Achromatopsia

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.

Phacoemulsification is a common cataract surgery performed under topical anesthesia, which is generally safe but can cause pain, anxiety, and discomfort for some patients. This research evaluates the effect of oral anti-anxiety medication, specifically Alprazolam, on improving patient satisfaction during second eye cataract surgery. The study aims to clarify whether oral sedation can reduce pain, anxiety, and side effects like nausea and dizziness compared to no sedation during phacoemulsification. Patients will receive either a single oral dose of Alprazolam or a placebo tablet 30 minutes before surgery. The dose of Alprazolam is 0.5 mg for most patients, with a reduced 0.25 mg dose for those over 65 years or with liver disease. The study compares these groups to assess how oral sedation affects anxiety, pain, and surgical satisfaction. This is an extension study involving patients undergoing surgery on their second eye with topical anesthesia. During the trial, participants will complete questionnaires assessing pain, anxiety, and overall satisfaction before and after surgery. Researchers will monitor intraoperative discomfort and postoperative anxiety levels. Data from approximately 250 patients will be collected and analyzed for these outcomes. This study runs from March 2025 to December 2027, focusing on improving cataract surgery experiences safely and conveniently.

Researchers are studying the relationship between clinical factors and the occurrence of adverse drug reactions in hospitalized patients who receive tropicamide/phenylephrine 8 mg/50 mg/mL. This study aims to identify which clinical determinants may increase the risk of these reactions and provide safety information for this commonly used ophthalmic drug combination. It also seeks to establish safety measures for patients undergoing eye examinations or surgery where these drugs are used. The study involves hospitalized patients scheduled for surgery who will receive two drops of tropicamide/phenylephrine 8 mg/50 mg/mL. Vital signs will be measured before administration and then at several time points afterward, including 5, 15, 30 minutes, and 1, 2, and 3 hours post-administration. Data will be collected using questionnaires and analyzed to find associations between clinical factors and adverse reactions. Participants will be monitored closely through vital sign checks and questionnaires during the study period. Researchers will evaluate adverse drug reactions and clinical determinants to understand their connection. The main outcome is to assess vital signs at set intervals following drug administration and identify any adverse reactions. The study is observational and aims to enhance the safety profile of this medication in hospitalized patients.

Researchers are developing and testing an 8-week group therapy program that combines cognitive behavioral therapy (CBT) with mindfulness techniques tailored for people adjusting to vision loss. The study aims to create an effective approach to help individuals cope with the emotional and psychological challenges related to vision impairment. The initial phase involves gathering input from focus groups and expert panels to shape the therapy curriculum. During the study, participants will first attend up to two focus group sessions to provide feedback on the therapy content. Following this, a pilot phase will involve 15-20 participants attending weekly group therapy sessions either in person or virtually, based on the developed 8-week curriculum. The therapy combines CBT and mindfulness practices designed specifically for vision loss adjustment. Participants will complete surveys and assessments before and after the therapy, including questions about their vision, demographic information, and experiences related to vision loss. The research team will measure changes in participants' adaptation to vision loss, depression levels, and anxiety before and after the intervention. The total participation period includes the initial feedback sessions and the 8-week therapy program.

Researchers are exploring a new multisensory rehabilitation approach for people with profound visual impairment, particularly those with ultra low vision. This study aims to fill gaps in current vision rehabilitation methods after treatments for end-stage eye diseases, aiming to improve functional abilities and quality of life for participants. The trial is interventional and evaluates two types of training programs designed to enhance sensory integration and residual vision use. Participants are randomly assigned to one of two training programs. One group receives Visual Information Training focused on improving the use of remaining vision to support daily activities. The other group undergoes Multimodal Training that teaches how to combine information from multiple senses to boost functional performance. Both trainings are behavioral interventions delivered during the study. Throughout the study, participants will be assessed at the start, and then after 1, 3, and 6 months to measure changes in visual ability, hand-eye coordination, and hearing ability. These assessments help track how well the training supports sensory function and overall visual performance. The study runs until July 2026, with ongoing evaluations to understand the impact of these rehabilitation strategies.

