drugset / Trial / NCT02407678
REP1 Gene Replacement Therapy for Choroideremia
Phase 2
Completed
30 enrolled
University of Oxford
Moorfields Eye Hospital NHS Foundation Trust · collabUniversity College, London · collab
RandomizedParallel-groupOpen-labelTreatment
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.
Timeline
- Start
- 2016-08-16
- Primary completion
- 2021-07-23
- Completion
- 2021-07-23
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAV2-REP1 | Gene therapy (AAV / viral vector) | — | Other |