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    EudraCT Number:2015-001266-26
    Sponsor's Protocol Code Number:GS-LHON-CLIN-03B
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-05-10
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2015-001266-26
    A.3Full title of the trial
    Sperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valutare l'efficacia di un'unica iniezione intravitreale di GS010 (rAAV2/2-ND4) in soggetti affetti, per un periodo maggiore di 6 mesi e fino a 12 mesi da neuropatia ottica ereditaria di Leber a causa della mutazione G11778A nel gene mitocondriale NADH deidrogenasi 4
    A Randomized, Double-Masked, Sham-Controlled, Pivotal Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 (rAAV2/2-ND4) in Subjects Affected for More Than 6 Months and To 12 Months by Leber Hereditary Optic Neuropathy Due to the G11778A Mutation in the Mitochondrial NADH Dehydrogenase 4 Gene
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase III gene therapy clinical trial in LHON Subjects Affected for more 7 months
    Studio clinico di terapia genica di fase III in soggetti affetti da LHON per un periodo maggiore di 7 mesi
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberGS-LHON-CLIN-03B
    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 SponsorGENSIGHT BIOLOGICS
    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 supportGENSIGHT BIOLOGICS
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRegulatory Pharma Net srl
    B.5.2Functional name of contact pointAnita Falezza
    B.5.3 Address:
    B.5.3.1Street AddressCorso Italia 108
    B.5.3.2Town/ cityPisa
    B.5.3.3Post code56125
    B.5.4Telephone number050 503954
    B.5.5Fax number050 2204315
    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/3/11/860
    D.3 Description of the IMP
    D.3.1Product nameRecombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene
    D.3.2Product code GS010
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1640969-63-6
    D.3.9.2Current sponsor coderAAv2/2-ND4
    D.3.9.3Other descriptive nameRAAV2/2-ND4 VECTOR
    D.3.9.4EV Substance CodeSUB129740
    D.3.10 Strength
    D.3.10.1Concentration unit titre titre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000000000000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    D. cell therapy medicinal product No
    D. therapy medical product Yes
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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
    Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene
    Neuropatia ottica ereditaria di Leber dovuta a mutazione nel gene mitocondriale NADH deidrogenasi 4
    E.1.1.1Medical condition in easily understood language
    LHON: Genetic disease of the optic nerve which leads to visual loss and development of blindness
    LHON: disturbo genetico del nervo ottico che porta a perdita della vista e sviluppo di cecità
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10062951
    E.1.2Term Leber's hereditary optic atrophy neuropathy
    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 evaluate the efficacy of GS010 compared with sham at Week 48 in the change from baseline of the Log of the Minimal Angle of Resolution (LogMAR) in subjects affected for more than 6 months and to 12 months by LHON.
    Valutare l'efficacia di GS010 rispetto alla simulazione in settimana 48 nella variazione dal basale del logaritmo del minimo angolo di risoluzione (LogMAR) in soggetti affetti da LHON da piu’ di 6 mesi e fino a 12 mesi.
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of GS010 compared with sham over the follow-up period and at Week 96 in the change from baseline of the LogMAR.
    To verify whether the efficacy at Week 48 and at Week 96 of GS010 compared with sham and measured by the change from baseline in the LogMAR is dependent upon the treatment of the better- or worse-seeing-eye.
    To verify whether the rate of responders at Week 48 and 96 is dependent upon the treatment received and whether the magnitude of the treatment effect is dependent the treatment of the better- or worse-seeing eye at entry.
    To assess the effect of GS010 on parameters measured with high resolution spectral-domain Optical Coherence Tomography (SDOCT).
    To assess the effect of GS010 on standardized automated visual fields obtained with the Humphrey Visual Field (HVF) Analyzer II
    To assess the effect of GS010 on contrast sensitivity measured with the Pelli-Robson chart
    To assess the effect of GS010 on color vision measured with the Farnsworth-Mu..
    Valutare l'efficacia di GS010 rispetto alla simulazione nel periodo di follow-up in settimana 96 nella variazione dal basale del LogMAR.
    Verificare se l'efficacia in Settimana 48 e in Settimana 96 di GS010 rispetto alla simulazione e misurata dalla variazione dal basale nel LogMAR dipenda dal trattamento dell'occhio che vede meglio o dell'occhio che vede peggio.
