Geographic AtrophyPhase 1NCT04615325
A Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Intravitreal Injections of RO7303359 in Participants With Geographic Atrophy Secondary to Age-related Macular Degeneration
A Phase 1 study testing RO7303359 in people with Geographic Atrophy, run by Hoffmann-La Roche at 12 sites in United States.
Trial summary
This multicenter study will investigate the safety, tolerability, pharmacokinetics, and immunogenicity of RO7303359 following single intravitreal (ITV) injection in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Geographic Atrophy
- What is being tested?
- RO7303359
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- 12 sitesUnited States
- Study dates
- 8 Dec 2020to 4 Jan 2023
- 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 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Ocular Inclusion Criteria Study Eye:
- Visual acuity: BCVA letter score of 19-48 ETDRS letters (Snellen equivalent of 20/125-20/400) using ETDRS charts at a starting distance of 4 meters
- Well-demarcated area of GA secondary to AMD in the absence of choroidal neovascularization (CNV)
- GA area must be >/= 0.5 disc area (1.25 mm^2)
Ocular Exclusion Criteria, Study Eye:
- GA in the study eye due to causes other than AMD
- History of vitrectomy surgery, submacular surgery, or other surgical intervention for AMD
- Previous laser photocoagulation for CNV, diabetic macular edema, retinal vein occlusion, or proliferative diabetic retinopathy
- Prior treatment with photodynamic therapy, external beam radiation therapy (for intraocular conditions), or transpupillary thermotherapy
Ocular Exclusion Criteria, Both eyes:
- Evidence of prior or active CNV
- Previous participation in interventional clinical trials for GA or dry AMD, except for vitamins and minerals, irrespective of the route of administration (i.e. ocular or systemic) within the last 6 months
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Western Carolina Retinal Associate PA
Asheville, North Carolina, 28803
Austin Clinical Research LLC
Austin, Texas, 78750
Retina Foundation of the Southwest
Dallas, Texas, 75231
Colorado Retina Associates, PC
Denver, Colorado, 80230
Florida Eye Associates
Melbourne, Florida, 32901
Northern California Retina Vitreous Associates
Mountain View, California, 94040
Tennessee Retina PC.
Nashville, Tennessee, 37203
University Retina and Macula Associates, PC
Oak Forest, Illinois, 60452
Sierra Eye Associates
Reno, Nevada, 89502
Associated Retinal Consultants PC
Royal Oak, Michigan, 48073
Retinal Consultants Med Group
Sacramento, California, 95825
California Retina Consultants - Santa Maria
Santa Maria, California, 93454
About this study
Official title: A Phase 1a, Multicenter, Open-label, Single-dose, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Intravitreal Injections of RO7303359 in Patients With Geographic Atrophy Secondary to Age-related Macular Degeneration
This multicenter study will investigate the safety, tolerability, pharmacokinetics, and immunogenicity of RO7303359 following single intravitreal (ITV) injection in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Participants will receive an ITV injection of RO7303359 in the single ascending dose stage and the maximum tolerated dose (MTD) or maximum tested dose (MTeD) of RO7303359 in the expansion stage.
- Conditions
- Geographic Atrophy
- Being tested
- RO7303359
- Medical subject headings
- Geographic Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RO7303359Drug
RO730359 will be administered as a single intravitreal injection.
| Study group | Role | What they receive |
|---|---|---|
| Single Ascending Dose Stage | Experimental | Participants will receive a single dose of RO7303359, in multiple escalating cohorts (A-D). Receives: Drug: RO7303359 |
| Expansion Cohort Stage | Experimental | Participants will receive the maximum tolerated dose (MTD) or the maximum tested dose (MTeD) as determined in the single ascending dose stage. Receives: Drug: RO7303359 |
| Optional Cohort E | Experimental | An optional additional cohort may be added with the dose not exceed the MTD or MTeD. Receives: Drug: RO7303359 |
| Optional cohort F | Experimental | An optional additional cohort may be added with the dose not exceed the MTD or MTeD. Receives: Drug: RO7303359 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Ocular Adverse Events
Measured: Up to 12 weeks
Percentage of Participants With Systemic Adverse Events
Measured: Up to 12 weeks
Percentage of Participants With Dose-limiting Adverse Events (DLAEs),
Measured: Up to 12 weeks
Percentage of Participants With Serious Adverse Events (SAEs)
Measured: Up to 12 weeks
Percentage of Participants With Adverse Events Leading to Study Discontinuation
Measured: Up to 12 weeks
Percentage of Participants With Adverse Events of Special Interest (AESIs)
Measured: Up to 12 weeks
Change from Baseline in Best Corrected Visual Acuity (BCVA) Score as Assessed by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart
Measured: Baseline, Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2020
- Study start8 Dec 2020
- Primary completion4 Jan 2023
- Study completion4 Jan 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Geographic AtrophyStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- 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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04615325
- Sponsor's protocol ID
- GR42163
- Status last verified
- Feb 2023
- First submitted
- 29 Oct 2020
- Last updated
- 1 Mar 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research\_and\_development/who\_we\_are\_how\_we\_work/clinical\_trials/our\_commitment\_to\_data\_sharing.htm).
