Arecibo

Researchers are evaluating the safety and effects of a gene therapy called 4D-150 in adults aged 50 years and older with neovascular wet age-related macular degeneration AMD who are currently receiving anti-VEGF treatments and have shown a positive response. This Phase 12 trial includes a dose-escalation stage, a randomized controlled expansion phase, and additional cohorts to optimize steroid use and extend the population studied. The study also includes substudies to assess dosing safety in the opposite eye and to examine vector shedding. Participants receive a single injection of 4D-150 into the eye on Day 1 at assigned dose levels. Some participants receive aflibercept injections as an active comparator. Following the injection, participants are monitored monthly for 24 months to track safety and treatment effects. Those who receive 4D-150 enter a long-term follow-up period lasting up to five years to evaluate extended safety and the duration of the gene therapys activity. The substudies include one-time dosing in the contralateral eye and assessments of vector shedding, with safety monitored for one year and continued follow-up through five years. Throughout the study, participants undergo regular eye function and structure tests, including visual acuity and retinal thickness measurements using imaging technologies. Researchers track adverse events and the need for additional aflibercept injections, as well as changes in vision and retinal health. Participants must comply with study procedures and agree to use barrier methods during and after treatment to prevent fluid transmission. The study ensures comprehensive safety monitoring, with evaluations continuing for up to five years after treatment administration.

Researchers are evaluating how well different doses of a medicine called BI 3812465 are tolerated in adults with diabetic macular edema that affects the center of the eye. This is the first time BI 3812465 is being given to humans. The study is a Phase IIIa trial conducted in two parts to investigate safety and tolerability of this treatment. In Part 1, a small group of participants receive low, medium, or high doses of BI 3812465 as injections into the back of the eye. Participants starting later receive higher doses only if lower doses are tolerated. In Part 2, a larger group is randomized into low, medium, or high dose groups, also receiving three eye injections of BI 3812465. The treatment is given sequentially with dose escalation based on safety. Participants stay in the study for up to seven months, attending 19 visits to the study site, with some visits possibly conducted at home. During these visits, doctors assess the severity of the eye condition and monitor any health problems related to the treatment. The study measures safety outcomes including dose limiting events and ocular adverse events over up to 169 days after treatment starts.

Researchers are evaluating FWY003 in adults aged 50 and older with geographic atrophy GA caused by age-related macular degeneration AMD. The study aims to understand the relationship between different doses of FWY003 and their effects on the size of GA lesions, as well as the safety and overall effectiveness of the treatment compared to a placebo. This is a randomized, multi-center, double-masked Phase 2 trial designed to gather detailed information about dosing and outcomes. Participants are randomly assigned to one of four groups receiving either one of three dose levels of FWY003 or a placebo. The treatment period lasts 18 months, during which doses are administered and monitored regularly. The study includes extensive assessments of visual function using various eye tests and imaging to measure changes in GA lesion areas. Blood samples are also collected to measure FWY003 levels. Throughout the study, participants undergo periodic evaluations including eye exams, imaging, and visual acuity tests to track changes in their condition and monitor for any adverse effects. Safety is closely observed from the first dose through month 19. The primary measurement is the change in GA lesion size over 18 months. Participants are followed for the full duration of the trial, which lasts until 2029, ensuring comprehensive monitoring of treatment effects and safety.

Researchers are studying an experimental drug called pozelimab, given as an injection into the eye, for people with geographic atrophy GA, a condition where parts of the retina gradually stop working and vision worsens. The study aims to assess how safe and tolerable pozelimab is when used in this way. It also looks at side effects, how the drug moves through the blood and eye fluid, and whether the body produces antibodies that might affect the drugs action. Participants will receive intravitreal injections of pozelimab according to the study protocol. The study is organized in parts, with dose escalation and repeated doses being evaluated. Participants are randomly assigned in a quadruple-masked way to receive treatment. The study focuses on adults aged 50 years and older with GA linked to age-related macular degeneration AMD. During the trial, participants will have their vision tested and eye health monitored through visual acuity measures and assessments of adverse events in the eyes and body. Blood and eye fluid samples will be collected to measure drug levels and antibody responses up to 16 weeks. The main safety outcomes involve tracking treatment-emergent adverse events in the study eye and systemically during the 8 and 16 week periods. The total participation duration can last up to 16 weeks with regular evaluations.

