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    Summary
    EudraCT Number:2020-002431-30
    Sponsor's Protocol Code Number:GT005-03
    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:2020-09-07
    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 ConcernedFrance - ANSM
    A.2EudraCT number2020-002431-30
    A.3Full title of the trial
    HORIZON: A Phase II, open-label, outcomes-assessor masked, multicentre, randomised,controlled study to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with geographic atrophy secondary to dry age-related macular degeneration
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to test the safety and effect of a treatment for people with sight loss due to dry Age-related Macular Degeneration
    A.4.1Sponsor's protocol code numberGT005-03
    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 SponsorGyroscope Therapeutics
    B.1.3.4CountryUnited Kingdom
    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 supportGyroscope Therapeutics
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGyroscope Therapeutics
    B.5.2Functional name of contact pointKathryn Parsley
    B.5.3 Address:
    B.5.3.1Street AddressGunnels Wood Road, Stevenage Bioscience Catalyst
    B.5.3.2Town/ cityStevenage, Hertfordshire
    B.5.3.3Post codeSG1 2FX
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+442071133568
    B.5.6E-mailk.parsley@gyroscopetx.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameRecombinant, non-replicating AAV2 expressing human CFI
    D.3.2Product code GT005
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubretinal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNone
    D.3.9.1CAS number None
    D.3.9.2Current sponsor codeGT005
    D.3.9.3Other descriptive nameGT005
    D.3.9.4EV Substance CodeSUB195182
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number5E10 to 2E11
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Age-related Macular Degeneration (AMD) presents as a progressive loss of vision in the centre of the retina (the macula) resulting in a blurred area or blank spot in the centre of vision. The primary clinical characteristic of late stage AMD is atrophy of the Retinal Pigment Epithelium, known as macular atrophy due to AMD, which leads to the gradual degeneration of nearby photoreceptors, resulting in thinning of the retina and a progressive visual impairment.
    E.1.1.1Medical condition in easily understood language
    Age-related Macular Degeneration (AMD) presents as a progressive loss of vision in the centre of the retina (the macula) resulting in a blurred area or blank spot in the centre of vision.
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10075719
    E.1.2Term Atrophic age-related macular degeneration
    E.1.2System Organ Class 100000004853
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The overall objective of the study is to evaluate the safety and efficacy (anatomical and functional visual outcomes) of two doses of GT005 in subjects with GA due to AMD.

    Primary objective:
    To evaluate the effect of GT005 on the progression of GA in subjects with GA due to AMD
    E.2.2Secondary objectives of the trial
    Secondary:
    - To evaluate the safety and tolerability of GT005
    - To evaluate the effect of GT005 on retinal anatomical measures
    - To evaluate the effect of GT005 on functional measures
    - To evaluate the effect of GT005 on functional vision
    - To evaluate the effect of GT005 on patient-reported outcomes
    - To evaluate the effect of GT005 on the progression of GA due to AMD, in each genetically defined subgroup(s)

    Exploratory:
    - To evaluate immunogenicity of GT005
    - To evaluate local and systemic changes in protein expression
    - To evaluate polygenic risk scores (derived of complement genetic variants)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Able and willing to give written informed consent
    2. Age ≥55 years
    3. Have a clinical diagnosis of bilateral GA secondary to AMD as determined by the Investigator
    4. Have GA lesion(s) total size between or equal to 1.25 mm2 to 17.5 mm2 in the study eye
    5. The GA lesion in the study eye must reside completely within the FAF image
    6. Have a BCVA of ≥34 letters (6/60 or 20/200 Snellen acuity equivalent), using ETDRS charts, in the study eye
    7. Meet the following AMD genetic subgroup criteria, as reviewed and confirmed by the Sponsor, and be allocated to one of the following groups below:
    a. CFI rare variant Type 2: CFI rare variant (minor allele frequency ≤1%) that has serum Factor I levels in that patient carrier measuring ≥11.6 μg/ml, and the variant is enriched in AMD and/or predicted to be functionally damaging in silico
    b. Complement rare: rare coding variant in either C3, CFB, CFH, CD59, or CD46 genes with a minor allele frequency ≤2% that is enriched in AMD
    and/or predicted to be damaging in silico
    c. CFH common: Carriers of one or two copies of either the C allele of rs1329428, or the C allele of rs1061170 (also excluding carriers of T allele
    of rs10033900, which define the CFI common group)
    d. CFI common: Carriers of one or two copies of the T allele of rs10033900
    8. Able to attend all study visits and complete the study procedures
    9. Women of child-bearing potential must have a negative pregnancy test within 2 weeks prior to randomisation or provide documentation of being surgically sterilised
