Actively Recruiting

Phase 2
Age: 55Years +
All Genders
ID06659445

A Phase 2 Study to Evaluate Safety and Efficacy of ONL1204 Compared to Sham and Avacincaptad Pegol in Patients With Geographic Atrophy Associated With Age-Related Macular Degeneration

Led by ONL Therapeutics · Updated on 2025-12-05

324

Participants Needed

28

Research Sites

N/A

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and effectiveness of intravitreal injections of ONL1204 in patients with geographic atrophy (GA) linked to age-related macular degeneration (AMD). This phase 2, randomized, double-masked, sham-controlled study aims to collect information on different doses and treatment frequencies of ONL1204, a new type of drug that may protect retinal cells. GA associated with AMD is a major cause of vision loss worldwide, influenced by aging, lifestyle factors, and genetics. Participants will receive one of several treatments: two doses of ONL1204 (100 µg or 200 µg) given every 12 weeks, a 200 µg dose every 24 weeks, sham injections every 12 or 24 weeks, or monthly injections of Avacincaptad Pegol. All treatments are delivered by intravitreal injection into the eye. The study includes six treatment groups to compare these different approaches. During the 48-week treatment period, participants will attend regular visits for injections and evaluations. Researchers will monitor vision, eye pressure, retinal health, and safety through various examinations and imaging. The main measurement is the primary endpoint assessed from enrollment until the end of treatment. Safety will also be closely observed. Participants must be at least 55 years old with specific eye health criteria to take part, and their involvement includes ongoing assessments to understand treatment effects and safety.

CONDITIONS

Brief Title

Study to Evaluate Efficacy and Safety of ONL1204 in Patients With GA Associated With AMD

Who Can Participate

Age: 55Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • 55 years of age or older at Screening
  • Able and willing to give informed consent and attend study visits
  • Women or intersex individuals must use two forms of contraception during the study or be postmenopausal for at least 12 months or surgically sterile; negative pregnancy tests required if of childbearing potential
  • Men or intersex individuals with partners of childbearing potential must use contraception and avoid sperm donation during and for 90 days after the study
  • Willing to continue or not use Age-related Eye Disease Study 2 (AREDS2) or similar nutraceutical therapy consistently during the study
  • Best-corrected visual acuity (BCVA) of 24 letters or more in the study eye at Screening
  • Intraocular pressure (IOP) of 22 mmHg or lower in the study eye at Screening
  • Non-foveal geographic atrophy (GA) associated with AMD without exudative macular neovascularization (eMNV) in the study eye, confirmed by central reading center
  • GA lesion size between 0.85 and 8.05 disc areas in the study eye as confirmed by central reading center
  • At least one focal lesion of 0.425 disc areas or larger if GA is multifocal
  • GA lesion border 1725 microns or less from the foveal center in the study eye
  • Entire GA lesion must be fully visualized on macula-centered fundus autofluorescence image and not contiguous with peripapillary atrophy
  • Presence of hyper-autofluorescence pattern in the junctional zone of GA
  • Best-corrected visual acuity (BCVA) of 19 letters or more in the fellow eye at Screening
Not Eligible

You will not qualify if you...

  • Participation in another investigational study or use of investigational drugs/devices within 6 months prior to enrollment or during the study without sponsor approval
  • Previous ophthalmic gene therapy or planned gene therapy participation during the study
  • Use of systemic complement inhibitors during the study
  • Any ocular or systemic condition making patient unsuitable for investigational drug treatment or safety/efficacy assessments
  • Treatment with ocular or systemic medications toxic to lens, retina, or optic nerve within 90 days prior to Screening or anticipated during the study
  • Previous treatment with pentosan polysulfate (Elmiron�)
  • Known allergy to fluorescein, povidone iodine, or ingredients of study drugs
  • Pregnancy, planning pregnancy, or nursing during the study
  • Active ocular or periocular infection in the study eye
  • Contraindication to intravitreal injection in the study eye
  • Media opacity limiting vision or retinal imaging in the study eye
  • Previous intravitreal pharmaceutical treatment in the study eye except FDA-approved complement inhibitors within 12 weeks prior to Screening
  • History of incisional retinal or glaucoma surgery in the study eye
  • Yttrium aluminum garnet capsulotomy in the study eye within 1 month prior to Screening
  • Planned or expected cataract surgery or aphakia without posterior capsule in the study eye
  • Evidence or history of wet AMD in the study eye
  • Other significant retinal diseases except benign degenerative conditions in the study eye
  • Intraocular surgery in the study eye within 3 months prior to Screening
  • Previous therapeutic radiation in the region of the study eye
  • Planned intravitreal injection in fellow eye with agents other than FDA-approved complement inhibitors during the study
  • Active intraocular inflammation or history of uveitis or endophthalmitis in either eye
  • GA in either eye due to causes other than AMD
  • Ophthalmic condition likely to require surgery during the study in either eye

