Arecibo

Researchers are investigating the safety and effects of 4D-150 gene therapy in adults aged 50 and older with neovascular (wet) age-related macular degeneration (AMD) who are already receiving anti-VEGF treatment and have shown a clinical response. This Phase 1/2 trial includes dose-escalation and randomized, controlled, masked expansion phases, aiming to evaluate 4D-150 administered by intravitreal injection in one eye, with additional substudies assessing dosing in the second eye and vector shedding. Participants will receive a one-time dose of 4D-150 by injection into the study eye, followed by monthly assessments for 24 months to monitor safety and effectiveness. Those who receive 4D-150 will then enter a long-term follow-up period up to 5 years to assess ongoing safety and the duration of treatment effects. Substudies include one for contralateral eye dosing and another to characterize vector shedding, with participants monitored regularly for safety through one year and continuing long-term follow-up through year 5. Throughout the study, participants will undergo tests of visual and retinal function and structure, with assessments performed monthly initially and safety monitored for up to five years. Researchers will track treatment-emergent adverse events, serious adverse events, and any significant changes in safety parameters. Participants must comply with study procedures and visits, and males receiving 4D-150 will be advised to use barrier methods during intercourse for six months to prevent fluid transmission.

Researchers are evaluating ABBV-RGX-314, a novel one-time gene therapy, for treating neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD causes vision loss due to abnormal blood vessel growth in the retina and affects millions in the United States, Europe, and Japan. Current treatments require frequent eye injections, which can be burdensome and may lead to reduced vision over time. This Phase 3 study aims to compare the effectiveness and safety of two doses of ABBV-RGX-314 against the standard anti-VEGF drug, aflibercept, in people with wet AMD. Participants will be randomly assigned to receive one of two doses of ABBV-RGX-314 gene therapy or aflibercept injections. The gene therapy involves a one-time subretinal injection delivering a gene that produces an anti-VEGF protein to help control abnormal blood vessels. In addition, a bilateral treatment substudy will examine safety and effectiveness when both eyes are treated in participants with wet AMD in both eyes. This substudy will enroll up to 15 participants for at least 50 weeks of follow-up. During the study, participants will have their vision measured regularly to assess changes in best-corrected visual acuity (BCVA). Safety will be monitored by recording any eye-related adverse events and serious side effects. Participants will be followed for up to 54 weeks or more to evaluate how well the gene therapy maintains or improves vision compared to aflibercept and to assess overall treatment safety and tolerability.

Researchers are studying patients with metastatic HER-2-positive breast cancer who are receiving trastuzumab-based treatments to understand the risk of heart problems related to their cancer therapy. The study includes two groups: one large observational group of patients already taking beta blockers, ACE inhibitors, or ARBs alongside their cancer treatment, and a smaller randomized group comparing patients who receive carvedilol, a heart medication, to those who do not. The trial aims to assess how often heart issues occur and whether carvedilol can help prevent heart damage from chemotherapy. It also investigates biomarkers and heart function measures as predictors of cardiac risk. In the randomized part, patients not already on beta blockers, ACE inhibitors, or ARBs are assigned to receive carvedilol twice daily or no additional treatment for up to 108 weeks, with treatment cycles repeated every 12 weeks if there is no disease progression or unacceptable side effects. Patients already taking these heart medications join the observational cohort and are monitored for up to 108 weeks without any change in their therapy. The study collects blood samples and performs regular heart imaging to evaluate heart function and strain. Participants will have regular echocardiograms every 12 weeks to monitor heart function, with both local and central readings compared. Blood samples are collected for biomarker analysis, and patient health status is assessed throughout the study. The main outcome measured is the time until any heart dysfunction is first detected, followed for up to 108 weeks. The study also tracks interruptions in cancer therapy due to heart problems and explores genetic and plasma markers that might predict heart risk. Participants are followed closely for safety and treatment effects during the entire study period.

Researchers are evaluating how well serum tumor marker directed disease monitoring (STMDDM) works for patients with hormone receptor positive, HER2 negative metastatic breast cancer. The study compares STMDDM with the usual care approach to see if overall survival is not worse using STMDDM. The trial also looks at healthcare costs, patient anxiety, quality of life, and preferences related to disease monitoring. Patients are randomly assigned to one of two groups. One group receives usual care with imaging at least every 12 weeks and other monitoring at the doctor's discretion for up to 312 weeks if the disease does not progress. The other group has their serum tumor markers checked every 4 to 8 weeks, with imaging only if markers are elevated, also for up to 312 weeks without progression. Additional assessments include quality-of-life and anxiety questionnaires. Throughout the study, participants undergo regular evaluations including imaging, blood tests for tumor markers, and patient-reported outcome questionnaires. Researchers track overall survival up to 312 weeks after randomization, along with healthcare costs and patient experiences. Participants must provide informed consent and are monitored for safety during the study period.

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 Phase 3b study is evaluating how safe and effective Surabgene Lomparvovec (ABBV-RGX-314), an investigational gene therapy, is in treating adults aged 50 and older who have previously been treated for nAMD. Approximately 561 participants will be enrolled at about 150 sites worldwide to compare different treatment approaches in a real-world setting. Participants will be randomly assigned to one of three groups. Two groups will receive a single subretinal injection of Surabgene Lomparvovec, while the third group will receive Ranibizumab injections as needed. Ranibizumab is given as an intravitreal injection into the jelly-like tissue inside the eye, whereas Surabgene Lomparvovec is injected between the retina and the back of the eye. The assessment period begins two weeks before treatment and continues for up to 5 years, allowing long-term monitoring of treatment effects. Participants will attend monthly visits at a hospital or clinic to monitor their eye health. These visits include medical assessments, blood tests, side effect checks, and questionnaires. Researchers will measure the annualized rate of anti-VEGF injections needed and record any adverse events over the 5-year period. The study involves a higher treatment burden than standard care, but aims to gather detailed information on long-term vision preservation and safety.