A phase 2 placebo-controlled clinical trial with ACU-4429 (visual cycle modulator) for the treatment of dry age related macular degeneration (AMD): effect on drusen volume, measured with an optical coherence tomography device and a newly developed algorithm.

• Applicant: Dr. Karen Schaal
• Subject Area: Ophthalmology
• Term: from 2013 to 2016
• Project identifier: Deutsche Forschungsgemeinschaft (DFG) - Project number 242255480

The intention of the presented research project is to find a treatment for non-exudative (dry) Age Related Macular Degeneration (AMD), which is currently not treatable, in contrast to the wet form of the disease. AMD is a chronic degenerative retinal disease and the leading cause of blindness in the elderly (over 50 years of age) of the industrialized world. Dry AMD affects approx. 90 % of all AMD-patients, who so far do not have a therapeutic option. The goal of my research project is to initiate a placebo-controlled phase 2 clinical trial with ACU-4429 (visual cycle modulator) as oral medication for the treatment of dry AMD and its effect on drusen growth as drusen are first signs of AMD. ACU-4429 is an isomerase-complex (RPE-65)-inhibitor, and a first phase 1 clinical trial on healthy subjects showed an effect on the accumulation of toxic end-products in the retinal pigment epithelial cell (RPE). ACU-4429 could therefore affect the development of drusen, and influence the course of the disease. Drusen volume is an important endpoint of the presented study and is measured with a newly developped algorithm incorporated in the Cirrus spectral domain optical coherence tomography device (SD-OCT). This algorithm generates a difference map between the real RPE of a patient and a virtuell RPE (free of any deformation), resulting in a drusen-volume map. Considering natural drusen dynamic, an effect of ACU-4429 on drusen growth (sign for disease progression) can be measured with this algorithm. The hypothesis of the project is to stop or at least to retard dry AMD progression with ACU-4429 medication.

• DFG Programme: Research Fellowships
• International Connection: USA
• Host: Professor Dr. Philip Rosenfeld