Clinical Research Directory

A Long-term Follow up Study of EXG102-031 in Patients With wAMD (Everest LTFU)

In neovascular (wet) age-related macular degeneration (nAMD), the macula, or the part of the eye that provides the clear, detailed central vision, is being affected by abnormal blood vessel growth and leakage. This leakage affects the vision over time and can lead to severe blurriness or blinding. EXG102-031 was made to block the extra vessel formation which would lead to less leakage affecting the vision. Before EXG102-031 can be tested for its efficacy (if it makes vision better), it must be tested to see if it is safely tolerated to confirm it can continue to be studied in more patients with nAMD. This study is designed to fulfill the long-term safety monitoring of EXG102-031. Participants that enroll in this long-term follow-up study have been treated with EXG102-031 under the main study (EXG102-031-211).

Study to Evaluate Long-Term Safety of Intravitreal OTX-TKI (Axitinib Implant) in Participants With Neovascular Age-Related Macular Degeneration Who Successfully Completed 2-Year OTX-TKI-2023-AMD-301 or OTX-TKI-2023-AMD-303 Study

Study to Evaluate Long-Term Safety of Intravitreal OTX-TKI (Axitinib Implant) in Participants with Neovascular Age-Related Macular Degeneration Who Successfully Completed 2-Year OTX-TKI-2023-AMD-301 or OTX-TKI-2023-AMD-303 Study

A Clinical Study of QL1207H Injection Versus Aflibercept for Neovascular Age-related Macular Degeneration

The goal of this clinical trial is to evaluate the efficacy and safety of QL1207H injection versus aflibercept 8 mg in patients with neovascular age-related macular degeneration. The main question it aims to answer is: • Whether the efficacy and safety of QL1207H and aflibercept 8 mg are similar. Participants will receive injection once every 4 weeks for 3 consecutive doses, followed by injection once every 16 weeks at maximum. Researchers will compare QL1207H group and aflibercept 8 mg group to see if they are similar in improving best corrected visual acuity.

Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-related Macular Degeneration (AQUARIUS)

This is a multi-center, randomized, double-masked, active-comparator-controlled, Phase 3 study in a broad participant population (treatment-naïve and treatment-experienced) with neovascular (wet) age-related macular degeneration (nAMD). The study will evaluate a single intravitreal (IVT) injection of Ixo-vec compared to intravitreal aflibercept (active comparator). The primary endpoint of this study is the mean change in best corrected visual acuity (BCVA) of Ixo-vec compared to an active comparator measured as an average at Weeks 52 and 56. Safety, tolerability, and efficacy will be evaluated throughout the study.

Speculum-Free Intravitreal Injection Using Cotton-Tipped Applicator Retraction: A Randomized Trial of Pain, Procedure Time, Patient Satisfaction, and Safety

This randomized controlled trial compares two techniques for eyelid retraction during intravitreal injection (IVI) of anti-VEGF agents: the standard wire eyelid speculum (Group A) versus cotton-tipped applicator retraction (Group B) in patients with neovascular AMD, diabetic macular edema, or retinal vein occlusion. The study evaluates four outcomes: (1) patient pain perception measured by a 10-cm visual analogue scale immediately after injection; (2) procedure duration from retraction device placement to removal; (3) patient satisfaction assessed by a 5-item Likert scale; and (4) safety including rates of subconjunctival hemorrhage, corneal abrasion, endophthalmitis, and intraocular pressure elevation. A novel syringe cap technique using the Terumo 31G insulin syringe plastic cap as an injection-site marker (3.5 mm for pseudophakic eyes, 5.0 mm for phakic eyes from the limbus) is employed in both groups, replacing the traditional caliper. Randomization is stratified by diagnosis and prior injection history using permuted block randomization (block sizes 4 and 6). The target sample size is 120 patients (60 per group) at Walailak University Hospital, Nakhon Si Thammarat, Thailand.

Efficacy and Safety of LX102 Gene Therapy in Patients With Neovascular Age-related Macular Degeneration (nAMD) (STELLAR)

This is a Phase III, randomized, open-label, active-controlled study to evaluate the efficacy and safety of subretinal injection of LX102 in participants with neovascular age-related macular degeneration. The study will evaluate a single subretinal injection of LX102 compared to an active comparator. The primary endpoint of this study is the mean change from D0 in BCVA based on an average at weeks 40 and 48.

RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD)

This interventional study is being conducted with an investigational gene therapy treatment called ABBV-RGX-314 (also known as RGX-314) and is being developed as a potential one-time gene therapy treatment for neovascular (wet) age-related macular degeneration (wet AMD or nAMD). The typical treatment for nAMD is frequent injections of anti-VEGF therapy. Researchers are testing ABBV-RGX-314 to see if it has similar effects as the current approved standard of care, such as Lucentis® or Eylea® injections. The duration of this study will be up to 52 weeks or for ranibizumab control participants who cross over to ABBV-RGX-314 after week 52, up to 80 weeks post-randomization. The primary outcome measure for this investigational study is to evaluate the mean change in best-corrected visual acuity (BCVA) for ABBV-RGX-314 compared with ranibizumab monthly at the Week 40 visit.

