Canadian company QLT has completed enrolment in its natural history study for inherited retinal diseases caused by mutations in the RPE65 and LRAT genes. This includes some forms of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). The aim of the retrospective study is to gather data on the natural progression of the diseases in people over time, including from childhood through to adulthood.
The study included both participants who had previously received treatment in QLT-sponsored clinical trials with QLT’s synthetic oral retinoid product, QLT091001, as well as participants who had not received any prior therapeutic treatment. The intention of this study was to better observe and understand the long term natural disease progression of untreated patients, and to provide comparative data to further assess the extent to which treatment with QLT091001 may prolong or improve visual function relative to the underlying natural disease progression. The company expects the study to provide important data to support the on-going development programme for its potential therapy QLT091001. In total, 59 participants across 10 clinical study sites in Europe, the U.S. and Canada were enrolled in the study, 25 of whom had participated in previous clinical studies with QLT091001, and 34 of whom had received no previous treatment. Results from this study are expected in the near future.