HORIZON: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005

Trial ID or NCT#

NCT04566445

Status

not recruiting iconNOT RECRUITING

Purpose

The purpose of this clinical study is to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with geographic atrophy secondary to age-related macular degeneration (AMD).

Official Title

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

Eligibility Criteria

Ages Eligible for Study: Older than 55 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. 1. Able and willing to give written informed consent2. Age ≥55 years3. a. In Stage 1: Have a clinical diagnosis of GA secondary to AMD in the study eye, as determined by the Investigator, and a diagnosis of AMD in the contralateral eye; b. In Stage 2: Have a clinical diagnosis of GA secondary to AMD in the study eye, as determined by the Investigator, that is non-foveal, as determined by the central reading centre, or has a CFI rare variant genotype and meets inclusion criteria 3a, and a diagnosis of AMD in the contralateral eye (except if monocular)4. GA lesion(s) within an acceptable size on FAF, in the study eye5. The GA lesion in the study eye must reside completely within the FAF image6. Up to 25% of the enrolled study population are permitted to have CNV in the fellow eye7. Have a BCVA of ≥24 letters (6/95 or 20/320 Snellen acuity equivalent), using ETDRS charts, in the study eye8. a. In Stage 1: Meet one of the pre-specified AMD genetic subgroup criteria; b. In Stage 2: Genotyping is not required for study eligibility9. Able to attend all study visits and complete the study procedures10. Women of child-bearing potential must have a negative pregnancy test within 2 weeks prior to randomisation (not required for postmenopausal women) or provide documentation of being surgically sterilised
Exclusion Criteria:
  1. 1. a. In Stage 1: Carriers of excluded genetic variants; b. In Stage 2: Subjects are excluded if they have a clinical diagnosis of Stargardt Disease or other retinal dystrophies2. Have a history, or evidence, of CNV in the study eye3. Presence of moderate/severe or worse non-proliferative, diabetic retinopathy in the study eye4. Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in the study eye5. History of intraocular surgery in the study eye within 12 weeks prior to Visit 16. Have clinically significant cataract that may require surgery during the study period in the study eye7. Presence of moderate to severe glaucomatous optic neuropathy, uncontrolled intraocular pressure (IOP), despite use of two or more topical agents; or a history of glaucoma-filtering or valve surgery8. Axial myopia of greater than -8 diopters in the study eye9. 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) formula10. Have received a gene or cell therapy at any time.11. Have a contraindication to the protocol specified corticosteroid regimen12. Are unwilling to use two forms of contraception (one of which being a barrier method) for 90 days post-dosing, if relevant13. 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 months14. 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

Investigator(s)

Vinit B. Mahajan, MD, PhD