Richmond

Researchers are evaluating the safety, tolerability, and early effects of 4D-710, an investigational gene therapy, in adults with cystic fibrosis CF lung disease who cannot use or tolerate existing CFTR modulator therapies. A sub-study also includes adults with advanced CF lung disease or frequent lung flare-ups who are currently on CFTR modulator therapy. This Phase 12 open-label trial aims to find appropriate dosing and assess potential benefits for these patient groups. Participants receive a single inhaled dose of 4D-710, which is a gene therapy designed to deliver a corrected version of the CFTR gene to lung cells. The study includes a dose exploration phase for those ineligible for modulator therapy, a dose expansion phase at selected doses, and a sub-study for participants on modulator therapy receiving various doses. Each participant undergoes one administration of the therapy during the trial. Throughout the study, participants are monitored for adverse events over a 60-month period. Evaluations include lung function tests, oxygen saturation measurements, and tracking of pulmonary exacerbations. Participants maintain their existing treatments if applicable, and researchers assess safety and early signs of effectiveness. The total study duration extends up to approximately nine years, including long-term observation after dosing.

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. The study compares two treatment combinations to see if more people respond to zilovertamab vedotin MK-2140 plus R-CHP versus polatuzumab vedotin plus R-CHP. This Phase 2 trial aims to assess the effectiveness and safety of these regimens in participants with newly diagnosed GCB DLBCL. Participants receive either zilovertamab vedotin along with rituximab, cyclophosphamide, doxorubicin, and prednisone R-CHP, or polatuzumab vedotin combined with R-CHP. Treatments are given by intravenous infusion on Day 1 of each 3-week cycle for up to 6 cycles, approximately 4 months, with prednisone or prednisolone taken orally for 5 days of each cycle. For participants with high-risk DLBCL, up to 2 additional cycles of rituximab or biosimilar are given. During the study, participants are monitored for response to treatment using Lugano Response Criteria, with follow-up lasting up to about 31 months for the primary outcome. Secondary outcomes include progression-free survival, overall survival, event-free survival, duration of complete response, adverse events, and quality of life assessments. Safety and health status are regularly checked through exams, lab tests, and questionnaires over several years, with total study participation extending up to 7 years.

This research aims to compare the safety and healing effects of a periodontal hydrogel dressing called Emanate Perio PODS in adults with generalized stage III periodontitis. The study evaluates whether individuals receiving this dressing after plaque and tartar removal experience better wound healing than those who receive no treatment. The trial focuses on healing in the periodontal pockets, which are considered chronic wounds compromised by bacteria. Participants are randomly assigned to one of two groups one group uses the Emanate Perio PODS device twice daily for 30 minutes immediately after toothbrushing and interdental cleaning for 15 days following scaling and root planing SRP. The other group receives no treatment after SRP. The dressing aims to protect the healing tissue from bacteria during the critical first 30 days post-treatment while the epithelium recovers. Use is limited to twice daily to match recommended oral hygiene routines and improve compliance. During the study, participants undergo periodontal evaluations and digital scans to monitor healing progress. The main outcome is the presence or absence of gingival bleeding 60 days after non-surgical therapy to assess wound healing effectiveness. Researchers also monitor safety and healing through additional measures at this timepoint. Participants are expected to follow study instructions and refrain from using certain oral care products during the trial, which lasts through the healing period assessed at 60 days.

Researchers are evaluating BGB-16673, an oral drug, in adults with various types of B-cell malignancies such as marginal zone lymphoma, follicular lymphoma, mantle cell lymphoma, chronic lymphocytic leukemia, Waldenstrm macroglobulinemia, diffuse large B-cell lymphoma, and Richters transformation. This study includes Phase 1 dose finding and safety expansion, followed by Phase 2 expansion cohorts to determine recommended doses and further assess safety and efficacy. The study is divided into several parts, starting with Phase 1 dose escalation to find safe dosage levels, including monotherapy dose escalation and safety expansion in selected doses. Phase 2 involves expansion cohorts where participants receive the recommended doses identified in Phase 1 for further safety and efficacy evaluation. Some cohorts include participants who have not received prior BTK inhibitors, and Japanese participants are also enrolled to assess safety. Treatments are orally administered. Participants will undergo regular assessments including monitoring for adverse events, disease response, and drug concentration levels in the blood at various time points. Researchers will measure outcomes such as overall response rate and progression-free survival over approximately three years. Safety and tolerability will be closely tracked, and quality of life questionnaires will be completed at scheduled intervals. Participation may last several years, including follow-up periods to monitor long-term effects.

Researchers are evaluating the efficacy and safety of volrustomig compared to observation in participants with unresected locally advanced head and neck squamous cell carcinoma LA-HNSCC who have not progressed after receiving definitive concurrent chemoradiotherapy cCRT. This phase III, randomized, open-label global study aims to assess whether volrustomig can improve outcomes in this patient population. Participants are randomly assigned to one of two groups those who receive volrustomig as sequential therapy, and those who undergo observation without additional treatment. The study compares these two approaches following prior curative concurrent chemoradiotherapy. The trial includes long-term follow-up to monitor patient outcomes. During the study, participants will be regularly assessed for progression-free survival, overall survival, physical functioning, and quality of life. Researchers will also monitor for the presence of anti-drug antibodies and adverse events related to volrustomig. Follow-up evaluations may continue for up to approximately eight years to fully understand the treatment impact and safety profile.

