Kansas City

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.

Researchers are evaluating sotatercept as a potential treatment for pulmonary arterial hypertension (PAH), a condition where blood vessels in the lungs thicken and narrow, causing high blood pressure in the lungs and overworking the heart. PAH symptoms include difficulty breathing and reduced ability to be active. Current standard treatments address symptoms but do not stop disease progression. This Phase 3 study focuses on the long-term safety and tolerability of sotatercept when added to standard PAH therapy. Participants in this long-term follow-up study receive sotatercept through subcutaneous injections every three weeks. Only individuals who completed prior sotatercept PAH studies without early discontinuation may join. This study continues the observation and assessment of participants over an extended period to learn about the effects and safety of sotatercept combined with background PAH treatments. During the study, participants will be regularly monitored for adverse events, treatment discontinuations, and the presence of anti-drug antibodies for up to approximately 90 months. Laboratory tests will evaluate blood components such as platelets, hemoglobin, creatinine, bilirubin, and liver enzymes. Changes from baseline in body weight, blood pressure, and electrocardiogram readings will also be tracked. The study involves adherence to visit schedules and compliance with study procedures to ensure comprehensive long-term safety data collection.

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 the safety, tolerability, pharmacokinetics, pharmacodynamics, and early antitumor effects of BGB-53038, a pan-KRAS inhibitor, in people with advanced or metastatic solid tumors that have KRAS mutations or amplifications. The study also explores using BGB-53038 alone or combined with tislelizumab in nonsquamous non-small cell lung cancer and with cetuximab in colorectal cancer. This first-in-human, open-label, multicenter study has two phases: Phase 1a for dose escalation and safety expansion, and Phase 1b for dose expansion. Participants receive BGB-53038 orally. In combination groups, tislelizumab and cetuximab are given by intravenous infusion to participants with specific cancers. The study evaluates different doses to find the maximum tolerated dose, recommended doses for further study, and overall response rates over approximately two years. Throughout the study, participants are closely monitored for adverse events, treatment responses, and drug effects. Researchers collect data on safety, tolerability, and preliminary antitumor activity. The total study duration includes dose escalation, expansion phases, and follow-up assessments lasting up to about two years.

Researchers are conducting a Phase 1, first-in-human study to evaluate YL217, an antibody-drug conjugate targeting the CDH17 protein, in patients with advanced solid tumors. This study aims to assess the safety, tolerability, pharmacokinetics, and efficacy of YL217, which has shown promising anti-tumor activity in preclinical models of colorectal and gastric cancers. The study addresses a significant unmet need in gastrointestinal cancer treatment by exploring a novel therapeutic approach. Participants will receive YL217 through intravenous infusion. The study is designed as an open-label, multicenter trial where patients with advanced solid tumors will be treated and monitored. The trial includes different parts, with eligibility based on tumor type and disease stage, ensuring patients have measurable tumor lesions and adequate organ function. The treatment period will involve careful dose administration and observation. Throughout the trial, participants will undergo various assessments to monitor safety and treatment response, including tracking dose-limiting toxicities, adverse events, and objective response rates for up to approximately three years. Researchers will collect data on tumor response and side effects, with regular clinical evaluations and laboratory tests. The study duration allows for long-term follow-up to understand the impact of YL217 on tumor progression and patient health.

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.

The trial investigates the use of volrustomig in participants with unresected locally advanced head and neck squamous cell carcinoma (LA-HNSCC) who have not shown disease progression after receiving definitive concurrent chemoradiotherapy (cCRT). The study aims to evaluate the efficacy and safety of volrustomig compared to observation in this patient population. Participants have tumors that express PD-L1 and the study is conducted as a Phase III, randomized, open-label, multi-center global trial. Participants are assigned to receive either volrustomig as sequential therapy following cCRT or to an observation group. The treatment period involves monitoring participants who have completed definitive cCRT but remain unresected and have no evidence of metastatic disease. The study focuses on participants with Stage III, IVA, or IVB LA-HNSCC according to AJCC criteria, who have not undergone tumor resection before cCRT and have not been treated with radiotherapy alone. During the study, participants are regularly evaluated for progression-free survival, with follow-up lasting up to approximately 8 years to assess long-term outcomes. Researchers will monitor safety and disease progression closely. The overall participation duration includes screening, treatment or observation, and extended follow-up to capture both efficacy and safety data over time.

Researchers are studying adults with confirmed Primary Biliary Cholangitis (PBC) and cirrhosis, a scarring of the liver caused by damage to bile ducts. PBC is a slowly progressing disease that causes bile acid buildup and further liver damage, which can lead to cirrhosis. This study aims to evaluate if elafibranor, a daily medication, can prevent worsening clinical outcomes such as the need for liver transplant or death, compared to a placebo. It also looks at the safety of long-term elafibranor use and its effect on symptoms like itching and tiredness. Participants will take either an 80 mg tablet of elafibranor or a matching placebo once daily for up to 3.5 years in a double-blind setup, meaning neither the participants nor researchers know who receives which treatment. This long-term treatment period is designed to monitor the drug's impact over time. The study includes two groups: one receiving elafibranor and the other receiving placebo, with treatment lasting up to approximately 42 months. During the study, participants will be regularly assessed from the start until 4 weeks after treatment ends, with a maximum involvement of 3.5 years. Researchers will measure event-free survival, tracking if participants avoid clinical events indicating disease worsening. Safety monitoring will include tracking side effects and overall health, while symptom impact will be evaluated. Participants will provide informed consent and follow the study protocol throughout this extended observation period.

Researchers are conducting a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and effectiveness of duvakitug in participants with moderately to severely active Ulcerative Colitis (UC). The study aims to assess how well duvakitug maintains clinical remission compared to a placebo over time. Participants will receive either duvakitug or placebo administered by subcutaneous injection during a 40-week pivotal maintenance period. Following this, eligible participants may join a 240-week open-label extension phase where they can continue receiving treatment. Participants who do not join the extension will complete a 45-day follow-up visit. The study includes up to 32 on-site visits, with 21 visits during the maintenance phase and 11 visits during the extension phase. Throughout the study, participants will be monitored for clinical remission using the modified Mayo Score by week 40. Safety and efficacy will be assessed regularly during office visits. The total study duration may last up to 286 weeks, including treatment, extension, and follow-up periods, ensuring thorough evaluation of long-term outcomes and safety of duvakitug in UC management.

This research aims to evaluate whether a home-based mobile health (mHealth) intervention can help preschool children aged 3 to 4 years improve their adherence to the 24-Hour Movement Guidelines. These guidelines cover physical activity, screen time, and sleep. The study focuses on children who currently meet zero or only one of these recommendations and seeks to determine if the intervention increases the number of children meeting all three guidelines. Researchers also want to find out if parents find the intervention easy to use and practical. Participants will be randomly assigned to either an intervention group or a waitlist control group. The intervention group will use the Shining Star mHealth app for 12 weeks, which provides short weekly lessons and behavior goals designed to promote healthy movement behaviors in children. The app includes gamification features, behavior trackers, a child-friendly "Kids Corner," reminders, and a parent chat forum. The control group will receive access to the app after the 12-week period. Throughout the study, children will wear accelerometers to track physical activity and sleep at baseline, week 6, and week 12. Parents will complete questionnaires about their child’s movement behaviors and development at these same times. Additional assessments include motor skills tests, feedback on cognitive development and behavior, and BMI measurements. The app’s usability and feasibility will be evaluated through weekly surveys and a system usability scale. The total study duration for participants is approximately 12 weeks.