Choroideremia // 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, a Biogen Company
  • Principal Investigator: Kimberly Stepien, MD
  • Study Coordinator: Nickie Stangel

Study objective:

To evaluate the efficacy and safety of a single sub-retinal injection of AAV2-REP1 in subjects with Choroideremia.

Study design:

Subjects will be randomized to receive either AAV2-REP1 treatment (high or low dose) or the control group. There will be 5-8 study visits over approximately 14 months.

Inclusion criteria:

  • Are male and ≥18 years of age
  • Have a documented genetically confirmed diagnosis of CHM
  • Have active disease clinically visible within the macular region in the study eye
  • Fulfill pre-defined visual acuity criteria

Exclusion criteria:

  • Have a history of amblyopia in the eligible eye
  • Are unwilling to use barrier contraception methods, or abstain from sexual intercourse, for a period of 3 months, if treated with AAV2-REP1
  • Have had previous intraocular surgery performed in the study eye within 3 months of Visit 1
  • Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously

Contact Nickie Stangel with questions: 608-263-8783. For more information about this study: visit