Study to Assess the Ability of ONL1204 to Slow the Progression of Atrophic Age-related Macular Degeneration

Overview

Sponsor-declared trial summary

Key facts

Sponsor

ONL Therapeutics Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11], Phenomena and Processes [G] - Ocular Physiological Phenomena [G14]

Trial duration

14 Apr 2026 → ongoing

Decision date (initial)

2025-08-11

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

ONL Therapeutics, Inc.

External identifiers

EU CT number

2024-513174-22-00

ClinicalTrials.gov

NCT06659445

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the efficacy of ONL1204 compared to sham injection in patients with geographic atrophy (GA) associated with AMD.

Conditions and MedDRA coding

Atrophic Age-related Macular Degeneration (AMD)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 55 years of age or older at Screening.
2. If currently using Age-related Eye Disease Study 2 (AREDS, AREDS2, or similar [see Protocol for formulation]) nutraceutical therapy at Screening, patient must be willing to continue use for the duration of the study. If not currently using AREDS2 or similar, patient must be willing to continue not to use therapy for the duration of the study. Patient must agree to choose either approach.
3. Non-foveal GA associated with AMD without eMNV in the study eye per Investigator’s evaluation and as confirmed by the central RC.
4. BCVA of 24 letters or more using ETDRS charts in the study eye at Screening.
5. BCVA of 19 letters or more using ETDRS charts in the fellow eye at Screening.

Exclusion criteria 9

1. Any ocular or systemic condition that, in the opinion of the Investigator, makes the patient unsuitable for treatment with an investigational drug or that would compromise the safety or efficacy assessments of the study.
2. Individuals who are currently pregnant, planning to become pregnant, or are nursing at Screening or during the study period.
3. Previous IVT pharmaceutical treatment in the study eye with any agent except regionally approved complement inhibitors and treatment with regionally approved complement inhibitors within 12 weeks prior to Screening.
4. Any current evidence or history in the study eye of exudative (“wet”) AMD, including any RPE rips or evidence of actively leaking neovascularization anywhere in the retina based on the FA or SD-OCT as assessed per the Investigator’s evaluation and confirmed by the central RC.
5. Intraocular surgery in the study eye (including lens replacement surgery) within 3 months prior to Screening. See Exclusion Criteria 13 and 14 pertaining to retinal and glaucoma surgery, respectively.
6. Planned or expected IVT injection in the fellow eye for treatment of GA with agents other than regionally approved complement inhibitors during the study period.
7. Active intraocular inflammation in either eye or a history of uveitis or endophthalmitis in either eye.
8. GA in either eye due to causes other than AMD.
9. Any ophthalmic condition in either eye that is likely to require surgery during the study period.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. GA lesion area assessment

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

ONL Therapeutics Inc.

Sponsor organisation

ONL Therapeutics Inc.

Address

524 South Main Street

City

Ann Arbor

Postcode

48104-2993

Country

United States

Scientific contact point

Organisation

ONL Therapeutics Inc.

Contact name

Dr. David Zacks

Public contact point

Organisation

ONL Therapeutics Inc.

Contact name

Lindsay Puscas

Third parties 5

Locations

9 EU/EEA countries · 57 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 169 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications