Richmond

This research aims to evaluate the safety, tolerability, and early effectiveness of 4D-710, an investigational gene therapy, in adults with cystic fibrosis (CF) lung disease who cannot use or tolerate CFTR modulator therapy. It also includes a sub-study to assess this gene therapy in adults with advanced CF lung disease or frequent lung flare-ups while on current modulator treatments. The study is a Phase 1/2, open-label, multicenter trial focused on this population. Participants receive a single inhaled dose of 4D-710, a gene therapy using an adeno-associated virus to deliver a modified CFTR gene. The trial has different groups: those who cannot use modulator therapy will get varying doses to find the best dose for further study, while those on modulator therapy receive selected doses in the sub-study. The treatment is given once, and doses are explored and expanded to identify recommended levels. During the trial, participants are monitored for up to 60 months to track any side effects and overall safety. The main measure is the number and severity of adverse events. Participants will have lung function tests, oxygen level checks, and other health assessments throughout the observation period. The study will gather detailed safety and tolerability data to guide future research on this gene therapy in CF lung disease.

Researchers are evaluating litifilimab (BIIB059), a monoclonal antibody, in adults with active cutaneous lupus erythematosus (CLE). This includes those with subacute or chronic CLE, with or without systemic lupus erythematosus (SLE), who have not responded well or tolerated antimalarial treatments. The study aims to assess how litifilimab affects skin disease activity using scoring tools such as CLA-IGA-R and CLASI, as well as its safety and impact on quality of life. The study has two parts: Part A and Part B. After screening, participants are randomly assigned to receive either litifilimab or a placebo injection under the skin every four weeks for 24 weeks in a double-blind setup. After this, all participants receive litifilimab for another 28 weeks. Those who finish may join a long-term extension study or enter a safety follow-up lasting up to 24 weeks. Treatment involves regular injections and monitoring during these periods. Participants will undergo assessments of skin symptoms, immune responses, and quality of life using questionnaires. Researchers will measure outcomes like the percentage of participants achieving low skin redness scores and significant reductions in skin disease activity. Safety monitoring continues through the study and follow-up, with total participation lasting up to 80 weeks.

Researchers are evaluating an investigational drug called OHB-607 to prevent Bronchopulmonary Dysplasia (BPD), a common chronic lung disease in extremely premature infants. The study compares infants receiving OHB-607 to those receiving standard neonatal care to see if the drug can reduce the incidence of severe BPD or death by 36 weeks postmenstrual age. This is a Phase 2b, randomized, open-label study involving infants born between 23 weeks 0 days and 27 weeks 6 days gestational age. Participants in the trial will be randomly assigned to one of two groups. One group will receive a continuous intravenous infusion of OHB-607 from birth until 29 weeks and 6 days postmenstrual age. The other group will receive standard neonatal care without the investigational drug. This approach allows researchers to compare the effects of OHB-607 against routine care practices for preventing lung disease in these infants. During the study, infants will be closely monitored through 36 weeks postmenstrual age and up to 24 months corrected age. Researchers will assess lung health, including the incidence and severity of BPD, time to weaning off respiratory support, and other complications such as intraventricular hemorrhage and retinopathy of prematurity. Developmental outcomes will also be measured using standardized scales at 24 months corrected age. Safety assessments and long-term follow-up are included to understand the drug's effects over time.

Researchers are studying a medicine called enlicitide to lower low-density lipoprotein cholesterol (LDL-C) in people with high cholesterol. This trial aims to learn if enlicitide given with rosuvastatin, a standard cholesterol-lowering treatment, works better than a placebo. The study is a phase 3, randomized, double-blind, placebo-controlled trial evaluating the combination's effect on LDL-C levels in adults with hyperlipidemia. Participants will be assigned to one of four groups: enlicitide with a placebo matching rosuvastatin, enlicitide with rosuvastatin, rosuvastatin with a placebo matching enlicitide, or placebos for both drugs. All treatments are taken orally once daily for up to 12 weeks. This setup helps researchers compare the effects of enlicitide alone, rosuvastatin alone, their combination, and placebo. During the study, participants will have their LDL-C levels measured at baseline and at weeks 8 and 12. Researchers will also monitor apolipoprotein B, non-high-density lipoprotein cholesterol, lipoprotein(a), and adverse events. The main outcome is the percent change in LDL-C at week 8. Safety and tolerability are assessed throughout the treatment and up to about 20 weeks. The total participation duration is up to 12 weeks of treatment with follow-up monitoring.

Researchers are evaluating treatments for germinal center B-cell-like diffuse large B-cell lymphoma (GCB DLBCL), a fast-growing blood cancer affecting immature B-cells. This phase 2 study compares the effects of zilovertamab vedotin combined with R-CHP chemotherapy versus polatuzumab vedotin combined with R-CHP in people newly diagnosed with this type of lymphoma. The goal is to see if the cancer responds better to one treatment over the other. Participants will be randomly assigned to receive either zilovertamab vedotin or polatuzumab vedotin, both given by intravenous infusion on the first day of each 3-week cycle for up to six cycles (about 4 months). Alongside these drugs, participants will receive cyclophosphamide, doxorubicin, and rituximab or a rituximab biosimilar by IV infusion, plus prednisone or prednisolone orally for five days each cycle. Those with high-risk DLBCL may receive two additional cycles of rituximab or its biosimilar. During the study, participants will have scans and tests to measure cancer response using established criteria over up to 31 months. Researchers will also monitor survival rates, quality of life, and side effects for up to several years. Safety assessments and treatment adherence will be tracked throughout the study and follow-up periods, ensuring thorough evaluation of each treatment's impact.

