- recruitingTherapeutic confirmatory (Phase III)
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study (SOLSTICE)
This study is a long-term follow-up for men who have already received gene therapy for rare inherited eye conditions (choroideremia or X-linked retinitis pigmentosa). Researchers want to track their eye health and sight over several years to ensure the treatment remains safe and effective.
France · Germany - recruiting
InsightRP2 Registry
The InsightRP2 Registry is an online database for people with a rare eye condition called RP2-associated Retinitis Pigmentosa. It aims to gather information to help scientists understand the disease better and develop new treatments, including gene therapy, to protect eyesight.
Germany