    Verificare se il tasso di responder in Settimana 48 e in Settimana 96 dipenda dal trattamento ricevuto e se l'ampiezza dell'effetto del trattamento sia dipendente dal trattamento dell'occhio che vede meglio o dell'occhio che vede peggio all'ingresso.
    Verificare l'effetto di GS010 sui parametri misurati con la tomografia a coerenza ottica a dominio spettrale (spectral-domain optical coherence tomography, SD-OCT) ad alta risoluzione.
    Valutare l'effetto di GS010 sui campi visivi automatizzati standardizzati ottenuti con l'Analizzatore II del Campo visivo Humphrey (Humphrey Visual Field, HVF).
    Valutare l'effetto di GS01 sulla sen..
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Selection Criteria:
    1. Age 18 years or older.
    2. Onset of vision loss based on medically documented history or subject testimony, in both eyes for 181 and ≤365 days in duration.
    3. Each eye of the subject maintaining visual ability to allow at least for counting of the examiner's fingers at any distance.
    4. Female subjects (if of childbearing potential) must agree to use effective methods of birth control up to 6 months after IVT injection and male subjects must agree to use condoms for up to 6 months after IVT injection.
    5. Ability to obtain adequate pupillary dilation to permit thorough ocular examination and testing.
    6. Signed written informed consent.

    Inclusion criteria:
    Subjects included in the study must satisfy all the following criteria at the Inclusion Visit (Visit 2).
    1. Documented results of genotyping showing the presence of the G11778A mutation in the ND4 gene and the absence of the other primary LHON-associated mutations (ND1 or ND6) in the subject's mitochondrial DNA.
    2. Review of all selection criteria to ensure continued compliance.
    3. Have a negative test for infection with human immunodeficiency virus (HIV).
    4. Have a negative pregnancy test for women of childbearing potential (a woman who is two years post-menopausal or surgically sterile is not considered to be of childbearing potential).
    Criteri di selezione
    I soggetti devono soddisfare tutti i seguenti criteri alla Visita di screening (Visita 1) per potere essere inclusi nello studio:
    1. Avere compiuto 18 anni.
    2. Insorgenza di perdita della vista basata su anamnesi documentata o testimonianza del soggetto, in entrambi gli occhi deve essere 181 a ≤365 giorni.
    3. Ciascun occhio del soggetto deve mantenere una capacità visiva che consenta di contare le dita dell'esaminatore a qualsiasi distanza.
    4. I soggetti di sesso femminile (se potenzialmente fertili) devono accettare di utilizzare metodi efficaci di controllo delle nascite fino a 6 mesi dopo l'iniezione IVT e quelli di sesso maschile devono accettare di utilizzare il preservativo fino a un massimo di 6 mesi dopo l'iniezione IVT.
    5. Capacità di ottenere un'adeguata dilatazione delle pupille per permettere un esame oculare e analisi approfonditi.
    6. Consenso informato scritto firmato.

    Criteri di inclusione
    I soggetti inclusi nello studio devono soddisfare tutti i seguenti criteri alla Visita di inclusione (Visita 2).
    1. Risultati documentati di genotipizzazione che mostrano la presenza della mutazione G11778A nel gene ND4 e l'assenza di altre mutazioni primarie associate a LHON (ND1 o ND6) nel DNA mitocondriale del soggetto.
    2. Analisi di tutti i criteri di selezione per assicurare la continuità della compliance.
    3. Risultato negativo del test per l'infezione da virus della immuno-deficienza umana (HIV).
    4. Risultato negativo del test di gravidanza per le donne potenzialmente fertili (la donna che sia in post-menopausa da 2 anni o chirurgicamente sterile non viene considerata potenzialmente fertile).
    E.4Principal exclusion criteria
    Non-selection criteria:
    Subjects who meet at least one of the following criteria at the Screening Visit (Visit 1) will not be included into the study.
    1. Any known allergy or hypersensitivity to GS010 or its constituents.
    2. Contraindication to IVT injection.
    3. IVT drug delivery to either eye within 30 days prior to the Screening Visit (Visit 1).
    4. Previous vitrectomy in either eye.
    5. Narrow angle in either eye contra-indicating pupillary dilation.
    6. Presence of disorders of the ocular media, such as the cornea and lens, which may interfere with visual acuity and other ocular assessments during the study period.
