Chapel Hill

Researchers are studying a new MRI technique called 19F MRI that uses a special inert gas to visualize lung ventilation in children and adolescents aged 6 to 17 years with cystic fibrosis (CF) who have normal lung function. This approach has not been tested in children before. The study also includes healthy children of the same age group for comparison. The goal is to see if this imaging method is feasible and to compare its sensitivity with other lung function tests like spirometry and multiple breath nitrogen washout (MBW) for detecting mild lung disease. Participants will inhale a biologically inert contrast gas called perfluoropropane while undergoing 19F MRI scans. Images will be taken both during breath-holds and with a new free-breathing technique that does not require holding breath, to see how well each method works. The study is cross-sectional and involves one visit to complete the imaging and lung function tests. During the study visit, children will perform spirometry and MBW tests alongside the MRI scan. Researchers will assess how well children tolerate the procedures and measure parental and child acceptability scores. The main outcomes include participation and completion rates over the two-year recruitment period and the feasibility of using this imaging method in pediatric patients. Safety and lung function stability will also be monitored.

Essential tremor (ET) is a common neurological disorder that can cause significant challenges in daily function and mental well-being, especially when medications fail to control symptoms effectively. This research investigates a new approach called 3-D tractography Vim-Focused Ultrasound Ablation (Vim-FUSA) to improve the accuracy and safety of brain ablation for ET. The study also explores how intraoperative magnetic resonance (i-MR) imaging can help distinguish ablated tissue from swelling and predict treatment outcomes. Participants will undergo a specialized MR-guided focused ultrasound procedure enhanced by 3-D tractography, which visually maps nerve pathways in the brain. The process includes head preparation, placement of a stereotactic frame, imaging, careful targeting with low-energy sonications, and therapeutic ablation of the ventral intermediate nucleus (Vim) responsible for tremor. Clinical testing will be performed during the procedure to monitor tremor and side effects. The study includes both experimental animal testing comparing 3-D tractography guided ablation versus standard methods, and a human phase-II study to assess safety, feasibility, and preliminary effectiveness. During the study, participants will have tremor assessments videotaped at baseline and 12 weeks after treatment. Researchers will monitor side effects during surgery and use intraoperative imaging to evaluate tissue changes. The main outcomes measured include the number of participants without side effects during surgery and changes in tremor severity from baseline to three months. Participants must be willing to attend all follow-up visits and undergo MR imaging for ongoing evaluation.

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 looking for new medicines to prevent HIV-1 (Human Immunodeficiency Virus Type 1) infection. The goals of this study are to learn: * If taking MK-8527 once a month works to prevent HIV-1 infection as well as or better than a standard (usual) pre-exposure prophylaxis (PrEP) taken once a day * About the safety of MK-8527 and if people tolerate it

Researchers are looking for ways to treat germinal center B-cell-like diffuse large B-cell lymphoma (GCB DLBCL). DLBCL is a fast-growing blood cancer that affects B-cells. GCB is a type of DLBCL that affects young B-cells that are still maturing. The goal of this study is to learn if more people who receive zilovertamab vedotin (MK-2140) and R-CHP have the cancer respond (go away) than those who receive polatuzumab vedotin and R-CHP.

Researchers are evaluating the safety, tolerability, pharmacometrics, and effectiveness of Claseprubart (DNTH103) in adults with multifocal motor neuropathy (MMN) in this Phase 2 clinical trial. The study aims to understand how this drug works and its safety profile compared to a placebo in people diagnosed with definite or probable MMN who have shown responsiveness to immunoglobulin (Ig) treatment. Participants receive an initial intravenous loading dose on Day 1, followed by subcutaneous doses of Claseprubart or placebo every two weeks from Week 1 through Week 15. This randomized, double-blind, placebo-controlled setup allows researchers to carefully compare the effects of the drug versus placebo over the course of the treatment period. Throughout the study, participants will be monitored for any treatment-emergent adverse events or serious adverse events from baseline to Week 17. Researchers will collect safety and efficacy data, including pharmacometric assessments, to evaluate the drug's impact. Participants are involved in regular assessments and follow-ups during this time to ensure thorough observation and data collection related to safety and treatment response.

