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

Primary Purpose

Dry Age-related Macular Degeneration, Geographic Atrophy, Gene Therapy

Status

Withdrawn

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

Intravitreal AAVCAGsCD59

Intravitreal Sham Injection

About this trial

This is an interventional treatment trial for Dry Age-related Macular Degeneration

Eligibility Criteria

65 Years - undefined (Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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)

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Active Comparator

Sham Comparator

Arm Label

Intravitreal AAVCAGsCD59 1.071x10e12 vg Injection

Intravitreal AAVCAGsCD59 3.56x10e11 vg Injection

Sham Intravitreal Injection

Arm Description

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

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

Intravitreal Sham injection administered once on Day 0

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Measure the number of treated eyes in the sham and AAVCAGsCD59-treated arms that convert from dry to wet age-related macular degeneration

Change in visual acuity of the AAVCAGsCD59 treated eye

Visual acuity measured on an Early Treatment Diabetic Retinopathy Study (ETDRS) chart will be compared at Day 0 and Month 24

Full Information

NCT ID

NCT04358471

First Posted

March 22, 2020

Last Updated

May 6, 2021

Sponsor

Hemera Biosciences

1. Study Identification

Unique Protocol Identification Number

NCT04358471

Brief Title

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

Official Title

A Phase 2 Multi-Center Trial Evaluating Intravitreal AAVCAGsCD59 Compared to Sham Injection for the Treatment of Advanced Dry Age-Related Macular Degeneration (AMD) With Geographic Atrophy (GA)

Study Type

Interventional

2. Study Status

Record Verification Date

May 2021

Overall Recruitment Status

Withdrawn

Why Stopped

The AAVCAGsCD59 asset has been transferred to Janssen Research and Development LLC

Study Start Date

July 31, 2021 (Anticipated)

Primary Completion Date

July 1, 2023 (Anticipated)

Study Completion Date

September 1, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Hemera Biosciences

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Model Description

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

Masking

ParticipantOutcomes Assessor

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Intravitreal AAVCAGsCD59 1.071x10e12 vg Injection

Arm Type

Active Comparator

Arm Description

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

Arm Title

Intravitreal AAVCAGsCD59 3.56x10e11 vg Injection

Arm Type

Active Comparator

Arm Description

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

Arm Title

Sham Intravitreal Injection

Arm Type

Sham Comparator

Arm Description

Intravitreal Sham injection administered once on Day 0

Intervention Type

Biological

Intervention Name(s)

Intravitreal AAVCAGsCD59

Other Intervention Name(s)

HMR59

Intervention Description

AAVCAGsCD59 is administered as an intravitreal injection in the enrolled eye

Intervention Type

Other

Intervention Name(s)

Intravitreal Sham Injection

Other Intervention Name(s)

Sham

Intervention Description

Sham injection mimics a real injection in the enrolled eye

Primary Outcome Measure Information:

Title

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

Description

Geographic atrophy will be measured based on imaging of the retina

Time Frame

24 Months

Secondary Outcome Measure Information:

Title

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

Description

Measure the number of treated eyes in the sham and AAVCAGsCD59-treated arms that convert from dry to wet age-related macular degeneration

Time Frame

24 Months

Title

Change in visual acuity of the AAVCAGsCD59 treated eye

Description

Visual acuity measured on an Early Treatment Diabetic Retinopathy Study (ETDRS) chart will be compared at Day 0 and Month 24

Time Frame

24 Months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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)

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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