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REP1 Gene Replacement Therapy for Choroideremia — REGENERATE  

Choroideremia Clinical Trial

Official title:
An Open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)

Clinical Trial Summary

The assessment of the efficacy (with respect to preservation of visual function and retinal structure) and safety of a single subretinal injection of AAV2.REP1 in participants with a confirmed diagnosis of choroideremia, as evaluated by various functional and anatomical outcomes measured over a number of time points up to 24 months post-treatment.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02407678
Study type Interventional
Source University of Oxford
Contact
Status Completed
Phase Phase 2
Start date August 16, 2016
Completion date July 23, 2021
View Details
See also
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Active, not recruiting NCT00427180 - IRIS PILOT - Extended Pilot Study With a Retinal Implant System N/A