    E.4Principal exclusion criteria
    1. Any carriers of the following genetic variants:
    a. ABCA4 rare (minor allele frequency ≤1%) coding variants predicted to be damaging in silico, either biallelic, or monoallelic in combination with a minor allele of a hypomorphic variant (rs1801466 or rs6657239)
    b. CFI rare variant Type 1 (minor allele frequency ≤1%) that has been tested to have a low Factor I level in serum of that patient (<11.6 μg/ml),
    2. Have evidence or history of CNV in either eye
    3. Presence of moderate/severe or worse non-proliferative diabetic retinopathy in the study eye
    4. Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in the study eye
    5. Have clinically significant cataract that may require surgery during the study period in the study eye
    6. Presence of moderate to severe glaucomatous optic neuropathy, uncontrolled intraocular pressure (IOP), use of two or more topical agents to control IOP, or a history of glaucoma-filtering surgery in the study eye
    7. Axial myopia of greater than -8 diopters in the study eye
    8. Have received any investigational product for the treatment of GA within the past 6 months, or 5 half-lives (whichever is longer) other than nutritional supplements such as the age-related eye disease study formula
    9. Have received a gene or cell therapy at any time
    10. Have a contraindication to the corticosteroid regimen
    11. Are unwilling to use two forms of contraception (one of which being a barrier method) for 90 days post-dosing, if relevant
    12. Have a history or presence of cutaneous squamous cell carcinoma
    13. Have any other significant ocular or non-ocular medical or psychiatric condition which, in the opinion of the Investigator, may either put the subject at risk or may influence the results of the study
    E.5 End points
    E.5.1Primary end point(s)
    Primary:
    - The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening, Weeks 12, 24, 36, 48 (EoS), Early termination visit (ETV)
    E.5.2Secondary end point(s)
    • Frequency of Treatment Emergent Adverse Events
    • Change on ophthalmic examination
    • Change in other parameters of safety, including imaging modalities, Best Corrected Visual Acuity (BCVA), vital signs, and laboratory
    assessments
    • Change in retinal microstructures on spectral domain optical coherence tomography (SD-OCT)
    • Change in area of nascent GA on SD-OCT
    • Change in GA morphology on colour fundus photography
    • Macular Sensitivity as assessed by Mesopic Microperimetry
    • Change in BCVA Score via the early treatment for diabetic retinopathy (ETDRS) chart
    • Change in Low Luminance Difference (LLD) via the ETDRS chart
    • Change in reading performance as assessed by Minnesota Low-Vision Reading Test (MNRead) Chart
    • Change in Functional Reading Independence Index
    • Change in quality of life measured on the Visual Functioning Questionnaire-25 (VFQ-25)
    • Change from baseline to Week 48 in GA
    area as measured by FAF
    E.5.2.1Timepoint(s) of evaluation of this end point
    • AEs-Screen, Randomisation, pre & post surgery/surgery, Day3, Wk 1, 2, 5, 8, 12, 24, 36, 48 (EoS), ETV
    •Ophthalmic exam-Screen, pre & post surgery, Wk 1, 5, 8, 12, 24, 36, 48, ETV
    •SD-OCT-Screen, post surgery, Wk 5, 12, 24, 36, 48, ETV
    •CFP-Screen, Wk 5, 48, ETV
    •Microperimetry-Screen, Wk 12, 24, 36, 48, ETV
    •BCVA with ETDRS-Screen, Wk 1, 5, 8, 12, 24, 36, 48, ETV
    •LLVA with ETDRS for LLD-Screen, Wk 12, 24, 36, 48, ETV
    •MNRead-Screen, Wk 24, 36, 48, ETV
    •FRI Index-Screen, Wk 24, 36, 48, ETV
    •VFQ-25-Screen, Wk 24, 36, 48, ETV
    •Vital signs – Screen, pre & post surgery, Wk 1, 5, 24, 48, ETV
    •Samples for Proteomics (blood or vitreous) – Screen, Pre surgery, Surgery, Wk 12, 24, 48, ETV
    •Immunogenicity – Pre surgery, Wk 5, 12, 24, 48, ETV
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    All involved with the study will be masked to the dose received for subjects allocated to GT005
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Untreated control group
    E.8.2.4Number of treatment arms in the trial2
    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 Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Australia
    France
    Germany
    Poland
    Spain
    United Kingdom
    United States
    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 years1
    E.8.9.1In the Member State concerned months11
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    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 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: 13
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 167
    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 state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 180
    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)
    All subjects who receive treatment with GT005, including those that have withdrawn from the study for any reason, will, subject to consent, be enrolled in a separate long-term follow-up study (ORACLE). The long-term follow-up study will evaluate the long-term safety and durability of GT005 after the final follow-up visit at Week 48.
    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 Decision2021-03-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-12-04
    P. End of Trial
    P.End of Trial StatusCompleted
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