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    Summary
    EudraCT Number:2017-002153-11
    Sponsor's Protocol Code Number:GS-LHON-CLIN-06
    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:2018-03-12
    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 number2017-002153-11
    A.3Full title of the trial
    Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials
    Follow-up a lungo termine di ND4 in soggetti affetti da neuropatia ottica ereditaria di Leber (LHON, Leber hereditary optic neuropathy) trattati con la terapia genica oculare GS010 nelle sperimentazioni cliniche di fase III RESCUE o REVERSE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials
    Follow-up a lungo termine di ND4 insoggetti affetti da neuropatia ottica ereditaria di Leber (LHON, Leber hereditary optic neuropathy) trattati con la terapia genica oculare GS010 nelle sperimentazioni cliniche di fase III RESCUE o REVERSE
    A.3.2Name or abbreviated title of the trial where available
    RESCUE and REVERSE Long-term Follow-up
    RESCUE and REVERSE Long-term Follow-up
    A.4.1Sponsor's protocol code numberGS-LHON-CLIN-06
    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 pointMedical Development 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 number0033143142592
    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.2.1.2Country which granted the Marketing AuthorisationItaly
    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 IMPIntravitreal use
    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 No
    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 del 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 long-term safety of intravitreal GS010 administration up to 5 years posttreatment in subjects who were treated in the RESCUE or REVERSE studies.
    Valutazione della sicurezza a lungo termine della somministrazione intravitreale di GS010 fino a 5 anni dopo il trattamento in soggetti che sono stati trattati negli studi RESCUE o REVERSE
    E.2.2Secondary objectives of the trial
    (1) To assess the long-term efficacy of intravitreal GS010 administration up to 5 years post-treatment in subjects who were treated in the RESCUE or REVERSE studies.
    (2) To assess the quality of life (QOL) in subjects who were treated with GS010 in the RESCUE or REVERSE studies for up to 5 years post-treatment.
    1)Valutazione dell'efficacia a lungo termine della somministrazione intravitreale di GS010 fino a 5 anni dopo il trattamento in soggetti che sono stati trattati negli studi RESCUE o REVERSE
    2)Valutazione della qualità della vita (QOL, quality of life) in soggetti che sono stati trattati con GS010 negli studi RESCUE o
    REVERSE fino a 5 anni dopo il trattamento.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject was treated with GS010 IVT injection in either of the RESCUE or REVERSE Phase III clinical studies.
    2. Subject of legal consent age has provided informed consent; subjects that are not of legal consent age have undergone their
    country-approved clinical trial enrollment consent process
    1. Il soggetto è stato trattato con una iniezione intravitreale (IVT) di GS010 negli studi clinici di Fase III RESCUE o REVERSE.
    2. Il soggetto di età del consenso legale ha fornito il consenso informato; i soggetti di età inferiore all'età del consenso legale
    hanno rispettato il procedimento di consenso per l'arruolamento nella sperimentazione clinica approvato dal loro paese.
    E.4Principal exclusion criteria
    1. Subject is unwilling or unable to comply with the protocol requirements.
    2. Subject has any medical or psychological condition that, in the opinion of the Investigator, may
    compromise his or her safe participation in the study.
    3. Subject is taking or intending to take idebenone during the long-term follow-up study period
    1. Il soggetto non desidera o non è in grado di conformarsi ai requisiti del protocollo.
    2. Il soggetto presenta una condizione medica o psicologica che, a giudizio dello sperimentatore, potrebbe compromettere la sicurezza della sua partecipazione allo studio.
    3. Il soggetto sta assumendo o intende assumere idebenone durante il periodo dello studio di follow-up a lungo termine.
    E.5 End points
    E.5.1Primary end point(s)
    Adverse events (AEs) or serious adverse events (SAEs) (ocular or systemic) related to IMP or administration procedure, as judged by the Investigator, reported during the long-term follow-up visits (2, 2.5, 3, 4, and 5 years) from the period of 96 weeks up to 5 years post-treatment and
    summarized descriptively by type, frequency (number, percentage), severity, causal relationship, and seriousness.
