Neovascular (Wet) Age-related Macular Degeneration (nAMD)Phase 1NCT05903794
A Study of EXG102-031 in Patients With wAMD (Everest)
A Phase 1 study testing EXG102-031 in people with Neovascular (Wet) Age-related Macular Degeneration (nAMD), run by Exegenesis Bio at 2 sites in United States.
Trial summary
In neovascular (wet) age-related macular degeneration (nAMD), the macula, or the part of the eye that provides the clear, detailed central vision, is being affected by abnormal blood vessel growth and leakage. This leakage affects the vision over time and can lead to severe blurriness or blinding. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Neovascular (Wet) Age-related Macular Degeneration (nAMD)
- What is being tested?
- EXG102-031
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- 24 Jul 2023to 16 Feb 2026
- 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
Who may be able to join
- Male or female, age ≥ 50 years of age;
- Diagnosis of nAMD and current active lesion in the study eye at Screening;
- An ETDRS BCVA letter scores between 73 and 19 letters in the study eye;
- Response to anti-VEGF treatment during Screening;
- The study eye must be a pseudophakic lens (post-cataract surgery status); and
- Voluntarily agree to participate in the clinical trial, understand the trial procedures, and be capable of signing the informed consent form before screening.
Who cannot join
- Presence of any ocular disease or history of disease in the study eye other than nAMD that may affect central visual acuity and/or macular detection, including retinal detachment, or in the opinion of the investigator could limit VA improvement in the study eye;
- Presence in the study eye of CNV or macular edema due to causes other than AMD;
- Presence in the study eye of scarring, fibrosis or atrophy involving the macula;
- Subretinal hemorrhage accumulating in the center of the macula of the test eye, with an area of hemorrhage ≥ 4 optic disc diameters;
- Active ocular infection in either eye;
- Presence of advanced glaucoma or uncontrolled glaucoma in the study eye;
- History of intraocular surgery in the study eye within 90 days of screening; or
- Prior receipt of any ocular or systemic gene therapy agent.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Erie Retina Research
Erie, Pennsylvania, 16507
Sierra Eye Associates
Reno, Nevada, 89502
About this study
Official title: An Open-label, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With EXG102-031 in Participants With Neovascular Age-related Macular Degeneration.
In neovascular (wet) age-related macular degeneration (nAMD), the macula, or the part of the eye that provides the clear, detailed central vision, is being affected by abnormal blood vessel growth and leakage. This leakage affects the vision over time and can lead to severe blurriness or blinding. EXG102-031 was made to block the extra vessel formation which would lead to less leakage affecting the vision. Before EXG102-031 can be tested for its efficacy (if it makes vision better), it must be tested to see if it is safely tolerated to confirm it can continue to be studied in more patients with nAMD.
Read the detailed description
Age-related macular degeneration (AMD) is a major cause of blindness and visual impairment in older adults. The wet form of AMD, also called neovascular AMD (nAMD) usually causes faster vision loss than the dry form. The most common current treatments of nAMD are products that inhibit vascular endothelial growth factor (VEGF) (including ranibizumab (LUCENTIS®, Genentech) and aflibercept (EYLEA®, Regeneron) and are delivered by intravitreal injections at 4 to 16 week intervals and continued indefinitely. This Phase I, open-label, multiple-cohort, dose-escalation study is designed to evaluate the safety and tolerability of EXG102-031 gene therapy in subjects with previously treated nAMD. Safety will be assessed over 52 weeks after the administration of EXG102-031.
- Being tested
- EXG102-031
- Medical subject headings
- Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EXG102-031Biological
EXG102-031 is a recombinant adeno-associated virus (rAAV) expressing an angiopoietin domain and VEGF receptor (ABD-VEGFR) fusion protein. EXG102-031 will be administered by subretinal injecting into one eye of each participant.
| Study group | Role | What they receive |
|---|---|---|
| EXG102-031 | Experimental | Each participant will receive a single subretinal injection of EXG102-031 in the study eye. Participants will be enrolled into one of four dosage groups sequentially, and the dose administered will be determined based on which study group the participant is enrolled into. Receives: Biological: EXG102-031 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of safety and tolerability
Frequency, type, and intensity of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs)
Measured: Throughout 52 weeks
Secondary outcomes (3)
Evaluation of potential efficacy
Change from baseline in best corrected visual acuity (BCVA) measured by ETDRS method
Measured: 52 weeks post administration
Evaluation of potential safety
Frequency, type, and intensity of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs)
Measured: Through week 24
Evaluation of supplementary therapy injections received
Average number of doses of anti-vascular endothelial growth factor (VEGF) supplemental therapy received
Measured: Throughout 52 weeks post administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2023
- Study start24 Jul 2023
- Primary completion16 Feb 2026
- Study completion16 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neovascular (Wet) Age-related Macular Degeneration (nAMD)Studies 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
- Not applicable
- Intervention model
- Sequential
- Model description
- Dose-escalation Study with four cohorts of three participants in each.
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Exegenesis Bio
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Arshad Khanani, MD, Principal investigator, Sierra Eye Associates
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05903794
- Sponsor's protocol ID
- EXG102-031 (211)
- Status last verified
- Apr 2026
- First submitted
- 6 Jun 2023
- Last updated
- 8 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With EXG102-031 in Participants With Neovascular Age-related Macular Degeneration.
