ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
X-linked RetinoschisisThis study will evaluate the safety and tolerability of ATSN-201 in male subjects 6 to 64 years old with RS1-associated X-linked retinoschisis (XLRS).
Safety and Efficacy Study of LX103 Treatment of X-Linked Retinoschisis (XLRS)
X-linked RetinoschisisThe goal of this study is to evaluate the safety and efficacy of LX103 treatment of X-linked retinoschisis. This study will enroll subjects aged ≥ 6 years old to receive a single unilateral intravitreal (IVT) injection of LX103 to evaluate its safety and efficacy.
Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients
X-linked RetinoschisisThis trail is meant to evaluate the safety and efficacy of ZM-01 of X-linked retinoschisis. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.
Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
RetinoschisisX-LinkedBackground: - X-linked juvenile retinoschisis (XLRS) is caused by changes in the RS1 gene. These changes cause abnormal function of the eye protein retinoschisin. Without normal retinoschisin, the layers of the retina split and vision is lost. Researchers want to try to introduce a healthy RS1 gene into eye cells, to see if this helps retinal cells make healthy retinoschisin. They will put the gene in a virus. The gene and virus package is known as a gene transfer vector (AAV-RS1 vector). Objectives: - To see if the AAV-RS1 vector is safe to use in people. Eligibility: - Adults 18 and older with a mutation of the RS1 gene, 20/63 vision or worse in one eye, and XLRS. Design: Participants will be screened with genetic tests to confirm XLRS. They will have a medical history and physical and eye exams. At visits 1-2, participants will have some or all of the following: Medical history Physical exam Blood and urine tests Tuberculosis skin test Eye exam Vision tests (for one test an intravenous line will be placed in the arm. A dye will be injected that will travel to the blood vessels in the eye). At visit 3, the AAV-RS1 vector will be injected with a needle in the study eye. Participants pupils will be dilated. They will get numbing eye drops. Visits 4-13 will occur in the 18 months after gene transfer. Many of the above tests will be repeated. Participants will discuss any side effects. Visits 14-17 will occur yearly between years 2 and 5. After year 5, participants will be contacted yearly by phone for up to 15 years.
Clinical and Genetic Studies of X-Linked Juvenile Retinoschisis
RetinoschisisThis study will explore the causes and eye problems of X-linked juvenile retinoschisis (XLRS), an inherited disease that causes vision loss primarily in young males. The vision loss, which worsens over time, is a result of schisis, or splitting, of the layers of the retina (tissue that lines the back of the eye). A better understanding of why and how XLRS develops might lead to improved treatments. Patients 9 months of age and older with XLRS and females who are suspected carriers of the gene responsible for the disease (such as the mother of the patient) may be eligible for this study. Other family members of patients also may be enrolled. Patients will undergo the following tests and procedures: Personal and family medical history to review past and current medical conditions and treatments, particularly regarding eye disease, and to construct a family tree. Eye examination to assess visual acuity (eye chart test) and examine pupils, lens, retina, and eye movements. The pupils will be dilated with drops for this examination. Photography of the retina to help evaluate the status of the retina. Specialized eye tests to evaluate color vision, field of vision, and ability to see in the dark. Electroretinogram (ERG) to examine what happens to the eyes after a flash of bright light. For this test, the patient sits in a dark room for 30 minutes with his or her eyes patched. Then, a small silver disk electrode is taped to the forehead, the eye patches are removed, the surface of the eye is numbed with eye drops and contact lenses are placed on the eyes. The patient looks inside a large empty bowl and then a light flashes, first in the dark and then with a light turned on inside the bowl. The contact lenses sense small electrical signals generated by the retina when the light flashes. Blood test to examine DNA for genetic study of XLRS. Family members will provide a blood sample for genetic study. ...
Inherited Retinal Degenerative Disease Registry
Eye Diseases HereditaryRetinal Disease26 moreThe My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation. It is a patient-initiated registry accessible via a secure on-line portal at www.MyRetinaTracker.org. Affected individuals who register are guided to create a profile that captures their perspective on their retinal disease and its progress; family history; genetic testing results; preventive measures; general health and interest in participation in research studies. The participants may also choose to ask their clinician to add clinical measurements and results at each clinical visit. Participants are urged to update the information regularly to create longitudinal records of their disease, from their own perspective, and their clinical progress. The overall goals of the Registry are: to better understand the diversity within the inherited retinal degenerative diseases; to understand the prevalence of the different diseases and gene variants; to assist in the establishment of genotype-phenotype relationships; to help understand the natural history of the diseases; to help accelerate research and development of clinical trials for treatments; and to provide a tool to investigators that can assist with recruitment for research studies and clinical trials.
Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)
X-linked RetinoschisisThis study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector expressing retinoschisin (rAAV2tYF-CB-hRS1) in patients with X-linked retinoschisis. Up to 27 participants will be enrolled and 3 dose levels will be evaluated in a dose escalation format.
Effects of Macular Buckle Versus Vitrectomy on Macular Schisis and Macular Detachment in Highly...
RetinoschisisMacular Detachment1 moreMacular schisis associated with macular detachment is a one of the complications of high myopia. There is controversy in the primary treatment for this situation. This study will compare the effects of macular buckling + gas injection versus vitrectomy + internal limiting membrane peeling + gas tamponade in a cohort of highly myopic eyes with macular retinal detachment associated with macular schisis.
Macular Detachment Associated With Intrachoroidal Cavitation
Macular DetachmentMacular Retinoschisis1 moreEvaluation of clinical characteristics and surgical outcome for macular detachment associated with peripapillary intrachoroidal cavitation with or without pathologic myopia.
Postoperative Pain Control Following Vitreoretinal Surgery
Retinal DetachmentProliferative Vitreoretinopathy1 moreThe aim of this study is to evaluate if patients receiving a steroid (triamcinolone acetonide) combined with local anesthesia and antibiotic following retina surgery have better postoperative pain control those receiving local anesthesia and antibiotic alone.