Last updated on August 2018

A Safety Study of Retinal Gene Therapy for Choroideremia

Brief description of study

This is a multi-centre, open-label, prospective, two-period, interventional safety study of bilateral use of AAV2-REP1 in adult male subjects with genetically confirmed CHM.

Detailed Study Description

This is a phase 2 clinical trial of a gene therapy vector made from adenoassociated 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 undergo a surgical procedure under general anaesthesia consisting of vitrectomy, retinal detachment and sub-retinal administration of AAV2-REP1.

Subjects will be treated bilaterally, with the interval between eyes determined by discussion between the investigator and Nightstar. Participating subjects will be required to attend follow up visits for 12 months following the second eye surgery.

Clinical Study Identifier: NCT03507686

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Study Site

Miami, FL United States
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