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 2
  • Study Type

    Interventional
  • Status

    Recruiting
  • Study Participants

    65
This 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.
Study Started
Dec 17
2018
Primary Completion
Oct 31
2022
Anticipated
Study Completion
Oct 31
2026
Anticipated
Last Update
Dec 22
2021

Biological GT005

A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor

Biological GT005

A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor

Biological GT005

A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor

Biological GT005

A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor

Device GT005

A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor

Device GT005

A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor

Device GT005

A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor

GT005 Dose 1 Experimental

A single dose of GT005 will be administered via subretinal injection

GT005 Dose 2 Experimental

A single dose of GT005 will be administered via subretinal injection

GT005 Dose 3 Experimental

A single dose of GT005 will be administered via subretinal injection

GT005 Dose 1, 2 or 3 Experimental

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

GT005 Dose 2 with Orbit Subretinal Delivery System Experimental

A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach

GT005 Dose 3 with Orbit Subretinal Delivery System Experimental

A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach

GT005 Dose 1, 2 or 3 with Orbit Subretinal Delivery Sysem Experimental

A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach

Criteria

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
No Results Posted