Cerulea Clinical Trials

Science and Research

Kyowa Kirin

A Phase 2, Multicenter, Randomized, Double-Masked, Parallel-Group Study to Assess the Efficacy and Safety of KHK4951, a Vascular Endothelial Growth Factor Receptor Inhibitor, in Patients with Diabetic Macular Edema


This study is investigating a new treatment for Diabetic Macular Edema (DME). The new treatment is an eye drop called KHK4951. DME is swelling in the macula, or central part of the retina (inner lining of the back of the eye that generates vision), and the main cause of vision impairment in diabetic patients. DME happens when high blood sugar levels affect the blood vessels in your eyes, making them weaker and more likely to leak fluid and/or blood. It may also cause new and more fragile blood vessels to grow where they shouldn’t grow. Leaking of fluid from blood vessels causes fluid build-up and thickens the retina which may result in loss of vision. Vascular endothelial growth factor (VEGF) is a protein that plays a significant role in this new blood vessel growth.
The Kyowa Kirin DME study is testing a new treatment called KHK4951, which is a topical eye drop formulation of tivozanib, a novel VEGFR inhibitor. KHK4951 is expected to stop the activation of the VEGF to its receptors (called anti VEGF treatment) and thus block new blood vessel formation. By slowing and preventing new blood vessels and the leaking fluid from these new vessels, this will help prevent the progression of diseases such as DME.

This study will assess the efficacy and safety of KHK4951 in the potential treatment for DME. KHK4951 is not approved for the treatment of DME [or any other condition] in Australia therefore it is an experimental treatment in this study. This study also uses aflibercept (eye injection) as the treatment before starting KHK4951 eye drop treatment, as well as rescue treatment, which means that aflibercept may be used to help your DME if KHK4951 is not helping your condition and if it is deemed necessary by your study doctor. Aflibercept is another anti-VEGF treatment approved by regulatory health agencies worldwide for the treatment of DME and other retinal eye diseases. As part of this study, suitable participants will be screened to determine their eligibility. If eligible, participants will be randomised to one of 3 treatment arms consisting of different dosages of KHK4951. The study runs for 12 months and would require participants to use the study eye drops daily.
For further information about this study please contact Cerulea on (03) 86385300 or email info@ceruleaclinicaltrials.org.au

  • Principal Investigator
Associate Professor Sanj Wickremasinghe

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View this study on ClinicalTrials.gov

See the ClinicalTrials.gov database listing for more detailed information about this study.