STAR trial (phase 3 gene therapy)

Purpose of the Study

The purpose of this study is to find out if vision in patients suffering from choroideremia can be preserved by replacing the defective gene using a gene therapy called Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein (REP1) or AAV2-REP1 in subjects with CHM and to evaluate the efficacy and safety of a single sub-retinal injection of AAV2-REP1.

The image comparison above shows the results from microperimetry superimposed on the patient’s retina .

Who will participate in this Study?

The cohort of patients will have been involved in the Natural History of Choroideremia study (NIGHT) first and eligible patients will be enrolled onto the STAR trial. It is planned that approximately 100 male subjects (50 subjects in the AAV2-REP1 group and 50 in the Control group) across approximately 15 hospitals/centres (sites) will participate in the study in Europe and North America. The expected participation in this study is 12 months in total and patients will be asked to visit the site six to nine times over this period.

This trial has closed to recruitment.