The Institut de la Màcula conducts a pioneering trial to halt the advance of Stargardt’s disease17/05/2018 · News
The Institut de la Màcula, directed by the ophthalmologist and investigator Jordi Monés MD, PhD, has begun a pioneering international trial that tests a new drug that helps to halt the advance of Stargardt’s disease (STGD). This serious genetic condition, which currently lacks a treatment or cure, is the most common macular dystrophy in children and adults.
This clinical trial, which is in phase 2b, aims to test the efficacy and safety of a drug called Zimura. It has been developed by Ophthotech Corporation, which specialises in the discovery and development of gene therapy solutions to treat ophthalmic diseases. There are very few lines of research into Stargardt’s disease and these are only conducted at international reference centres. Together with the Institut de la Màcula, prestigious centres from the USA, UK, France, Germany and Israel have been selected as collaborators in the trial.
The drug has been designed to regulate the overactivity of the complement system (a key component in innate immune responses). The inflammation caused by this overactivation has been linked to the development of retinal diseases as it can attack healthy cells and affect vision. Zimura, which is administered through intravitreal injections, blocks a complement protein known as C5. In laboratory studies, the inhibition of C5 prevented the degeneration of retinal cells.
Participation in this clinical trial is in accordance with rigorous protocols and selection criteria. For example, those affected should present two mutations of the ABCA4 gene. The duration of the trial, which began this week, is 18 months. The medical team of the Institut de la Màcula, which is located in the Teknon Medical Centre, will administer the drug and be responsible for the monitoring of all patients.
Here you have all the information: Clinical trial OPH2005: Efficacy and safety of Zimura in the treatment of Stargardt’s diseaseStargardt disease