Natural History of the Progression of Choroideremia Study

Active Clinical Trials // Feb 12 2018

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.