    7. Vision disorders, other than LHON, involving visual disability or with the potential to cause further vision loss during the trial period.
    8. Causes of optic neuropathy other than LHON and glaucoma.
    9. Subjects with known mutations of other genes involved in pathological retinal or optic nerve conditions.
    10. Presence of ocular or systemic disease, other than LHON, whose pathology or associated treatments might affect the retina or the optic nerve.
    11. History of amblyopia associated with a Snellen visual acuity equivalent of worse than 20/80 (equivalent to 6/24 at 6 meters, decimal acuity 0.25, LogMAR +0.6) in the affected eye.
    12. Presence of ocular conditions, which in the opinion of the Investigator will prevent good quality SD-OCT imaging from being obtained.
    13. Presence, in either eye, of uncontrolled glaucoma, defined as an IOP greater than 25 mmHg, despite maximal medical therapy with IOP lowering agents.
    14. Active ocular inflammation or history of idiopathic or autoimmuneassociated uveitis.
    15. Subjects participating in another clinical trial and receiving an IMP within 90 days prior to the Screening Visit (Visit 1).
    16. Previous treatment with an ocular gene therapy product.
    17. Subjects who have undergone ocular surgery of clinical relevance (per Investigator opinion) within 90 days preceding the Screening Visit (Visit 1).
    18. Female Subjects who are or who intend to breast feed during the trial period.

    Exclusion criteria:
    Subjects who meet at least one of the following criteria at the Inclusion Visit (Visit 2) will not be included in the study.
    1. Any non-selection criteria which may have appeared after the screening visit.
    2. Subjects taking idebenone who have not completely discontinued the idebenone at least 7 days prior to Visit 2. If the subject has not discontinued idebenone at least 7 days prior to Visit 2, the visit may be delayed until the 7-day period is complete.
    3. Presence, at the time of study inclusion, of infectious conjunctivitis, keratitis, scleritis or endophthalmitis in either eye.
    4. Presence of systemic illness, including alcohol and drug abuse (except nicotine), or medically significant abnormal laboratory values that are deemed by the Investigator to preclude the subject's safe participation in the study.
    5. Presence of illness or disease that, in the opinion of the Investigator, include symptoms and/or the associated treatments that can alter visual function, for instance cancers or pathology of the central nervous system.
    6. Any medical or psychological condition that, in the opinion of the Investigator, may compromise the safe participation of the subject in the study or would preclude compliance with the study protocol or ability of the subject to successfully complete the study.
    7. Subjects unable or unwilling to comply with the protocol requirements.
    Criteri di non selezione
    I soggetti che soddisfano almeno uno dei seguenti criteri alla Visita di screening (Visita 1) non verranno inclusi nello studio:
    1. Qualunque allergia o ipersensibilità nota a GS010 o ai suoi costituenti.
    2. Controindicazione all'iniezione IVT.
    3. Somministrazione di farmaco IVT in uno degli occhi nei 30 giorni precedenti alla Visita di screening (Visita 1).
    4. Precedente vitrectomia in uno degli occhi.
    5. Angolo stretto in uno degli occhi che è una controindicazione alla dilatazione della pupilla.
    6. Presenza di patologie del bulbo oculare, come la cornea e il cristallino, che possono interferire con l'acuità visiva e con altre valutazioni oculari durante il periodo di studio.
    7. Patologie della vista, diverse da LHON, che comportano disabilità visiva o con la potenzialità di determinare un'ulteriore perdita della vista durante il periodo della sperimentazione.
    8. Cause di neuropatia ottica diverse da LHON e glaucoma.
    9. Soggetti con mutazioni note di altri geni coinvolti in condizioni patologiche retiniche o del nervo ottico.
    10. Presenza di malattia oculare o sistemica, diversa da LHON, la cui patologia o i cui trattamenti associati possano interessare la retina o il nervo ottico.
    11. Anamnesi di ambliopia associata a un'acuità visiva di Snellen equivalente a o peggiore di 20/80 (equivalente a 6/24 a 6 metri, acuità decimale 0,25, LogMAR +0,6) nell'occhio affetto.
