adults with Dravet syndrome. Epilepsia. 2025 Jun;66(6):1919-1932. doi: 10.1111/epi.18342. Epub 2025 Mar 12.
- Derived from this study: PubMed 34768178Cross JH, Galer BS, Gil-Nagel A, Devinsky O, Ceulemans B, Lagae L, Schoonjans AS, Donner E, Wirrell E, Kothare S, Agarwal A, Lock M, Gammaitoni AR. Impact of fenfluramine on the expected SUDEP mortality rates in patients with Dravet syndrome. Seizure. 2021 Dec;93:154-159. doi: 10.1016/j.seizure.2021.10.024. Epub 2021 Nov 2.
- Individual participant dataSharing planned: 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 prespecified 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.
Related trials for Dravet Syndrome
RecruitingPhase 1/2
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Dravet Syndrome
Active, not recruitingPhase 3
A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
Dravet Syndrome
Enrolling by invitationPhase 3
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)
Developmental and Epileptic Encephalopathy
Enrolling by invitation
Clinical Utility of Reduced EEG Home Monitoring in Fenfluramine Titration for Dravet and LGS
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)
Active, not recruitingPhase 3
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
Dravet Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
UCB Cares study director
001 844 599 2273
67 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ZX008 (Fenfluramine Hydrochloride) in people with Dravet Syndrome, run by Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. at 67 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 67 sites in United States, Germany, Italy, France, United Kingdom and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Sep 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Open-label Extension (OLE) Treatment Period (time frame: From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02823145, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jul 2025.