    Gli eventi avversi o gli eventi avversi gravi (SAE, serious adverse events) (oculari o sistemici)
    collegati al farmaco sperimentale (IMP) o alla procedura di somministrazione, come da parere dello sperimentatore, riportati
    durante le visite di follow-up a lungo termine (2; 2,5; 3; 4 e 5 anni) dal periodo di 96 settimane fino a 5 anni dopo il trattamento e
    riepilogate in modo descrittivo per tipo, frequenza (numero, percentuale), gravità, relazione causale e serietà.
    E.5.1.1Timepoint(s) of evaluation of this end point
    2, 2.5, 3, 4, and 5 years visits.
    visite a 2, 2.5, 3, 4, e 5 anni.
    E.5.2Secondary end point(s)
    1)Ocular or vision-related adverse events reported during the post-treatment long-term follow-up
    visits from the period of 96 weeks up to 5 years and summarized descriptively by type, frequency (number, percentage), severity, causal relationship, and seriousness. 2) Change in best corrected visual acuity (BCVA) reported with Logarithm of the Minimal Angle of Resolution (LogMAR), change of parameters measured with Humphrey™ visual field (HVF) 30-2, and change of parameters measured with spectral-domain optical coherence tomography (SDOCT)
    in all-, best- and worst-eyes treated with GS010 IVT injection compared to all-, best-, and worst-eyes treated with sham IVT injection respectively, from the RESCUE or REVERSE study baselines and pooled, to each follow-up study time points (2, 2.5, 3, 4, and 5 years). 3) Response status of eyes treated with GS010 IVT injection compared to eyes treated with sham IVT injection, including all-, best- and worst-eyes receiving GS010 or sham respectively, with
    responder eyes defined by: a. An improvement of at least 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (equivalent to a decrease of at least 0.3 LogMAR) compared to baseline, or having a Snellen acuity equivalent better than 20/200. The McNemar test will be used for comparisons from the
    RESCUE or REVERSE study baselines and pooled to each of the follow-up study time points (2, 2.5, 3, 4, and 5 years). b. Eyes that lose less than the 15 ETDRS letters (equivalent to an increase of less than 0.3 LogMAR) compared to the RESCUE or REVERSE study baselines and pooled. 4) Response status of subjects whose ETDRS scores of the treated eye are at least 15 letters better than the sham eye or whose treated eye has a LogMAR acuity of at least 0.3 LogMAR better than the sham eye at each long-term follow-up visit (2, 2.5, 3, 4, and 5 years) in the RESCUE and REVERSE studies and pooled. 5) Time course of the response in eyes treated with GS010 IVT injection compared to eyes treated
    with sham IVT injection, for the BCVA reported with LogMAR, for parameters measured with HVF 30-2 and for parameters measured with SD-OCT, with comparisons from the RESCUE or REVERSE study baselines and pooled, to each follow-up study time point (2, 2.5, 3, 4, and
    5 years) using mixed model repeated measure.
    6) Visual improvement as measured by LogMAR by analysis of covariance (ANCOVA) to determine
    the difference in improvement for ‘best-GS010 eyes’ compared to ‘best-sham eyes’ and for ‘worst-GS010 eyes’ compared to worst-sham eyes, with comparisons from the RESCUE or REVERSE study baselines and pooled to each follow-up study time point (2, 2.5, 3, 4, and 5 years).
    7) Change of ganglion cell layer thickness/volume and topographical map and other parameters
    measured by SD-OCT (e.g., retinal nerve fiber layer and quadrantal thickness analyses) for best-
    GS010 eyes compared to best-sham eyes and for worst-GS010 eyes compared to worst-sham eyes, from the RESCUE or REVERSE study baselines and pooled, to each follow-up study time point
    (2, 2.5, 3, 4, and 5 years) using a mixed model of ANCOVA. 8) Quality of life as measured with the Visual Functioning Questionnaire 25 (VFQ-25) and 36-Item Short Form Health Survey, version 2 (SF-36-v2) subject-rated instruments and summarized descriptively with within-group comparisons from the RESCUE or REVERSE study baselines and pooled, to each follow-up study time point (2, 2.5, 3, 4, and 5 years).
