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    Summary
    EudraCT Number:2017-002204-27
    Sponsor's Protocol Code Number:GS030_CLIN_001
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2018-09-07
    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 number2017-002204-27
    A.3Full title of the trial
    A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects with Retinitis Pigmentosa
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase I/II gene therapy clinical trial in Retinitis Pigmentosa subjects
    A.4.1Sponsor's protocol code numberGS030_CLIN_001
    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 Manager
    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 number+33176217220
    B.5.5Fax number+33149230116
    B.5.6E-mailsbentobji@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/3/16/1693
    D.3 Description of the IMP
    D.3.1Product nameAdeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene
    D.3.2Product code GS030-DP
    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) Yes
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberGene therapy medicinal product Reference of the procedure : EMA/CAT/368630/2016
    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
    Retinitis Pigmentosa
    E.1.1.1Medical condition in easily understood language
    Retinitis Pigmentosa
    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 evaluate the safety and tolerability of escalating doses of GS030-DP administered via a single intravitreal injection (IVT) and repeated light stimulation using GS030-MD in subjects with non-syndromic retinitis pigmentosa.
    E.2.2Secondary objectives of the trial
    - Evaluate the treatment effect of GS030 as assessed by vision and ocular measures, including visual acuity, visual function, orientation and mobility, and quality of life
    - Compare visual acuity, visual function, orientation and mobility before and after gene transfer, with GS030-MD turned ON and turned OFF
    - Evaluate immune response (humoral and cellular) to recombinant adeno-associated viral vector derived from serotype 2 (rAAV2.7m8), and to ChrimsonR-tdTomato (ChR-tdT) protein.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Signed informed consent form
    - Age ≥18 years to ≤75 years at the time of ICF signature
    - Diagnosis of non-syndromic RP defined as: Clinical diagnosis of non-syndromic RP based on history, mid-peripheral visual dysfunction, and fundoscopic appearance, or diagnosis of non-syndromic RP is confirmed on full-field ERG
    - Visual acuity: Visual acuity in the dose-escalation cohorts of no better than LP, or Visual acuity in the extension cohort of no better than CF pending review of dose-escalation cohort data by the DSMB
    - Relatively preserved ganglion cell layer volume and retinal nerve fiber layer thickness, as measured with spectral domain optical coherence tomography (SD-OCT)
    - Retains memory of former useful vision
    - Ability to obtain adequate pupillary dilation to permit thorough ocular examination and testing
    - Negative serum pregnancy test for women of childbearing age only
    - Women and men of reproductive potential must agree to use highly effective contraception when sexually active. This applies to the time period between signing of the informed consent form and 12 months after the GS030-DP IVT
    - Ability to wear, utilize, and follow all instructions on proper use of the GS030-MD
    - Interpupillary distance of ≥51 mm and ≤72 mm
    - Refractive error of the study eye between -6 diopters and +6 diopters
    - Review of all selection criteria to ensure continued compliance at Visit 2 through Visit 4
    - Have a negative urine pregnancy test at Visit 4 for women of childbearing potential (women who are 2 years post-menopausal or surgically sterile are not considered to be of childbearing potential)
    - Have a negative test result for infection with HIV (results from test performed at Visit 1)
    - Ability to tolerate repeated light stimuli produced by the GS030-MD, as assessed in the inclusion phase (Visit 2 through Visit 4)
    E.4Principal exclusion criteria
    - Prior receipt of any gene therapy
    - Subjects participating in another clinical trial and receiving an investigational medicinal product within 90 days prior to Visit 1
    - Subjects with systemic disease or other pathology other than that related to diagnosis of non-syndromic RP whose symptoms or associated treatments may affect vision, for example cancers or pathology of the central nervous system
    - Subjects with systemic disease or other medical or psychiatric conditions that preclude safe participation in the study
    - Subjects who are taking photosensitizing drugs used in psoralen plus ultraviolet light (PUVA) therapy for psoriasis, eczema, vitiligo, and other similar diseases, or photosensitizing drugs used for photodynamic therapy in the treatment of cancer or eye diseases
    - Subjects receiving immunosuppressive therapies, other than the immune modulating regimen described in this protocol
    - Subjects of reproductive potential unwilling to use effective contraception for the 12 months after administration of GS030-DP
    - Subjects who are pregnant or breastfeeding
    - Subjects who are unwilling or unable to comply with the study protocol
    - Subjects with any condition that would not allow them to complete follow-up examinations during the study and, in the opinion of the investigator, would make them unsuitable for the study
    - Subject is human immunodeficiency virus (HIV) positive
    - Subject with known allergy to corticosteroids, or who will be unable to tolerate the corticosteroid regimen
    - Subjects who have undergone significant ocular surgery (per investigator determination) within 3 months prior to Visit 1
    - Presence of narrow iridocorneal angles contraindicating pupillary dilation
    - Presence of disorders of the ocular media which interfere with visual acuity and other ocular assessments, including SD-OCT, during the study period
    - Presence of any systemic or ocular diseases, or pathologies, other than non-syndromic RP, or their associated therapies, that can cause or have the potential to cause vision loss
    - Prior vitrectomy or vitreomacular surgery
    - Presence of vitreo-macular adhesion or traction, epiretinal membrane macular pucker and macular hole, evident by ophthalmoscopy and/or by SD-OCT examinations and assessed by the investigator to significantly affect central vision
    - Current evidence of retinal detachment assessed by the investigator to significantly affect central vision
    - Active ocular inflammation or recurrent history of idiopathic or autoimmune-associated uveitis
    - Hypersensitivity to GS030-DP or to any of the ingredients
    - Any non-selection criteria which become applicable after the selection visit
    - Presence, at the time of study inclusion, of infectious conjunctivitis, keratitis, scleritis, or endophthalmitis in either eye
    - 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
    - Presence of illness or disease that, in the opinion of the investigator, include symptoms and/or associated treatments that can alter visual function, for instance cancers or pathology of the central nervous system
    - 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
    - The subject is unable or unwilling to comply with the protocol requirements
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability of GS030 treatment at Week 52/Year 1 based on local and systemic safety issues, specifically those related to IVT of GS030-DP and the subsequent repeated use of GS030-MD, as assessed
    by incidence of adverse events (AEs)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 52 / Year 1
    E.5.2Secondary end point(s)
    • Assessment of the treatment effect on visual function, functional vision and mobility, with the change from baseline to Week 52 of parameters measured with FrACT, Humphrey visual field 10-
    2, full field threshold stimulus test (FST), square localization test, direction of motion test, door task, and line task
    • Assessment of the treatment effect on structural changes of the posterior pole of the fundus from baseline to Week 52 with parameters measured with SD-OCT, color fundus photography, and
    fundus auto fluorescence (FAF)
    • Assessment of the treatment effect on QoL changes from baseline to Week 52 with the Visual Function Questionnaire-25 (VFQ-25) and Short-Form Survey 36 Version 2 (SF-36v2)
    • Humoral and cellular immune responses to rAAV2.7m8 and ChR-tdT protein
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 52 / Year 1
    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 Yes
    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) Yes
    E.7.1.1First administration to humans Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 EEA2
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    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.1.2.1Number of subjects for this age range: 14
    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 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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 18
    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 Retinitis Pigmentosa 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 Decision2018-05-23
    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 StatusTrial now transitioned
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