Pensacola

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 a culturally-tailored, home-based physical activity program designed to improve physical fitness in Hispanic or Latino/Latina adolescent and young adult childhood cancer survivors. These survivors may face long-term effects such as weight gain, fatigue, and reduced fitness after cancer treatment, with Hispanic or Latino/Latina individuals potentially at higher risk. The study aims to increase moderate to vigorous physical activity (MVPA) through a mobile health and social media intervention. The study has two stages. Stage 1 involves developing the intervention using feedback from 20 Latinx survivors who speak either English or Spanish. Stage 2 is a randomized controlled trial comparing the intervention group with a control group that only uses a Fitbit tracker. The intervention group receives Fitbit trackers, weekly reminders, goal-setting sessions, social media peer support 2-3 times a week, badges, monthly Zoom meetings, and may choose a physical activity partner who also receives support. After 12 weeks, a 4-week maintenance phase continues these supports with less structure. The control group wears a Fitbit daily for 12 weeks without additional support. Participants wear Fitbit trackers daily, attend weekly sessions, post on social media, and complete interviews and questionnaires. Researchers measure changes in physical activity levels, sedentary time, quality of life, and cardiometabolic health indicators. Data is collected using Fitbit devices, interviews, and surveys, with follow-up over 12 weeks plus maintenance. Safety and acceptability of the intervention are also assessed throughout the study.

The purpose of this study is to assess the long-term safety and tolerability after an intravitreal injection (a shot of medicine into the eye) of JNJ-81201887 administered in parent clinical studies.

Researchers are investigating the effects of multiple doses of vosoritide and comparing its therapeutic dose to human growth hormone (hGH) in children with idiopathic short stature (ISS). This Phase 2 study aims to understand how these treatments influence growth in this population. After an initial observation period of at least 6 months to measure baseline growth, participants are randomly assigned to receive either vosoritide, placebo, or hGH (the latter only in the United States). Those in the vosoritide and placebo groups undergo up to 6 months of randomized treatment, followed by open-label vosoritide until they reach near-final adult height or at least age 16 for females or 18 for males. Participants assigned to hGH receive open-label treatment for a minimum of 4 years. Throughout the study, safety is carefully monitored with clinical and imaging assessments focused on hips and lower extremities, as well as watching for hypotension, fractures, and slipped capital femoral epiphysis. An independent Data Monitoring Committee reviews safety data regularly. Study visits include a treatment completion visit about 4 weeks after the last dose, and follow-up assessments may continue annually through the end of the study. Key outcome measures include changes in annualized growth velocity at 6 months and changes in height and height Z-score after 4 years.

Researchers are conducting a Phase 2, multicenter platform study to evaluate the safety and effectiveness of several investigational treatments for adults with moderately to severely active Inflammatory Bowel Disease (IBD), including Crohn's Disease and Ulcerative Colitis. The study focuses on assessing multiple experimental oral or injectable therapies to better understand their effects on these conditions. Participants will receive one of the study drugs, MT-501 or MT-201, as part of this evaluation. The study aims to gather data on how these treatments perform in terms of safety, how the body processes them (pharmacokinetics), and their biological effects (pharmacodynamics). Treatment effects will be measured over a period of up to 13 weeks. During the study, participants will be monitored for any side effects, serious adverse events, and laboratory test changes. Researchers will also assess the participants' clinical remission status and improvements seen through endoscopic evaluations at 12 to 13 weeks. The total involvement duration includes screening and treatment periods, with careful tracking of outcomes and safety throughout.

Researchers are evaluating the effectiveness, safety, tolerability, and pharmacodynamics of multiple doses of APL-3007 combined with Syfovre/Pegcetacoplan (APL-2) in patients aged 60 years and older diagnosed with geographic atrophy secondary to age-related macular degeneration. This Phase 2, randomized, placebo-controlled, multicenter, masked study focuses on measuring changes in retinal pigment epithelium lesions using advanced artificial intelligence-based SD-OCT imaging. Participants will receive either the combination of APL-3007 with pegcetacoplan (APL-2) or a placebo. The study includes a treatment period with multiple doses administered, aiming to assess the impact on geographic atrophy lesions over a 12-month period. Syfovre injections at 6-8 week intervals prior to enrollment are part of the inclusion criteria. During the study, participants will undergo various eye imaging assessments such as OCT and FAF to monitor lesion size and progression. Researchers will evaluate changes in lesions at month 12 compared to baseline. Safety and tolerability will be closely monitored through laboratory tests, clinical evaluations, and vaccination status requirements. The study duration includes regular visits for treatment administration and monitoring over at least one year.