    12. Presenza di patologie oculari, che nel parere dello Sperimentatore impediranno l'ottenimento di immagini SD-OCT di buona qualità.
    13. Presenza, in uno dei due occhi, di glaucoma incontrollato, definito come una pressione intraoculare (intraocular pressure, IOP) superiore a 25 mmHg, nonostante la terapia medica massima con agenti per la riduzione della IOP.
    14. Infiammazione oculare attiva o anamnesi di uveite idiopatica o autoimmune-associata.
    15. Soggetti che partecipano a un'altra sperimentazione clinica e che assumono un IMP nei 90 giorni precedenti alla Visita di screening (Visita 1).
    16. Precedente trattamento con prodotto di terapia genica oculare.
    17. Soggetti sottoposti a chirurgia oculare di rilevanza clinica (in base al parere dello Sperimentatore) nei 90 giorni precedenti alla Visita di screening (Visita 1).
    18. Soggetti di sesso femminile che sono in allattamento o intendono allattare durante il periodo della sperimentazione.

    Criteri di esclusione
    I soggetti che soddisfano almeno uno dei seguenti criteri alla Visita di inclusione (Visita 2) non verranno inclusi nello studio.
    1. Qualsiasi criterio di non selezione che possa essere comparso dopo la visita di screening.
    2. Soggetti che assumono idebenone che non hanno interrotto completamente l'idebenone almeno 7 giorni prima della Visita 2. Se il soggetto non ha interrotto l'idebenone almeno 7 giorni prima della Visita 2, la visita può essere posticipata finché il periodo di 7 giorni non sia raggiunto.
    3. Presenza, al momento dell'inclusione nello studio, di congiuntivite infettiva, cheratite, sclerite o endoftalmite in uno dei due occhi.
    4. Presenza di malattia sistemica, compreso abuso di droghe (esclusa la nicotina) e di alcool, o valori di laboratorio anormali e clinicamente significativi che nel parere dello Sperimentatore precludano la sicurezza della partecipazione del soggetto allo studio.
    5. Presenza di patologia o malattia che, nel parere dello Sperimentatore, includa sintomi e/o trattamenti associati che possano alterare la funzione visiva, ad esempio tumori o patologie del sistema nervoso centrale.
    6. Qualsiasi condizione medica o psicologica che, nel parere dello Sperimentatore, possa compromettere la sicurezza della partecipazione del soggetto allo studio o che potrebbe precludere l'ottemperanza al protocollo dello studio o la capacità del soggetto di completare lo studio con successo.
    7. Soggetti incapaci di o non disposti a rispettare i requisiti del protocollo.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint will be the ETDRS visual acuity (quantitative score) at Week 48 after IVT injection. The subjects’ LogMAR scores, which are derived from the number of letters they read on the ETDRS chart, will be used for statistical analysis purposes. The change from baseline in each eye will be the primary response of interest.
    L'endpoint primario sarà l'acuita visiva con ETDRS (punteggio quantitativo) in Settimana 48 dopo l'iniezione IVT. I punteggi LogMAR dei soggetti, che sono ottenuti dal numero di lettere che essi leggono sulla tavola ETDRS, verranno utilizzati a fini di analisi statistica. La variazione dal basale in ciascun occhio sarà la risposta primaria di interesse.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Full analysis = 48 weeks
    Full analysis = 48 settimane
    E.5.2Secondary end point(s)
    Measure of color vision with the Farnsworth-Munsell 100 Hue color vision test at Week 48 and Week 96.
    Misurazione della visione dei colori con il test di Farnsworth-Munsell 100 Hue della visione dei colori in Settimana 48 e in Settimana 96.
    E.5.2.1Timepoint(s) of evaluation of this end point
    48 and 96 weeks after the injection of GS010
    48 e 96 settimane dopo l'iniezione di GS010
    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 No
    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. 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E. description
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    United Kingdom
    United States
    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
    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 years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 1
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 38
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 Yes
    F.3.3.2Women of child-bearing potential using contraception No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 28
    F.4.2.2In the whole clinical trial 40
    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)
    Terapie standard dei pazienti affetti da LHON
    Standard follow-up of LHON patients
    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 Decision2016-03-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-12-16
    P. End of Trial
    P.End of Trial StatusCompleted
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