Intravitreal AAVCAGsCD59 for Advanced Dry Age-related Macular Degeneration (AMD) With Geographic Atrophy (GA)

Hemera Biosciences

Status and phase: Withdrawn; Phase 2

Conditions: Intravitreal Injection; Gene Therapy; Geographic Atrophy; Dry Age-related Macular Degeneration

Treatments: Biological: Intravitreal AAVCAGsCD59; Other: Intravitreal Sham Injection

Study type: Interventional

Funder types: Industry

Identifiers

NCT04358471

HMR-2001

Details and patient eligibility

About

Patients with advanced dry AMD with GA meeting inclusion criteria will be randomized in one eye in a 1:1:1 ratio comparing intravitreal high or low dose AAVCAGsCD59 with a sham injection. All enrolled subjects will be followed for 24 months to evaluate reduction in GA growth and safety of intravitreal AAVCAGsCD59.

Sex: All

Ages: 65+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Advanced dry AMD with GA in the study eye
2. BCVA in the study eye of 80 or less ETDRS letters (Snellen equivalent 20/25 or worse)
3. Total cumulative GA lesion size 2.5 mm2 to 12.5 mm2 in the study eye as confirmed by the reading center during the Screening Period.

Exclusion criteria

1. GA secondary to non-AMD etiologies in the study eye (i.e. myopia, inherited retinal diseases).
2. GA associated with the presence of an RPE rip.
3. GA contiguous with peripapillary atrophy.
4. Active CNV secondary to wet AMD in the study eye and currently receiving anti-VEGF ocular treatment within the previous 18 months.
5. Subretinal fibrosis in the macula from CNV both clinically and imaged on SD-OCT in the macula.
6. Previous macular laser photocoagulation (i.e. focal or grid laser for macular edema), photodynamic therapy (PDT), ocular/orbital radiation, laser to CNV, or subretinal surgery for CNV in the study eye.
7. History of conditions in the study eye which might alter visual acuity or interfere with study testing including proliferative diabetic retinopathy (PDR), clinically significant macular edema (CSME), central retinal vein occlusion (CRVO), hemi retinal vein occlusion (HRVO), macular branch retinal vein occlusion, and optic neuropathy.
8. Active uncontrolled glaucoma with at least one of the following: IOP>30 mmHg despite maximum medical treatment with glaucoma medications, cup-to-disc ratio of >0.9, visual field defects secondary to glaucoma that involve the macula, or optic atrophy from glaucoma.
9. Active acute or chronic infectious uveitis, retinitis, or conjunctivitis (excluding blepharitis)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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0 participants in 3 patient groups

Intravitreal AAVCAGsCD59 1.071x10e12 vg Injection

Active Comparator group

Description:

Intravitreal AAVACGsCD59 at a dose of 1.071x10e12 vg administered once on Day 0

Treatment:

Biological: Intravitreal AAVCAGsCD59

Intravitreal AAVCAGsCD59 3.56x10e11 vg Injection

Active Comparator group

Description:

Intravitreal AAVACGsCD59 at a dose of 3.56x10e11 vg administered once on Day 0

Treatment:

Biological: Intravitreal AAVCAGsCD59

Sham Intravitreal Injection

Sham Comparator group

Description:

Intravitreal Sham injection administered once on Day 0

Treatment:

Other: Intravitreal Sham Injection