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - 48 weeks

Participants receive intravitreal injections of ONL1204, Avacincaptad Pegol, or sham injections according to their assigned group to evaluate safety and efficacy.

Injections every 12 weeks, 24 weeks, or monthly depending on treatment group

Trial Site Locations

Total: 28 locations

1

Associated Retina Consultants

Gilbert, Arizona, United States, 85297

Actively Recruiting

2

Associated Retina Consultants

Phoenix, Arizona, United States, 85020

Actively Recruiting

3

Doheny Image Reading Center

Pasadena, California, United States, 91103

Actively Recruiting

4

Bay Area Retina Associates

Walnut Creek, California, United States, 94598

Actively Recruiting

5

Retina Consultants of Southern Colorado

Colorado Springs, Colorado, United States, 80909-1183

Actively Recruiting

6

Retina Group of New England

Waterford, Connecticut, United States, 06385

Actively Recruiting

7

Florida Retina Institute

Orlando, Florida, United States, 32806

Actively Recruiting

8

Retina Associates, Ltd.

Elmhurst, Illinois, United States, 60126

Actively Recruiting

9

Retina Partners Midwest, P.C.

Carmel, Indiana, United States, 46032

Actively Recruiting

10

Retina Research Institute at New England Retina Consultants

Springfield, Massachusetts, United States, 01107

Actively Recruiting

11

Associated Retinal Consultants, P.C.

Royal Oak, Michigan, United States, 48073

Actively Recruiting

12

The Retina Institute

St Louis, Missouri, United States, 63128

Actively Recruiting

13

Sierra Eye Associates

Reno, Nevada, United States, 89502

Actively Recruiting

14

Retina Associates of Western NY

Rochester, New York, United States, 14620

Actively Recruiting

15

Long Island Vitreoretinal Consultants

Westbury, New York, United States, 11590

Actively Recruiting

16

North Carolina Retina Associates, S.C.

Wake Forest, North Carolina, United States, 27587

Actively Recruiting

17

Erie Retina Research, LLC (Clinic)

Erie, Pennsylvania, United States, 16505

Actively Recruiting

18

Charleston Neuroscience Institute

Mt. Pleasant, South Carolina, United States, 29464

Actively Recruiting

19

Palmetto Retina Center

West Columbia, South Carolina, United States, 29169

Actively Recruiting

20

Retina Consultants of Texas

Beaumont, Texas, United States, 77707

Actively Recruiting

21

Retina Consultants of Texas

Bellaire, Texas, United States, 77401

Actively Recruiting

22

Retina Consultants of Texas

Katy, Texas, United States, 77494

Actively Recruiting

23

Retina Associates of South Texas, PA

San Antonio, Texas, United States, 78240

Actively Recruiting

24

Retina Consultants of Texas

San Antonio, Texas, United States, 78240

Actively Recruiting

25

Retina Consultants of Texas

The Woodlands, Texas, United States, 77384

Actively Recruiting

26

Pacific Northwest Retina

Bellevue, Washington, United States, 98004

Actively Recruiting

27

Pacific Northwest Retina, PLLC

Silverdale, Washington, United States, 98383

Actively Recruiting

28

Clinique d'ophtalmologie des Laurentides

Boisbriand, Quebec, Canada, J7H0E8

Actively Recruiting

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Research Team

S

Study Coordinator

How is the study designed?

Study Type

INTERVENTIONAL

Masking

DOUBLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

6

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