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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04744662




Registration number
NCT04744662
Ethics application status
Date submitted
13/11/2020
Date registered
9/02/2021
Date last updated
6/03/2024

Titles & IDs
Public title
ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy Associated With Age-related Macular Degeneration
Scientific title
A Phase 1b Multicenter, Randomized, Controlled, Single-dose Study of the Safety and Tolerability of ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy (GA) Associated With Age-related Macular Degeneration (AMD
Secondary ID [1] 0 0
ONL1204-GA-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Geographic Atrophy 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ONL1204 Ophthalmic solution
Treatment: Surgery - sham injection

Experimental: Treatment Group A - ONL1204 Ophthalmic solution (dose A) administered by intravitreal injection

Experimental: Treatment Group B - ONL1204 Ophthalmic solution (dose B) administered by intravitreal injection

Sham Comparator: Treatment Group C - sham injection without penetrating the eye


Treatment: Drugs: ONL1204 Ophthalmic solution
Liquid formulation administered by intravitreal (IVT) injection

Treatment: Surgery: sham injection
sham injection is done by touching the eye surface with a syringe without a needle
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Treatment: Surgery
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Best Corrected Visual Acuity
Timepoint [1] 0 0
up to 48 weeks
Primary outcome [2] 0 0
Intraocular pressure
Timepoint [2] 0 0
up to 48 weeks
Primary outcome [3] 0 0
Slit lamp biomicroscopy
Timepoint [3] 0 0
up to 48 weeks

Eligibility
Key inclusion criteria
- 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

- 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.
Minimum age
55 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- 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

- 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/07/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
29/02/2024
Sample size
Target
Accrual to date
Final
28
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Chatswood Retina - Chatswood
Recruitment hospital [2] 0 0
Retina and Eye Consultants Hurtsville - Hurstville
Recruitment hospital [3] 0 0
Retinology Institute - Glen Iris
Recruitment hospital [4] 0 0
Queensland Eye Institute - Melbourne
Recruitment hospital [5] 0 0
Center for Eye Rearch Australia - Melbourne
Recruitment hospital [6] 0 0
Sunshine Eye Surgeons - St Albans
Recruitment postcode(s) [1] 0 0
2067 - Chatswood
Recruitment postcode(s) [2] 0 0
2220 - Hurstville
Recruitment postcode(s) [3] 0 0
3146 - Glen Iris
Recruitment postcode(s) [4] 0 0
4101 - Melbourne
Recruitment postcode(s) [5] 0 0
VIC 3002 - Melbourne
Recruitment postcode(s) [6] 0 0
3021 - St Albans
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland
Country [2] 0 0
New Zealand
State/province [2] 0 0
Christchurch

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
ONL Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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. 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 single-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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04744662
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Robyn Guymer, MD
Address 0 0
Center for Eye Research Australia
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04744662