Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43851   clinical trials with a EudraCT protocol, of which   7283   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2017-002153-11
    Sponsor's Protocol Code Number:GS-LHON-CLIN-06
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2018-02-14
    Trial results View 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 ConcernedUK - MHRA
    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.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.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 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 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
    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.1.2 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 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 posttreatment 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 contrast sensitivity measured with the Pelli-Robson chart 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 the follow-up study time points (2, 3, 4, and 5 years).

    3) 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).

    4) Response status of eyes treated with GS010 IVT injection compared to eyes treated with sham IVT injection, including all-, best- and worsteyes 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.

    5) 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.

    6) 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 followup study time point (2, 2.5, 3, 4, and
    5 years) using mixed effects models with repeated measures (MMRM).

    7) Time course of the response in eyes treated with GS010 IVT injection compared to eyes treated with sham IVT injection, for contrast sensitivity measured with the Pelli-Robson chart with comparisons from the RESCUE or REVERSE study baselines and pooled, to follow-up study time points (2, 3, 4, and 5 years) using MMRM.

    8) Visual improvement as measured by LogMAR by analysis of covariance (ANCOVA) to determine the difference in improvement for all GS010 eyes compared to all-sham eyes, 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).

    9) 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 all-GS010 eyes compared to all-sham eyes, 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 MMRM.

    10) 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 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.8.3 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
    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 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 Decision2018-03-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-01-17
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat Apr 20 10:02:00 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA