Clinical Trials // Inherited Retinal Degeneration // Kimberly Stepien // Ongoing // Sep 30 2020

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

Study objective:

This study will evaluate the efficacy and safety of a single sub-retinal injection of AAV2-REP1 in subjects with choroideremia (CHM).

Study design:

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