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)]
Life Biosciences Inc.
18 participants
Mar 2, 2026
INTERVENTIONAL
Conditions
Summary
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
Eligibility
Inclusion Criteria17
- 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 Criteria19
- 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).
- \- Diagnosed with glaucoma before age 40.
- 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.
Interventions
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)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07290244