A single-center, placebo-controlled dose-escalating and repeated-dosing study of the safety, tolerability, and pharmacokinetics of orally-administered ACU-3223 in healthy adults of 55 years of age or older. This is the first ever human trial for a class of compounds known as Visual Cycle Modulators (VCMs). These compounds reduce the activity of the rod (nighttime) visual system by competing for key enzymes in the visual cycle. The goal is to “slow down” the rods, reducing the metabolic load on the cones, and, hopefully, slowing down or preventing age-related macular degeneration (AMD). The general approach is based on the work of Travis, Birch and Palczewski, who showed that inhibiting the rod visual cycle protects the retina in the recessive Stargardt mouse model.

A phase II study of implants of encapsulated human NTC-201 cells releasing ciliary neurotrophic factor (CNTF) in participants with geographic atrophy due to macular degeneration using visual acuity as the primary outcome. CNTF is a potent protective factor for retinal neurons, but until recently, there has been no way to provide slow steady release to the human retina. The drug cannot be taken orally because it is too big to cross the blood-retinal barrier. We are using a sustained-release capsule containing human retinal epithelial cells which have been genetically-transfected to produce CNTF. This study is attempting to determine whether sustained CNTF for 1 year prevents progression in ayes with geographic atrophy due to AMD.

Electroretinographic measurements in patients with age-related macular degeneration initiating treatment with vascular endothelial growth factor inhibitors. This study is designed to help determine why some patients benefit from the new anti-VEGF inhibitors (Avastin, Macugen and Lucentis), while some do not. We are looking at multiple aspects of macular physiology before and during treatment.

Hereditary Retinal Degenerations

A Phase II Clinical Trial to investigate the effectiveness and safety of high dose docosahexaenoic acid (DHA) in early-stage X-linked retinitis pigmentosa. This 4-year prospective, randomized, placebo-controlled trial has enrolled 60 male patients (age 7-32 y) from across the U.S. and Canada. Patient enrollment is expected to close in March, 2008. The study is designed to test whether the nutritional intervention will slow the loss of visual function associated with this degenerative disease and thus, provide an extended period of usable vision. This trial is a collaborative venture between Retina Foundation researchers (Hoffman, Birch, & Wheaton) and retinal specialists (Fish & Spencer) at Texas Retina Associates. The project is funded, in part by the U.S. Food and Drug Administration, Martek Biosciences, and the Foundation Fighting Blindness. A detailed description of the trial is posted on the U.S. government’s ClinicalTrials.gov website at http://clinicaltrials.gov/ct/show/NCT00100230?order=2.

Argus™ II retinal implant system by Second Sight feasibility protocol attempting to provide visual stimulation by implanting an artificial chip in patients with end stage retinitis pigmentosa. Artificial retinas have evolved to the point where it is time to carefully assess the benefits that they can provide to blind patients. The Aggus system involves 60 electrofes placed on the retinal surface to “replace” degenerate photoreceptors. We are working extensively with a small number of blind patients to optimize the stimulation for each individual.

A phase II study of implants of encapsulated human NTC-201 cells releasing ciliary neurotrophic factor (CNTF) in participants with retinitis pigmentosa using visual acuity as the primary outcome. As a potent protective agent, CNTF has been shown to prevent retinal degeneration in animal models of retinitis pigmentosa. In this trial, we are attempting to improve ventral vision in patients with fairly advanced retinitis pigmentosa.

A phase II study of implants of encapsulated human NTC-201 cells releasing ciliary neurotrophic factor (CNTF) in participants with retinitis pigmentosa using visual field sensitivity as the primary outcome. Using the same rationale as above, we are attempting to slow the constriction of visual field loss in young patients with retinitis pigmentosa. Patients have the encapsulated CNTF cells implanted in one eye, with the fellow eye serving as a control.

Pediatric Eye Research

• an observational study of infantile, acquired non-accommodative, and acquired partially-accommodative esotropia
• a randomized trial to compare near versus distance activities while patching for amblyopia
• a randomized trial comparing atropine to atropine plus a reduced-plus lens for the sound eye as treatments for amblyopia in children 3 to <7 years old
• a randomized trial comparing patching versus atropine for amblyopia in 7 to <13 year olds
• a study of nerve fiber layer thickness in amblyopia
• a prospective study of surgical procedures for the treatment of persistent nasolacrimal duct obstruction in children less than four years old
• a randomized trial of the effect of progressive addition lenses versus single vision lenses on low myopia associated with large accommodative lags and near esophoria in children
• long term follow up of amblyopia treatment
• an evaluation of treatment of amblyopia in 7 to <18 year olds - recurrence observation phase
• bilateral refractive amblyopia treatment study: response to treatment of previously untreated presumed bilateral refractive amblyopia
• a prospective study of primary surgical treatment of nasolacrimal duct obstruction in children less than four years old

Long-term visual, cognitive, and growth outcomes and LCP-supplementation of infant formula

Allergy and asthma in children who were fed LCP-supplemented infant formula during the first year of life