Choroideremia
Conditions
Keywords
retinal degeneration, eye, REP-1 gene
Brief summary
The purpose of this study is to understand the rate of progression of all stages of choroideremia using a variety of assessments performed in the clinic including visual field measures, specialized photography of the eye and participant-reported visual problems
Interventions
OTHERObservation
Sponsors
4D Molecular Therapeutics
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
(Primary Cohort): * Healthy individuals (at least 14 years) with choroideremia (20/200 or better vision) willing to participate in an observational study and meeting the eligibility criteria. Inclusion Criteria (Expansion Cohort): * Healthy individuals (at least 18 years) with choroideremia (20/200 or better vision in both eyes) willing to participate in an observational study and meeting the eligibility criteria.
Exclusion criteria
(Primary and Expansion Cohort): 1. Prior therapy with an AAV vector-based treatment 2. Pre-existing eye conditions that would: (1) preclude future planned treatment in a therapeutic intent clinical trial (i.e. intravitreal injection), (2) interfere with the interpretation of study endpoints, and/ or put patient at risk for surgical complications 3. Complicating systemic diseases that would preclude future enrollment in a therapeutic intent clinical trial 4. Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Rate of progression of disease | 4 years | A variety of psychophysical, anatomical and image-based endpoints |
Countries
Canada, United States
Outcome results
None listed