Trials / Completed
CompletedNCT02407678
REP1 Gene Replacement Therapy for Choroideremia
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)
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 30 (estimated)
- Sponsor
- University of Oxford · Academic / Other
- Sex
- Male
- Age
- 18 Years – 90 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | AAV-mediated REP1 gene replacement | AAV vector carrying human REP1 gene is delivered into the treated eye by subretinal injection |
Timeline
- Start date
- 2016-08-16
- Primary completion
- 2021-07-23
- Completion
- 2021-07-23
- First posted
- 2015-04-03
- Last updated
- 2021-08-05
Locations
2 sites across 1 country: United Kingdom
Source: ClinicalTrials.gov record NCT02407678. Inclusion in this directory is not an endorsement.