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

NCT06770933 | Recruiting Phase 2 FDA Drug

• 1 other identifier: VX01-DR-201
• Study Type: interventional
• Target: 100
• Locations: 5 countries
• Sites: 26

Brief 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.

Conditions

Diabetic Retinopathy; NPDR - Non Proliferative Diabetic Retinopathy

Keywords

NPDR; Diabetic Retinopathy; AOC3; VAP-1; SSAO; VAP1; Amine Oxidase Copper containing 3; Amine Oxidase; HPAO

Outcome Measures

Primary Outcomes (1)

• Evaluate the efficacy of oral doses of VX-01 in subjects compared to placebo following 1 year of treatment.

  The endpoint of this objective is the proportion of subjects who do not develop a worsening from Baseline in binocular ETDRS DRSS at Week 52. The diabetic retinopathy severity scale (DRSS) is a scale healthcare professionals use to measure the severity and progression of a person's diabetic retinopathy. The main DRSS is the Early Treatment Diabetic Retinopathy Study (ETDRS) scale which will be used in this study.

  From enrollment to the end of treatment at week 52

Secondary Outcomes (4)

• To evaluate the efficacy of VX-01 in subjects with moderate to severe NPDR without CI-DME by determining the overall change from Baseline in BCVA letter scores.

  From enrollment to the end of treatment at week 52

• To evaluate the efficacy of VX-01 in subjects with moderate to severe NPDR without CI-DME by determining the overall change from Baseline in the ETDRS DRSS scores.

  From enrollment to the end of treatment at week 52

• Incidence of Treatment-Emergent Adverse Events (TEAEs) when taking multiple oral doses of VX-01.

  From enrollment to the end of treatment at week 52

• Number of subjects developing moderate to high risk proliferative diabetic retinopathy

  From enrollment to the end of treatment at week 52

Study Arms (2)

VX-01

EXPERIMENTAL

Cohort 1 will include 50 subjects who will be randomized to take investigational drug VX-01 (film-coated tablets) at dose of 150 mg, administered BID.

Drug: VX-01

Placebo

PLACEBO COMPARATOR

Cohort 2 will include 50 subjects who will be randomized to receive the placebo drug (film-coated tablets), that will be administered BID.

Drug: Placebo

Interventions

VX-01 DRUG

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.

VX-01

Placebo DRUG

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.

Placebo

Eligibility Criteria

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

You may qualify if:

• Written informed consent must be obtained from the subject prior to any study-related procedures.
• Subject must be aged \> 18 years at the time of Screening.
• Subject must have a body mass index (BMI) of between 18 and 40 kg/m2, inclusive.
• Subject has a documented diagnosis of T1DM or T2DM.
• Subject has moderate to severe NPDR, as determined by a Central Reading Centre (CRC) using DRSS in at least one eye
• Subject must have clear ocular media and be able to undergo adequate pupil dilation to allow adequate fundus imaging of both eyes.
• Female subject must be either:
• Of non-childbearing potential: post-menopausal or documented surgically sterile post hysterectomy (at least 1 month prior to Screening)
• Or, if of childbearing potential, must have a negative serum pregnancy test at Screening and must use 2 acceptable forms of contraception, starting at Screening and throughout the study period and for 28 days after the final IP administration.
• Female subject must not be breastfeeding at Screening or during the study period, and for 28 days after the final IP administration.
• Male subject must be surgically sterile (\> 30 days since vasectomy with no viable sperm), or if engaged in sexual relations with a female of childbearing potential, the couple should agree to use 2 acceptable contraceptive methods from Screening, during the study, and for 28 days after last IP administration.
• Female subject must not donate ova or male subject must not donate sperm starting at Screening and throughout the study period, and for 28 days after the final IP administration.
• Subject must have Best Corrected Visual Acuity (BCVA) assessed by Early Treatment Diabetic Retinopathy Study (ETDRS) protocol letters score of ≥ 70 letters in study eye, and ≥ 20 letters in the non-qualified fellow eye.
• Subject must have the ability, in the opinion of the Investigator, and willingness to return for all scheduled visits and perform all assessments.
• Subject agrees not to participate in another interventional study after signing the informed consent and until the End of Study (EOS) visit has been completed.

You may not qualify if:

