Eye degenerative disorder

This study tests a gene therapy called ATSN-201 in males aged 6 and older with X-linked retinoschisis, a genetic eye condition that leads to vision loss. The therapy delivers a working copy of the RS1 gene to the retina. The trial has three parts: first, finding the right dose; s…

This early-stage trial tests whether adding special cells (DUOC-01) into the spinal fluid is safe for children with inherited metabolic diseases that damage the brain. Participants are ages 1 week to 21 years and are already receiving a standard umbilical cord blood transplant. T…

This study tests a new way to implant a corrective lens inside the eye for people with high myopia (severe nearsightedness). The new method uses a saltwater solution instead of a gel to keep the eye stable during surgery. Half of the participants will have the new method in one e…

This trial investigates whether a low dose of the drug conbercept can effectively treat retinopathy of prematurity (ROP), an eye disease that can cause blindness in preterm infants. The study will enroll 146 preterm babies with type 1 ROP and compare the low-dose treatment agains…

This study tests if a special red light device can slow down damage to the back of the eye in people with severe nearsightedness (pathologic myopia). About 158 adults will use the device or a fake one for 3 minutes, twice a day, 5 days a week for a year. Researchers will check if…

This early-stage study tests a new medicine called FLQ-101 in 18 premature babies born at 26-27 weeks who are at high risk for retinopathy of prematurity (ROP), an eye disease that can cause blindness. The main goal is to check if the drug is safe and how it works in the body. Re…

This study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…

This study tests whether a probiotic called MR-61 can help slow the worsening of nearsightedness (myopia) in children and teens aged 3 to 18. Participants will take either the probiotic or a placebo daily for a period, and researchers will measure changes in their vision and eye …

This study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…

This study tests whether early physiotherapy, taught to parents, can improve movement and development in babies at risk for problems like cerebral palsy. Fifty infants from the NICU will be randomly assigned to get either the special program or standard care. Researchers will mea…

This study tests whether a handheld, ultra-widefield eye scanner (OCT) can accurately identify preterm infants with severe retinopathy of prematurity (ROP), a condition that can harm vision. The scanner takes detailed images of the retina without touching the eye, potentially red…

This study is testing a new, noninvasive imaging device called HH-SECTR to see inside the eyes of premature infants with retinopathy of prematurity (ROP), a condition that can affect vision. The goal is to see if this technology can provide clearer, more detailed images of the re…

This study will enroll 500,000 people with high myopia and cataracts to develop an artificial intelligence model that helps doctors calculate the correct lens power and grade cataracts more accurately. The goal is to improve vision outcomes and reduce complications after surgery.…

This study is testing whether artificial intelligence can analyze eye images to better detect and predict vision-threatening changes in people with high myopia (severe nearsightedness). Researchers will collect eye scans from 1,000 patients and use AI to identify a condition call…

This study tests a laser treatment called OSC/SS to prevent retinal detachment in people with Stickler Syndrome, a genetic condition that raises the risk of vision loss. About 500 children and adults will receive the procedure in one or both eyes and be followed for 5 years. Rese…

This study tests whether an oral blood pressure drug, propranolol, can prevent severe eye disease (retinopathy of prematurity) in extremely premature infants. About 276 babies born before 28 weeks and weighing under 1250 grams will receive either propranolol or a placebo. The goa…

This early-stage study tests a topical spray called ELU42 on 15 adults with diabetic foot ulcers (Wagner grade 1-2) that have lasted 4 to 52 weeks. The spray is applied three times a week for six weeks. The main goals are to check safety and measure how much the wound shrinks. Th…

Researchers are building a global registry to collect patient-reported surveys, genetic test results, and medical records from people with Charcot-Marie-Tooth disease and related inherited neuropathies. The study is open to children and adults with a confirmed or suspected diagno…

This observational study collects data from UK preterm babies with retinopathy of prematurity (ROP) who are treated with the drug aflibercept (Eylea) using a paediatric dosing device. The main goal is to count how many babies receive this treatment and decide if there are enough …

This study looks at whether certain eye features can predict how well people recover after surgery for myopic foveoschisis, a complication of severe nearsightedness that can threaten sight. Researchers will follow 30 adults aged 40-90 who need vitrectomy, a standard procedure to …

This study looks at 200 highly myopic adults in France, the Netherlands, and Spain to understand why some develop a condition called posterior staphyloma, where the back of the eye bulges outward. Researchers will analyze blood samples and eye images to find genetic and molecular…

This study builds a global database of people diagnosed with or at risk for Krabbe disease, a rare nerve disorder. Researchers will track symptoms, genetic mutations, and enzyme levels to find clues that predict when the disease starts and how severe it becomes. The registry also…

This study looks at how often oxygen levels drop in premature infants when they receive an eye injection of ranibizumab for retinopathy of prematurity (ROP). Researchers reviewed records from 74 infants treated across multiple hospitals, tracking oxygen saturation during the proc…

This pilot study is testing how well children aged 6 to 16 adapt to Essilor Stellest myopia-control spectacle lenses. The goal is to see if kids find them comfortable and easy to wear in real life, which is important for long-term use. Fifty children with progressive myopia will …

This study is creating a large database of eye measurements from 3,000 people with inflammatory eye diseases like diabetic retinopathy and age-related macular degeneration. Researchers will use advanced imaging to collect detailed information from both the front and back of the e…

This study follows preterm infants who were part of an earlier study to see if a new, portable eye imaging device can detect hidden signs of retinopathy of prematurity (ROP), a vision-threatening condition. Researchers will compare the new imaging to standard photos and track chi…

This registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …

This study follows 60,000 people with cataracts, including those with high myopia (severe nearsightedness), to see how their vision changes after cataract surgery. Researchers will also look at genetic and environmental factors that might affect cataract development and surgical …

This study enrolls 25 people with severe retinal diseases like retinitis pigmentosa and Stargardt disease to test new ways of measuring visual function. Researchers will evaluate how well participants can perform various vision tests, such as navigating obstacles or detecting lig…

This study follows 1500 people with rare genetic brain disorders to learn how these diseases progress. Researchers measure thinking, movement, and daily living skills over time, and also look at brain scans and body fluids. The goal is to better understand the diseases and how tr…

This study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…

This study aims to understand how blood vessels around the fovea (a key part of the eye for sharp vision) develop in infants with retinopathy of prematurity, a leading cause of childhood blindness. Researchers will use a special imaging technique (OCTA) to track changes in blood …

This study looks at why some premature babies develop retinopathy of prematurity (ROP), an eye condition that can affect vision. Researchers think fatty acids from the mother may play a role. They will collect blood, placenta, and cord samples from 135 mothers and their preterm o…