    1. Eventi avversi oculari o correlati alla vista riportati durante le visite di follow-up a lungo termine dopo il trattamento dal periodo di 96 settimane fino a 5 anni e riepilogati in modo descrittivo per tipo, frequenza (numero, percentuale), gravità, relazione causale e serietà. 2. Variazione nella migliore acuità visiva corretta (BCVA, best corrected visual acuity) riportata con logaritmo dell'angolo di risoluzione minimo (LogMAR, Logarithm of the Minimal Angle of Resolution), variazione del campo visivo -2 e variazione dei parametri misurati con tomografia a coerenza ottica nel dominio spettrale (SD-OCT, spectral domain optical coherence tomography) sia nell'occhio migliore che nel peggiore trattato con iniezione intravitreale (IVT) di GS010 comparata sia all'occhio migliore che al peggiore trattato con iniezione intravitreale (IVT) sham dai basali e dai dati aggregati degli studi RESCUE e REVERSE per ciascun punto temporale (2; 2,5; 3; 4 e 5 anni).
    Stato di risposta degli occhi trattati con iniezione intravitreale (IVT) di GS010 comparato agli occhi trattati con iniezione intravitreale (IVT) sham, comprendendo sia gli occhi migliori che i peggiori i quali abbiano ricevuto rispettivamente GS010 e sham, con occhi rispondenti definiti da: a. Un miglioramento di almeno 15 lettere dello studio sul trattamento precoce della retinopatia diabetica (ETDRS, Early Treatment Diabetic Retinopathy Study) (equivalenti a una riduzione di almeno 0,3 LogMAR) comparata al basale o il possesso di un'acuità visiva Snellen migliore di 20/200. Sarà impiegato il test McNemar per le comparazioni dai basali e dai dati aggregati degli studi RESCUE o REVERSE, in ciascun punto temporale dello studio di follow-up (2; 2,5; 3; 4 e 5 anni). b. Gli occhi che perdono meno di 15 lettere ETDRS (equivalenti a un incremento inferiore a 0,3 LogMAR) comparati ai basali e ai dati aggregati degli studi RESCUE o REVERSE.Stato di risposta dei soggetti il cui punteggio ETDRS dell'occhio trattato sia di almeno 15 lettere migliore rispetto all'occhio trattato con sham o la cui acuità LogMAR sia di almeno 0,3 LogMAR migliore rispetto all'occhio trattato con sham, in ciascuna visita di follow-up a lungo termine (2; 2,5; 3; 4 e 5 anni) negli studi e nei dati aggregati di RESCUE e REVERSE. 5. Decorso temporale della risposta negli occhi trattati con iniezione intravitreale (IVT) di GS010 comparata agli occhi trattati con iniezione intravitreale (IVT) sham, per la BCVA riportata con LogMAR, per parametri misurati con HVF 30-2 e per parametri misurati con SD-OCT, con comparazioni dai basali e dai dati aggregati degli studi RESCUE o REVERSE per ciascun punto temporale del follow-up (2; 2,5; 3; 4 e 5 anni) usando una misurazione ripetuta a modello misto (MMRM, mixed model repeated measure). Miglioramento della vista come misurato dal LogMAR mediante analisi della covarianza (ANCOVA, analysis of covariance) per determinare la differenza nel miglioramento dei migliori occhi trattati con GS010 rispetto ai migliori occhi trattati con sham, con comparazioni dai basali e dai dati aggregati dello studio RESCUE o REVERSE per ciascun punto temporale dello studio di follow-up (2; 2,5; 3; 4 e 5 anni). 7. Variazione di spessore/volume dello strato cellulare del ganglio (GCL, ganglion cell layer) e della mappa topografica e altri parametri mediante SD-OCT (ad esempio, analisi dello spessore dello strato della fibra nervosa della retina e del quadrante) per i migliori occhi trattati con GS010 rispetto ai migliori occhi trattati con sham e i peggiori occhi trattati con GS010 rispetto ai peggiori occhi trattati con sham, dai basali e dai dati aggregati dello studio RESCUE o REVERSE, in ciascun punto temporale dello studio di follow-up (2; 2,5; 3; 4 e 5 anni).
    E.5.2.1Timepoint(s) of evaluation of this end point
    2, 2.5, 3, 4, and 5 years visits.
    visite a 2, 2.5, 3, 4, e 5 anni.
    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 Yes
    E.6.13.1Other scope of the trial description
    Follow up without IMP
    Follow up senza IMP
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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.8.3 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.8.5The 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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    France
    Germany
    Italy
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    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 years5
    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 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 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: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 6
    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 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 40
    F.4.2.2In the whole clinical trial 76
    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 Decision2018-03-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-07-12
    P. End of Trial
    P.End of Trial StatusCompleted
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