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

NCT04358471 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: HMR-2001
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Evaluate the change in Geographic Atrophy area (mm2) measured at Day 0 and compared to the measurement at Month 24

  Geographic atrophy will be measured based on imaging of the retina

  24 Months

Secondary Outcomes (2)

• Incidence of conversion from dry to wet age-related macular degeneration

  24 Months

• Change in visual acuity of the AAVCAGsCD59 treated eye

  24 Months

Study Arms (3)

Intravitreal AAVCAGsCD59 1.071x10e12 vg Injection

ACTIVE COMPARATOR

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

Biological: Intravitreal AAVCAGsCD59

Intravitreal AAVCAGsCD59 3.56x10e11 vg Injection

ACTIVE COMPARATOR

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

Biological: Intravitreal AAVCAGsCD59

Sham Intravitreal Injection

SHAM COMPARATOR

Intravitreal Sham injection administered once on Day 0

Other: Intravitreal Sham Injection

Interventions

Intravitreal AAVCAGsCD59 BIOLOGICAL

AAVCAGsCD59 is administered as an intravitreal injection in the enrolled eye

Also known as: HMR59

Intravitreal AAVCAGsCD59 1.071x10e12 vg Injection; Intravitreal AAVCAGsCD59 3.56x10e11 vg Injection

Intravitreal Sham Injection OTHER

Sham injection mimics a real injection in the enrolled eye

Also known as: Sham

Sham Intravitreal Injection

Eligibility Criteria

• Age: 65 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Older Adult (65+)

You may qualify if:

• Advanced dry AMD with GA in the study eye
• BCVA in the study eye of 80 or less ETDRS letters (Snellen equivalent 20/25 or worse)
• 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.

You may not qualify if:

• GA secondary to non-AMD etiologies in the study eye (i.e. myopia, inherited retinal diseases).
• GA associated with the presence of an RPE rip.
• GA contiguous with peripapillary atrophy.
• Active CNV secondary to wet AMD in the study eye and currently receiving anti-VEGF ocular treatment within the previous 18 months.
• Subretinal fibrosis in the macula from CNV both clinically and imaged on SD-OCT in the macula.
• 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.
• 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.
• 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.
• Active acute or chronic infectious uveitis, retinitis, or conjunctivitis (excluding blepharitis)

Sponsors & Collaborators

• Hemera Biosciences: lead

MeSH Terms

Conditions

Geographic Atrophy

Interventions

salicylhydroxamic acid

Condition Hierarchy (Ancestors)

Macular Degeneration; Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Subjects will be assigned to low dose AAVCAGsCD59, high dose AAVCAGsCD59, or sham arm.

Study Record Dates

• First Submitted: March 22, 2020
• First Posted: April 24, 2020
• Study Start: July 31, 2021
• Primary Completion: July 1, 2023
• Study Completion: September 1, 2023
• Last Updated: May 10, 2021

Record last verified: 2021-05

Data Sharing

• IPD Sharing: Will not share