Avoralstat for Diabetic Macular Edema

Recruiting at 7 trial locations

Overseen By BioCryst Pharmaceuticals, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: BioCryst Pharmaceuticals

Disqualifiers: Retinal surgery, Untreated diabetes, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: Single dose

9 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called avoralstat to determine its safety and effectiveness for people with diabetic macular edema (DME), a condition that causes eye swelling and can lead to vision problems. The study involves a one-time injection of avoralstat directly into the eye. Suitable candidates for this trial have DME with vision issues requiring treatment and have not received more than three anti-VEGF injections. The researchers aim to find a new way to help people with DME maintain or improve their vision. As a Phase 1 trial, this research focuses on understanding how avoralstat works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you have started new diabetes medications within the last 3 months, you may not be eligible to participate.

Is there any evidence suggesting that avoralstat is likely to be safe for humans?

Research shows that avoralstat is being tested for its safety and potential to help with diabetic macular edema (DME), an eye condition. In this study, participants received a single dose of avoralstat, and researchers closely monitor its safety. Previous studies suggest that avoralstat is generally well-tolerated, with no major safety concerns reported so far. However, since this is an early study, the main goal is to ensure the treatment is safe for people with DME. Participants might help identify any side effects not yet noticed. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for diabetic macular edema, which often involve anti-VEGF injections or corticosteroids, Avoralstat offers a novel approach. Avoralstat is administered as a single dose through a suprachoroidal injection, which directly targets the back of the eye and may enhance drug delivery efficiency. Researchers are excited about Avoralstat because it has the potential to reduce inflammation by specifically inhibiting kallikrein, a different mechanism than current options, which might lead to improved outcomes and fewer side effects.

What evidence suggests that avoralstat might be an effective treatment for diabetic macular edema?

Research has shown that avoralstat, administered as a single-dose suprachoroidal injection in this trial, might help treat diabetic macular edema (DME), a condition where fluid leaks into the retina and causes vision problems. Studies in rabbits demonstrated that avoralstat can stop this fluid from leaking. This finding is promising because it suggests the drug might help keep the retina dry and improve vision in humans. Avoralstat is known for its effectiveness, meaning it can work well even in small doses. While more studies are needed in people, these early findings are encouraging. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

This trial is for adults with Diabetic Macular Edema (DME) who need treatment and have a blood sugar control measure (HbA1c) below 10%. They should have some vision loss due to DME, mild-to-severe non-proliferative diabetic retinopathy, and specific levels of central subfield thickness. People with conditions that could interfere with the study or pose risks are excluded.

Inclusion Criteria

My HbA1c level is below 10%.

My vision in the study eye ranges from slightly impaired to significantly impaired.

My eye's central thickness is between 325 and 600 μm.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single suprachoroidal injection of avoralstat at one of three dose levels (low, medium, high)

Single dose

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 weeks

Multiple visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Avoralstat

Trial Overview

The study is testing avoralstat's safety and effectiveness in treating DME. Participants will receive a single dose of avoralstat. The main focus is on how well it works and any potential side effects in adult participants.

How Is the Trial Designed?

Group I: Single dose suprachoroidal injection of avoralstat Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

BioCryst Pharmaceuticals

Lead Sponsor

Trials

55

Recruited

4,400+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07228559

A Study of Avoralstat In Participants With Diabetic Macular ...

The goal of this study is to assess the safety and therapeutic potential of a single dose of avoralstat in adult participants with DME. Detailed Description.

2.

ophthalmologytimes.com

ophthalmologytimes.com/view/asrs-2025-asrs-2025-suprachoroidal-avoralstat-stops-vascular-leakage-in-a-well-established-rabbit-model

ASRS 2025: Suprachoroidal avoralstat stops vascular ...

This 3x3 dose-escalation study will involve recently diagnosed DME patients and aims to further assess safety, durability, and early efficacy ...

3.

ir.biocryst.com

ir.biocryst.com/news-releases/news-release-details/biocryst-and-clearside-biomedical-enter-partnership-develop

BioCryst and Clearside Biomedical Enter Partnership to ...

Avoralstat has high potency and low solubility, which are two characteristics important to achieving potential efficacy with reduced dosing ...

4.

theglobeandmail.com

theglobeandmail.com/investing/markets/stocks/BCRX/pressreleases/36627532/biocrysts-avoralstat-study-a-new-hope-for-diabetic-macular-edema/

BioCryst's Avoralstat Study: A New Hope for Diabetic ...

The study aims to assess the safety and therapeutic potential of a single dose of avoralstat in adults with Diabetic Macular Edema (DME), a ...

5.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC5567804/

Comparative Effectiveness Trial for Diabetic Macular Edema

The primary outcome was mean visual acuity change from baseline to one year. All three treatment groups had improvement at one month with substantial ...