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    Summary
    EudraCT Number:2017-002153-11
    Sponsor's Protocol Code Number:GS-LHON-CLIN-06
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-10-03
    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 ConcernedFrance - ANSM
    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
    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
    A.3.2Name or abbreviated title of the trial where available
    GS010 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 pointISABELLE PENGUE KOYI
    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 number33176217233
    B.5.5Fax number33149230116
    B.5.6E-mailipengue@gensight-biologics.com
    D. IMP Identification
    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
    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
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    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 post-treatment in subjects who were treated in the RESCUE or REVERSE studies.
    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
    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.
    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.
    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
    E.5.1.1Timepoint(s) of evaluation of this end point
    2, 2.5, 3, 4, and 5 years visits
    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).

    E.5.2.1Timepoint(s) of evaluation of this end point
    2, 2.5, 3, 4, and 5 years visits
    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 study without 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) 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 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.8.2.4Number 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 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 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
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    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 state
    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 Decision2018-02-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-01-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-05-25
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