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    Summary
    EudraCT Number:2017-003712-39
    Sponsor's Protocol Code Number:GT005-01
    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:2017-12-12
    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 ConcernedUK - MHRA
    A.2EudraCT number2017-003712-39
    A.3Full title of the trial
    FOCUS: An open label first in human Phase I/II multicentre study to evaluate the safety, dose response and efficacy of GT005 administered as a single subretinal injection in subjects with Macular Atrophy due to 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 new treatment for people with sight loss due to dry Age-related Macular Degeneration
    A.4.1Sponsor's protocol code numberGT005-01
    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+440207113 3568
    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 nameGT005
    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 INNThere is no recommended INN
    D.3.9.2Current sponsor codeGT005
    D.3.9.3Other descriptive namerAAV2 delivery vector comprising a nucleotide sequence encoding CFI (rAAV2.CFI)
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2000000000000
    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 objectives of the study are to evaluate the safety, dose response and efficacy (anatomical and functional visual outcomes) of three doses of GT005 in genetically defined subjects with Macular Atrophy due to Age-related Macular Degeneration (AMD).

    Primary Objective:
    To evaluate the safety of three doses of GT005 by dose and by route of administration.
    E.2.2Secondary objectives of the trial
    To evaluate the anatomical and functional visual outcomes of three doses of GT005

    To evaluate vector shedding and complement factor expression
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Able and willing to give consent to the study participation
    2. Presence of Bilateral GA due to AMD on Colour Fundus Photography
    (CFP)

    3. Cohorts 1 to 3: GA lesions total size in the treatment eye must be ≥
    1.25mm2 and ≤17.5mm2

    Cohort 4 : GA lesions total size must be ≥1.25mm2 and ≤17.5mm2 for both eyes.

    4. Cohort 1 to 3: The GA lesion in the treatment eye must reside
    completely within the FAF fundus image
    Cohort 4: The GA lesion must reside completely within the FAF fundus
    image for both eyes

    5. Cohorts 1 to 3: BCVA of ≤50 letters (6/36 Snellen acuity equivalent or
    worse) using ETDRS charts in the treatment eye
    Cohort 4: BCVA of ≥34 letters (6/60 Snellen acuity equivalent or better)
    using ETDRS charts in both eyes.

    6. Aged ≥55 years

    7. Able to attend all study visits and complete the study procedures

    8. Women of child-bearing potential need to have a negative urine
    pregnancy test within one month prior to receiving the drug
    E.4Principal exclusion criteria
    1. Have evidence or history of CNV in either eye
    2. Presence of moderate/severe or worse non-proliferative, diabetic retinopathy in either eye
    3. Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in either eye
    4. History of intraocular surgery in the study eye within 12 weeks prior to Screening (Visit 1). Yttrium aluminium garnet capsulotomy is permitted if performed >10 weeks prior to Visit 1
    5. Have clinically significant cataract that may require surgery during the study period in either eye
    6. Presence of moderate to severe glaucomatous optic neuropathy in either eye, uncontrolled IOP despite the use of more than two topical agents; a history of glaucoma-filtering or valve is also excluded
    7. Axial myopia of greater than -8 diopters in the either 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 (AREDS) formula
    9. Have received a gene or cell therapy at any time
    10. Have a contraindication to the specified protocol 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
    11. Have a history or presence of cutaneous squamous cell carcinoma
    12. Active malignancy within the past 12 months, except for: appropriately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, or prostate cancer with a stable prostate-specific antigen (PSA) >= 12 months.
    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)
    To evaluate the safety of two three doses of GT005 by collection of TEAE & TESAE within the first year following dosing.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The Week 48 visit will be the time point used for the primary endpoint of the study. At the Week 48 visit, subjects will be invited to consent to the additional follow-up .Week 240 (Year 5) will be the end of the study.
    E.5.2Secondary end point(s)
    To evaluate the anatomical and functional visual outcomes of two three doses of GT005

    Local and systemic changes in protein expression and vector shedding
    E.5.2.1Timepoint(s) of evaluation of this end point
    Samples of blood, urine, tears and saliva will be collected for vector shedding analysis prior to discharge.
    Week 240 (Year 5) will be the end of the study.
    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.8.2.4Number of treatment arms in the trial4
    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 concerned10
    E.8.5The trial involves multiple Member States No
    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
    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
    The study will have ended after the last subject has completed the last study visit at 5 years post GT005 administration
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months5
    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: 32
    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 state30
    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)
    After finalisation of this study, subjects will be invited and encouraged to participate in a four year follow up study.
    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 Decision2018-02-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-04-20
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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