Single Intravitreal (IVT) Injection of 4D-150 in Patients With Macular Neovascularization Secondary to Age-Related Macular Degeneration

A Phase 3, Randomized, Double-Masked, Active-Controlled Trial in Adults with Macular Neovascularization Secondary to Age-Related Macular Degeneration

To Evaluate the Safety, Pharmacokinetics, and Efficacy of GB10 Intravitreal Injection in Patients With Neovascular Age-related Macular Degeneration (nAMD)

This study aims to preliminarily evaluate the efficacy and safety of GB10 intravitreal (IVT) injection for the treatment of patients with neovascular age-related macular degeneration (nAMD). It consists of two parts, single-ascending-dose escalation (SAD) and multiple-ascending-dose escalation (MAD). In SAD, a single IVT of up to 6 doses will be administered to up to 36 treatment-naïve or previously treated patients with nAMD. If the lowest dose is considered safe without dose-limiting toxicity, escalation will proceed to the next higher dose level. At the end of SAD, the two doses that best balance efficacy and safety will be selected and entered into MAD. In MAD, a single IVT of 2 doses will be administered to 12 treatment-naïve or previously treated patients with nAMD, who will be enrolled across the low- to high-dose levels. After GB10 intervention, the participants will undergo tests to evaluate the PK/PD characteristics of GB10 and ocular and non-ocular safety.

Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration

This is a multi-center, randomized, double-masked, active-comparator-controlled, Phase 3 study in a broad participant population (treatment-naïve and treatment-experienced) with neovascular (wet) age-related macular degeneration (nAMD). The study will evaluate a single intravitreal (IVT) injection of Ixo-vec compared to an active comparator. The primary endpoint of this study is the mean change in best corrected visual acuity (BCVA) of Ixo-vec compared to an active comparator measured at an average of Weeks 52 and 56. Safety, tolerability, and efficacy will be evaluated throughout the study.

Study to Assess the Efficacy & Safety of KHK4951 in Patients With Neovascular Age-Related Macular Degeneration

The purpose of this study is to evaluate efficacy and safety of KHK4951 eye drops in patients with nAMD.

Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With nAMD

This is a prospective, observational study designed to evaluate the long-term safety and efficacy of RGX-314. Eligible participants are those who were previously enrolled in a clinical study of nAMD in which they received suprachoroidal space (SCS) administration of RGX-314. Enrollment of each participant in the current study should occur after the participant has completed either the end of study or early discontinuation visit in the previous (parent) clinical study. Participants will be followed for up to 5 years after RGX-314 administration (inclusive of the parent study). As such, the total study duration for each participant may vary depending on when they enroll in the current study following RGX-314 administration in the parent study.

This Project At LMU Looks At How Using AI 2nd Opinion Report to Analyze Retinal Eye Scans Impact Doctors' Decisions About Treatment for Patients with a Specific Eye Disease (nAMD)

This is a research plan from the University of Munich (LMU) that aims to study how the use of AI reports can impact ophthalmologists' decisions regarding treatment for patients with neovascular age-related macular degeneration (nAMD). This disease is a leading cause of vision loss, and while anti-VEGF treatments are effective, they require careful monitoring and retreatment decisions to maximize benefits. The study will involve up to 1000 ophthalmologists with varying levels of expertise. These ophthalmologists will review SD-OCT scans and make treatment decisions before and after reviewing AI-generated reports. The primary objective is to compare these decisions and see how the AI reports influence them. Secondary objectives include assessing the accuracy and safety of the AI reports.

Open-laBel Dose-escalation Study for CRISPR/cas13- Rna TargetInG THerapy for the Treatment of Neovascular Age-related Macular Degeneration in Phase I Trial

Age-related macular degeneration (AMD) leads to severe and irreversible vision loss, while neovascular AMD (nAMD) accounts for 80-90% of AMD blindness. Current anti-VEGF therapies are the standard of care, but these therapies require life-long repeated intraocular injections. These frequent intravitreal injections increase the risk of complications, including submacular hemorrhage, intraocular hypertension, inflammation, and retinal detachment. Therefore, repeated treatments for nAMD place a substantial burden on healthcare systems, patients, and their caregivers. Additionally, approximately 25-35% of individuals with aggressive nAMD show suboptimal responses to the anti-VEGF therapies, experience treatment-extended failure, or require intensive, frequent intraocular injections, and do not prevent irreversible vision loss. HG202 is a CRISPR/Cas13 RNA-editing therapy delivered through one single AAV vector to partially knock down the expression of VEGFA and thus inhibit CNV formation in AMD. The long-term, stable delivery of HG202 following a one-time gene-editing therapy treatment for nAMD may potentially reduce the frequent injections and the potential risks of currently available anti-VEGF therapies since it does not rely on the long-term expression of anti-VEGF antibodies.