Firearm violence is a serious public health issue in the United States, especially among youth aged 13 to 17. This research aims to evaluate an innovative hospital-based intervention called Elevate Virtual Reality VR designed to prevent firearm-related violence, injury, and death among young people. The study is conducted in Virginia, where firearm-related fatalities and hospitalizations have significantly increased, creating an urgent need for effective prevention strategies. The Elevate VR program is a brief 40-minute intervention combining positive psychology, motivational goal setting through gameplay, psychoeducation, cognitive-behavioral therapy, and dialectical behavioral therapy. Participants include youth aged 13 to 17 who were treated for intentional or unintentional injuries, as well as youth recruited from community partners and caregivers aged 18 and older. Questionnaires assessing firearm-related violence, violent crime, attitudes, risky behaviors, personality traits, and future aspirations are completed at the start and at 1 and 3 months after baseline. Participants will complete the Elevate VR intervention and various assessments during the study period, with follow-ups at 1 and 3 months to evaluate changes in firearm-related violence and related outcomes. The study measures include firearm violence assessments, violent crime evaluations, and attitudes toward firearms. This study is sponsored by Virginia Commonwealth University and aims to inform future efforts to reduce firearm-related harm among youth.

This research aims to evaluate elafibranor, a study drug, compared to a placebo in adults with Primary Biliary Cholangitis PBC and cirrhosis, a liver disease causing bile duct damage and scarring. The trial focuses on whether elafibranor can better prevent worsening of the disease, including progression leading to liver transplant or death, and also assesses long-term safety and symptom impact such as itching and tiredness. Participants will be randomly assigned to take either an 80 mg tablet of elafibranor or a matching placebo tablet once daily, orally, with or without food. This double-blind treatment period can last up to 3.5 years for each participant, with tablets taken at approximately the same time each morning. The study is designed to compare these two groups over the long term. During the study, participants will undergo regular assessments including physical exams, vital signs, electrocardiograms, laboratory tests, and symptom questionnaires at intervals up to 3.5 years. Researchers will measure liver function tests, symptom scales, liver stiffness, and clinical outcomes related to disease progression. Safety is monitored through adverse event tracking and laboratory parameters, with follow-up extending to four weeks after the last dose. Overall participation may last up to 3.5 years.

Researchers are evaluating whether retatrutide and tirzepatide can prevent major adverse liver outcomes in adults with metabolic dysfunction-associated steatotic liver disease MASLD who are at high risk based on non-invasive tests. This Phase 3 randomized controlled trial aims to assess these treatments compared to placebo in about 4,500 adults over approximately 224 weeks. The study is sponsored by Eli Lilly and Company and focuses on liver disease progression and related health measures. Participants will be randomly assigned to receive retatrutide, tirzepatide, or placebo, all administered by subcutaneous injection. The trial includes two placebo groups corresponding to each experimental drug. After completing the main study, eligible participants may join a 2-year extension where all will receive either retatrutide or tirzepatide regardless of their initial assignment. During the study, participants may attend around 25 to 30 clinic visits for health monitoring, study procedures, and assessments of liver function and disease status. Researchers will measure the time to major adverse liver outcomes, changes in liver fibrosis scores, liver stiffness, liver fat content, liver enzyme levels, body weight, and cardiovascular events. Monitoring will continue from baseline through study completion, with detailed evaluations at multiple timepoints including week 104.

Researchers are studying the use of unlicensed cryopreserved cord blood units CBUs for transplantation in both children and adults with blood cancers and other related disorders. This observational study involves patients with hematologic malignancies and various inherited and acquired disorders affecting the blood and immune system. The main goal is to monitor how well neutrophil recovery occurs after transplantation using these unlicensed CBUs in multiple institutions. Participants receive unlicensed cryopreserved CBUs as part of their transplant treatment. The study includes patients of any age receiving these CBUs for approved indications. The protocol focuses on the access and distribution of these unlicensed units rather than a specific treatment intervention. The study gathers data from recipients who receive these CBUs, tracking outcomes after transplantation. Participants are monitored for neutrophil recovery at 60 and 100 days after transplant, defined by a neutrophil count of at least 500mm3. Researchers also collect information on infection transmission, infusion reactions, survival rates at one year, and incidence of acute and chronic graft versus host disease. Platelet recovery is also evaluated. Safety and efficacy outcomes are followed over time to better understand the effects of unlicensed CBUs in this patient population.

Researchers are studying bleximenib, an oral drug, in participants with acute leukemia to find the best dose and evaluate its safety and effectiveness. The study includes Phase 1 dose escalation to find recommended doses and Phase 1 dose expansion and Phase 2 to assess safety, tolerability, and anti-leukemia activity. Participants include both pediatric and adult patients with relapsed or refractory acute leukemia, especially those with specific genetic alterations. In Phase 1 Part 1, participants receive increasing doses of bleximenib orally to identify recommended doses based on tolerance. In Phase 1 Part 2, participants receive bleximenib at these doses to further evaluate safety. Phase 2 participants take the recommended dose to study the drugs effect on leukemia. The study monitors participants up to 4 years and 9 months for safety and treatment response. Participants will undergo assessments including monitoring adverse events, dose-limiting toxicities, and treatment responses. Blood tests will measure drug levels and leukemia remission rates. Safety and efficacy are tracked throughout treatment and follow-up, with a focus on remission rates and survival outcomes. The total study duration extends to September 2030, allowing long-term evaluation of bleximenib.