Researchers are evaluating a new approach to prevent cardiovascular events in patients at increased risk due to age and conditions like type 2 diabetes, prediabetes, or metabolic syndrome but without known symptomatic cardiovascular disease. The study compares a Cleerly Coronary Artery Disease (CAD) Staging System-based care strategy with standard risk factor-based care to see if the former can better reduce cardiovascular events. The Cleerly system uses imaging to visualize and quantify coronary artery disease and guides personalized treatment and education based on this assessment. The trial uses the Cleerly CAD Staging System device, which employs a proprietary algorithm to detect and stage coronary artery disease and generate a risk score to guide treatment decisions. Participants receive either this stage-based care or the usual care based on traditional risk factors. The study is prospective, randomized, and pragmatic, designed to follow patients over an average of 3.5 years to compare cardiovascular event outcomes between these two care approaches. Participants will be monitored through cardiovascular event tracking throughout the study period. Data collected includes imaging results, risk scores, and treatment adherence to evaluate the impact of the care strategies. The primary outcome is the comparison of cardiovascular event risk between the Cleerly stage-based care and risk factor-based care groups. The study also includes ongoing safety monitoring and personalized management by a cardiologist-led team via digital communication devices.

Researchers are evaluating azetukalner as a monotherapy in adults diagnosed with moderate-to-severe Major Depressive Disorder (MDD). This Phase 3, multicenter, randomized, double-blind, placebo-controlled study aims to assess the clinical efficacy, safety, and tolerability of azetukalner compared to placebo. Participants must be adults between 18 and 74 years old, experiencing a current major depressive episode confirmed by standard diagnostic criteria and lasting between 6 weeks and 24 months. Participants are randomly assigned to receive either azetukalner 20 mg or a placebo, both taken orally once daily with food (preferably with the evening meal) for 6 weeks. The study compares these two groups to determine the impact of azetukalner on depressive symptoms. The trial maintains double-blinding to ensure unbiased assessment of outcomes. During the study, participants undergo regular assessments including evaluations of depressive symptoms using the Hamilton Depression Rating Scale (HAMD-17) at baseline and at Week 6. Safety and tolerability are monitored throughout the treatment period. The total participation time corresponds to the 6-week treatment phase, during which symptom changes and adverse events are closely observed.

Researchers are evaluating corneal endothelial cells in people aged 50 and older who have neovascular age-related macular degeneration (nAMD). The study focuses on participants treated with the Port Delivery System (PDS) refilled every 24 weeks. This Phase IV, open-label trial aims to understand changes in corneal endothelial cell density over time in the eye receiving treatment compared to the fellow eye. The study involves delivering ranibizumab 100 mg/mL via the PDS implant. Supplemental treatment with intravitreal injections of ranibizumab (0.5 mg of a 10 mg/mL formulation) in the study eye may be given if needed. If participants stop the study treatment, they may receive intravitreal ranibizumab injections based on the investigator's judgment. Treatment and monitoring occur over at least 48 weeks. Participants will undergo detailed eye examinations including specular microscopy to measure corneal endothelial cell density at baseline and week 48. Historical visual acuity and imaging data will be reviewed. Researchers will monitor safety, disease activity, and treatment response through visual acuity assessments, optical coherence tomography, and other imaging techniques. The main outcome is the percent change in corneal endothelial cell density in the treated eye compared to the fellow eye after 48 weeks.

Researchers are evaluating the safety and effectiveness of divarasib combined with pembrolizumab compared to pembrolizumab with pemetrexed and either carboplatin or cisplatin. The study focuses on adults with advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) that has a specific KRAS G12C mutation. This is a Phase III trial aiming to improve first-line treatment options for these patients. Participants will receive one of two treatment combinations. One group will take divarasib orally once daily along with pembrolizumab given through an intravenous infusion every three weeks. The other group will receive pembrolizumab with pemetrexed and either carboplatin or cisplatin, all administered by intravenous infusion every three weeks. Treatment schedules and dosages are carefully monitored during the study. Throughout the study, participants will be regularly assessed for progression-free survival and overall survival, with follow-up lasting up to approximately five years. Researchers will perform various evaluations including tumor measurements and safety monitoring. This long-term observation helps to understand the treatments' effects and safety over time, supporting informed decisions for future lung cancer therapies.