Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12625000877482
Ethics application status
Approved
Date submitted
1/07/2025
Date registered
12/08/2025
Date last updated
12/08/2025
Date data sharing statement initially provided
12/08/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
Observational Study in Patients with Geographic Atrophy Secondary to Age-related Macular Degeneration
Scientific title
A Multicenter, OBSERVational Study in PatiEnts with Geographic Atrophy
Secondary to Age-related Macular Degeneration (OBSERVE)
Secondary ID [1] 314634 0
KRIYA-825-901
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Geographic Atrophy 337782 0
Condition category
Condition code
Eye 334135 334135 0 0
Diseases / disorders of the eye

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Geographic atrophy (GA) is an advanced form of age-related macular degeneration (AMD) characterized by atrophy of retinal tissues and typically occurs in individuals over the age of 50 years. In GA, the buildup of protein and lipid aggregates known as drusen leads to the progressive loss of photoreceptors (PR) and retinal pigment epithelium (RPE) in the macula. Progressive PR degeneration, formation of atrophic lesions, and ultimately vision loss are key phenotypes of GA.

This is a 2-part multicenter observational study to assess ocular images collected from participants diagnosed with GA secondary to AMD. The duration of observation is 6 months (Part 1) or 18 months (Part 2).

No treatment or experimental intervention will be given in this study. In both Part 1 and Part 2, longitudinal GA progression will be evaluated. In Part 1, data will be collected retrospectively from participants’ medical records; additional ocular images and ophthalmic assessments (optical coherence tomography [OCT] and fundus autofluorescence [FAF]) may also be collected through 6 months. If Part 1 participants are confirmed to meet lesion-specific criteria based on these assessments, they will be asked to screen for Part 2, where further ocular images and ophthalmic assessments (OCT, FAF, and best-corrected visual acuity [BCVA]) will be collected through 18 months. Additionally, a blood sample will be obtained to assess adeno-associated virus (AAV) neutralizing antibody (Nab) titers in serum. Part 2 will occur as soon as eligible study participants are identified from Part 1.

Ophthalmic assessments will include BCVA (Part 2 only: Months 0, 3, 6, 12, 18; 5-10 minutes per eye), OCT (Part 1: Months 0, 6; Part 2: Months 0, 3, 6, 12, 18; 5-10 minutes per eye), and FAF (Part 1: Months 0, 6; Part 2: Months 0, 3, 6, 12, 18; 5 minutes per eye). One blood sample will be taken in Part 2 (Month 0). The progression of participants’ GA will be observed from these data.
Intervention code [1] 331366 0
Not applicable
Intervention code [2] 331367 0
Early Detection / Screening
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 341952 0
Changes in photoreceptor (PR) and retinal pigment epithelium (RPE) area in patients with geographic atrophy, assessed together as a composite measure
Timepoint [1] 341952 0
6 months post-Baseline in Part 1; 3, 6, 12, and 18 months post-Baseline in Part 2
Primary outcome [2] 342228 0
Geographic atrophy lesion progression rate
Timepoint [2] 342228 0
6 months post-Baseline in Part 1; 3, 6, 12, and 18 months post-Baseline in Part 2
Primary outcome [3] 342444 0
AAV neutralizing antibody levels in serum
Timepoint [3] 342444 0
Baseline in Part 2
Secondary outcome [1] 448707 0
Nil
Timepoint [1] 448707 0
Nil

Eligibility
Key inclusion criteria
Inclusion Criteria
Part 1 (per Medical Chart Review):
1. Participant is able and willing to give informed consent to study participation
2. Clinical diagnosis of GA of the macula secondary to AMD

Part 2:

1. Participant must be between 55 to 80 years of age (inclusive).
2.. Clinical diagnosis of GA of the macula secondary to AMD.
3. The GA lesion must meet specific study-defined characteristics for PR and RPE area,
GA area/size, lesion growth rate, and hyperautofluorescence pattern.

The same group of participants will be used for both parts of the study. Part 1 participants who meet lesion-specific inclusion criteria will be asked to screen for Part 2.
Minimum age
55 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
General Exclusion Criteria (Both Parts)
Ocular Conditions:
• Active or history of macular atrophy secondary to a condition other than age-related macular degeneration such as Stargardt disease, cone-rod dystrophy, or toxic maculopathies like hydroxychloroquine maculopathy in either eye.
• High myopia (greater than 6 diopters or an axial length greater than 26 mm).
• Exudative age-related macular degeneration diagnosis or any history of or active macular neovascularization in either eye and/or retinal angiomatous proliferation associated with age-related macular degeneration or any other cause including any evidence of retinal pigment epithelium rips or evidence of neovascularization.
• Aphakia.
• History of vitrectomy, retinal detachment, or corneal transplant in either eye.
• Small cup to disc ratio (less than 0.1).
• Active or history of uveitis.
• Any ocular condition that prevents adequate imaging as determined by the site investigator.
• Ocular or periocular infection within 3 months of screening or active uncontrolled intraocular inflammation.
Prior/Concomitant Therapy:
• Intraocular surgery (including lens replacement surgery) within 3 months prior to screening.
• History of laser therapy in the macular region.
• Use of subconjunctival gentamicin and intracameral vancomycin within 30 days prior to screening.

