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    Summary
    EudraCT Number:2019-001331-31
    Sponsor's Protocol Code Number:ZX008-1900
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-17
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2019-001331-31
    A.3Full title of the trial
    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
    Studio di estensione in aperto volto a valutare la sicurezza a lungo termine di ZX008 (fenfluramina cloridrato) soluzione orale come terapia aggiuntiva per le crisi convulsive in pazienti con patologie epilettiche rare come le encefalopatie epilettiche, tra cui la sindrome di Dravet e la sindrome di Lennox-Gastaut
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    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
    Studio per indagare la sicurezza a lungo termine di ZX008 (fenfluramina cloridrato) soluzione orale in bambini e adulti con encefalopatie epilettiche, tra cui la sindrome di Dravet e la sindrome di Lennox-Gastaut
    A.3.2Name or abbreviated title of the trial where available
    -
    -
    A.4.1Sponsor's protocol code numberZX008-1900
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03936777
    A.5.4Other Identifiers
    Name:IND NumberNumber:125797; 132604
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorZogenix International Limited
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportZogenix, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationZogenix, Inc.
    B.5.2Functional name of contact pointKunal Kanani
    B.5.3 Address:
    B.5.3.1Street Address5959 Horton Street, Suite 500
    B.5.3.2Town/ cityEmeryville
    B.5.3.3Post codeCA 94608
    B.5.3.4CountryUnited States
    B.5.4Telephone number0015103389964
    B.5.5Fax number000000
    B.5.6E-mailkkanani@zogenix.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/13/1219; EU/3/17/1836
    D.3 Description of the IMP
    D.3.1Product nameFenfluramina cloridrato
    D.3.2Product code [ZX008]
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNFenfluramina cloridrato
    D.3.9.1CAS number 404-82-0
    D.3.9.2Current sponsor codeZX008
    D.3.9.3Other descriptive nameFENFLURAMINE HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB02115MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Dravet syndrome or Lennox-Gastaut syndrome
    Sindrome di Dravet o sindrome di Lennox-Gastaut
    E.1.1.1Medical condition in easily understood language
    Dravet syndrome or Lennox-Gastaut syndrome
    Sindrome di Dravet o sindrome di Lennox-Gastaut
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10048816
    E.1.2Term Lennox-Gastaut syndrome
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10073682
    E.1.2Term Dravet syndrome
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the long-term safety and tolerability of ZX008
    Valutare la sicurezza e la tollerabilità a lungo termine di ZX008
    E.2.2Secondary objectives of the trial
    To assess the effect of ZX008 on the following effectiveness measures:
    - Investigator assessment of convulsive seizure response (<25%, >= 25%,>= 50%, >= 75%, or 100% [ie, seizure-free] improvement)
    - Clinical Global Impression – Improvement (CGI-I) rating, global and symptomatic, as assessed by the investigator
    - CGI-I rating, global and symptomatic, as assessed by the parent/caregiver
    Valutare l’effetto di ZX008 sulle seguenti misure di efficacia:
    - Valutazione dello sperimentatore della risposta alle crisi convulsive (miglioramento del <25%, >= 25%, >= 50%, >= 75%, o 100% [ovvero, libero da crisi convulsive])
    - Valutazione dell’impressione clinica globale – Miglioramento (Clinical Global Impression– Improvement, CGI-I), globale e sintomatica, valutata dallo sperimentatore
    - Valutazione della CGI-I, globale e sintomatica, valutata dal genitore/dalla persona che assiste il soggetto
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Male or non pregnant, non lactating 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
    - Soggetto di sesso maschile o di sesso femminile non in stato di gravidanza né in allattamento
    - Completamento soddisfacente dello studio core
    - Ha un raro disturbo convulsivo, come l'encefalopatia epilettica ed ha completato con successo altri studi clinici sponsorizzati da Zogenix con
    ZX008
    - La persona che assiste il soggetto è disposta e in grado di rispettare la conformità alle procedure dello studio, al programma delle visite e alla contabilità del farmaco dello studio.
    E.4Principal exclusion criteria
    • Current cardiac valvulopathy or pulmonary hypertension that is clinically significant
    • Moderate or severe hepatic impairment
    • Receiving prohibited medication (please see protocol section 5.6.2), within 14 days of receiving ZX008
    - Il soggetto presenta un’attuale valvulopatia o ipertensione polmonare clinicamente significativa
    - Insufficienza epatica moderata o grave
    - Somministrazione di farmaci vietati (si faccia riferimento alla sezione 5.6.2 del protocollo) entro 14 giorni dal trattamento con ZX008
    E.5 End points
    E.5.1Primary end point(s)
    The safety endpoints of the study are:
    • AEs
    • Laboratory safety (hematology, chemistry)
    • Vital signs (blood pressure, heart rate, temperature, and respiratory rate)
    • Physical examination
    • Neurological examination
    • Electrocardiogram (ECGs)
    • Doppler echocardiogram (ECHOs)
    • Body weight/height
    • Chest x-ray (subjects in France and Netherlands only)
    • Electroencephalogram (EEG) (in Italy only)
    Gli endpoint di sicurezza dello studio sono:
    • EA
    • Sicurezza di laboratorio (ematologia, ematochimica)
    • Parametri vitali (pressione sanguigna, frequenza cardiaca, temperatura corporea e frequenza respiratoria)
    • Esame obiettivo
    • Esame neurologico
    • Elettrocardiogramma (ECG)
    • Ecocardiogramma Doppler (ECHO)
    • Peso corporeo / altezza
    • Radiografia del torace (soggetti solo in Francia e Paesi Bassi)
    • Elettroencefalogramma (EEG) (solo in Italia)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 36 months
    Fino a 36 mesi
    E.5.2Secondary end point(s)
    The effectiveness endpoints of the study are:
    • CGI-I, global and symptomatic, as assessed by parent/caregiver
    • CGI-I, global and symptomatic, as assessed by investigator (or designee)
    • Percent improvement in seizure burden as assessed by the investigator (or designee)
    Gli endpoint di efficacia dello studio sono:
    • CGI-I, globale e sintomatica, valutata dal genitore/dalla persona che assiste il soggetto
    • CGI-I, globale e sintomatica, valutata dallo sperimentatore (o da un suo incaricato)
    • Percentuale di miglioramento del carico delle crisi convulsive, valutata dallo sperimentatore (o da un suo incaricato)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to 36 months
    Fino a 36 mesi
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned9
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA43
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Canada
    Japan
    Mexico
    United States
    Belgium
    Denmark
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    Sweden
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days22
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 253
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 192
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 205
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Parent or Legal Guardian will sign consent in case Adult patients will be incapable to give a consent
    In caso di pazienti adulti incapaci di dare un consenso il genitore o il tutore legale firmeranno il consenso
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state41
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 330
    F.4.2.2In the whole clinical trial 650
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Standard care
    Standard care
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-12-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-12-11
    P. End of Trial
    P.End of Trial StatusOngoing
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