A Randomized, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial Of Retinal Gene Therapy For Choroideremia Using An Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1) (STAR)
Sponsor: NightstaRx Ltd
Principal Investigator: Kimberly Stepien, MD
Study Coordinator: Nickie Stangel
This study will evaluate the efficacy and safety of a single sub-retinal injection of AAV2-REP1 in subjects with choroideremia (CHM).
This is an open label, outcomes-assessor masked, prospective, randomized, parallel controlled group, multi-center, global, interventional study. The study consists of 8 visits with a 12-month evaluation period.
Diagnosis and Key Eligibility Criteria:
Males ≥18 years of age with genetically-confirmed diagnosis of CHM, active disease clinically visible within the macular region in the study eye, and Snellen score of 20/200 to 20/40. Past investigational therapy is allowed with a 12 week washout period.
Duration of study: Approximately 3 years
For more information, contact Nickie Stangel 608-263-8783