Cerulea Clinical Trials

Science and Research


A Phase 2, Double-Masked, Placebo-Controlled, Dose Range Finding Study of Danicopan (ALXN2040) in Patients with Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD).


Age-related Macular Degeneration (AMD) is a degenerative disease of the retina that causes a progressive, painless loss of central vision, affecting the ability to see fine detail, drive, read and recognize faces. There are typically two types of AMD: dry AMD and neovascular/wet AMD. Some patients with AMD will develop geographic atrophy (GA), where specific retinal cells waste away and die (atrophy). It is a disease that gradually worsens, with no approved therapy to slow or stop the process of continual retinal cell loss.

The ALXN2040-GA-201 trial is testing a new potential treatment for Geographic atrophy. The drug being investigated in this study is an oral medication called Danicopan, a complement factor D inhibitor. This is a phase 2 trial which aims to assess the efficacy of Danicopan in slowing the progression of geographic atrophy, while also testing the safety of the drug. As part of the study, suitable participants will be screened to determine their eligibility. For the first year, eligible participants will be assigned to Danicopan or placebo, and for the 2nd year participants will be assigned to a specified dosage of Danicopan. This study would require participants to attend CERA approximately every 2 months for the 2-year duration of the trial. For further information about this study please contact Cerulea on (03) 8638 5300 or email info@ceruleaclinicaltrials.org

  • Principal Investigator
Professor Robyn Guymer AM

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View this study at ClinicalTrials.gov

This database listing provides more detailed information about this study.