Clinical Trials

The UCSF Retinal Degenerations Clinic participated in 3 multi-center clinical trials for patients with inherited retinal degenerations. Two Phase II/III clinical trials evaluated the effect that a growth factor called ciliary neurotrophic factor (CNTF) has on retinal function in patients with early and late stages of RP. A Phase I study evaluated the safety and efficacy of a new generation epiretinal prosthetic device to stimulate visual perception in patients with end-stage RP. These clinical trials are among the first to study possible treatments for patients with these blinding diseases.

Research Studies

High-Resolution Imaging Research In Inherited Retinal Degenerations Study

The purpose of this study is to determine whether the structure and function of the human retina can be studied with high resolution in patients with inherited retinal degenerations using the Adaptive Optics Scanning Laser Ophthalmoscope (AOSLO).


Rate of Progression in USH2A-RElated Retinal Degeneration Study

The Rate of Progression in USH2A-Related Retinal Degeneration (RUSH2A) study is a 4-year, natural history study documenting the longitudinal effects of retinitis pigmentosa (RP) in people with mutations in the USH2A gene. With a better understanding of USH2A-related RP, we hope to accelerate the development of potential treatments, identify clear sub-populations for future treatment trials, and determine which types of tests are most sensitive and useful for monitoring disease progression. RUSH2A is currently enrolling patients with visual acuity of 20/80 or better in at least one eye.


Natural History of the Progression of X-Linked Retinitis Pigmentosa Study

The Natural History of the Progression of X-Linked Retinitis Pigmentosa (XOLARIS) study focuses on patients with retinitis pigmentosa (RP) inherited in an X-linked fashion (XLRP). This 2-year, observational study provides extremely valuable information on the clinical features, disease progression rates, and demographic and physiological characteristics of XLRP. The goal of this study is to ultimately facilitate the development of treatment trials for this rare and severe disease. We are currently enrolling patients for XOLARIS.