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

March 2020


  • Genetic: AAV2-REP1

Study ID

Nightstar Therapeutics -- NSR-REP-01



Trial ID


Study Type


Trial Phase

Phase 3

Enrollment Quota



Nightstar Therapeutics

Inclusion Criteria

  • 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.

Exclusion 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)

Study Location Distance Name Phone Email
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

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