VX-01 for Diabetic Retinopathy

Recruiting at 30 trial locations

Overseen By Steffy George

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Vantage Biosciences Ltd

Must not be taking: Anticoagulants, Steroids, GLP-1 modulators

Disqualifiers: Uncontrolled diabetes, Hypertension, Glaucoma, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

100 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, you cannot participate if you are currently using certain medications like coumarin anticoagulants (e.g., Warfarin) or strong inhibitors of the P-glycoprotein transporter. It's best to discuss your specific medications with the study team.

What data supports the effectiveness of the drug VX-01 for Diabetic Retinopathy?

Research shows that dexamethasone, a component of VX-01, is effective in treating diabetic macular edema, which is related to diabetic retinopathy. Studies have found that dexamethasone implants can improve vision and reduce swelling in the eye for patients with similar conditions. ^{1 2 3 4 5}

What safety data exists for VX-01 (dexamethasone intravitreal implant) in humans?

The dexamethasone intravitreal implant, also known as Ozurdex, has been studied for safety in conditions like macular edema and age-related macular degeneration. Some reported side effects include increased eye pressure and cataract formation, but it is generally considered safe for use in these eye conditions. ^{1 6 7 8 9}

What is the purpose of this trial?

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.

Eligibility Criteria

This trial is for adults over 18 with a BMI of 18-40 kg/m2 who have Type 1 or Type 2 Diabetes Mellitus and moderate to severe Non-Proliferative Diabetic Retinopathy. Participants must not be pregnant, breastfeeding, and agree to use contraception if applicable. They should have clear eyes for imaging and no recent surgeries affecting reproductive status.

Inclusion Criteria

I have signed a consent form for this study.

I am over 18 years old.

I have been diagnosed with type 1 or type 2 diabetes.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive daily oral doses of VX-01 or placebo for 52 weeks

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

Treatment Details

Interventions

• VX-01

Trial Overview The study tests the effectiveness and safety of VX-01, an oral medication taken daily for one year against a placebo in treating Diabetic Retinopathy. It will also look at how the body processes the drug (pharmacokinetics) and its effects on the disease (pharmacodynamics).

Participant Groups

Group I: VX-01 Experimental Treatment 1 Intervention

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.

Group II: Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Vantage Biosciences Ltd

Lead Sponsor

Trials

1

Recruited

100+

Vantage Biosciences Australia Pty Ltd

Industry Sponsor

Trials

1

Recruited

100+

Findings from Research

In a study of seven patients with refractory wet age-related macular degeneration (AMD), the dexamethasone intravitreal implant (DXI) effectively reduced intra/sub-retinal fluid (IRF/SRF) in 71.4% of eyes within 6 weeks, indicating its potential as an adjunctive therapy.

While visual acuity remained stable after DXI treatment, there was a significant reduction in central retinal thickness (CRT) over time, suggesting that DXI can help improve anatomical outcomes in patients who have not responded adequately to other treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dexamethasone intravitreal implant as adjunct therapy for patients with wet age-related macular degeneration with incomplete response to ranibizumab. Calvo, P., Ferreras, A., Al Adel, F., et al.[2022]

In a pilot study involving 16 eyes of 15 patients with wet age-related macular degeneration resistant to anti-VEGF therapy, the combination of a dexamethasone implant and anti-VEGF treatment led to a significantly higher rate of complete retinal fluid resorption (72.7% vs. 40% in the control group).

While the combination treatment improved retinal fluid resorption, it did not result in significant changes in best corrected visual acuity compared to the control group, indicating that while the treatment may help with fluid management, it does not necessarily enhance visual function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravitreal Dexamethasone in Patients with Wet Age-Related Macular Degeneration Resistant to Anti-VEGF: A Prospective Pilot Study. Giancipoli, E., Pinna, A., Boscia, F., et al.[2022]

In a study of 44 eyes with diabetic macular edema (DME) unresponsive to ranibizumab, treatment with intravitreal dexamethasone implants significantly improved best-corrected visual acuity (BCVA) and reduced central macula thickness (CMT) at 1 and 3 months.

Patients with subfoveal neuroretinal detachment (SND) and hyperreflective retinal spots (HRS) showed a more pronounced response to dexamethasone, indicating that these inflammatory features may help predict which patients will benefit most from this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dexamethasone for unresponsive diabetic macular oedema: optical coherence tomography biomarkers. Bonfiglio, V., Reibaldi, M., Pizzo, A., et al.[2019]

In a study of 15 patients with non-infectious posterior segment uveitis, the dexamethasone intravitreal implant (Ozurdex) significantly reduced central retinal thickness (CRT) and improved best-corrected visual acuity (BCVA) after the first treatment, with 91.4% of eyes showing CRT reduction and 80% showing BCVA improvement.