• Ophthalmic:
• Presence of CI-DME (with central subfield thickness \[CST\] measured greater than 325 μm on spectral domain optical coherence tomography \[SD-OCT\]) threatening the center of the macula (within 1,000 μm of the foveal center) in either eye, or presence of DME requiring treatment.
• Presence of moderate to high-risk PDR (DRSS level 65 or higher).
• Any prior treatment (in either eye) with:
• Focal or grid laser photocoagulation within the past 6 months prior to Screening or pan-retinal photocoagulation (PRP) at any time.
• Systemic or intravitreal anti-vascular endothelial growth factor (VEGF) agents within the last 12 months prior to Screening.
• Intraocular, sub-tenon or periocular steroids, including triamcinolone and dexamethasone implant within the last 6 months, or suprachoroidal triamcinolone within the last 3 months prior to Screening.
• Fluocinolone implant within the last 3 years prior to Screening.
• Prior treatment for NPDR with any other treatment which is not labelled for NPDR within 1 year prior to Screening (e.g., calcium dobesilate, fibrate medication).
• Vitrectomy at any timepoint prior to Screening.
• Yttrium-Aluminium-Granate (YAG) capsulotomy within 3 months prior to Screening.
• Active uveitis, vitritis, or infection in either eye including infectious conjunctivitis, keratitis, scleritis, or endophthalmitis.
• History of corneal transplant and/or vitrectomy or any other ocular incisional surgery in either eye (e.g., shunt surgery). Note: Subjects who have had cataract or refractive surgery in either that was more than 3 months prior to Screening may be permitted at the discretion of the Investigator.
• Uncontrolled glaucoma, as evidenced by intraocular pressure (IOP) \> 25 mmHg despite up to 4 glaucoma medications, or evidence of glaucomatous visual field loss or has advanced glaucoma (e.g., prior shunt surgery) in either eye.
• Clinically significant ocular disease in either eye that in the opinion of the Investigator would preclude participation in the study.

Sponsors & Collaborators

• Vantage Biosciences Ltd: lead
• Vantage Biosciences Australia Pty Ltd: collaborator

Study Sites (26)

Retina-Vitreous Associates Medical Group

Beverly Hills, California, 90211, United States

NOT YET RECRUITING

Stanford Byers Eye Institute

Palo Alto, California, 94303, United States

NOT YET RECRUITING

California Retina Consultants- Santa Barbara

Santa Barbara, California, 93103, United States

NOT YET RECRUITING

Florida Retina Institute - Jacksonville Southside

Jacksonville, Florida, 32216, United States

NOT YET RECRUITING

Retina Associates

Elmhurst, Illinois, 60126, United States

NOT YET RECRUITING

Cumberland Valley Retina Consultants

Hagerstown, Maryland, 21740-5940, United States

NOT YET RECRUITING

Erie Retina Research

Erie, Pennsylvania, 16507, United States

NOT YET RECRUITING

Piedmont Eye Center

Lynchburg, Virginia, 24502, United States

NOT YET RECRUITING

Eye Clinic Albury Wodonga

Albury, New South Wales, 2640, Australia

RECRUITING

Retina And Eye Consultants Hurstville

Hurstville, New South Wales, 2220, Australia

RECRUITING

Marsden Eye Specialists

Parramatta, New South Wales, 2150, Australia

RECRUITING

Sydney Eye Hospital

Sydney, New South Wales, 2000, Australia

RECRUITING

Sydney Retina Clinic

Sydney, New South Wales, 2000, Australia

RECRUITING

Sydney West Retina

Westmead, New South Wales, 2145, Australia

RECRUITING

University of the Sunshine Coast Clinical Trials (Birtinya)

Birtinya, Queensland, 4575, Australia

RECRUITING

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

RECRUITING

Prince of Wales Hospital The Chinese University of Hong Kong

Shatin, Hong Kong

RECRUITING

HKU Eye Centre

Wong Chuk Hang, Hong Kong

NOT YET RECRUITING

University Malaya Medical Centre

Petaling Jaya, Kuala Lumpur Federal Territory of Kuala Lumpur, 59100, Malaysia

NOT YET RECRUITING

Hospital Pulau Pinang

George Town, Pulau Pinang, 10450, Malaysia

NOT YET RECRUITING

Hospital Shah Alam

Shah Alam, Selangor, 40000, Malaysia

NOT YET RECRUITING

Hospital Al-sultan Abdullah Uitm

Sungai Buloh, Selangor, 47000, Malaysia

NOT YET RECRUITING

Hospital Selayang

Selayang Baru Utara, Malaysia

NOT YET RECRUITING

Seoul National University Bundang Hospital

Seongnam-si, South Korea

NOT YET RECRUITING

Asan Medical Center

Seoul, South Korea

NOT YET RECRUITING

Samsung Medical Center

Seoul, South Korea

NOT YET RECRUITING

MeSH Terms

Conditions

Diabetic Retinopathy

Condition Hierarchy (Ancestors)

Retinal Diseases; Eye Diseases; Diabetic Angiopathies; Vascular Diseases; Cardiovascular Diseases; Diabetes Complications; Diabetes Mellitus; Endocrine System Diseases

Central Study Contacts

Steffy George

CONTACT

+44 7450953382; steffy.george@vantage-biosciences.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a multi-center, double-masked, randomized, placebo-controlled, parallel group study, where subjects will be randomized 1:1 to 1 of 2 study cohort of VX-01 and placebo.

Study Record Dates

• First Submitted: December 16, 2024
• First Posted: January 13, 2025
• Study Start: February 11, 2025
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: June 18, 2025

Record last verified: 2025-06

Locations

US (8); KR (3); AU (8); MY (5); HK (2)