NCT06770933

A Study to Evaluate the Efficacy and Safety of Orally Administered VX-01

A Phase II, Double-Masked, Randomised, Placebo-Controlled, Parallel Design Study to Evaluate the Efficacy and Safety of Orally Administered VX-01 in Diabetic Retinopathy OF Non-Proliferative Type (NPDR)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 100 (estimated)
• Sponsor: Vantage Biosciences Ltd · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to evaluate the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of VX-01 as stand-alone treatment for Diabetic Retinopathy of Non-Proliferative Type (NPDR). The primary objective of the study is to evaluate the efficacy of daily oral doses of VX-01 versus placebo following 52 weeks of treatment.

Detailed description

This is a Phase 2, multi-center, double-masked, randomized, placebo-controlled, parallel group study to evaluate the efficacy of oral doses of VX-01 in subjects with moderate to severe NPDR, without CI-DME. Approximately 100 male and female subjects aged ≥ 18 years with a documented diagnosis of Type 1 Diabetic Mellitus or Type 2 Diabetic Mellitus with moderate to severe NPDR (without CI-DME) will be enrolled, if they meet all the eligibility criteria for the study. Subjects will be randomized 1:1 to 1 of 2 study cohorts: * Cohort 1 (n = 50): VX-01 (film-coated tablets, 150 mg administered BID) * Cohort 2 (n = 50): Placebo (film-coated tablets, administered BID) Subjects will be stratified by the presence or absence of proliferative diabetic retinopathy (PDR) and by glycated hemoglobin (HbA1c) of ≥ 8.5% or \< 8.5% at Screening. All subjects will take 1 tablet of VX-01 or placebo BID for 52 consecutive weeks. All subjects will be followed for 12 weeks after completion of treatment at Week 52. The Sponsor, study site staff, monitors, personnel, and subjects will be masked to treatment assignment during the entirety of the study.

Conditions

• Diabetic Retinopathy
• NPDR - Non Proliferative Diabetic Retinopathy

Interventions

Type	Name	Description
DRUG	VX-01	There is no physical difference in VX-01 and the placebo. The only difference lies in active ingredient found in VX-01, which is the compound that will be evaluated in the course of this study.
DRUG	Placebo	Placebo will be supplied as a tablet identical to test drug but without VX-01. Placebo packaging will be identical to IP in order to keep study personnel and subjects masked.

Timeline

• Start date: 2025-02-11
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2025-01-13
• Last updated: 2025-06-18

Locations

26 sites across 5 countries: United States, Australia, Hong Kong, Malaysia, South Korea

Regulatory

• FDA-regulated drug study