Researchers are studying individuals with generalized stage III periodontitis to compare the safety and healing effects of a new periodontal hydrogel dressing called Emanate Perio PODS. This device is being evaluated to see if it improves wound healing after plaque and tartar removal from teeth compared to no treatment. The study focuses on the critical healing period following non-surgical periodontal therapy and aims to protect healing tissue from harmful bacteria. The trial involves two groups: one group uses the Emanate Perio PODS device twice daily for 30 minutes each session for 15 days after scaling and root planing (SRP), while the other group receives no additional treatment after SRP. The dressing is applied immediately after toothbrushing and interdental cleaning to shield the healing periodontal pocket during the first 30 days post-treatment, especially during the initial phases when the tissue is vulnerable. Participants will be monitored over 60 days after their non-surgical therapy. Researchers will assess healing by checking for gingival bleeding and other signs of wound repair. The study includes regular periodontal evaluations, intraoral digital scans, and requires participants to follow oral hygiene instructions carefully. The goal is to evaluate the effectiveness and safety of the dressing in supporting tissue healing after SRP with minimal burden on participants.

Researchers are evaluating BGB-16673, an orally administered Bruton Tyrosine Kinase targeted protein degrader, in adults with various B-cell malignancies including relapsed or refractory forms of marginal zone lymphoma, follicular lymphoma, mantle cell lymphoma, chronic lymphocytic leukemia/small lymphocytic lymphoma, Waldenstrf6m macroglobulinemia, diffuse large B-cell lymphoma, and Richter's transformation. The study aims to find the recommended dose and assess the safety, tolerability, and response rates in this population through a phase 1/2 open-label trial. The trial includes several parts: a phase 1 monotherapy dose finding with dose escalation and safety expansion, followed by phase 2 expansion cohorts. Participants receive BGB-16673 orally at various dose levels to determine the maximum tolerated dose and the recommended dose for further study. Specific groups include Japanese participants and those who have not previously received a Bruton Tyrosine Kinase inhibitor. Dose escalation and safety data are collected to guide dosing recommendations, with some cohorts focused on particular lymphoma subtypes and treatment histories. Participants will be monitored from the first dose of BGB-16673 until 30 days after the last dose or before starting new anticancer therapies, for up to 47 weeks in phase 1 and approximately three years in phase 2. Assessments include adverse event tracking, response rates, pharmacokinetics, and quality of life questionnaires. Various measures such as plasma drug concentration, protein degradation, and progression-free survival are evaluated periodically. Safety and efficacy data support long-term monitoring and dose adjustments throughout the study period.

Researchers are evaluating the effectiveness and safety of volrustomig compared to observation in adults with unresected locally advanced head and neck squamous cell carcinoma (LA-HNSCC) who have not shown disease progression after receiving definitive concurrent chemoradiotherapy (cCRT). This phase III global study focuses on patients with specific stages of LA-HNSCC and aims to better understand treatment options after initial therapy. Participants are randomly assigned to one of two groups: one group receives volrustomig, a drug being studied, while the other group is placed under observation without additional treatment. The study is open-label and conducted at multiple centers worldwide. The treatment or observation period follows the completion of definitive cCRT with curative intent. During the study, participants undergo regular assessments to monitor progression-free survival, overall survival, physical functioning, and quality of life. Researchers will also track the presence of anti-drug antibodies and measure drug concentrations in blood samples. Safety is monitored by recording adverse events over a period of up to approximately 8 years, allowing long-term evaluation of outcomes and participant well-being.

This research aims to evaluate the effectiveness of Elevate VR, a hospital-based brief violence intervention using virtual reality, to prevent firearm-related violence, injury, and death among youth. Firearm violence is a major public health concern, especially for young people, with rates of firearm-related deaths and injuries increasing nationally and particularly in Virginia. The study addresses the urgent need for new and innovative interventions to reduce firearm-related harm and its significant economic impact. Participants include youth aged 13 to 17 years who have received hospital treatment for intentional or unintentional injuries or those recruited from community sources, along with their caregivers aged 18 or older. The Elevate VR program consists of a 40-minute intervention based on positive psychology, motivational goal setting through gameplay, psychoeducation, and cognitive-behavioral and dialectical behavioral therapies. The study also involves completing questionnaires assessing firearm-related violence, attitudes, beliefs, and behaviors. During the study, participants will complete assessments at baseline and again at 1 and 3 months to measure firearm-related violence, violent crime involvement, firearm attitudes, re-injury, risky behaviors, personality traits, and future aspirations. These evaluations will help determine the intervention's impact. The study is non-randomized and does not involve masking. The overall goal is to gather data to support effective violence prevention methods among youth in high-risk communities.

Researchers are evaluating the use of elafibranor in adults with Primary Biliary Cholangitis (PBC) who also have cirrhosis, a serious liver condition involving scarring. PBC is a slowly progressing disease that damages bile ducts, causing bile acids to build up and harm the liver further. This study aims to see if elafibranor can better prevent worsening of the disease, including the need for liver transplant or death, compared to a placebo. The safety of long-term use and effects on symptoms like itching and tiredness will also be assessed. Participants will be randomly assigned to take either one 80 mg tablet of elafibranor or a matching placebo tablet once daily, taken orally with or without food, at about the same time each morning. The treatment period can last up to 3.5 years in a double-blind setting, meaning neither participants nor researchers know who receives the drug or placebo. This design allows a direct comparison of elafibranor's impact on disease progression and safety over a long term. Throughout the study, participants will undergo regular assessments including blood tests, physical exams, vital signs, ECGs, and liver imaging to monitor liver function and stiffness. Researchers will track a range of outcomes such as survival without clinical events, changes in liver and blood markers, symptom severity, and quality of life measures. Safety monitoring will continue until 4 weeks after the last dose. Each participant may be involved for up to 3.5 years from baseline to final evaluation.