Evaluating ER-100 for Safety in People With Glaucoma or Non-Arteritic Anterior Ischemic Optic Neuropathy (Optic Nerve Conditions)

A Phase 1 Single Dose Study to Evaluate the Safety and Tolerability of ER-100 in Optic Neuropathies [Open Angle Glaucoma (OAG) and Non-arteritic Anterior Ischemic Optic Neuropathy (NAION)]

Evaluating ER-100 for Safety in People With Glaucoma or Non-Arteritic Anterior Ischemic Optic Neuropathy (Optic Nerve Conditions) (NCT07290244) is a Phase 1 interventional studying Open Angle Glaucoma (OAG) and NAION( Non-arteritic Anterior Ischemic Optic Neuropathy), sponsored by Life Biosciences Inc.. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

The goal of this clinical trial is to evaluate the safety and tolerability of a single dose of ER-100 in adults with optic nerve conditions, specifically Open Angle Glaucoma (OAG) and Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION). The main questions it aims to answer are: * Is ER-100 safe when given as a single dose to people with OAG or NAION * What side effects may occur, if any, after taking ER-100? Participants will: * Receive a single dose of ER-100 * Undergo safety assessments including detailed eye examination and laboratory tests * Provide body fluid samples (tears, saliva, feces, urine) to help researchers understand how the drug is processed and cleared from the body * Complete questionnaires about their quality of life * Be followed for up to 5 years to monitor long-term health and vision outcomes

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Open Angle Glaucoma (OAG), a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 18 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: - Have clear eye structures and be able to have your pupils safely dilated so the doctor can examine the back of your eye. - Able to understand the study and sign a consent form. - Be between 40 and 85 years old. - Willing and able to follow the study schedule, including all visits and tests, and speak a language for which the study materials are available. - If a participant can become pregnant, must agree to use a condom and one highly effective form of birth control during sex for at least 4 months after receiving the study drug (ER-100). For participants with open-angle glaucoma (OAG): - Diagnosis of open-angle glaucoma in the study eye. - Eye pressure must be less than 30 mmHg, measured with a standard test. - Visual field test must show moderate to advanced vision loss (MD score between -6 and -20 dB). - Not expected to need glaucoma surgery in the study eye within 2 months after receiving ER-100. - Have reasonably good vision in the study eye (at least 20/80 on a standard eye chart). For participants with NAION (non-arteritic anterior ischemic optic neuropathy): - Had a sudden, painless loss of vision in one eye within 14 days before receiving ER-100, confirmed by a specialist. Having had NAION in the other eye is okay. - The affected eye must show swelling of the optic nerve. - Visual field test must show vision loss consistent with optic nerve damage (MD worse than -3.0 dB). - If only one eye is affected, there must be a difference in pupil response between the two eyes. - Have vision in the affected eye between 20/40 and 2/500 on a standard eye chart. Who Should NOT Join This Trial: - History of optic neuritis (inflammation of the optic nerve) or repeated episodes of eye inflammation (uveitis) not caused by injury or surgery. - Allergic reactions to tetracycline antibiotics or steroid medications. - Moderate to severe cataracts, macular problems, or corneal issues that could interfere with eye testing. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: * Have clear eye structures and be able to have your pupils safely dilated so the doctor can examine the back of your eye. * Able to understand the study and sign a consent form. * Be between 40 and 85 years old. * Willing and able to follow the study schedule, including all visits and tests, and speak a language for which the study materials are available. * If a participant can become pregnant, must agree to use a condom and one highly effective form of birth control during sex for at least 4 months after receiving the study drug (ER-100). For participants with open-angle glaucoma (OAG): * Diagnosis of open-angle glaucoma in the study eye. * Eye pressure must be less than 30 mmHg, measured with a standard test. * Visual field test must show moderate to advanced vision loss (MD score between -6 and -20 dB). * Not expected to need glaucoma surgery in the study eye within 2 months after receiving ER-100. * Have reasonably good vision in the study eye (at least 20/80 on a standard eye chart). For participants with NAION (non-arteritic anterior ischemic optic neuropathy): * Had a sudden, painless loss of vision in one eye within 14 days before receiving ER-100, confirmed by a specialist. Having had NAION in the other eye is okay. * The affected eye must show swelling of the optic nerve. * Visual field test must show vision loss consistent with optic nerve damage (MD worse than -3.0 dB). * If only one eye is affected, there must be a difference in pupil response between the two eyes. * Have vision in the affected eye between 20/40 and 2/500 on a standard eye chart. Exclusion Criteria: * History of optic neuritis (inflammation of the optic nerve) or repeated episodes of eye inflammation (uveitis) not caused by injury or surgery. * Allergic reactions to tetracycline antibiotics or steroid medications. * Moderate to severe cataracts, macular problems, or corneal issues that could interfere with eye testing. * Unable to keep your eyes focused on a target during testing. * Had cataract surgery or other eye surgery (including laser procedures) within 3 months before receiving the study drug. * Had cancer (except for basal cell skin cancer) within the past 5 years. * Have Type 1 diabetes, or poorly controlled Type 2 diabetes (A1c greater than 7 despite treatment). * Have memory or thinking problems that prevent you from understanding the study or completing the required tests. * Pregnant or breastfeeding. * Have a weakened immune system, including a history of organ transplant, or test positive for HIV, hepatitis B or C, or tuberculosis. * Have any other condition that, in the opinion of the study doctor, could increase your risk from the study drug or procedures, affect the study results, or make it hard for you to complete the study. * Have macular disease, advanced diabetic eye disease, or other eye conditions that limit vision in the study eye. * Eye pressure at screening is 30 mmHg or higher. * Taking certain medications (warfarin, dilantin, carbamazepine, or barbiturates) within 14 days before starting the study or during the first 8 weeks. * Have any other eye or vision problem that, in the opinion of the study doctor, could affect safety or interfere with vision testing. * Have previously received any gene therapy using adeno-associated virus (AAV). Additional Exclusion Criteria for Participants with Open-Angle Glaucoma (OAG): \- Diagnosed with glaucoma before age 40. Additional Exclusion Criteria for Participants with NAION: * Show signs of giant cell arteritis (a type of blood vessel inflammation), based on abnormal blood tests. * Had NAION start in both eyes at the same time.

Treatments Being Tested

GENETIC

ER-100 epigenetic therapy

ER-100 is an investigational AAV-based epigenetic therapy administered via intravitreal injection to one eye. It uses a modified adeno-associated virus (AAV) vector to deliver instructions for producing three transcription factors-OCT4, SOX2, and KLF4 (collectively referred to as OSK)-intended to reverse age-related epigenetic changes in retinal cells. Systemic doxycycline is administered for 8 weeks (56 days) to activate OSK expression. ER-100 does not alter the participant's existing genes, and the AAV vector has been engineered to eliminate its ability to cause infectious disease.

Locations (2)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Global Research Management, Inc.

Glendale, California, United States

Charleston Neuroscience Institute

Charleston, South Carolina, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT07290244), the sponsor (Life Biosciences Inc.), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT07290244 clinical trial studying?

The goal of this clinical trial is to evaluate the safety and tolerability of a single dose of ER-100 in adults with optic nerve conditions, specifically Open Angle Glaucoma (OAG) and Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION). The main questions it aims to answer are: * Is ER-100 safe when given as a single dose to people with OAG or NAION * What side effects may occur, if any, after taking ER-100? Participants will: * Receive a single dose of ER-100 * Undergo safety assessments including detailed eye examination and laboratory tests * Provide body fluid samples (tears, saliv… The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT07290244?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT07290244?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT07290244. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT07290244. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.