Title
FocuS: First in Human Study to Evaluate the Safety and Efficacy of GT005 Administered in Subjects With Dry AMD
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 AMD
Phase
Phase 1/Phase 2Lead Sponsor
Gyroscope TherapeuticsStudy Type
InterventionalStatus
RecruitingIndication/Condition
Dry Age-related Macular Degeneration Retinal Disease Eye Diseases Retinal Degeneration Geographic Atrophy Macular Atrophy ...Study Participants
65This is an open label first in human Phase I/II multicentre study of GT005 in subjects with Macular Atrophy due to AMD
This study will evaluate the safety, the dose response and efficacy (anatomical and functional visual outcomes) of three doses of GT005 administered as a single subretinal injection in genetically defined subjects with Macular Atrophy due to Age-related Macular Degeneration (AMD). Following consent, subjects will undergo a number of ophthalmic and clinical assessments to determine eligibility for inclusion in the study. Once eligibility is confirmed, subjects will be enrolled, receive treatment, and will be followed for 48 weeks.
A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor
A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor
A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor
A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor
A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor
A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor
A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor
A single dose of GT005 will be administered via subretinal injection
A single dose of GT005 will be administered via subretinal injection
A single dose of GT005 will be administered via subretinal injection
A single dose of GT005 will be administered via subretinal injection. This dose will be determined by dose levels determined to be tolerable in Arms 1,2 and 3
A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach
A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach
A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach
Inclusion Criteria Able and willing to give consent to study participation Presence of Bilateral GA due to AMD on Color Fundus Photography (CFP) Cohorts 1 to 3: GA lesions total size in the treatment eye must be ≥1.25mm2 and ≤17.5mm2 Cohorts 4 to 7: GA lesions total size must be ≥1.25mm2 and ≤17.5mm2 for both eyes Cohorts 1 to 3: the GA lesion in the treatment eye must reside completely within the FAF fundus image Cohorts 4 to 7: The GA lesion must reside completely within the FAF fundus image for both eyes Cohorts 1 to 3: BCVA of ≤50 letters (6/36 Snellen acuity equivalent or worse) using ETDRS charts in the treatment eye Cohorts 4 to 7: BCVA of ≥24 letters (6/95 and 20/320 Snellen acuity equivalent or better) using ETDRS charts in both eyes Aged ≥55 years Able to attend all study visits and complete the study procedures Exclusion Criteria Have evidence or history of choroidal neovascularization (CNV) in either eye Presence of moderate/severe or worse non-proliferative, diabetic retinopathy in either eye Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in either eye History of intraocular surgery in the study eye within 12 weeks prior to Screening (Visit 1). Yttrium aluminum garnet capsulotomy is permitted if performed >10 weeks prior to Visit 1 Have clinically significant cataract that may require surgery during the study period in either eye 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 surgery is also excluded Axial myopia of greater than -8 diopters in the either eye Cohorts 5 to 7 only: presence of metallic objects or implanted stimulator devices in or near the head, including cochlear implants, deep brain stimulators, vagus nerve stimulators, and other implanted electrodes or stimulators
