Retinitis Pigmentosa

The purpose of this project is to capture research images from microscope integrated Optical Coherence Tomography (MIOCT) integrated into a Zeiss Artevo 800 surgical microscope with an add-on investigational 3D OCT scanner (hereafter called the 4D MIOCT) in participants undergoing clinically-indicated surgical procedures for a range of ocular diseases. The researchers will evaluate normal and abnormal microanatomy of the eye, image during surgical procedures, and track subretinal injections for therapeutic delivery during surgery (volumes measured/analyzed from the OCT images after surgery). This study is an observational imaging study with no treatment interventions for research purposes. The population is 5 adult ophthalmic surgical patients scheduled for eye surgery at Duke Eye Center, Durham. Up to 8 patients may be enrolled due to potential for surgery scheduling changes that would not allow research imaging on a surgery day for up to 3 patients. Research activities consist of investigational 4D MIOCT integrated into Zeiss Artevo 800 imaging of the eyes during surgery and collection of clinical data and other imaging from the participant's medical record, clinical visit and surgical procedure. Additional imaging of the participant's eye performed for clinical care will be extracted from the medical record for comparison to the intraoperative images. In this initial pilot, comparisons will enable design of future studies for accuracy, precision and reproducibility of the research device in eye surgery. For study participants there is no additional risk to the participant beyond what is normal for their ophthalmic surgery. No medications or surgical interventions/activities will be performed for research purposes. Images will be captured during the standard care ophthalmic microsurgery. This research will utilize the data gathered during OCT imaging performed as described above. The data collected from the OCT systems will be analyzed offline to allow for image processing and alternate visualizations of the area under study. The data gathered from 4D MIOCT imaging will be compared to existing clinical studies (performed as part of standard of care) on the participant, should they exist, for the purpose of identifying whether new information is gained by 4D MIOCT. Researchers will review the participant's Medical Record for up to three eye care visits prior to surgery and record information related to ocular health, eye examinations and imaging and prior ophthalmic treatment. No additional clinical studies will be performed for the purpose of this study.

This is a randomized, double-blind, parallel study that will be conducted in patients with Diabetic Macular Edema (DME) to assess efficacy and safety between test and reference products. Total 70 subjects with DME are planned to be randomized into this study. Enrolled subjects will receive total 3 doses of 0.5 mg Intravitreal injection of either test or reference product once every 4 Weeks (Day 0, Week 4±3 days and Week 8±3 days). All subjects will have their first injection of ranibizumab on Day 0 and undergo a safety visit 48hrs after the first injection. At subsequent visits, the subject will have a safety follow up prior to each intravitreal injection. Also the subject will have a safety follow up visit 48hrs after second and third injection. The final visit or end of study visit will be on Week 12±3 days for efficacy and safety evaluation.

Octant, Inc. is evaluating OCT-980, an oral small molecule corrector for the treatment of patients with rhodopsin (RHO)-associated autosomal dominant retinitis pigmentosa (RHO-adRP). There is no known cure and no currently approved therapy for RHO-adRP, a disease of the retina characterized by progressive loss of vision. This study is comprised of a Phase 1a, to be conducted in Australia; and a Phase 1b/2, to be conducted in the US. The phase 1a study will be a single dose, double-masked, placebo-controlled ascending dose escalation (SAD +/- food effect) to evaluate the safety, tolerability, and PK of OCT-980 in healthy volunteers. The phase 1b/2 study will be open-label, multi-center multiple ascending dose escalation (MAD) to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of multiple-ascending doses of OCT-980 for up to 48 weeks, in participants with a genetic diagnosis of Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa (RP).

XLRP is a genetic (inherited) eye disease that affects cells in the retina (the lining of the back of the eye that detects light). It causes night blindness and gradual worsening of your vision. The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.

All enrolled and randomised study participants will attend study visits every 3 weeks during treatment (12 weeks) and follow-up (12 weeks) for PK, safety, tolerability, and efficacy assessments. The Screening period may be up to 45 days. Total duration of the main study may be up to 30 weeks. For participants who received placebo in the main study and choose to participate in the open label (OL) extension, duration of participation will be a further 24 weeks with total participation dependent upon time between completion of the main study and initiation of the OL extension.

This is a Phase 1/2 repeat-dose, open-label, two-arm, parallel group safety and efficacy study of two doses of VP-001 (30 μg and 75 μg) in participants with confirmed PRPF31 mutation-associated retinal dystrophy, including participants previously treated with VP001 in the PLATYPUS Study or WALLABY Study for a minimum of 8 weeks.

Administering subretinal injection of LX107 injection (a gene therapy drug) to patients with retinal dystrophy caused by AIPL1 gene mutation to evaluate its efficacy and safety.

This is a Phase 1/2a, open-label, single-arm, uncontrolled, dose-escalation study evaluating 2 dose levels of iPSC-derived retinal sheets (DSP-3077) administered with a single subretinal uniocular injection in adults with RP. The total duration of participant participation will be approximately 67 months from start of Screening through end of Extension Observation Period Part B. After an initial 2-week period of frequent visits after surgery, the visit frequency will be approximately monthly through Month 4, every 3 months through Month 24, and every 6 months through Month 60. After completion of Study DE101101 (Month 60 Visit or ET Visit) and under a separate process, the Sponsor will collect long-term data following treatment with DSP-3077, annually from 6 years to 15 years after DSP-3077 administration to further characterize the long-term safety of DSP-3077. Participants will be treated in 3 cohorts, with each cohort defined by visual acuity (VA) criteria and dose level of DSP-3077. Each cohort will include 4 participants for a total of 12 participants. Primary objective is to evaluate the safety and tolerability of 2 dose levels of DSP-3077 in adults with RP. Other objectives are to evaluate the safety, engraftment, and potential therapeutic response of 2 dose levels of DSP-3077 in adults with RP and to evaluate DSP-3077-delivery device performance.

Phase 1 of the study includes up to 4 planned dose levels to be administered across up to 4 cohorts. Participants are assigned to receive OpCT-001 in an open-label manner. Dose escalation in Phase 1 is being conducted using a standard 3+3 scheme in which a total of up to 24 legally blind participants (\~3 to 6 per cohort) will receive OpCT-001. Phase 2 is planned to enroll a maximum of 15 participants per cohort in 2 cohorts to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data. Phase 2 participants will be randomized 1:1 to either dose-level cohort. Phase 2 participants and investigators and study site personnel outside of the surgical team will be masked to the Phase 2 OpCT-001 dose-level assignments.

Objective: The objective of the study is to collect adaptive optics (AO) retinal images from human subjects with outer retinal diseases (diseases of the outer retina including photoreceptor, retinal pigment epithelium (RPE), basement membrane or choroidal pathologies) to develop new diagnostic methods, biomarkers, and clinical endpoints. Study Population: Up to fifty (50) healthy volunteers without eye disease (Cohort 1) and up to fifty (50) affected participants with any type of outer retinal disease (Cohort 2) will be enrolled. Design: This is a longitudinal study protocol where participants will be imaged with investigational multimodal AO (mAO) retinal imaging systems that include optical coherence tomography (OCT) and scanning laser ophthalmoscopy (SLO) channels over three years. High resolution OCT and SLO videos will be collected while the instruments automatically detect and correct for image distortion caused by ocular aberrations. In general, videos of different retinal cellular structures will be acquired from several retinal locations using various imaging modes. Outcome Measures: The primary outcomes for this protocol are development of new diagnostic methods and disease biomarkers, investigation of cellular morphological and functional changes due to various outer retinal diseases, and development of new AO clinical endpoints for novel therapies.