RecruitingPhase 1

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)]

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
40 Years to 85 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Visits mentioned: 15 visits, 1 visit
  • Time periods mentioned: 8 weeks

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Systemic doxycycline is administered for 8 weeks (56 days) to activate OSK expression."
  • "A total of 15 clinic visits is planned for the study: 10 in the first 6 months, then one visit per year through Year 5."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests the safety of one dose of ER-100 and checks for side effects. It is for adults ages 40 to 85 with open-angle glaucoma or NAION, two conditions that damage the nerve that carries signals from the eye to the brain. Participants will have eye exams and other tests, with follow-up for up to 5 years.

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

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be 40 to 85 years old, understand the study, and sign a consent form.
  • Be able to attend all visits and tests and speak a language used in the study materials.
  • Have eyes clear enough for the doctor to examine the back of the eye, and be able to safely use drops that widen the pupils.
  • If you can become pregnant, agree to use a condom and one highly effective birth control method during sex for at least 4 months after ER-100.
  • For glaucoma: Have open-angle glaucoma in the study eye, with eye pressure below 30 mmHg and moderate to advanced loss on a side-vision test (MD score from -6 to -20 dB).
  • For glaucoma: Have eye-chart vision of 20/80 or better in the study eye and not expect to need glaucoma surgery within 2 months after ER-100.
  • For NAION: Have sudden, painless vision loss in one eye within 14 days before ER-100, confirmed by a specialist, with swelling of the eye nerve. Past NAION in the other eye is allowed.
  • For NAION: Have eye-chart vision between 20/40 and 2/500 and side-vision loss from nerve damage (MD score worse than -3 dB). If only one eye is affected, the pupils must respond differently to light.

Who can't join

  • Past inflammation of the eye nerve, or repeated inflammation inside the eye not caused by injury or surgery.
  • Eye problems that limit vision or affect testing, trouble keeping your eyes focused during tests, eye pressure of 30 mmHg or higher, or eye surgery, including laser treatment, within 3 months before ER-100.
  • Allergy to tetracycline antibiotics or steroids, or use of warfarin, Dilantin, carbamazepine, or barbiturates within 14 days before starting the study or during the first 8 weeks.
  • Cancer within the past 5 years, except basal cell skin cancer; type 1 diabetes; or poorly controlled type 2 diabetes (A1c blood sugar result above 7 despite treatment).
  • Pregnancy or breastfeeding; a weakened immune system, including a past organ transplant; or a positive test for HIV, hepatitis B or C, or tuberculosis.
  • Memory or thinking problems that prevent study participation, or another condition the study doctor believes could make the study unsafe, affect results, or prevent completion.
  • Past gene therapy using AAV, a virus used to carry genes into cells. For glaucoma: A diagnosis before age 40.
  • For NAION: Signs of giant cell arteritis, a condition that inflames blood vessels, based on abnormal blood tests; or NAION that started in both eyes at the same time.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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.

Study sites (4)

CA

MA

NY

SC