Natural History of the Progression of Choroideremia Study
Multicenter Observational Study of Choroideremia
Sponsor: Night Therapeutics
Principal Investigator: Kim Stepien, MD
Study Coordinator: Nickie Stangel
Study Objective:
To assess the rate of progression of choroideremia over 20 months utilizing both functional and anatomical outcome measures.
Study Design:
This is a prospective, observational multicenter study. There will be three cohorts to this study, mild, moderate, and severe. Subjects will need to have best-corrected visual acuity equal to or better than 20/200. The study consist of up to six visits over a 20-month study period with two optional study visits.
Diagnosis and Key Eligibility Criteria:
Are male and over the age of 18. Have a clinical phenotype and confirmed genetic diagnosis of Choroideremia. Have active disease clinically visible within the macular region.
Duration of Study: 20 months
For more information, contact Nickie Stangel 608-263-8783.