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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)]

Status: Recruiting
Location: See all (2) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1
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
Participation Requirements
Sex: All
Minimum Age: 40
Maximum Age: 85
Healthy Volunteers: f
View:

• 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.

Locations
United States
California
Global Research Management, Inc.
RECRUITING
Glendale
South Carolina
Charleston Neuroscience Institute
RECRUITING
Charleston
Contact Information
Primary
Life Biosciences
ER100trial@lifebiosciences.com
(857) 400-9245
Time Frame
Start Date: 2026-03-02
Estimated Completion Date: 2032-03
Participants
Target number of participants: 18
Treatments
Experimental: OAG - Low Dose ER-100 (2 x 10^11 vg/eye)
Participants with Open Angle Glaucoma will receive a low dose of ER-100 administered to one eye. ER-100 is delivered via a modified adeno-associated virus (AAV) vector and activated by systemic doxycycline taken for 8 weeks (56 days). This dose level begins with a sentinel participant followed by additional participants after DSMB review.
Experimental: OAG - High Dose ER-100 (6 x 10^11 vg/eye)
Participants with Open Angle Glaucoma will receive a higher dose of ER-100 administered to one eye. ER-100 is delivered via a modified AAV vector and activated by systemic doxycycline for 8 weeks. This dose level also begins with a sentinel participant and proceeds following DSMB review.
Experimental: NAION - Selected Dose ER-100
Participants with Non-Arteritic Anterior Ischemic Optic Neuropathy will receive ER-100 at a dose selected based on safety and tolerability data from the OAG cohort. ER-100 is administered to one eye and activated by systemic doxycycline for 8 weeks. Initial enrollment is limited to three participants, with potential expansion to six following DSMB review.
Sponsors
Leads: Life Biosciences Inc.

This content was sourced from clinicaltrials.gov