Researchers are evaluating a phase II, randomized, double-blind, placebo-controlled study involving 48 adults aged 18 to 70 years who have recently been diagnosed with acute HIV infection. The study aims to assess whether adding a combination of HIV-specific broadly neutralizing antibodies (bNAbs) to standard antiretroviral therapy (ART) is safe and whether this combination can delay the return of detectable HIV viral levels, reduce viral reservoirs, and improve HIV-specific immune responses compared to ART with placebo. Participants receive either the combination bNAbs or placebo along with ART at the start of the study. The bNAbs include VRC07-523LS given as a 10 mg/kg intravenous infusion over 15 to 30 minutes and PGT121.414.LS given as a 5 mg/kg intravenous infusion over 30 to 60 minutes, both administered once at entry. ART consists of daily oral tablets containing bictegravir, emtricitabine, and tenofovir alafenamide. The study includes multiple steps: initial treatment with ART and antibody or placebo infusions (Step 1), analytic treatment interruption (ATI) to monitor viral rebound (Step 2 and Step 3), and ART restart when criteria are met (Step 4). Throughout the study, participants undergo regular visits with laboratory tests to monitor HIV viral load, CD4+ T-cell counts, safety labs, and pregnancy tests when applicable. Researchers track the occurrence of significant adverse events related to the study antibodies and measure the time it takes for HIV viral levels to rebound after stopping ART. Participants must adhere to contraceptive requirements and use barrier methods to prevent HIV transmission during ATI. The total study duration includes treatment, interruption, and follow-up phases lasting up to 24 weeks or longer depending on individual progress.

Researchers are evaluating the safety and potential benefits of VHB937 in people aged 50 to 85 years with early Alzheimer's disease, including those diagnosed with Mild Cognitive Impairment due to Alzheimer's or mild Alzheimer's disease. This Phase II, multicenter, randomized, double-blind, placebo-controlled study aims to assess how VHB937 affects memory, thinking abilities, daily activities, and brain changes, while also studying how the body processes and responds to the treatment. The study includes an initial 72-week double-blind phase followed by an extension period. Participants will receive either VHB937 solution for infusion or a placebo solution through infusion during the 72-week double-blind phase. The study compares these two groups to evaluate the effects and safety of VHB937 in early Alzheimer's disease. After the double-blind phase, participants may continue in an extension period for further observation. Treatment involves regular infusions under controlled conditions throughout the study. During the study, participants and their study partners will attend visits for assessments including memory and cognitive tests, evaluations of daily functioning, brain imaging, and biomarker analysis from cerebrospinal fluid or PET scans. Researchers will monitor safety, record any side effects, and track changes using the Clinical Dementia Rating scale (CDR) over 72 weeks. The study requires a reliable partner to accompany participants to visits, and overall participation includes monitoring during treatment and the extension phase to thoroughly assess VHB937's effects and safety.

This research aims to evaluate a decision-making support intervention called the University of Rochester-Geriatric Oncology assessment for Acute Myeloid Leukemia (UR-GOAL) in older adults diagnosed with acute myeloid leukemia (AML) and their caregivers. The study is a multicenter randomized controlled trial conducted at four sites, comparing UR-GOAL to an attention control. The purpose is to see if UR-GOAL can reduce patient distress, improve shared decision making between patients and healthcare providers, and reduce decisional conflict. Participants will be randomly assigned to either the UR-GOAL intervention or the attention control group. Those in the UR-GOAL group will watch an AML educational video, complete preference assessments, and receive a summary report to review and discuss during upcoming clinical visits. Caregivers, if involved, will also view the video and receive a summary report. Oncologists will be provided with the summary report to support discussions with patients. This approach is behavioral and designed to enhance decision-making support. During the study, participants will be assessed using the Distress Thermometer Questionnaire at one month to measure distress levels. The study involves patient and caregiver participation in viewing materials and discussing care plans with oncologists. The researchers will monitor shared decision making, patient distress, and decisional conflict to understand the impact of the intervention. The total participation duration aligns with the study visits and assessments over the course of initial treatment decisions.