Retinitis pigmentosa

This study tests whether NPI-001 tablets can safely slow or stop vision loss in people with Usher syndrome, a rare genetic condition that causes blindness and hearing loss. About 49 adults will take either the study drug or a placebo for 24 months. The main goal is to see if the …

This early-stage study tested a gene therapy called CPK850 in 12 adults with a rare inherited eye disease (RLBP1 retinitis pigmentosa) that causes progressive vision loss and night blindness. The treatment was injected under the retina to deliver a working copy of the faulty gene…

This early-stage study tested the safety of a new drug called VP-001, given as an injection into the eye, for people with a rare genetic eye disease (PRPF31 mutation) that causes vision loss. Six participants aged 12 and older received multiple doses. The main goal was to check f…

This early-stage study tested a new drug, VP-001, given as an injection into the eye to see if it is safe for people with a rare genetic eye disease caused by a PRPF31 mutation. Seventeen adults took part. The main goal was to check for side effects, not to measure vision improve…

This study tested an experimental drug called KIO-301, given as an injection into the eye, in 32 adults with retinitis pigmentosa or choroideremia—two inherited conditions that cause progressive vision loss. The main goal was to check safety and tolerability at increasing doses. …

This phase II trial tested an experimental drug called ADX-2191 in 8 adults with retinitis pigmentosa caused by specific genetic mutations. Participants received monthly injections into one eye for up to six months. The main goal was to check safety, but researchers also looked a…

This Phase 3 trial tests a gene therapy called botaretigene sparoparvovec for X-linked retinitis pigmentosa, a genetic condition that causes progressive vision loss. The study involves 105 participants who receive the therapy via injection under the retina. The main goal is to se…

This study followed 42 people who received a gene therapy for X-linked retinitis pigmentosa, an inherited eye disease that causes blindness. The goal was to check the treatment's safety over up to 5 years. Researchers also looked at whether patients' vision and ability to move ar…

This completed Phase 1/2a trial tested a gene therapy called RST-001 (AGN-151597) in 14 people with advanced retinitis pigmentosa, a genetic eye disease that causes severe vision loss. All participants had very poor vision (hand motion or worse) at the start. The study aimed to c…

This study tested an artificial intelligence (AI) system called Retina4IRD that helps doctors diagnose inherited retinal diseases, which are rare eye conditions that can cause vision loss. The AI looks at eye images and patient information to suggest which gene might be causing t…

This study tested whether playing slow tempo music before and during an eye injection (intravitreal injection) can reduce anxiety and pain. 144 adults took part, with half listening to music and the other half receiving standard care. Researchers measured anxiety using saliva tes…

This trial tests a new device called P200TxE that takes detailed images of the back of the eye. Researchers want to see how well it captures images in people with retinal diseases like glaucoma or macular degeneration. The study involves adults aged 22 and older who have been dia…

This study tested whether artificial intelligence (AI) can help doctors detect retinal diseases from ultra-widefield eye images. 462 images were read by four eye doctors with different experience levels, both with and without AI assistance. The goal was to see if AI improves accu…

This study tested a new type of camera that can see individual cells in the retina, which standard clinical cameras cannot do. Five people with retinal disease (inherited degeneration or toxicity) had their eyes scanned with the device. The goal was to compare image quality and o…

This study collected information from nearly 1,000 people in France with inherited retinal diseases (IRDs) to better understand the genetics behind these conditions. Researchers looked at genetic test results and disease types to help build a national registry. The goal is to imp…

This study created a national DNA and blood repository for inherited eye diseases like retinitis pigmentosa. Over 6,600 participants provided blood samples and eye exam data to help researchers identify genetic causes. The stored samples are available to scientists without person…

This study looked at 10 people with rare genetic conditions that cause very low cholesterol and trouble absorbing vitamins. Researchers measured a protective pigment in the eye called macular pigment, which may be low in these patients and could explain why some still develop vis…

This completed study looked at how well different eye tests can track changes in retinitis pigmentosa (RP), a group of blinding eye diseases. Researchers tested 49 people with RP and healthy volunteers using exams like vision tests, retinal imaging, and electrical signal recordin…

This completed study tracked 87 patients who received Luxturna gene therapy for a rare inherited eye condition that causes blindness. Researchers monitored side effects and pregnancy outcomes for up to 5 years after treatment. The goal was to gather real-world safety data, not to…