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. MedDRA version: 20.0 Level: LLT Cla
Conditions
Interventions
Product Name: GT005
Product Code: GT005
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: There is no recommended INN
Current Sponsor code: GT005
Other descriptive name: rAAV2 deliver
Sponsors
Gyroscope Therapeutics
Eligibility
Sex/Gender
All
Inclusion criteria
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 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 13 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 32
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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. ;Secondary Objective: To evaluate the anatomical and functional visual outcomes of three doses of GT005 To evaluate vector shedding and complement factor expression;Primary end point(s): To evaluate the safety of two three doses of GT005 by collection of TEAE & TESAE within the first year following dosing.;Timepoint(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. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary 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 ;Timepoint(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. | — |
Countries
United Kingdom, United States
Contacts
Public ContactKathryn Parsley
Gyroscope Therapeutics
Outcome results
None listed