Retinitis pigmentosa

This pilot study tests whether injections of platelet-rich plasma (PRP) made from a patient's own blood can help preserve vision in people with retinitis pigmentosa or a related condition called EMAP. Thirty participants receive three injections over four months, and researchers …

This pilot study tests whether adalimumab, an anti-inflammatory drug, can help preserve vision in people with retinitis pigmentosa or a related condition called EMAP. Thirty participants will receive three injections into the eye over four months. The main goal is to see if the t…

This study tests a gene therapy called laruparetigene zovaparvovec (AGTC-501) in 10 males aged 12-50 with X-linked retinitis pigmentosa, a genetic condition that leads to vision loss. The therapy is injected into both eyes to deliver a working copy of the RPGR gene. The main goal…

This phase 3 trial tests whether an oral antioxidant called N-acetylcysteine (NAC) can slow the loss of vision in people with retinitis pigmentosa, an inherited eye disease that causes gradual blindness. About 485 adults aged 18 to 65 are taking NAC or a placebo for several years…

This study is a follow-up to a Phase 3 trial testing a gene therapy called AAV5-hRKp.RPGR for people with X-linked retinitis pigmentosa, a genetic eye disease that causes vision loss. The treatment aims to slow or stop vision decline by delivering a working copy of the RPGR gene …

This study tests a gene therapy called botaretigene sparoparvovec in the second eye of people with X-linked retinitis pigmentosa, a genetic condition that causes vision loss. The 24 participants previously received the therapy in one eye and are now being followed for safety and …

This Phase 3 study tests a gene therapy called AAV5-hRKp.RPGR in 4 Japanese people with X-linked retinitis pigmentosa, a genetic condition that causes progressive vision loss. The therapy is injected into the eye to deliver a working copy of the RPGR gene. The main goal is to che…

This study tests an experimental drug called VP-001 for people with a rare genetic eye disease that causes vision loss. The drug is injected into the eye and aims to slow or stop further damage. Researchers will check safety and measure any changes in eyesight over about two year…

This early-stage trial tests a new oral drug called OCT-980 for people with a specific inherited eye disease (autosomal dominant retinitis pigmentosa) that causes gradual vision loss and blindness. The study first gives a single dose to healthy volunteers to check safety, then mu…

This study tests a gene therapy called SPVN06 for retinitis pigmentosa, an inherited eye disease that causes gradual vision loss. The treatment is given as a single injection under the retina. The trial includes 33 adults with advanced disease and will check safety and whether it…

This program offers an experimental treatment called OCU400 to up to 75 adults with retinitis pigmentosa (RP) who cannot join the main Phase 3 study. Participants must be 18 or older, have a confirmed genetic diagnosis of RP (except a specific mutation), and have remaining light-…

This study tests a gene therapy called AGTC-501 in males aged 12 to 50 with X-linked retinitis pigmentosa, a genetic eye disease that causes vision loss. Participants receive one of two doses of the therapy or no treatment, and researchers measure changes in vision under low ligh…

This study tests a gene therapy called OCU400 for people with retinitis pigmentosa, an inherited eye disease that causes vision loss. The treatment is injected under the retina to help light-sensitive cells work better. The trial enrolled 140 people aged 3 and older and measures …

This early-stage trial tests a combination treatment for retinitis pigmentosa, a genetic disease that causes blindness. Ten adults receive a single injection of GS030 gene therapy into one eye, then use special glasses that send light signals to the treated retina. The main goal …

This study tests a new gene therapy for people with chronic granulomatous disease (CGD), a genetic disorder that weakens the immune system and makes them prone to serious infections. The treatment takes the patient's own blood stem cells, adds a missing gene in the lab, and puts …

This early-phase trial tests a single injection of a gene therapy called RTx-015 in 10 adults with retinitis pigmentosa or choroideremia, two inherited conditions that cause vision loss. The therapy delivers a gene that makes retinal cells produce a light-sensitive protein, poten…

This early-stage trial is testing whether it is safe to inject a person's own bone marrow stem cells into their eye to treat vision loss from conditions like macular degeneration and diabetic retinopathy. Fifteen adults with severe vision loss will receive the injection and be mo…

This study tests an experimental drug called RP1 that is injected directly into skin cancer tumors. The goal is to see if it can shrink or eliminate the cancer before surgery. Up to 12 adults with a type of skin cancer called cutaneous squamous cell carcinoma will take part. The …

This study is testing the long-term safety of a high-dose antioxidant called N-Acetylcysteine (NAC) in 30 adults with retinitis pigmentosa, an inherited eye disease that causes gradual vision loss and blindness. Participants take 1800 mg of NAC twice daily for two years, with reg…

This study tests a treatment called transcorneal electrical stimulation to see if it can improve vision in people with retinitis pigmentosa, an eye disease that causes vision loss. Twenty-one adults with the condition received the therapy, and researchers measured changes in thei…

This study was designed to see if a new method of measuring the visual field—by projecting vision zones on the ground—helps people with retinitis pigmentosa understand their remaining sight and improve their walking rehabilitation. The trial was withdrawn before enrolling any par…

This study collects information from about 889 people with inherited retinal diseases (IRDs) to understand how vision changes over time. Participants have their eyesight and visual field tested regularly. The goal is to learn more about the natural course of these rare eye condit…

This study aims to find better ways to measure how retinitis pigmentosa (a genetic eye disease that causes vision loss) progresses over time. Researchers will use advanced imaging and vision tests in 40 people with specific genetic forms of the disease. The goal is to identify re…

This study follows 50 people with a rare inherited eye condition called PRPF31-related retinal dystrophy (RP11) to see how their vision changes over time. Researchers will measure things like visual acuity, retinal thickness, and quality of life using eye exams and questionnaires…

This study looks at a rare, inherited eye disease called CNGB1 retinitis pigmentosa that causes gradual vision loss and blindness. Researchers want to learn how the disease progresses by using eye exams and imaging tests over three years. The goal is to find the best ways to meas…

This study follows 103 adults with a genetic eye condition called retinitis pigmentosa caused by EYS mutations. Researchers will track changes in vision and eye structure over 4 years. The goal is to better understand how the disease progresses so that future clinical trials can …

This study follows 12 adults with severe retinitis pigmentosa, a genetic eye disease that causes vision loss. Researchers want to find out which vision tests people with very low sight can reliably perform and how their vision changes over one year. The goal is to improve future …

This study follows 127 people with a genetic eye condition called USH2A-related retinal degeneration, which can cause vision loss and hearing problems. Researchers measure changes in vision, retinal sensitivity, and mobility over several years. The goal is to understand how the d…

This study follows 44 people with a rare genetic form of Usher syndrome caused by PCDH15 mutations. Over 48 months, researchers measure how their vision changes using eye exams and imaging. The goal is to identify the best ways to track disease progression, which will help design…

This study offers free vision screenings to underserved adults over 40 living in affordable housing in Harlem and Washington Heights. The goal is to find eye problems like glaucoma, cataracts, and diabetic retinopathy early and connect people with follow-up care. About 749 partic…