Gainesville

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 4D-710, an investigational gene therapy, in adults with cystic fibrosis (CF) lung disease who cannot use or tolerate CFTR modulator therapy. This Phase 1/2, multicenter, open-label trial also includes a sub-study assessing 4D-710 in adults with advanced CF lung disease or frequent lung flare-ups despite using CFTR modulators. The study aims to assess the safety, tolerability, and early effectiveness of this gene therapy in these populations. The trial involves a single dose of 4D-710, which is a gene therapy using a specialized virus to deliver a modified CFTR gene. Participants receive this treatment once, and those in the sub-study must be on a stable CFTR modulator regimen for at least 60 days before screening and continue it during a 24-month observation period. The study monitors participants with CF lung disease ranging from moderate to advanced stages. During the study, participants undergo regular evaluations including lung function tests, oxygen level checks, and monitoring for adverse effects. Researchers track the occurrence and severity of any side effects over a 60-month period. The study also includes assessments of lung health, medication adherence, and clinical status to understand the therapy's impact and safety over time.

This research aims to evaluate the effects of litifilimab (BIIB059), a monoclonal antibody, in adults with active subacute or chronic cutaneous lupus erythematosus (CLE), with or without systemic lupus erythematosus (SLE). Participants have active skin symptoms of CLE that have not improved with antimalarial therapy or had difficulties continuing that treatment. The study focuses on reducing skin disease activity using several scores including CLA-IGA-R and CLASI, while also assessing safety, immune response, and quality of life. Participants will be randomly assigned to receive either litifilimab or a placebo injection under the skin every four weeks during a 24-week double-blind period where neither participants nor researchers know which treatment is given. After this, all participants will receive litifilimab injections every four weeks for an additional 28 weeks. Those who complete the treatment may join a long-term extension study or enter a follow-up safety period lasting up to 24 weeks. Total participation may last up to 80 weeks. Throughout the study, researchers will monitor skin disease activity using the CLA-IGA-R erythema score and the CLASI-A activity score to see how many participants improve. They will also assess safety, tolerability, immune system effects, and participants' quality of life using questionnaires. These evaluations occur regularly during both treatment periods and follow-up to understand the impact of litifilimab on CLE symptoms and overall health.

Researchers are evaluating a community health worker-delivered mental health counseling intervention aimed at reducing anxiety and depression in people living with HIV in Florida. This pilot randomized controlled trial tests an adapted positive affect counseling program delivered remotely through a mobile health (mHealth) app. The purpose is to assess the preliminary effectiveness of this approach in improving mental health outcomes among adults with HIV who experience anxiety and depression. Participants will be randomly assigned to one of two groups. The intervention group will receive approximately five weekly 60-minute individual counseling sessions via secure video calls on the PositiveLinks app, along with enhanced mental health resources within the app. If a participant does not show improvement, they will be referred to a licensed mental health professional. The control group will use the standard version of the PositiveLinks app alongside usual mental health care available in the HIV clinic. During the study, data will be collected from participants' electronic medical records and the mHealth app for about six months after enrollment. Researchers will measure changes in anxiety and depression levels at three months to evaluate the intervention's impact. Participants will provide informed consent before joining the study, and their progress will be monitored through app usage and clinical assessments to ensure safety and effectiveness.

Researchers are investigating sacituzumab tirumotecan (MK-2870) alone or combined with other treatments to treat certain gastrointestinal cancers. These include colorectal cancer that cannot be removed by surgery or has spread, advanced pancreatic ductal adenocarcinoma, and biliary tract cancer. The study aims to understand the safety and tolerability of sacituzumab tirumotecan and measure how many participants respond to the treatment by having their cancer shrink or disappear. Participants may receive sacituzumab tirumotecan by intravenous infusion alone or with other anticancer drugs such as fluorouracil (5-FU), leucovorin or levoleucovorin, cisplatin, and pembrolizumab. Rescue medications like diphenhydramine, H2 receptor antagonists, acetaminophen, dexamethasone, and a steroid mouthwash are given to prevent infusion reactions and oral side effects. Supportive care treatments for side effects, including antidiarrheal and antiemetic agents, are allowed throughout the study. During the study, researchers monitor participants for dose-limiting toxicities within about 4 weeks and track adverse events, treatment discontinuations, and tumor response over up to approximately 63 months. Assessments include safety evaluations and measuring cancer response using standardized criteria. This long-term follow-up helps evaluate both the effectiveness and safety of the treatments being studied.

