Sacramento

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 the use of [68Ga]Ga DOTA-5G PET/CT imaging in patients diagnosed with metastatic or advanced invasive lobular breast cancer (LBC). This phase I study aims to assess whether this imaging agent can detect cancer lesions, its safety and tolerability, and if it is more sensitive than the current standard 18F-FDG PET/CT imaging. The study plans to enroll 30 patients over a 24-month period to investigate these questions. Participants will receive an injection of up to 5 mCi of [68Ga]Ga DOTA-5G. Imaging will be performed at 1 hour and again at 2 hours after the injection to detect cancer lesions. The study focuses on observing the ability of this imaging to identify lesions and monitoring any adverse events for up to 7 days following the injection to evaluate safety. During the study, participants will undergo PET/CT scans at specified times after receiving the imaging agent. Researchers will collect data on lesion detection and track any side effects using a standardized criteria for adverse events. The main outcomes include the imaging agent's ability to detect lesions 2 hours post-injection and its safety profile within 7 days of administration. Total participation will include these imaging visits and follow-up for safety assessments.

Researchers are evaluating a cancer vaccine called Labvax 3(22)-23 and GM-CSF, alone or combined with pembrolizumab, in treating advanced or metastatic adenocarcinoma, a type of cancer where tumor cells produce excess labyrinthin protein. Labyrinthin is found on adenocarcinoma tumor cells but not on normal tissues, and this study aims to teach the immune system to target these tumor cells. The trial includes both phase 1 and phase 2 parts, with phase 2 focusing on participants with lung and non-lung adenocarcinomas who may receive pembrolizumab to enhance cancer-fighting effects. In phase 1, up to 10 participants receive Labvax 3(22)-23 injections into the skin and GM-CSF injections under the skin at weeks 1, 2, 4, 8, and 12, with evaluations over 16 weeks including follow-up 4 weeks after the last injection. Phase 2 involves up to 67 participants who receive pembrolizumab intravenously every 3 weeks for up to 12 cycles and Labvax 3(22)-23 plus GM-CSF at specific weeks between weeks 7 and 18. Treatment lasts up to 34 weeks if tolerated without tumor growth, followed by safety follow-up and chart reviews for up to 12 months. Participants will undergo exams, scans, and lab tests to monitor side effects, immune response, and tumor response throughout the study. Safety is closely tracked during and after treatment, including dose-limiting toxicities and adverse events in phase 1, and objective response rates up to 12 months in phase 2. The study also monitors participants' performance status, organ function, and life expectancy to ensure safety and eligibility.

Researchers are studying a medicine called enlicitide to reduce low-density lipoprotein cholesterol (LDL-C) in adults with high cholesterol (hyperlipidemia). This trial aims to find out if taking enlicitide together with rosuvastatin, a standard cholesterol-lowering drug, works better than a placebo in lowering LDL-C levels. The study is a Phase 3 trial that is randomized, double-blind, and placebo-controlled to ensure accurate and unbiased results. Participants will receive oral tablets of enlicitide or placebo along with oral capsules of rosuvastatin or placebo. The study compares the effect of enlicitide plus rosuvastatin against placebo to evaluate their impact on LDL-C. The treatment period lasts 8 weeks, during which participants take their assigned medications as directed. During the study, researchers will measure the average percent change in LDL-C from the start of the trial to week 8. Participants will be monitored for safety and any side effects throughout the study. The total participation time includes screening, treatment, and follow-up assessments to evaluate the medicines' effects and safety in adults aged 18 to 64 with hyperlipidemia.

Researchers are investigating ways to prevent cytomegalovirus (CMV) infection in children and adolescents who have received a kidney transplant and weigh less than 40 kilograms. This Phase 1 study aims to understand how the drug letermovir behaves in the body over time and to evaluate its safety and tolerability in this young population. Participants receive letermovir orally, either as tablets or pellets, or through a gastrostomy or nasogastric tube if pellets are used. The treatment is given once daily for seven consecutive days. This study is open-label and single-arm, meaning all participants receive the same treatment, and the study monitors them closely throughout this period. During the study, participants will have blood samples collected before the first dose and at several points up to 24 hours after dosing on Day 7 to measure how the drug is processed by the body. Researchers will also monitor kidney function stability, CMV DNA levels, and any side effects to assess safety. The study focuses on children and adolescents younger than 18 years and weighing between 2.5 and less than 40 kilograms, with a total participation time covering at least seven days of treatment and associated assessments.