The effectiveness of the DEX implant was maintained with repeat use, showing similar improvements in CRT and VA, indicating it can be a valuable adjunct treatment alongside other therapies for managing macular edema.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravitreal Dexamethasone Implant for the Treatment of Macular Edema in Chronic Non-infectious Uveitis. Tsang, AC., Virgili, G., Abtahi, M., et al.[2018]

A single dexamethasone intravitreal implant significantly reduced central macular thickness (CMT) in patients with diabetic macular edema (DME), with a notable decrease observed 2 months after injection.

While visual acuity improved for many patients, with 30% gaining more than 2 logMAR lines, a recurrence of macular edema occurred in 79% of eyes by 4 months, indicating the need for ongoing monitoring and potential additional treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of dexamethasone intravitreal implant for the treatment of diabetic macular edema. Bonnin, S., Dupas, B., Sanharawi, ME., et al.[2018]

In a study of 61 eyes treated with intravitreal dexamethasone implants for macular edema, 12% experienced elevated intraocular pressure (IOP) above 25 mmHg, particularly in patients with central retinal vein occlusion and glaucoma, indicating a need for careful monitoring in these subgroups.

The incidence of cataract surgery was notably high at 24% among treated phakic eyes, with the likelihood increasing with the number of implants, suggesting that repeated treatments may lead to a higher risk of cataract formation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

OBSERVED COMPLICATIONS FROM DEXAMETHASONE INTRAVITREAL IMPLANT FOR THE TREATMENT OF MACULAR EDEMA IN RETINAL VEIN OCCLUSION OVER 3 TREATMENT ROUNDS. Reid, GA., Sahota, DS., Sarhan, M.[2015]

In a pilot study involving 10 patients with neovascular age-related macular degeneration, the combination of dexamethasone intravitreal implant (DXI) and ranibizumab did not show significant improvements in visual acuity or central macular thickness compared to ranibizumab alone after 9 months.

One patient experienced elevated intraocular pressure after receiving the DXI, indicating a potential adverse effect consistent with known risks of dexamethasone treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ozurdex in age-related macular degeneration as adjunct to ranibizumab (The OARA Study). Chaudhary, V., Barbosa, J., Lam, WC., et al.[2022]

In a study of 50 patients with diabetic macular edema, both ranibizumab (RZB) and dexamethasone implant (DEX) treatments significantly improved visual acuity and central macular thickness over 6 months, with DEX showing a greater improvement in visual acuity.

The DEX group had a lower retreatment rate compared to the RZB group, although two patients in the DEX group experienced increased intraocular pressure, indicating a potential safety concern.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Anatomical and functional changes after dexamethasone implant and ranibizumab in diabetic macular edema: a retrospective cohort study. Mastropasqua, L., Di Staso, S., D'Aloisio, R., et al.[2020]

In a study comparing 37 treatment-naive diabetic macular edema patients, the intravitreal dexamethasone implant (IVDEX) showed significantly greater improvement in best-corrected visual acuity (BCVA) compared to intravitreal ranibizumab (IVR) at 2 and 3 months after treatment.

Both treatments reduced hyperreflective spots (HRSs) in the retina, but IVDEX was more effective in decreasing the total number of HRSs, particularly in the inner retinal layers, suggesting it may be a better option for patients with an inflammatory component in their condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evaluation of retinal inflammatory biomarkers after intravitreal steroid implant and Ranibizumab injection in diabetic macular edema. Muftuoglu, IK., Tokuc, EO., Sümer, F., et al.[2022]

References

1. England pubmed.ncbi.nlm.nih.gov

Dexamethasone intravitreal implant as adjunct therapy for patients with wet age-related macular degeneration with incomplete response to ranibizumab. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Intravitreal Dexamethasone in Patients with Wet Age-Related Macular Degeneration Resistant to Anti-VEGF: A Prospective Pilot Study. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Dexamethasone for unresponsive diabetic macular oedema: optical coherence tomography biomarkers. [2019]

4. England pubmed.ncbi.nlm.nih.gov

Intravitreal Dexamethasone Implant for the Treatment of Macular Edema in Chronic Non-infectious Uveitis. [2018]

5. United States pubmed.ncbi.nlm.nih.gov

Efficacy of dexamethasone intravitreal implant for the treatment of diabetic macular edema. [2018]

6. United States pubmed.ncbi.nlm.nih.gov

OBSERVED COMPLICATIONS FROM DEXAMETHASONE INTRAVITREAL IMPLANT FOR THE TREATMENT OF MACULAR EDEMA IN RETINAL VEIN OCCLUSION OVER 3 TREATMENT ROUNDS. [2015]

7. England pubmed.ncbi.nlm.nih.gov

Ozurdex in age-related macular degeneration as adjunct to ranibizumab (The OARA Study). [2022]

8. China pubmed.ncbi.nlm.nih.gov

Anatomical and functional changes after dexamethasone implant and ranibizumab in diabetic macular edema: a retrospective cohort study. [2020]

9. United States pubmed.ncbi.nlm.nih.gov

Evaluation of retinal inflammatory biomarkers after intravitreal steroid implant and Ranibizumab injection in diabetic macular edema. [2022]