This research aims to evaluate the effectiveness and safety of leriglitazone in adult male patients diagnosed with cerebral adrenoleukodystrophy (cALD), a progressive neurological condition. The study is a Phase 3 clinical trial focusing on males aged 18 years and older who have specific brain lesions related to cALD. It excludes patients who are candidates for or willing to undergo hematopoietic stem cell transplantation (HSCT). Participants will be randomly assigned to receive either leriglitazone, given once daily at a dose of 15 mg/ml with an initial volume of 10 ml, or a matching placebo that looks and tastes the same but contains no active drug. The treatment period includes planned assessments at 18, 27, and 36 months, with the primary measure being the time until death or becoming bedridden requiring permanent ventilatory support. Throughout the study, participants will be monitored regularly to assess neurological function and overall health. Researchers will collect data on brain lesion progression, functional disabilities, and cognitive status to evaluate treatment impact and safety. The total duration of treatment and follow-up spans up to 36 months, with interim analyses at 18 and 27 months to evaluate ongoing results.

Researchers are evaluating a new treatment approach for patients with triple negative breast cancer (TNBC), an aggressive form of breast cancer that often leads to deadly metastatic disease within two years of diagnosis, especially if there is no complete response to initial chemotherapy. This study focuses on patients with locally advanced TNBC who remain at high risk despite surgery and other treatments, aiming to eliminate remaining cancer cells and improve survival rates. It involves both phase 1 and phase 2 clinical trials to explore this approach in metastatic and earlier stage TNBC patients. The study includes two main phases. Phase 1 enrolls patients with metastatic TNBC or hormone-resistant, HER2/neu-negative breast cancer and tests escalating doses of sarilumab combined with capecitabine chemotherapy over cycles lasting 21 days. Sarilumab is given subcutaneously every three weeks before capecitabine, which is taken orally twice daily for 14 days per cycle. Phase 2 enrolls stage I to III TNBC patients with high-risk residual disease after neoadjuvant therapy, receiving sarilumab plus capecitabine for the first four of eight cycles, with sarilumab dosed at the phase 1 determined level. Blood and optional bone marrow samples are collected during treatment. A parallel biological study observes patients receiving standard capecitabine alone. Participants will undergo regular assessments including blood tests to measure treatment effects and safety, with optional bone marrow sampling. The study will monitor dose tolerance and toxicity during phase 1, and the reduction of circulating and disseminated tumor cells in phase 2. Follow-up includes observing participants for up to 14 weeks from baseline to evaluate outcomes and side effects. Total participation duration depends on the phase and treatment schedule, with close monitoring to assess treatment response and safety.

Researchers are evaluating the long-term safety and effects of nerandomilast in people with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF) who have previously completed treatment with nerandomilast in earlier studies. The study aims to understand how well participants tolerate nerandomilast over time, and whether it helps improve lung function, delays symptom worsening, reduces hospital visits, or impacts survival. This is a Phase 3 open-label extension trial. Participants take nerandomilast tablets daily for up to 1 year and 10 months while continuing their usual pulmonary fibrosis treatments. The study follows an open-label design where all participants receive nerandomilast. There are no placebo or comparator groups in this extension phase. Throughout the study, participants regularly visit their doctors for health assessments and lung function tests. Doctors monitor any health problems or side effects experienced during treatment. The main outcome measured is whether participants experience any adverse events up to the final follow-up visit, which occurs at week 99. This close monitoring helps evaluate the long-term safety and potential benefits of nerandomilast in this patient group.

Researchers are evaluating the safety and effectiveness of rilvegostomig compared to pembrolizumab, both combined with platinum-based doublet chemotherapy, as initial treatments for patients with metastatic non-squamous non-small cell lung cancer (mNSCLC) whose tumors express PD-L1. This Phase III, randomized, double-blind, global study focuses on patients whose tumors meet the PD-L1 expression threshold of 1% or higher and do not have certain genetic mutations or rearrangements that would require other targeted therapies. Participants receive either rilvegostomig or pembrolizumab intravenously on the first day of each 21-day treatment cycle. Both groups also receive platinum-based chemotherapy drugs such as carboplatin or cisplatin, administered intravenously up to four cycles, along with pemetrexed given intravenously on Day 1 of each cycle. The study monitors these treatments as first-line therapy for metastatic non-squamous NSCLC. During the study, participants undergo regular assessments including imaging scans to measure tumor size and response, as well as evaluations of organ and bone marrow function. Researchers track overall survival and progression-free survival for up to approximately five years. Safety is closely monitored throughout, and patients are followed long-term to assess outcomes related to treatment effectiveness and tolerability.

This is a Phase III, two-arm, randomized, double-blind, global, multicenter study assessing the efficacy and safety of rilvegostomig compared to pembrolizumab, both in combination with platinum-based doublet chemotherapy, as a first-line (1L) treatment for patients with squamous metastatic non-small cell lung cancer (mNSCLC) whose tumors express PD-L1 (tumor cells (TC) ≥ 1%).