Researchers are evaluating patients with metastatic HER-2-positive breast cancer who are receiving trastuzumab-based therapy and are at risk of heart problems. The study includes two groups one large observational group taking beta blockers, ACE inhibitors, or ARBs alongside trastuzumab, and a smaller randomized group comparing the effects of carvedilol versus no treatment. The aim is to understand the occurrence of heart issues and whether carvedilol might help prevent cardiac side effects from chemotherapy. Participants are assigned to one of three arms based on their current medications. Patients not on beta blockers, ARBs, or ACE inhibitors are randomized to either receive carvedilol orally twice daily or no study intervention. Those already taking these heart medications enter an observational arm without additional treatment. Treatment and observation continue for up to 108 weeks unless disease progression or unacceptable side effects occur. Throughout the study, participants undergo heart function monitoring with echocardiograms every 12 weeks and provide blood samples for biomarker analysis. Researchers track the time to the first sign of heart dysfunction and any cardiac events, as well as adherence to medication and side effects. The study also collects data to develop models predicting heart risk and banks samples for future research. Participant involvement may last over two years with regular assessments to monitor safety and heart health.

Researchers are evaluating how well serum tumor marker directed disease monitoring STMDDM works compared to usual care in patients with hormone receptor positive, HER2-negative metastatic breast cancer. This trial aims to see if monitoring with serum tumor markers can provide similar overall survival outcomes to the standard approach, which involves regular imaging scans. The study also looks at healthcare costs, patient anxiety, and quality of life related to these monitoring methods. Participants are randomly assigned to one of two groups. In the usual care group, patients receive imaging studies at least every 12 weeks and may have serum tumor marker tests as determined by their doctor. In the STMDDM group, patients have blood tests for specific tumor markers every 4 to 8 weeks, and imaging scans are only done if these markers indicate a possible progression of disease. Both groups continue their monitoring for up to 312 weeks unless the disease progresses. During the study, participants undergo regular assessments including blood tests for tumor markers, imaging scans as needed, and questionnaires about anxiety and quality of life. Researchers track overall survival for up to 312 weeks and compare healthcare costs and patient-reported outcomes for up to 48 to 102 weeks. The study also collects data on how often and by what methods disease monitoring is performed, along with patient and physician preferences related to monitoring.

Neovascular age-related macular degeneration nAMD, also called wet AMD, involves abnormal growth of new blood vessels in the retina, the light-sensitive tissue at the back of the eye. This study aims to evaluate the safety and effectiveness of Surabgene Lomparvovec ABBV-RGX-314, an investigational gene therapy, in adults aged 50 and older with previously treated nAMD. Approximately 561 participants will be enrolled worldwide to compare this gene therapy with standard treatment. Participants will be randomly assigned to one of three groups. Two groups will receive a single subretinal injection of Surabgene Lomparvovec at different doses, and the third group will receive Ranibizumab injections as needed through an intravitreal route. The study includes an assessment period starting two weeks before treatment and continues for up to five years. Participants will attend monthly visits at hospitals or clinics for treatment and evaluation. During the study, participants will undergo medical assessments, blood tests, side effect monitoring, and complete questionnaires to track treatment effects and safety. The primary outcomes include the rate of intravitreal anti-VEGF injections up to week 54 and adverse events observed up to five years. Secondary outcomes focus on changes in visual acuity and injection rates over three years. This long-term follow-up will help understand the treatment impact and preservation of vision.

Diabetic Retinopathy DR is a common eye condition caused by diabetes that damages blood vessels in the retina, potentially leading to vision problems or blindness if untreated. This trial evaluates surabgene lomparvovec sura-vec, a one-time gene therapy injected into the suprachoroidal space SCS, for adults with non-proliferative DR NPDR without center-involved diabetic macular edema CI-DME. The study includes Phase 2b, Phase 3, and a bilateral treatment portion, enrolling about 576 adult participants in the United States and Puerto Rico. Participants are randomized to receive either a single SCS injection of sura-vec with prophylactic steroids or a sham injection with artificial tears in one eye during the Phase 2b and Phase 3 portions. Those assigned to sham may later cross over to receive sura-vec. In the bilateral treatment portion, participants receive sura-vec and steroids in both eyes. Follow-up for all participants continues for up to five years after treatment to monitor effects and safety. During the study, participants attend regular clinic visits for medical assessments including vision tests, blood tests, and safety monitoring. Researchers evaluate vision changes using the Diabetic Retinopathy Severity Scale DRSS, track vision-threatening events, and monitor for side effects such as ocular inflammation. Questionnaires are also completed to assess participant experience. The study aims to measure improvements in diabetic retinopathy severity and the occurrence of adverse events over time.