Geographic AtrophyPhase 1NCT04744662
ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy Associated With Age-related Macular Degeneration
A Phase 1 study testing ONL1204 Ophthalmic solution in people with Geographic Atrophy, run by ONL Therapeutics at 8 sites in 2 countries.
Trial summary
The purpose of this study is to evaluate the safety and tolerability of intravitreal injection of ONL1204 Ophthalmic Solution in patients with geographic atrophy associated with AMD. GA associated with AMD is one of the world's leading causes of visual disability. Read more about this study
- Who is it for?
- Ages 55 years and older
- Conditions the study is for
- Geographic Atrophy
- What is being tested?
- ONL1204 Ophthalmic solution
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 28 participants
- Where
- 8 sitesin 2 countries
- Study dates
- 8 Jul 2021to 29 Feb 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 55 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females, ≥ 55 years old
- Able to give informed consent and attend study visits
- Bilateral GA secondary to AMD without choroidal neovascularization in either eye
- ETDRS BCVA 20/400 (Snellen equivalent) or better in both eyes
- GA ≥1 disc area (DA) (DA, 2.5 mm2)
- If GA in study eye is multifocal, at least one focal lesion must have a DA of ≥ 1.25 mm2
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- Entire GA area must be visible within the standard FAF field of view
- Presence of banded or diffuse hyperautofluoresence adjacent to GA lesion in study eye
- Female subjects must be:
- Women of non-childbearing potential, or
- WOCBP with a negative pregnancy test at screening and willing to use permissible methods of contraception for the duration of the study
- Males with female partners of childbearing potential must agree to use permissible methods of contraception and agree to refrain from donating sperm for the duration of the study.
Who cannot join
- GA in either eye due to causes other than AMD
- Participation in other ophthalmic clinical trials or use of any other investigational drugs or devices in study eye or systemically for 6 months prior to enrollment, or anticipated participation in other ophthalmic clinical trials or use of any other investigational drugs or devices in study eye or systemically during the study period
- Intraocular inflammation in the study eye
- Ocular or periocular infection in the study eye
- Media opacity that would limit baseline visual acuity or clinical visualization of the retina at baseline
- Hyperautofluoresence adjacent to GA lesion in study eye that is focal only
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- Previous IVT treatment, history of retinal surgery, or other retinal therapeutic procedures in the study eye
- Systemic immunosuppression that may interfere with retinal and cytokine expression including but not limited to glucocorticoids (eg, oral prednisone or dexamethasone); antimetabolites (eg, methotrexate, mycophenolate mofetil, and azathioprine); T-cell inhibitors (eg, cyclosporine, tacrolimus, sirolimus); alkylating agents (eg, cyclophosphamide and chlorambucil); and biologic agents (eg, tumor necrosis factor inhibitors, interferons, lymphocyte inhibitors, and interleukin inhibitors)
- Prior history of systemic use of pentosan polysulfate sodium (trade name Elmiron®)
- Any ocular or systemic condition that in the opinion of the Investigator makes the subject unsuitable treatment with an investigational agent or that would compromise the safety and efficacy assessments of the trial
- An unwillingness to elect to either a) use Age-related Eye Disease Study 2 (AREDS2) formula nutraceutical therapy for the duration of the study or b) choose not to use such therapy for the duration of the study
Trial locations and contacts
8 sites in 2 countries.
8 sites in 2 countries
Australia6 sites
Chatswood Retina
Chatswood, New South Wales, 2067
Retinology Institute
Glen Iris, Victoria, 3146
Retina and Eye Consultants Hurtsville
Hurstville, New South Wales, 2220
Queensland Eye Institute
Melbourne, Victoria, 4101
Center for Eye Rearch Australia
Melbourne, Victoria, VIC 3002
Sunshine Eye Surgeons
St Albans, Victoria, 3021
New Zealand2 sites
Southern Eye Specialists
Christchurch, 8013
Eye Institute Limited
Remuera, Auckland, 1050
About this study
Official title: A Phase 1b Multicenter, Randomized, Controlled, Multi-dose Study of the Safety and Tolerability of ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy (GA) Associated With Age-related Macular Degeneration (AMD
The purpose of this study is to evaluate the safety and tolerability of intravitreal injection of ONL1204 Ophthalmic Solution in patients with geographic atrophy associated with AMD.