Researchers are looking for new ways to treat neovascular age-related macular degeneration (NVAMD). Available standard (usual) treatments for NVAMD, such as aflibercept, may not work for every person. Researchers want to learn if a trial medicine called tiespectus (also called MK-8748 or EYE201) can treat NVAMD. The goal of this trial is to learn if tiespectus works as well as aflibercept to treat NVAMD.

Researchers are evaluating intravitreal EYE103 in participants with neovascular age-related macular degeneration (NVAMD) or macular edema following branch retinal vein occlusion (BRVO). This Phase 2, randomized, dose-masked study includes four patient cohorts: treatment-naive NVAMD participants, incomplete responder (IR) NVAMD participants as monotherapy, IR NVAMD participants receiving EYE103 combined with aflibercept 2.0 mg, and treatment-naive BRVO participants. The study aims to assess safety and efficacy of different doses of EYE103 in these conditions. Participants in each cohort will be randomly assigned to receive either a low or high dose of EYE103 via intravitreal injection. All participants will receive three injections spaced four weeks apart. IR NVAMD participants in the combination therapy cohort will also receive an injection of aflibercept 2.0 mg on Day 1. The timing of enrollment into each cohort is determined by the Sponsor. Participants will undergo safety and efficacy assessments at each injection visit, with some cohorts returning two weeks after injections for further evaluations. Assessments include measuring best-corrected visual acuity using the ETDRS chart, slit-lamp biomicroscopy, fundoscopy, and spectral domain optical coherence tomography (SD-OCT) to measure central subfield thickness. The study concludes at Week 12, which is the end-of-study visit for all participants.

Researchers are investigating whether buntanetap/Posiphen can help treat early Alzheimer's disease in adults aged 55 to 85 years. This Phase 3 study aims to find out if buntanetap/Posiphen improves thinking abilities and daily functioning compared to a placebo. It also evaluates the safety of buntanetap/Posiphen by monitoring any medical issues that participants may experience during the trial. Participants will take either a 30 mg capsule of buntanetap/Posiphen or a placebo capsule by mouth once daily for 18 months. The study includes regular clinic visits at screening, enrollment, and months 1, 3, 6, 9, 12, 15, and 18. During some visits, participants will have brain MRI scans. The study uses a double-blind design, meaning neither participants nor researchers know who receives the active drug or placebo. Throughout the study, participants will complete tests and questionnaires to measure cognitive function and daily living activities, including the ADAS-Cog13 and ADCS-iADL scales. Phone calls before and after visits help track progress and adherence. Safety is closely monitored with ongoing assessments from screening through the 18-month treatment period.

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 expression of delandistrogene moxeparvovec, a gene transfer therapy, in males with Duchenne Muscular Dystrophy (DMD). This open-label Phase 1 study includes participants across different cohorts based on ambulatory status and age. Enrollment for Cohorts 1 through 7 is complete, and Cohort 8 is currently enrolling new participants. Participants receive a single intravenous infusion of delandistrogene moxeparvovec. The study involves multiple cohorts differentiated by age and motor function abilities, including ambulatory and non-ambulatory participants. Some cohorts require stable glucocorticoid use, while others do not yet require steroid treatment. Cohort 8 participants must meet specific motor function scores and are monitored for specific safety concerns such as acute liver injury. Throughout the study, participants undergo assessments including measurement of dystrophin expression by Western blot at baseline and week 12. Safety monitoring includes tracking acute liver injury up to week 72 for Cohort 8. The total participation duration can be up to 156 weeks. The study evaluates changes in dystrophin protein levels as the primary outcome, along with safety and tolerability of the gene therapy over time.