Publications
Pearlman JA, Sheth VS, Khanani AM, Indjeian VB, Brunstein F, Kuruvilla D, Maia M, Dere R, Ma L, Chen H, Datta S, Willis JR, Wiley HE. Phase I Study of the Anti-interleukin 33 Fragment Antigen-Binding Region RO7303359 in Geographic Atrophy Secondary to Age-Related Macular Degeneration. Ophthalmol Sci. 2025 Apr 20;5(5):100800. doi: 10.1016/j.xops.2025.100800. eCollection 2025 Sep-Oct.
PubMed 40538770
Study at a glance
From the registry record, last updated by the sponsor on 1 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1a, Multicenter, Open-label, Single-dose, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Intravitreal Injections of RO7303359 in Patients With Geographic Atrophy Secondary to Age-related Macular Degeneration
- ClinicalTrials.gov ID
- NCT04615325
- Acronym / sponsor ID
- GR42163
- Conditions
- Geographic Atrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 8 Dec 2020 (actual)
- Primary completion
- 4 Jan 2023 (actual)
- Study completion
- 4 Jan 2023 (actual)
- First posted
- 4 Nov 2020 (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.
This multicenter study will investigate the safety, tolerability, pharmacokinetics, and immunogenicity of RO7303359 following single intravitreal (ITV) injection in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Participants will receive an ITV injection of RO7303359 in the single ascending dose stage and the maximum tolerated dose (MTD) or maximum tested dose (MTeD) of RO7303359 in the expansion stage.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Single Ascending Dose Stage | Experimental | Drug: RO7303359Participants will receive a single dose of RO7303359, in multiple escalating cohorts (A-D). |
| Expansion Cohort Stage | Experimental | Drug: RO7303359Participants will receive the maximum tolerated dose (MTD) or the maximum tested dose (MTeD) as determined in the single ascending dose stage. |
| Optional Cohort E | Experimental | Drug: RO7303359An optional additional cohort may be added with the dose not exceed the MTD or MTeD. |
| Optional cohort F | Experimental | Drug: RO7303359An optional additional cohort may be added with the dose not exceed the MTD or MTeD. |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| RO7303359RO730359 will be administered as a single intravitreal injection. | Drug | Expansion Cohort Stage, Optional Cohort E, Optional cohort F, Single Ascending Dose Stage |
Primary outcome
Percentage of Participants With Ocular Adverse Events
Time frame: Up to 12 weeks
Percentage of Participants With Systemic Adverse Events
Time frame: Up to 12 weeks
Percentage of Participants With Dose-limiting Adverse Events (DLAEs),
Time frame: Up to 12 weeks
Percentage of Participants With Serious Adverse Events (SAEs)
Time frame: Up to 12 weeks
Percentage of Participants With Adverse Events Leading to Study Discontinuation
Time frame: Up to 12 weeks
Percentage of Participants With Adverse Events of Special Interest (AESIs)
Time frame: Up to 12 weeks
Change from Baseline in Best Corrected Visual Acuity (BCVA) Score as Assessed by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart
Time frame: Baseline, Week 12
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 1 Mar 2023.
- Derived from this study: PubMed 40538770Pearlman JA, Sheth VS, Khanani AM, Indjeian VB, Brunstein F, Kuruvilla D, Maia M, Dere R, Ma L, Chen H, Datta S, Willis JR, Wiley HE. Phase I Study of the Anti-interleukin 33 Fragment Antigen-Binding Region RO7303359 in Geographic Atrophy Secondary to Age-Related Macular Degeneration. Ophthalmol Sci. 2025 Apr 20;5(5):100800. doi: 10.1016/j.xops.2025.100800. eCollection 2025 Sep-Oct.
- Individual participant dataSharing planned: No. Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).
Related trials for Geographic Atrophy
RecruitingPhase 1
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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
Active, not recruitingPhase 1/2
Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy
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Active, not recruitingPhase 4
A Study About the Safety of ASP3021 Eye Injections and if They Help People in Japan With Vision Loss From Age-related Macular Degeneration
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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
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.
Clinical Trials study director
Hoffmann-La Roche
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RO7303359 in people with Geographic Atrophy, run by Hoffmann-La Roche at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 1 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. 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. 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?
Percentage of Participants With Ocular Adverse Events (time frame: Up to 12 weeks). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04615325, which also lists 1 linked publication.
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 1 Mar 2023.