GA associated with AMD is one of the world's leading causes of visual disability. It is a progressive disease with no approved therapy to slow or arrest the process of continual photoreceptor and retinal epithelial (RPE) cell loss. A safe and effective therapy for GA will have vast societal benefits. ONL1204 is being developed for this purpose. ONL1204 is a first-in-class inhibitor of fragment apoptosis stimulator receptor-mediated cell death in development for to reduce rates of vision in patients with GA associated with AMD. ONL1204 has demonstrated protection of multiple retinal cell types in several preclinical models of acute ocular injury and the protection of RPE in AMD models. ONL1204 Ophthalmic Solution is currently in a Phase 1 clinical study in patients with macula-off retinal detachment to evaluate safety and tolerability of a multi-dose of ONL1204 Ophthalmic Solution. The study is ongoing and uses the same doses and route of administration as this Phase 1b study in patients with GA.
Read the detailed description
In this study patients will be enrolled and followed for 24 weeks (natural history phase) at which point they will be randomized to 1 of 2 doses of ONL1204 Ophthalmic Solution or sham. Weeks 24 to 48 make up the Treatment Phase. Patients will be on study for a total of 48 weeks.
- Conditions
- Geographic Atrophy
- Being tested
- ONL1204 Ophthalmic solution, sham injection
- Medical subject headings
- Geographic Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ONL1204 Ophthalmic solutionDrug
Liquid formulation administered by intravitreal (IVT) injection
sham injectionProcedure or surgery
sham injection is done by touching the eye surface with a syringe without a needle
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group A | Experimental | ONL1204 Ophthalmic solution (dose A) administered by intravitreal injection Receives: Drug: ONL1204 Ophthalmic solution |
| Treatment Group B | Experimental | ONL1204 Ophthalmic solution (dose B) administered by intravitreal injection Receives: Drug: ONL1204 Ophthalmic solution |
| Treatment Group C | Sham comparator | sham injection without penetrating the eye Receives: Procedure: sham injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Best Corrected Visual Acuity
ETDRS chart (number of letters read)
Measured: up to 48 weeks
Intraocular pressure
Intraocular pressure recorded in mmHg
Measured: up to 48 weeks
Slit lamp biomicroscopy
Slit lamp using US FDA clinical grading scale 0-4; 0=normal, 4=very severe changes)
Measured: up to 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Feb 2021
- Study start8 Jul 2021
- Primary completion29 Feb 2024
- Study completion29 Feb 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Geographic AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- In this study patients will be enrolled and followed for 24 weeks (natural history phase) at which point they will be randomized to 1 of 2 doses of ONL1204 Ophthalmic Solution or sham. Weeks 24 to 48 make up the Treatment Phase. Patients will be on study for a total of 48 weeks.
- Masking
- Double blind
- Who is masked
- Participant, Care provider
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- ONL Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Robyn Guymer, MD, Principal investigator, Center for Eye Research Australia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04744662
- Sponsor's protocol ID
- ONL1204-GA-001
- Status last verified
- Nov 2024
- First submitted
- 13 Nov 2020
- Last updated
- 19 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b Multicenter, Randomized, Controlled, Multi-dose Study of the Safety and Tolerability of ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy (GA) Associated With Age-related Macular Degeneration (AMD
- ClinicalTrials.gov ID
- NCT04744662
- Acronym / sponsor ID
- ONL1204-GA-001
- Conditions
- Geographic Atrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- ONL TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 8 Jul 2021 (actual)
- Primary completion
- 29 Feb 2024 (actual)
- Study completion
- 29 Feb 2024 (actual)
- First posted
- 9 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the safety and tolerability of intravitreal injection of ONL1204 Ophthalmic Solution in patients with geographic atrophy associated with AMD.
