Efficacy and Safety of AAV2-REP1 for the Treatment of Choroideremia
The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of AAV2-REP1 in subjects with Choroideremia.
Study Start Date
December, 11 2017
Estimated Completion Date
- Genetic: AAV2-REP1
Nightstar Therapeutics -- NSR-REP-01
- Are willing and able to give informed consent for participation in the study.
- Are male and ?18 years of age.
- Have a genetically-confirmed diagnosis of CHM.
- Have active disease clinically visible within the macular region in the study eye.
- Fulfill pre-defined visual acuity criteria.
- Have a history of amblyopia in the eligible eye.
- Are unwilling to use barrier contraception methods, for a period of 3 months, if treated with AAV2-REP1.
- Previous intraocular surgery performed in the study eye within 3 months of Visit 1.
- Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may either put the subjects at risk because of participation in the study, or may influence the results of the study, or the subject's ability to participate in the study.
- 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.
18 Years and older
Accepts Healthy Volunteers
Study Locations and Contact Information (7)
|Columbia University Medical Center Edward S Harkness Eye Inst - New York, New York||181.2 miles||None||None||None|
|The Johns Hopkins Hospital - Baltimore, Maryland||356.0 miles||None||None||None|
|University of Wisconsin - Madison, Wisconsin||933.6 miles||None||None||None|
|Bascom Palmer Eye Institute - Miami, Florida||1,259.8 miles||None||None||None|
|Retina Foundation of the Southwest - Dallas, Texas||1,545.0 miles||None||None||None|
|Oregon Health and Science University - Portland, Oregon||2,535.1 miles||None||None||None|
|Jules Stein Eye Institute University of California - Los Angeles, California||2,605.2 miles||None||None||None|