Retinitis Pigmentosa

Researchers are evaluating the first use of a new imaging technology called 4D Microscope Integrated Optical Coherence Tomography (4D MIOCT) combined with a Zeiss Artevo 800 surgical microscope during eye surgeries. This study focuses on adult patients undergoing clinically indicated surgery for various eye diseases, aiming to assess how easy the system is to use, its safety, and how well it visualizes the eye's structures during both front and back segment procedures. The study involves using the investigational 4D MIOCT device integrated into the Zeiss Artevo 800 microscope to capture detailed images of the eye during surgery. This imaging is performed in the operating room as part of the standard surgical care, with no additional treatment or interventions added. Up to eight patients may be enrolled, and images will be taken of one or both eyes during surgery to observe normal and abnormal microanatomy, as well as track any injections under the retina. Participants will have their eye imaged during surgery, and researchers will collect related clinical data and previous eye care records for comparison. The images and data will be analyzed after surgery to explore new details captured by the 4D MIOCT system. Researchers will measure ease of use, surgical workflow impact, and the presence of normal and abnormal microanatomy based on the images. There is no extra risk beyond the usual surgery, and no new medications or procedures will be performed solely for research purposes.

Diabetic Macular Edema (DME) is a complication of diabetes that causes swelling in the central part of the retina, leading to vision loss. This condition results from changes in the small blood vessels of the retina, causing leakage and retinal thickening, often linked to increased levels of vascular endothelial growth factor (VEGF). DME is a common cause of blindness in people with diabetes, and it is especially significant in Bangladesh due to the high number of adults living with diabetes. This study compares the effectiveness and safety of two treatments for DME: a proposed biosimilar of Ranibizumab and Lucentis, both given as 0.5 mg injections into the eye every four weeks. A total of 70 adults with DME will be randomly assigned to receive one of these treatments in three doses over eight weeks. Safety visits will occur 48 hours after each injection, and the study concludes with an evaluation visit at week 12. Participants will undergo vision tests and imaging of the retina at the start and end of the study to measure changes in visual acuity and retinal thickness. Safety will be monitored throughout with follow-up visits shortly after each injection. Researchers will track how many patients maintain or improve their vision and assess any side effects. The total participation time for each patient is approximately 12 weeks.

X-linked retinitis pigmentosa (XLRP) is an inherited eye condition that affects the retina, leading to night blindness and gradual vision loss. This Phase 2 study evaluates the investigational drug laruparetigene zovaparvovec (AGTC-501) to see if it is safe and whether it can preserve or improve vision and other symptoms in male participants with XLRP. The trial focuses on males aged 12 to 50 years who have a genetic diagnosis of XLRP related to the RPGR gene. Participants will undergo surgery called pars plana vitrectomy to receive laruparetigene zovaparvovec by injection under the retina in both eyes. The first treated eye is dosed on Day 1, and the second eye is treated either 30 to 60 days later or sooner (7 to 14 days) depending on safety reviews. The study drug is a biological treatment delivered through a subretinal injection. Researchers will monitor participants for up to 5 years after treatment. During the study, participants will have regular eye exams and vision tests, including measures of visual acuity, light sensitivity, and retinal structure. Safety will be closely monitored by tracking any serious or significant side effects over the first year and longer term. Questionnaires and virtual reality tests will assess daily vision-related functions. The main outcome is the number of participants experiencing severe treatment-related adverse events within 12 months. The total participation time may last several years with multiple follow-up visits.

Researchers are evaluating the safety, tolerability, and effectiveness of up to three doses of KIO-301 given by injection into both eyes every six weeks in patients with late-stage retinitis pigmentosa (RP). The study includes patients who have no light perception or low vision. This Phase II trial aims to better understand how KIO-301 works in this population. Participants will receive either 50 micrograms or 100 micrograms of KIO-301 or a placebo injected into both eyes once every six weeks for a total of three injections. The study includes a screening period of up to 45 days, followed by a treatment and follow-up period lasting 12 weeks each. Those who received placebo and opt to join the open-label extension may participate for an additional 24 weeks. During the study, participants will attend visits every three weeks for safety, tolerability, and effectiveness checks, including blood tests and eye exams. Researchers will monitor the effects from baseline through 12 weeks after the last injection, totaling up to 30 weeks for the main study. Those in the extension phase will have ongoing assessments, with total participation depending on timing between study phases.

Researchers are evaluating the use of LX107, a gene therapy drug, in patients with retinal dystrophy caused by mutations in the AIPL1 gene. This early phase 1 clinical trial aims to assess the safety and effectiveness of this treatment for individuals aged 4 years and older who have a confirmed diagnosis of this inherited retinal disease. The study is sponsored by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine. Participants will receive a single subretinal injection of LX107 in one eye, designated as the study eye. The study includes three dose groups: low dose (3x10^9 viral genomes per eye), medium dose (1x10^10 viral genomes per eye), and high dose (3x10^10 viral genomes per eye). Treatment will be administered on Day 0, and patients will be monitored closely throughout the study. During the study, participants will undergo evaluations such as functional MRI scans, mobility tests, full-field sensitivity threshold tests, visual acuity assessments, and visual field measurements at 6 and 12 months after treatment. Safety will be closely monitored by tracking treatment-emergent adverse events over 6 months. Participants will also be followed long-term according to the study protocol to assess treatment effects and safety over time.

