Eye degenerative disorder

This study tests a gene therapy called PBKR03 for babies with early infantile Krabbe disease, a severe genetic disorder that damages the brain and nerves. The treatment delivers a working copy of the GALC gene to the brain and body. Researchers will check safety and find the best…

This study tests whether dual focus contact lenses (MiSight) can slow the progression of nearsightedness (myopia) in university students aged 18 to 21. Participants wear either the test lenses or standard single vision lenses for at least 10 hours a day. Researchers measure chang…

This study follows 75 Japanese babies with retinopathy of prematurity (ROP) who are being treated with Eylea, a drug injected into the eye. ROP causes abnormal blood vessel growth in the retina and can lead to blindness. The study collects real-world data on side effects and how …

This study tests a one-time gene therapy infusion (FBX-101) given after a standard stem cell transplant in 6 children with infantile Krabbe disease, a severe genetic disorder affecting the nervous system. The therapy uses a harmless virus to deliver a working copy of the GALC gen…

This study tests whether eye pressure-lowering medication can slow the progression of severe nearsightedness (high myopia) in adults. Researchers will measure changes in eye length and vision over time. The trial includes 152 participants aged 18 to 65 with worsening high myopia …

This study tests whether taking branched-chain amino acid (BCAA) supplements for six months can slow the progression of myopia (nearsightedness) in young adults aged 18 to 25. Participants either receive BCAA supplements plus standard glasses or a placebo plus glasses. The goal i…

This study tests whether special new spectacle lenses can slow the worsening of nearsightedness (myopia) in children. About 342 kids aged 7 to 13 will wear either the new lenses or standard single-vision glasses for one year, then all switch to the new lenses for another year. Th…

This study tests a new surgical technique to close macular holes in people with high myopia without using a gas bubble. The standard approach requires a gas bubble that blurs vision and forces patients to keep their head down for days. The new method uses a gel-like substance to …

This study tests whether a special lens called Blue Star MAX-5 can slow the progression of nearsightedness (myopia) in children aged 8 to 11. Two hundred kids will wear either the new lens or standard glasses and be checked every six months for two years. The goal is to see if th…

This study tests whether Essilor Stellest spectacle lenses can slow the progression of nearsightedness (myopia) in adults aged 18 to 40. Each participant wears a Stellest lens in one eye and a standard lens in the other, allowing direct comparison. The trial measures changes in e…

This study tests whether lower doses of the drug bevacizumab (Avastin) can effectively treat a severe form of retinopathy of prematurity (ROP) in premature infants. The trial includes 77 babies with type 1 ROP in zone I, the most dangerous type. Each infant receives a single inje…

This study tests a new eye drop called SHJ002 to see if it can safely slow down worsening nearsightedness (myopia) in children ages 3 to 12. The drop works by targeting a specific molecule in the eye. Researchers will compare it to a standard treatment, atropine, and measure chan…

This study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to get the donor cells to take hold while keeping side effects low. Participants receive chemotherapy drugs before the transplant to prepare their bo…

This study tests special 3D-designed defocus lenses to see if they can slow down myopia (nearsightedness) in children aged 6 to 14. The lenses are meant to control how the eye grows, reducing the need for stronger glasses over time. Researchers will track changes in vision and ey…

This study tests whether special open-ring spectacle lenses can slow the progression of nearsightedness (myopia) in Chinese children aged 6 to 12. A total of 180 children will wear one of three lens designs for 12 months, with some continuing for another year. The goal is to see …

This study tests whether special glasses with a double helix lens design can slow the progression of myopia (nearsightedness) in children and adolescents aged 6 to 13. About 236 participants will wear either the new glasses or standard single-vision glasses for over 10 hours a da…

This early-phase trial tests a single infusion of a gene therapy called FBX-101 in 9 people with Krabbe disease who have already received a stem cell transplant. The therapy uses a harmless virus to deliver a working copy of the GALC gene, aiming to improve motor function and saf…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study looks at whether a special brain scan called diffusion tensor imaging (DTI) can help doctors find Krabbe disease in newborns and decide who needs urgent treatment. About 100 babies with a positive newborn screen or family history of Krabbe disease will be scanned and f…

This study tests an artificial intelligence system called i-ROP DL that analyzes images of the back of the eye. The goal is to see if it helps eye doctors more accurately diagnose retinopathy of prematurity (ROP), a condition that can cause blindness in premature infants. Researc…

This study aimed to see if a surgical procedure called scleral buckling could prevent retinal detachment in people with Stickler syndrome, a genetic condition that raises the risk of vision loss. The plan was to treat one eye in patients aged 5 to 35 who had already lost vision i…

This study tests whether giving premature babies a pacifier dipped in breast milk during eye exams for retinopathy of prematurity can reduce pain and improve comfort. About 56 infants born before 32 weeks will be randomly assigned to receive either standard care or the pacifier. …

This study looks at X-linked juvenile retinoschisis (XLRS), a rare inherited eye disease that causes vision loss in young males. Researchers will examine 351 participants, including affected males and female carriers, using eye exams, retinal photos, and genetic tests. The goal i…

This study looks at how a new type of eyeglass lens affects the back layer of the eye (the choroid) in children with worsening nearsightedness. Ten children already in a larger study will have their eye thickness measured with a special camera. The goal is to understand if the le…

This study follows about 100 premature babies who were treated for retinopathy of prematurity (ROP) in an earlier study. Researchers will check their eyesight and overall development over time to see how they are doing. No new treatment is given in this study.

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…