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    Summary
    EudraCT Number:2017-002187-40
    Sponsor's Protocol Code Number:GS-LHON-CLIN-05
    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:2021-01-29
    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 number2017-002187-40
    A.3Full title of the trial
    Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year
    Efficacia e sicurezza dell’iniezione intravitreale bilaterale di GS010: sperimentazione randomizzata, in doppio cieco, controllata con placebo in soggetti affetti da neuropatia ottica ereditaria di Leber dovuta alla mutazione G11778A nel gene ND4 per un massimo di un anno
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy and Safety of Bilateral Intravitreal Injection of GS010
    Efficacia e sicurezza dell’iniezione intravitreale bilaterale di GS010
    A.3.2Name or abbreviated title of the trial where available
    REFLECT
    REFLECT
    A.4.1Sponsor's protocol code numberGS-LHON-CLIN-05
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03293524
    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.1.3.4CountryFrance
    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.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGENSIGHT-BIOLOGICS
    B.5.2Functional name of contact pointRegulatory Affairs Director
    B.5.3 Address:
    B.5.3.1Street Address74 rue du Faubourg Saint-Antoine
    B.5.3.2Town/ cityPARIS
    B.5.3.3Post code75012
    B.5.3.4CountryFrance
    B.5.4Telephone number0033176217233
    B.5.5Fax number0033149230116
    B.5.6E-mailipengue@gensight-biologics.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/3/11/860
    D.3 Description of the IMP
    D.3.1Product nameRecombinant AAV vector serotype 2 containing the human wild typemitochondrial 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 IMPIntravitreal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNlenadogene nolparvovec
    D.3.9.1CAS number 1640969-63-6
    D.3.9.2Current sponsor codeGS010
    D.3.10 Strength
    D.3.10.1Concentration unit titre titre
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number100000000
    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.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboIntravitreal use
    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 assess the efficacy of intravitreal GS010 compared to placebo intravitreal injection in second affected/not yet affected eyes, at 1,5-Year post-treatment, utilizing the change from baseline of the best-corrected visual acuity (BCVA) reported with the Log of the Minimal Angle of Resolution LogMAR), in ND4 LHON subjects with vision loss up to one year.
    Valutare l’efficacia dell’iniezione intravitreale di GS010 rispetto all’iniezione intravitreale di placebo nel secondo occhio affetto/nell’occhio non ancora affetto a 1 anno e mezzo post-trattamento, utilizzando la variazione rispetto al basale della migliore acuità visiva corretta (BCVA) riportata con il logaritmo dell’angolo minimo di risoluzione (LogMAR), in soggetti con LHON dovuta a mutazione nel gene ND4 con perdita della vista fino a un anno.
    E.2.2Secondary objectives of the trial
    1.Assess safety and tolerability of bilateral and unilateral intravitreal injection of GS010. 2.Compare time course of the BCVA LogMAR response in second affected/not yet affected eyes treated with GS010 compared to placebo treatment.3.Assess the efficacy of intravitreal GS010 compared to placebo intravitreal injection in second affected/not yet affected eyes. 4.compare the time course of the response in second affected/not yet affected eyes treated with GS010 compared to placebo treatment and to estimate the magnitude of treatment effect at 1.5 and 2-years posttreatment 5.Verify whether a difference exists at 1.5 and 2-years post-treatment, between first affected eyes and second affected/not yet affected eyes treated with GS010, utilizing both an intra-subject and inter-subject analysis.6.Assess rate of responders in first affected eyes treated with GS010.7.Assess humoral and cellular immune responses 8.Assess impact of bilateral intravitreal GS010 administration on Quality of Life
    1.Valutare sicurezza e tollerabilità dell’iniez intravitr bilat e unilat di GS010.2.Confrontare decorso temp della risp BCVA riportata con LogMAR nel 2°occhio affetto/non ancora affetto trattati con GS010 risp al placebo.3.Valutare a 1.5 e 2 anni post-tratt l’efficacia dell’iniez intravit di GS010 rispetto placebo nel 2°occhio affetto/non ancora affetto,determ la differenza nel tasso di occhi rispondenti. 4.Confrontare il decorso temporale della risposta nel 2°occhio affetto/non ancora affetto trattati con GS010 rispetto al placebo e stimare l’entità dell’effetto del trat a 1.5 e 2 anni post-trat.5.Verificare se c'è differenza a 1.5 e 2 anni post-trattamento tra 1°occhio affetto e 2°occhio affetto/non ancora affetto trat con GS010 utilizzando un’analisi intra e inter-soggetto. 6.Valutare a 1.5 e 2 anni tasso di rispondenti nel 1°occhio affetto trattato con GS010.7.Valutare risp immunitarie umorali e cellulari ad AAV2 dopo somm intravit unilat e bilat.8.Valutare impatto somm intravit
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age 15 years or older on the date of signed informed consent.
