Phase 2
N=6
Choroideremia Gene Therapy Clinical Trial
Choroideremia
Bottom Line
View on ClinicalTrials.gov: NCT02553135 ↗Enrolled (actual)
6
Serious AEs
0.0%
Results posted
Jul 2019
Primary outcome: Primary: Change in Best Corrected Visual Acuity From Baseline — 3.0 Letters
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Injection of AAV2-REP1 (10e11 vg) (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Byron Lam
- Primary completion
- Feb 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Best Corrected Visual Acuity From Baseline |
3.0 | — |
| SECONDARY Change in Retinal Macular Autofluorescence From Baseline |
-3.4; 2.8 | — |
| SECONDARY Changes in Microperimetry From Baseline |
0.823 | — |
| SECONDARY Number of Participants Who Experience an Adverse Event |
6 | — |
Summary
Phase II gene therapy study, involving a total of 6 male patients with choroideremia. The study will be conducted at the Bascom Palmer Eye Institute, University of Miami. Patients will be required to attend a total of 11 study visits over a 24 month period with an additional 3 year follow-up.
Eligibility Criteria
Inclusion Criteria
- 18 Years and older
- Male
- Able to give informed consent
- Genetically confirmed diagnosis of choroideremia
- Active disease visible clinically within the macula region
- Best-corrected visual acuity equal to or worse than 20/32 but better than or equal to 20/200 in the study eye.
Exclusion Criteria
- Female
- Under the age of 18
- History of amblyopia in the study eye
- Men unwilling to use barrier contraception methods
- Relevant grossly asymmetrical disease or other ocular morbidity which might confound use of the fellow eye as a long-term control
- Any other significant ocular and non-ocular disease/disorder or retinal surgery
- Contraindication to use of medications or contrast agents
- Participated in research study involving an investigational product in the past 12 weeks
- Having had gene or cellular therapy at any time prior to this study.
Data sourced from ClinicalTrials.gov (NCT02553135). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.