Age - Related Macular Degeneration (AMD), Geographic Atrophy (GA)
Conditions
Keywords
Phase 2, ONL1204, Geographic Atrophy (GA), Age-related Macular Degeneration (AMD)
Brief summary
The purpose of this study is to collect efficacy and safety information of intravitreal injection for 3 experimental arms, including 2 dose levels of ONL1204 and 2 treatment frequencies in patients with GA associated with AMD. GA associated with AMD is one of the world's leading causes of visual disability and legal blindness globally. Associated with aging, cigarette smoking, obesity, diets low in certain nutrients, a lifestyle related to cardiac risk, and a growing list of genetic factors, AMD is becoming an increasingly prevalent public health concern, especially as the global population ages. ONL1204 is a first-in-class inhibitor of fragment apoptosis stimulator receptor-mediated cell death that has demonstrated protection of multiple retinal cell types in numerous preclinical models of retinal disease, including models of dry AMD.
Interventions
DRUGONL1204 Opthalmic solution
Liquid formulation ONL1204 Ophthalmic Solution administered by intravitreal (IVT) injection
DRUGAvacincaptad Pegol intravitreal solution
Liquid formulation Avacincaptad Pegol administered by intravitreal (IVT) injection
OTHERSham
Sham injection
Sponsors
ONL Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
55 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. 55 years of age or older at Screening. 2. Able and willing to give informed consent and attend study visits. 3. Women or intersex individuals must be willing to use 2 forms of effective contraception during the study and for at least 90 days following the last dose of study drug, be postmenopausal for at least 12 months prior to study entry, or surgically sterile. If of childbearing potential, a negative urine pregnancy test is required at Screening and prior to the administration of study drug at each visit. 4. Men or intersex individuals with partners of childbearing potential must be willing to use permissible methods of contraception and refrain from sperm donation during the study and for at least 90 days following the last dose of study drug. 5. If currently using Age-related Eye Disease Study 2 (AREDS, AREDS2, or similar nutraceutical therapy at Screening, patient must be willing to continue use for the duration of the study. If not currently using AREDS2 or similar, patient must be willing to continue not to use therapy for the duration of the study. Patient must agree to choose either approach. Study Eye Inclusion Criteria: 6. BCVA of 24 letters or more using ETDRS charts in the study eye at Screening. 7. Intraocular pressure (IOP) of 22 mmHg or lower in the study eye at Screening. The GA lesion must meet all the following criteria: 8. Non-foveal GA associated with AMD without eMNV in the study eye per Investigator's evaluation and as confirmed by the central RC. 9. GA ≥0.85 disc area (DA) (2.125 mm2) and ≤8.05 DA (20.125 mm2) in the study eye as confirmed by the central RC. 10. If GA in the study eye is multifocal, at least 1 focal lesion must be ≥0.425 DA (1.063 mm2) as confirmed by the central RC. 11. Non-foveal GA (GA lesion up to the fovea, but not subfoveal) with the lesion's closest border 1725 microns or less from the foveal center in the study eye, as confirmed by the central RC. 12. The entire GA lesion in the study eye must be completely visualized on the macula-centered FAF image, must be able to be imaged in its entirety, and must not be contiguous with any areas of peripapillary atrophy per the Investigator's evaluation and as confirmed by the central RC. 13. A pattern of hyper-autofluorescence in the junctional zone of GA in the study eye must be present as confirmed by the central RC. Fellow Eye Inclusion Criterion: 14. BCVA of 19 letters or more using ETDRS charts in the fellow eye at Screening.
Exclusion criteria
1. Current or planned participation in another investigational clinical study or use of any other investigational drugs or devices at least 6 months prior to enrollment or during the study period without prior written Sponsor approval. 2. Previous ophthalmic disease gene therapy or planned participation in any gene therapy clinical study during the study period. 3. Current or planned use of systemic complement inhibitors during the study period. 4. Any ocular or systemic condition that, in the opinion of the Investigator, makes the patient unsuitable for treatment with an investigational drug or that would compromise the safety or efficacy assessments of the study. 5. Treatment with any ocular or systemic medication that is known to be toxic to the lens, retina, or optic nerve (including, but not limited to, aminoglycosides, vancomycin, hydroxychloroquine, interferon, tacrolimus, cisplatin, bis-chloroethyl nitrosourea, carmustine, ethambutol, and tamoxifen) within 90 days prior to Screening or anticipated during the study period. 6. Any previous treatment of pentosan polysulfate (Elmiron®) is exclusionary. 7. Known allergy to fluorescein, povidone iodine, or any ingredients of the study drug or avacincaptad pegol intravitreal solution. 8. Individuals who are currently pregnant, planning to become pregnant, or are nursing at Screening or during the study period. Study Eye
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Primary Endpoint | From enrollment to the end of treatment at 48 weeks. | GA lesion area |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Secondary Endpoints (Safety) | From enrollment to the end of treatment at 48 weeks. | Incidence of new active exudative macular neovascularization (eMNV) in the study eye |
Countries
Austria, Canada, Czechia, Germany, Greece, Hungary, Poland, Portugal, Spain, Switzerland, United Kingdom, United States
Contacts
CONTACTStudy Coordinator
Outcome results
None listed