    2. Clinically manifested vision loss due to ND4 LHON, to any extent, in at least one eye.
    3. Vision loss duration of < = 365 days (i.e. < = 1 year) in each affected eye at Inclusion Visit (Visit 2).
    4. Female subjects (if of childbearing potential) must agree to use effective methods of birth control for up to 6 months after Treatment Visit(s) 3 and male subjects must agree to use condoms for up to 6 months after Treatment Visit(s) 3.
    5. Ability to obtain adequate pupillary dilation to permit thorough ocular examination and visual testing.
    6. Subject – and parent/legal guardian if the subject is under 18 years of age – has provided signed, written informed consent.
    1.Perdita della vista di durata < =365 giorni (ovvero, < =1 anno) in ciascun occhio affetto alla visita di inclusione (Visita 2).
    2. Ciascun occhio del soggetto deve mantenere un’acuità visiva tale da distinguere almeno il movimento di una mano di fronte al viso (HM), come definito dalle procedure operative standard (SOP) dello studio per il test dell’acuità visiva.
    3. Risultati di genotipizzazione documentati che mostrino la presenza della mutazione G11778A nel gene ND4 e l’assenza di altre mutazioni primarie associate alla LHON (ND1 o ND6) nel DNA mitocondriale del soggetto.
    4. Negatività al test per l’infezione da virus dell’immunodeficienza umana (HIV).
    5. I soggetti di sesso femminile in età fertile devono risultare negativi al test di gravidanza (una donna da due anni in post-menopausa o resa sterile chirurgicamente non viene considerata in età fertile).
    6. Revisione di tutti i criteri di selezione per garantire la conformità costante.
    E.4Principal exclusion criteria
    1.Light Perception (LP) or No Light Perception (NLP) visual acuity in any eye, as defined by the study’s standard operating procedure (SOP) for visual acuity testing.
    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, Visit 2 may be delayed until the 7-day period is complete as long as the study visit windows are adhered to.
    3.Presence of active infectious conjunctivitis, keratitis, scleritis or endophthalmitis in either eye.
    4.Presence of alcoholism, alcohol dependence, or alcohol or drug abuse (excluding nicotine).
    5.Presence of systemic illness or medically significant abnormal laboratory values that are deemed by the Investigator to preclude the subject’s safe participation in the study.
    6.Any medical or psychological condition that, in the opinion of the Investigator, may lead to non-tolerance of the treatment regimen, 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. Any non-selection criteria which may have appeared after the screening visit
    1.Acuità visiva con percezione della luce (LP) o percezione dell’assenza di luce (NLP) in un qualsiasi occhio, come definito dalle procedure operative standard (SOP) dello studio per il test dell’acuità visiva.
    2.Soggetti che assumono idebenone e che non lo hanno interrotto completamente almeno 7 giorni prima della Visita 2. Se il soggetto non ha interrotto idebenone almeno 7 giorni prima della Visita 2, quest’ultima potrà essere ritardata fino al completamento del periodo di 7 giorni, a condizione che vengano rispettate le finestre temporali per la visita dello studio.
