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ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy Associated With Age-related Macular Degeneration

Primary Purpose

Geographic Atrophy

Status
Active
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
ONL1204 Ophthalmic solution
sham injection
Sponsored by
ONL Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Geographic Atrophy

Eligibility Criteria

55 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

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

Sites / Locations

  • Chatswood Retina
  • Retina and Eye Consultants Hurtsville
  • Retinology Institute
  • Queensland Eye Institute
  • Center for Eye Rearch Australia
  • Sunshine Eye Surgeons
  • Eye Institute Limited
  • Southern Eye Specialists

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Sham Comparator

Arm Label

Treatment Group A

Treatment Group B

Treatment Group C

Arm Description

ONL1204 Ophthalmic solution (dose A) administered by intravitreal injection

ONL1204 Ophthalmic solution (dose B) administered by intravitreal injection

sham injection without penetrating the eye

Outcomes

Primary Outcome Measures

Best Corrected Visual Acuity
ETDRS chart (number of letters read)
Intraocular pressure
Intraocular pressure recorded in mmHg
Slit lamp biomicroscopy
Slit lamp using US FDA clinical grading scale 0-4; 0=normal, 4=very severe changes)

Secondary Outcome Measures

Full Information

First Posted
November 13, 2020
Last Updated
July 13, 2023
Sponsor
ONL Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT04744662
Brief Title
ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy Associated With Age-related Macular Degeneration
Official 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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 8, 2021 (Actual)
Primary Completion Date
March 31, 2024 (Anticipated)
Study Completion Date
September 30, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ONL Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Geographic Atrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
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
ParticipantCare Provider
Allocation
Randomized
Enrollment
28 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Treatment Group A
Arm Type
Experimental
Arm Description
ONL1204 Ophthalmic solution (dose A) administered by intravitreal injection
Arm Title
Treatment Group B
Arm Type
Experimental
Arm Description
ONL1204 Ophthalmic solution (dose B) administered by intravitreal injection
Arm Title
Treatment Group C
Arm Type
Sham Comparator
Arm Description
sham injection without penetrating the eye
Intervention Type
Drug
Intervention Name(s)
ONL1204 Ophthalmic solution
Intervention Description
Liquid formulation administered by intravitreal (IVT) injection
Intervention Type
Procedure
Intervention Name(s)
sham injection
Intervention Description
sham injection is done by touching the eye surface with a syringe without a needle
Primary Outcome Measure Information:
Title
Best Corrected Visual Acuity
Description
ETDRS chart (number of letters read)
Time Frame
up to 48 weeks
Title
Intraocular pressure
Description
Intraocular pressure recorded in mmHg
Time Frame
up to 48 weeks
Title
Slit lamp biomicroscopy
Description
Slit lamp using US FDA clinical grading scale 0-4; 0=normal, 4=very severe changes)
Time Frame
up to 48 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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. 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robyn Guymer, MD
Organizational Affiliation
Center for Eye Research Australia
Official's Role
Principal Investigator
Facility Information:
Facility Name
Chatswood Retina
City
Chatswood
State/Province
New South Wales
ZIP/Postal Code
2067
Country
Australia
Facility Name
Retina and Eye Consultants Hurtsville
City
Hurstville
State/Province
New South Wales
ZIP/Postal Code
2220
Country
Australia
Facility Name
Retinology Institute
City
Glen Iris
State/Province
Victoria
ZIP/Postal Code
3146
Country
Australia
Facility Name
Queensland Eye Institute
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
4101
Country
Australia
Facility Name
Center for Eye Rearch Australia
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
VIC 3002
Country
Australia
Facility Name
Sunshine Eye Surgeons
City
St Albans
State/Province
Victoria
ZIP/Postal Code
3021
Country
Australia
Facility Name
Eye Institute Limited
City
Remuera
State/Province
Auckland
ZIP/Postal Code
1050
Country
New Zealand
Facility Name
Southern Eye Specialists
City
Christchurch
ZIP/Postal Code
8013
Country
New Zealand

12. IPD Sharing Statement

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ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy Associated With Age-related Macular Degeneration

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