ER-100 for Glaucoma

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Overseen ByAnita Treohan
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ER-100, a potential drug, to determine its safety and possible side effects for individuals with specific eye conditions. Participants will receive a single dose of ER-100 and undergo close monitoring to assess its impact on their health and vision over time. The trial targets adults with either Open Angle Glaucoma, where fluid pressure in the eye damages the optic nerve, or Non-Arteritic Anterior Ischemic Optic Neuropathy, a sudden vision loss due to reduced blood flow to the optic nerve. Individuals with vision issues related to these conditions might be suitable for this study. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the unique opportunity to be among the first to receive this new drug.

Is there any evidence suggesting that ER-100 is likely to be safe for humans?

Research has shown that ER-100, a treatment being tested for eye conditions like Open Angle Glaucoma (OAG) and Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION), is currently under study to determine its safety for humans. As a Phase 1 trial, ER-100 is being tested in people for the first time to assess its safety.

Currently, no specific safety results exist from studies on ER-100. However, the primary goal of Phase 1 is to determine if people can take ER-100 without major issues. In this trial, researchers administer ER-100 as a single dose and closely monitor for any side effects. They also conduct detailed eye exams and lab tests to ensure it is well-tolerated.

Participants receive ER-100 through a method that uses a virus to deliver genes into cells and take doxycycline (an antibiotic) for eight weeks to activate the treatment. This careful approach helps researchers identify potential side effects early on. Overall, more data from this ongoing trial will provide clearer safety insights.12345

Why do researchers think this study treatment might be promising for glaucoma?

Researchers are excited about ER-100 for glaucoma because it offers a novel approach by using a modified adeno-associated virus (AAV) vector to deliver treatment directly to the eye, which is then activated by systemic doxycycline. Unlike standard treatments like eye drops or oral medications that aim to reduce eye pressure, ER-100 potentially targets the root cause of the disease. This gene therapy approach could offer longer-lasting effects and improved outcomes for patients with open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION). By directly addressing the underlying issues at a molecular level, ER-100 represents a promising step forward in treating these vision-threatening conditions.

What evidence suggests that ER-100 might be an effective treatment for optic nerve conditions?

Research has shown that ER-100, when tested on animals similar to humans, successfully targets specific eye cells and helps reduce vision loss. This trial will evaluate ER-100 for conditions like Open Angle Glaucoma (OAG) and Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION). Participants with OAG will receive either a low or high dose of ER-100, while those with NAION will receive a selected dose based on safety and tolerability data. The treatment uses a modified virus to deliver the therapy, which doxycycline then activates. Although human studies have provided limited information, these early results offer a promising basis for ER-100's potential to protect vision in people with these conditions.12678

Who Is on the Research Team?

SR

Sharon Rosenzweign-Lipson, PhD

Principal Investigator

Life Biosciences Inc.

Are You a Good Fit for This Trial?

Adults aged 40-85 with Open Angle Glaucoma or Non-Arteritic Anterior Ischemic Optic Neuropathy, having eye pressure under 30 mmHg and moderate to advanced vision loss. They must not need glaucoma surgery within 2 months post-treatment, be able to use effective birth control if applicable, have reasonable vision (at least 20/80), and can follow the study schedule.

Inclusion Criteria

My eyes can be safely dilated for an exam.
I am between 40 and 85 years old.
I can attend all study visits, follow the schedule, and understand the study materials.
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Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of ER-100 and undergo safety assessments including detailed eye examination and laboratory tests. Systemic doxycycline is administered for 8 weeks to activate OSK expression.

8 weeks
9 visits in the first 6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment, with assessments including visual acuity and intraocular pressure.

5 years
1 visit per year through Year 5, plus 3 remote visits

What Are the Treatments Tested in This Trial?

Interventions

  • ER-100

Trial Overview

The trial is testing ER-100 epigenetic therapy's safety in a single dose for people with optic nerve conditions. It involves detailed eye exams, lab tests, body fluid samples collection for drug processing analysis, quality of life questionnaires, and up to a five-year follow-up for health and vision outcomes.

How Is the Trial Designed?

3

Treatment groups

Experimental Treatment

Group I: OAG - Low Dose ER-100 (2 x 10^11 vg/eye)Experimental Treatment1 Intervention
Group II: OAG - High Dose ER-100 (6 x 10^11 vg/eye)Experimental Treatment1 Intervention
Group III: NAION - Selected Dose ER-100Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Life Biosciences Inc.

Lead Sponsor

Citations

Evaluating ER-100 for Safety in People With Glaucoma or ...

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, ...

Review of evidence for treatments of acute non arteritic ...

To review treatment modalities that have been studied in acute non arteritic anterior ischemic optic neuropathy (NAION).

A Randomized Sham-Controlled Phase 2/3 Trial of QPI ...

Nonarteritic anterior ischemic optic neuropathy can be a visually devastating disease because 20% to 30% of patients have a final visual acuity of 20/200 or ...

Life Biosciences Presents at AAO Highlighting Progress of ...

Collectively, these results demonstrate that ER-100 successfully targets retinal ganglion cells and reduces the loss of visual function in a NHP ...

Risk of Nonarteritic Anterior Ischemic Optic Neuropathy in ...

This cohort study investigates whether semaglutide prescription is associated with increased risk of nonarteritic anterior ischemic optic ...

Non-Arteritic Anterior Ischemic Optic Neuropathy (NA-AION)

Non-arteritic anterior ischemic optic neuropathy (NA-AION) is a major cause of blindness or severely impaired vision in adults, representing the most common ...

Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION)

Obstructive sleep apnoea and increased risk of non-arteritic anterior ischaemic optic neuropathy. The British journal of ophthalmology. 2018 ...

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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 ...