- ClinicalTrials.gov ID
- NCT05903794
- Acronym / sponsor ID
- EXG102-031 (211)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Exegenesis BioIndustry (pharma, biotech, devices)
- Study start
- 24 Jul 2023 (actual)
- Primary completion
- 16 Feb 2026 (actual)
- Study completion
- 16 Feb 2026 (actual)
- First posted
- 15 Jun 2023 (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.
In neovascular (wet) age-related macular degeneration (nAMD), the macula, or the part of the eye that provides the clear, detailed central vision, is being affected by abnormal blood vessel growth and leakage. This leakage affects the vision over time and can lead to severe blurriness or blinding. EXG102-031 was made to block the extra vessel formation which would lead to less leakage affecting the vision. Before EXG102-031 can be tested for its efficacy (if it makes vision better), it must be tested to see if it is safely tolerated to confirm it can continue to be studied in more patients with nAMD.
Detailed description
Age-related macular degeneration (AMD) is a major cause of blindness and visual impairment in older adults. The wet form of AMD, also called neovascular AMD (nAMD) usually causes faster vision loss than the dry form. The most common current treatments of nAMD are products that inhibit vascular endothelial growth factor (VEGF) (including ranibizumab (LUCENTIS®, Genentech) and aflibercept (EYLEA®, Regeneron) and are delivered by intravitreal injections at 4 to 16 week intervals and continued indefinitely. This Phase I, open-label, multiple-cohort, dose-escalation study is designed to evaluate the safety and tolerability of EXG102-031 gene therapy in subjects with previously treated nAMD. Safety will be assessed over 52 weeks after the administration of EXG102-031.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Dose-escalation Study with four cohorts of three participants in each.
| Arm | Type | Receives |
|---|---|---|
| EXG102-031 | Experimental | Biological: EXG102-031Each participant will receive a single subretinal injection of EXG102-031 in the study eye. Participants will be enrolled into one of four dosage groups sequentially, and the dose administered will be determined based on which study group the participant is enrolled into. |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female, age ≥ 50 years of age;
- Diagnosis of nAMD and current active lesion in the study eye at Screening;
- An ETDRS BCVA letter scores between 73 and 19 letters in the study eye;
- Response to anti-VEGF treatment during Screening;
- The study eye must be a pseudophakic lens (post-cataract surgery status); and
Key exclusion criteria (8 listed)
- Presence of any ocular disease or history of disease in the study eye other than nAMD that may affect central visual acuity and/or macular detection, including retinal detachment, or in the opinion of the investigator could limit VA improvement in the study eye;
- Presence in the study eye of CNV or macular edema due to causes other than AMD;
- Presence in the study eye of scarring, fibrosis or atrophy involving the macula;
- Subretinal hemorrhage accumulating in the center of the macula of the test eye, with an area of hemorrhage ≥ 4 optic disc diameters;
- Active ocular infection in either eye;
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 |
|---|---|---|
| EXG102-031EXG102-031 is a recombinant adeno-associated virus (rAAV) expressing an angiopoietin domain and VEGF receptor (ABD-VEGFR) fusion protein. EXG102-031 will be administered by subretinal injecting into one eye of each participant. | Biological | EXG102-031 |
Primary outcome
Evaluation of safety and tolerability
Time frame: Throughout 52 weeks
Frequency, type, and intensity of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs)
3 secondary outcomes
Evaluation of potential efficacy
Time frame: 52 weeks post administration
Evaluation of potential safety
Time frame: Through week 24
Evaluation of supplementary therapy injections received
Time frame: Throughout 52 weeks post administration
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 8 Apr 2026.
- Individual participant dataSharing planned: No
Related trials for Neovascular (Wet) Age-related Macular Degeneration (nAMD)
Not yet recruitingPhase 2
A Study to Evaluate the Efficacy and Safety of VIS-101 in Participants With Neovascular Age-Related Macular Degeneration
Neovascular Age Related Macular Degeneration(nAMD)
RecruitingPhase 1
Safety and Efficacy Study of IVB103 Injection in Subjects With Neovascular (Wet) Agerelated Macular Degeneration (nAMD).
nAMD
Active, not recruitingPhase 2
Safety and Efficacy of ADVM-022 in Treatment-Experienced Patients With Neovascular Age-related Macular Degeneration [LUNA]
Neovascular Age-related Macular Degeneration
Active, not recruitingPhase 3
4D-150 in Patients With Macular Neovascularization Secondary to Age-Related Macular Degeneration
Macular Neovascularization Secondary to Age-Related Macular Degeneration
RecruitingPhase 3
Single Intravitreal (IVT) Injection of 4D-150 in Patients With Macular Neovascularization Secondary to Age-Related Macular Degeneration
Neovascular Age-Related Macular Degeneration (nAMD)
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.
Arshad Khanani, MD principal investigator
Sierra Eye Associates
2 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 EXG102-031 in people with Neovascular (Wet) Age-related Macular Degeneration (nAMD), run by Exegenesis Bio at 2 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?
The sponsor lists 6 inclusion and 8 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 8 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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?
Evaluation of safety and tolerability (time frame: Throughout 52 weeks). 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 8 Apr 2026.