Part 1 Specific Exclusions

• Active or history of ocular disease that in the opinion of the investigator compromises or confounds image data analyses, including but not limited to moderate or severe non-proliferative diabetic retinopathy, uveitis, endophthalmitis, other macular disease (clinically significant epiretinal membrane, full thickness macular hole), or uncontrolled glaucoma/ocular hypertension.
• Active or history of any ocular condition other than geographic atrophy secondary to age-related macular degeneration that required or will require surgery or medical intervention or, in the opinion of the investigator, could interfere with data interpretation.

Part 2 Specific Exclusions

Ocular Conditions:
• Presence of an active ocular disease that in the opinion of the investigator compromises or confounds visual function and retinal image quality.
• Any ocular condition other than geographic atrophy secondary to age-related macular degeneration that may require surgery or medical intervention during the study period or, in the opinion of the investigator, could compromise visual function during the study period.
Other Medical Conditions:
• Medical, cognitive or psychiatric conditions that, in the opinion of the investigator, make consistent study assessment and follow-up period unlikely.
Prior/Concomitant Therapy:
• Active or have history of usage of hydroxychloroquine, pentosan polysulfate sodium, and alkyl nitrites.
• Intravitreal steroid injection in 6 months prior to screening.
Prior/Concurrent Clinical Study Experience:
• Prior experience in another interventional clinical study for intraocularly or suprachoroidally administered therapies in either eye within the past 12 months of date of screening.
• Prior experience in another interventional clinical study for geographic atrophy in either eye including investigational oral medication and placebo within the past 12 months from the last dosing at date of screening.
• Prior exposure to other adeno-associated virus or adenovirus-based gene therapies or complement inhibitors.

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
15/09/2025
Actual
Date of last participant enrolment
Anticipated
18/06/2026
Actual
Date of last data collection
Anticipated
18/12/2027
Actual
Sample size
Target
120
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,WA,VIC
Recruitment outside Australia
Country [1] 27152 0
Canada
State/province [1] 27152 0
Country [2] 27153 0
Israel
State/province [2] 27153 0
Country [3] 27154 0
New Zealand
State/province [3] 27154 0
Country [4] 27155 0
United Kingdom
State/province [4] 27155 0

Funding & Sponsors
Funding source category [1] 319195 0
Commercial sector/Industry
Name [1] 319195 0
Kriya Therapeutics, Inc
Country [1] 319195 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
Kriya Therapeutics, Inc
Address
Country
United States of America
Secondary sponsor category [1] 321662 0
Commercial sector/Industry
Name [1] 321662 0
PPD Australia Pty Ltd
Address [1] 321662 0
Country [1] 321662 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 317777 0
Bellberry Human Research Ethics Committee H
Ethics committee address [1] 317777 0
Ethics committee country [1] 317777 0
Australia
Date submitted for ethics approval [1] 317777 0
08/04/2025
Approval date [1] 317777 0
03/06/2025
Ethics approval number [1] 317777 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 142114 0
Dr Andrew Chang
Address 142114 0
Sydney Retina Clinic and Day Surgery, Level 13, Park House, 187 Macquarie St, Sydney NSW 2000
Country 142114 0
Australia
Phone 142114 0
+61 2 9221 3755
Fax 142114 0
Email 142114 0
[email protected]
Contact person for public queries
Name 142115 0
Medical Affairs
Address 142115 0
Kriya Therapeutics, Inc 4105 Hopson Road Morrisville, NC 27560
Country 142115 0
United States of America
Phone 142115 0
+001 650 268 4273
Fax 142115 0
Email 142115 0
[email protected]
Contact person for scientific queries
Name 142116 0
Medical Affairs
Address 142116 0
Kriya Therapeutics, Inc 4105 Hopson Road Morrisville, NC 27560
Country 142116 0
United States of America
Phone 142116 0
+001 650 268 4273
Fax 142116 0
Email 142116 0
[email protected]

Data sharing statement
Will the study consider sharing individual participant data?
No
No IPD sharing reason/comment: Individual participant data will not be made available. Aggregate study results may be shared through peer-reviewed publications and regulatory filings.




What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.