Researchers are evaluating the safety, tolerability, and clinical responses after a single dose of DSP-3077 in adults with retinitis pigmentosa (RP). This Phase 1/2a, open-label, single-arm study involves three groups defined by their visual acuity and dose level of DSP-3077. The study aims to assess safety, engraftment, and potential therapeutic effects of DSP-3077 retinal sheets delivered subretinally. Participants will receive a single subretinal injection of DSP-3077 in one eye, with two dose levels tested across three cohorts. Each cohort will have 4 participants, totaling 12 adults. Cohorts 1 and 2 receive lower and higher doses respectively, while cohort 3 involves participants with better visual acuity receiving a high dose. The treatment uses a specialized delivery device for the injection. The study lasts about 67 months, starting with a 2-week period of frequent visits post-surgery, then monthly visits until month 4, followed by visits every 3 months up to month 24, and every 6 months up to month 60. Long-term safety data will be collected annually from years 6 to 15 after treatment. Researchers will monitor ocular adverse events, serious adverse events, and treatment tolerability throughout the study.

Researchers are studying OpCT-001, a cell therapy made from photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs), in adults with primary photoreceptor disease. This Phase 1/2a study aims to evaluate the safety, tolerability, and effects of OpCT-001 on visual function, functional vision, and retinal structure in about 54 adults with confirmed genetic diagnoses of photoreceptor diseases such as retinitis pigmentosa and Usher syndrome. The study is divided into two parts. Phase 1 involves up to 24 legally blind participants receiving OpCT-001 at up to 4 different dose levels via subretinal injection, using a dose-escalation design. Phase 2 will enroll up to 30 participants randomized to two dose levels selected from Phase 1 data, with masking applied to participants and most study staff. Both phases focus on monitoring safety and assessing effects on vision and retinal anatomy. Participants will receive OpCT-001 through subretinal injections and be followed for up to 52 weeks to monitor treatment-emergent adverse events and changes in retinal layer thickness using imaging methods like spectral domain optical coherence tomography. Visual acuity and retinal structure will be assessed regularly. The study includes genetic confirmation of disease, eye exams, and ongoing safety monitoring, with the total participation duration spanning about one year after treatment.

Researchers are investigating outer retinal diseases, which affect parts of the eye such as photoreceptors, the retinal pigment epithelium, basement membrane, or choroid. The study aims to collect detailed retinal images using adaptive optics to develop new diagnostic tools, biomarkers, and clinical endpoints. Both healthy volunteers and individuals with these diseases are included to support the comparison and understanding of retinal changes over time. Participants will undergo imaging with advanced adaptive optics devices that combine scanning laser ophthalmoscopy and optical coherence tomography. These systems capture high-resolution videos of retinal cells while correcting for eye-related distortions. Imaging will focus on several key areas in the macula and will be repeated over a three-year period to track changes in retinal cell structure and function. During the study, participants will receive imaging sessions where measurements such as photoreceptor density, retinal pigment epithelial cell density, organelle movement, and photoreceptor function will be recorded. Some measurements will be repeated to assess consistency. The study monitors these cellular features to better understand disease progression and to aid future therapy development. Participation may last up to three years with periodic imaging visits.

Researchers are studying adaptive optics, a new imaging technology, to improve the diagnosis, treatment, and management of eye diseases affecting the retina. The study involves both people over age 12 with various eye diseases and healthy volunteers over age 12 to compare retinal images between diseased and normal eyes. The goal is to find better ways to detect retinal problems earlier than current methods allow. Participants will undergo eye exams including pupil dilation and adaptive optics imaging, where they look into a special instrument that captures high-resolution images of the retina. Some may also have additional tests like perimetry, color vision tests, and electroretinograms using numbing eye drops and special contact lenses. This observational study plans to enroll a total of 600 participants over five years, with 350 having eye diseases and 250 healthy volunteers. During the study visits, participants will provide medical and eye history and information about their medications. Researchers will assess the adaptive optics images qualitatively and quantitatively to compare normal and diseased retinal states. The study includes regular eye exams and imaging assessments, with the primary outcome focused on evaluating these adaptive optics images. Participation involves one or more visits, with detailed eye examinations and imaging procedures to monitor retinal health.

Researchers are evaluating a new handheld optical coherence tomography (OCT) device designed to automatically align with the eye to capture detailed retinal images. This pilot study aims to assess this advanced imaging technology in healthy adult volunteers, adult patients, and pediatric patients with eye and retinal diseases, addressing current challenges in imaging uncooperative patients, especially children. The study is observational and led by Duke University, focusing on improving imaging speed, alignment, and data quality for retinal assessment. The study uses a Swept Source OCT system with enhanced hand-held probe technology that auto-aligns to the patient’s eye and detects image quality in real time, automatically saving optimal images. Participants include 20 healthy adults, 20 adult patients, and 10 pediatric patients from ophthalmology clinics. Imaging will be performed during clinical visits, with up to four single imaging sessions over two years to capture detailed retinal structure and blood flow. Participants will undergo retinal imaging using the handheld OCT device during their clinical visits. Researchers will analyze images for retinal microanatomy abnormalities and retinal thickness at specific eye regions, collecting data securely for further analysis. The study will monitor outcomes over multiple sessions for up to two years, with no known risks reported from prior handheld OCT use. The total participation duration depends on the number of imaging sessions completed within the study timeframe.