    3.Presenza di congiuntivite, cheratite, sclerite o endoftalmite infettiva attiva in uno dei due occhi.
    4.Presenza di alcolismo, dipendenza dall’alcol oppure abuso di alcol o droghe (esclusa la nicotina).
    5.Presenza di malattia sistemica o valori di laboratorio anomali clinicamente significativi che, secondo il parere dello sperimentatore, precludono la partecipazione sicura del soggetto allo studio.
    6.Qualsiasi condizione medica o psicologica che, secondo il parere dello sperimentatore, possa comportare la non tolleranza del regime di trattamento, possa compromettere la partecipazione sicura del soggetto allo studio o che precluderebbe la conformità al protocollo dello studio o la capacità del soggetto di completare lo studio con successo.
    7.Qualsiasi criterio di non selezione che potrebbe essere stato riscontrato dopo la visita di screening.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint will be the BCVA reported with LogMAR at 1-Year post-treatment in second affected/not yet affected eyes of ND4 LHON subjects with vision loss up to one year. The change from baseline (Visit 2) in second affected/not yet affected eyes receiving GS010 and placebo will be the primary response of interest. LogMAR BCVA will be used forstatistical purposes
    L’endpoint primario sarà la BCVA riportata con il LogMAR a 1 anno e mezzo post-trattamento nel secondo occhio affetto/nell’occhio non ancora affetto di soggetti con LHON dovuta a mutazione nel gene ND4 con perdita della vista fino a un anno. La variazione rispetto al basale (Visita 2) nel secondo occhio affetto/nell’occhio non ancora affetto che riceve GS010 e placebo sarà la risposta primaria di interesse. La BCVA riportata con il LogMAR sarà usata per scopi statistici.
    E.5.1.1Timepoint(s) of evaluation of this end point
    at 1,5-Year post-treatment in second affected/not yet affected eyes of ND4 LHON subjects
    Ad 1 anno e mezzo post-trattamento nel secondo occhio affetto/nell’occhio non ancora affetto di soggetti con LHON dovuta a mutazione nel gene ND4
    E.5.2Secondary end point(s)
    LogMAR BCVA for second affected/not yet affected eyes receiving GS010 versus placebo and also for first affected eyes receiving GS010. The change from baseline of the LogMAR BCVA will be used for statistical analyses. Response status over time for second affected/not yet affected eyes receiving GS010 versus placebo and also for first affected eyes receiving GS010.
    BCVA riportata con il LogMAR per il secondo occhio affetto/l’occhio non ancora affetto che riceve GS010 rispetto a placebo e anche per il primo occhio affetto che riceve GS010. La variazione rispetto al basale della BCVA riportata con il LogMAR sarà usata per le analisi statistiche. Lo stato della risposta sarà valutato per il secondo occhio affetto/l’occhio non ancora affetto che riceve GS010 rispetto al placebo e anche per il primo occhio affetto che riceve GS010.
    E.5.2.1Timepoint(s) of evaluation of this end point
    BCVA : each timepoint of the follow-up period and at 2-years post-treatment, Response status over time and at 1,5 and 2-years post-treatment.
    BCVA: ad ogni punto temporale del periodo di follow-up e a 2 anni post-trattamento. Stato della risposta nel tempo e a 1 anno e mezzo e 2 anni post-trattamento.
    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 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 Yes
    E.8.2.3Other No
    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 EEA6
    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
    Belgium
    France
    Italy
    Netherlands
    Spain
    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 months6
    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 months6
    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 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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 76
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 48
    F.4.2.2In the whole clinical trial 90
    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 Follow-up of LHON patients
    Follow-up Standard dei pazienti affetti da LHON
    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 Decision2018-12-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-20
    P. End of Trial
    P.End of Trial StatusOngoing
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