Last updated on August 2018

Efficacy and Safety of AAV2-REP1 for the Treatment of Choroideremia

Brief description of study

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.

Detailed Study Description

This is a phase 3 clinical trial of a gene therapy vector made from adeno-associated virus (AAV) called AAV2-REP1 for the treatment of Choroideremia. Participating subjects will be required to attend a screening visit during which their suitability for the study will be assessed, and eligible subjects will be randomized to either AAV2-REP1 treatment or the control group.

Clinical Study Identifier: NCT03496012

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Study Site

Miami, FL United States
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Recruitment Status: Open

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