GA associated with AMD is one of the world's leading causes of visual disability. It is a progressive disease with no approved therapy to slow or arrest the process of continual photoreceptor and retinal epithelial (RPE) cell loss. A safe and effective therapy for GA will have vast societal benefits. ONL1204 is being developed for this purpose. ONL1204 is a first-in-class inhibitor of fragment apoptosis stimulator receptor-mediated cell death in development for to reduce rates of vision in patients with GA associated with AMD. ONL1204 has demonstrated protection of multiple retinal cell types in several preclinical models of acute ocular injury and the protection of RPE in AMD models. ONL1204 Ophthalmic Solution is currently in a Phase 1 clinical study in patients with macula-off retinal detachment to evaluate safety and tolerability of a multi-dose of ONL1204 Ophthalmic Solution. The study is ongoing and uses the same doses and route of administration as this Phase 1b study in patients with GA.
Detailed description
In this study patients will be enrolled and followed for 24 weeks (natural history phase) at which point they will be randomized to 1 of 2 doses of ONL1204 Ophthalmic Solution or sham. Weeks 24 to 48 make up the Treatment Phase. Patients will be on study for a total of 48 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, care provider)
- Number of arms
- 3
- Model description
- In this study patients will be enrolled and followed for 24 weeks (natural history phase) at which point they will be randomized to 1 of 2 doses of ONL1204 Ophthalmic Solution or sham. Weeks 24 to 48 make up the Treatment Phase. Patients will be on study for a total of 48 weeks.
| Arm | Type | Receives |
|---|---|---|
| Treatment Group A | Experimental | Drug: ONL1204 Ophthalmic solutionONL1204 Ophthalmic solution (dose A) administered by intravitreal injection |
| Treatment Group B | Experimental | Drug: ONL1204 Ophthalmic solutionONL1204 Ophthalmic solution (dose B) administered by intravitreal injection |
| Treatment Group C | Sham comparator | Procedure: sham injectionsham injection without penetrating the eye |
Who may qualify
- Ages
- 55 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Males and females, ≥ 55 years old
- Able to give informed consent and attend study visits
- Bilateral GA secondary to AMD without choroidal neovascularization in either eye
- ETDRS BCVA 20/400 (Snellen equivalent) or better in both eyes
- GA ≥1 disc area (DA) (DA, 2.5 mm2)
Key exclusion criteria (11 listed)
- GA in either eye due to causes other than AMD
- Participation in other ophthalmic clinical trials or use of any other investigational drugs or devices in study eye or systemically for 6 months prior to enrollment, or anticipated participation in other ophthalmic clinical trials or use of any other investigational drugs or devices in study eye or systemically during the study period
- Intraocular inflammation in the study eye
- Ocular or periocular infection in the study eye
- Media opacity that would limit baseline visual acuity or clinical visualization of the retina at baseline
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ONL1204 Ophthalmic solutionLiquid formulation administered by intravitreal (IVT) injection | Drug | Treatment Group A, Treatment Group B |
| sham injectionsham injection is done by touching the eye surface with a syringe without a needle | Procedure or surgery | Treatment Group C |
Primary outcome
Best Corrected Visual Acuity
Time frame: up to 48 weeks
ETDRS chart (number of letters read)
Intraocular pressure
Time frame: up to 48 weeks
Intraocular pressure recorded in mmHg
Slit lamp biomicroscopy
Time frame: up to 48 weeks
Slit lamp using US FDA clinical grading scale 0-4; 0=normal, 4=very severe changes)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2024.
Related trials for Geographic Atrophy
Active, not recruitingPhase 1/2
Phase 1/2 Study of VOY-101 in Patients With Advanced Non-Neovascular Age-Related Macular Degeneration
Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 1/2
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Dry Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 3
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
Age-related Macular Degeneration (AMD), Geographic Atrophy (GA)
RecruitingPhase 1
Phase 1 Study of C.001 in Retinal Degeneration
Geographic Atrophy, Stargardt Disease
RecruitingPhase 2/3
A Long-term Extension Study of JNJ-81201887 (AAVCAGsCD59) Parent Studies in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
Geographic Atrophy, Macular Degeneration
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robyn Guymer, MD principal investigator
Center for Eye Research Australia
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ONL1204 Ophthalmic solution in people with Geographic Atrophy, run by ONL Therapeutics at 8 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Australia, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Best Corrected Visual Acuity (time frame: up to 48 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Nov 2024.