A Study of Avoralstat In Participants With Diabetic Macular Edema

NCT07228559 | Recruiting Phase 1 FDA Drug

• 1 other identifier: BCX4161-111
• Study Type: interventional
• Target: 9
• Locations: 2 countries
• Sites: 8

Brief Summary

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

Conditions

Diabetic Macular Edema (DME)

Keywords

single ascending dose; safety; pharmacokinetics; suprachoroidal injection

Outcome Measures

Primary Outcomes (1)

• The safety and tolerability of avoralstat in participants with DME. Incidence and severity of ocular (study eye) and systemic treatment emergent adverse events (TEAEs).

  From screening through EOS (Week 24)

Secondary Outcomes (2)

• Therapeutic potential of avoralstat in participants with DME. Trends in central subfield thickness (CST) as assessed by spectral domain optical coherence tomography (SD-OCT).

  From baseline through Weeks 12 and 24

• Therapeutic potential of avoralstat in participants with DME. Trends in Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA).

  From baseline through Weeks 12 and 24

Study Arms (1)

Single dose suprachoroidal injection of avoralstat

EXPERIMENTAL

Drug: avoralstat

Interventions

avoralstat DRUG

BCX4161 for suprachoroidal injection

Single dose suprachoroidal injection of avoralstat

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female aged 18 years or older
• Glycated hemoglobin A (HbA1c) \< 10% at screening
• Diagnosis of DME with center involvement requiring medical treatment within 6 months of screening and decreased visual acuity attributable to DME
• Mild-to-severe non-proliferative diabetic retinopathy (NPDR) with DRSS \< 61 in the study eye at screening
• BCVA between 35 and 80 ETDRS letters (20/25 to 20/200 Snellen equivalent), inclusive, in the study eye at screening
• CST measured by Heidelberg OCT between 325 and 600 μm in the study eye at screening

You may not qualify if:

• Participants who have previously received more than 3 anti-VEGF injections
• Any history of retinal surgery or other surgical intervention for diabetic ocular complications or anticipated need for use of laser photocoagulation course of any of the procedures listed in the criteria in the study eye during the study period
• Current medically untreated diabetes mellitus or previous medically untreated diabetes mellitus with initiation of oral or injectable anti-diabetic medication ≤ 3 months prior to screening
• Active intraocular or periocular infection or active intraocular inflammation in the study eye
• Intraocular surgery ≤ 3 months prior to screening or anticipated need for ocular surgery in the study eye during the study period

Sponsors & Collaborators

• BioCryst Pharmaceuticals: lead

Study Sites (8)

Investigative Site 6

Sacramento, California, 95825, United States

RECRUITING

Investigative Site 7

Katy, Texas, 77494, United States

RECRUITING

Investigative Site 8

The Woodlands, Texas, 77384, United States

RECRUITING

Investigative Site 1

Sydney, New South Wales, Australia

RECRUITING

Investigative Site 2

Sydney, New South Wales, Australia

RECRUITING

Investigative Site 3

Sydney, New South Wales, Australia

RECRUITING

Investigative Site 4

Adelaide, South Australia, Australia

RECRUITING

Investigative Site 5

Melbourne, Victoria, Australia

RECRUITING

MeSH Terms

Interventions

avoralstat

Central Study Contacts

BioCryst Pharmaceuticals, Inc.

CONTACT

+1 919 859 1302; clinicaltrials@biocryst.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 26, 2025
• First Posted: November 14, 2025
• Study Start: October 2, 2025
• Primary Completion: May 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: February 4, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

AU (5); US (3)