Researchers are studying early visual function in infants who may develop cerebral visual impairment due to brain or eye-related problems. The study focuses on babies from neonatal age up to 36 months old who are at risk because of conditions like brain lesions or ocular diseases. This pilot study aims to better understand how vision develops in these young patients over time. The study observes infants without testing any treatments or interventions. Researchers will assess visual abilities and correlate these findings with brain MRI results to understand the relationship between brain structure and vision function. Participants will be followed and evaluated regularly over a period of five years to track their visual development and neurological status. During the study, infants will undergo visual function assessments and brain imaging evaluations. The researchers will collect data on visual performance scores and compare them with MRI results to better understand visual impairment risks. The total participation includes assessments up to 36 months of age, with long-term follow-up planned for five years to monitor outcomes and correlations between visual function and brain health.

This research aims to improve visual health knowledge and access among children and adolescents aged 3 to under 18 with intellectual disabilities or developmental delays. It addresses challenges faced by this group in receiving medical information and preventive care due to communication barriers and limited support tools. The program includes developing easy-to-read materials and inclusive technologies to support education and early detection of visual health issues. Participants will be randomly assigned to one of three groups: standard visual health education using conventional materials with instructor explanations, Easy-to-Read instructor-guided education with simplified images and plain language, or Easy-to-Read audio-based self-learning using a voice-assisted pen device for independent study. The study involves an initial pre-assessment phase, followed by a pre-test, instructional phase, and post-test, all within a one-month window. Throughout the study, participants will undergo assessments including adaptive behavior scales, cognitive or developmental evaluations, and visual tests. Researchers will measure improvements in eye health knowledge using pre- and post-tests. The study also monitors participants' comprehension and allows a repeat instructional session if needed. The total participation includes a 50-minute baseline assessment and same-day instructional and testing sessions lasting about 20 minutes.

This research aims to investigate the relationship between blood serum biomarkers and how patients with diabetic macular edema (DME) respond to treatments with anti-VEGF or dexamethasone implants. The study compares these treatments to a group of patients who have not previously received treatment for DME. Researchers will also assess eye imaging markers using OCT scans to better understand this condition. Participants will be divided into three groups: those receiving anti-VEGF treatment, those receiving dexamethasone implants, and those who are treatment-naive. Blood samples will be taken at the start for all groups, and again after four weeks for the untreated group. OCT scans of the retina and choroid will be performed at baseline and after four weeks to evaluate specific imaging markers related to DME. During the study, participants will undergo blood tests to measure levels of several biomarkers including VEGF, IL-6, IL-8, MCP-1, Ang-2, PlGF, TNF-a, and ICAM-1. OCT scans will be analyzed for retinal layer disruptions and fluid presence. Researchers will classify participants based on their response to treatment and monitor changes over the four-week period. The total participation time varies based on group and treatment status.

Falls are a major health concern for older adults, especially those with glaucoma who experience peripheral vision loss that affects their mobility and balance. This research evaluates a new, enjoyable video game-based intervention designed to improve movement and postural control in glaucoma patients with moderate to severe peripheral vision loss. The study aims to reduce fall risk and improve quality of life by incorporating this intervention into existing vision and balance rehabilitation programs. This clinical trial randomly assigns 56 glaucoma patients with stable peripheral vision loss to two groups. One group participates in 20 sessions of standing, physically interactive action video-game training using a Nintendo Switch, with games involving muscle stretching and strengthening exercises, conducted twice weekly for 10 weeks. The other group receives 20 sessions of conventional physical training, also lasting 45 minutes twice per week for 10 weeks. Both interventions are compared to assess their effects on mobility and balance. Participants will be evaluated before starting, after 10 sessions, and after 20 sessions. Assessments include a primary mobility test combining walking and timed up and go tasks, as well as secondary tests of static and dynamic balance, visual cognition, reaction time, cognitive reflection, and questionnaires on fear of falling and quality of life. The study includes regular monitoring of participant progress over the 10-week intervention period.