Kansas City

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 12, 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 investigating new treatments for Pulmonary Arterial Hypertension PAH, a condition where the blood vessels in the lungs thicken and narrow, causing high blood pressure in the lungs and making it difficult for the heart to work properly. PAH can lead to breathing difficulties and reduced activity levels. While current treatments help manage symptoms, they do not stop the disease from progressing. Sotatercept is a study medicine designed to target specific proteins involved in PAH, and this long-term follow-up study aims to assess its safety and tolerability when used alongside standard PAH treatments over an extended period. Participants who have completed previous sotatercept PAH studies may join this open-label follow-up study. Those from blinded studies will start sotatercept at 0.3 mgkg by subcutaneous injection every three weeks and may increase to 0.7 mgkg. Participants from unblinded studies will continue their current dose and may also increase to 0.7 mgkg. The study focuses on monitoring sotatercept use combined with background PAH therapy to observe long-term effects. Throughout the study, participants will undergo regular assessments including tracking adverse events, laboratory tests for blood and chemistry markers, body weight, blood pressure, and electrocardiograms up to approximately 7 years. Additional measurements include walking distance tests, heart function markers, and risk scores related to PAH. Safety monitoring and tolerance to the medication will be closely observed over time to better understand sotatercepts long-term impact in managing PAH.

Researchers are evaluating Claseprubart DNTH103 in adults with multifocal motor neuropathy MMN to assess its safety, tolerability, pharmacometrics, and effectiveness. This Phase 2 randomized, double-blinded, placebo-controlled study aims to better understand how this drug works for people with MMN and to monitor any treatment-related side effects. The sponsor of this trial is Dianthus Therapeutics. Participants receive either Claseprubart or a placebo. On the first day, they get an intravenous loading dose, followed by subcutaneous doses every two weeks from Week 1 to Week 15. The study includes multiple groups receiving either 300 mg or 600 mg doses of Claseprubart, or placebo, to compare outcomes. This design helps researchers evaluate different doses and their effects. During the study, participants will be closely monitored from baseline up to Week 17 for side effects and treatment response, including grip strength, muscle function scores, disability scales, and quality of life measures. Some assessments continue up to Week 52 in an open-label extension period. Blood samples will be collected to analyze drug concentration and immune responses. Safety and effectiveness data are gathered through various clinical tests and questionnaires, ensuring comprehensive participant evaluation throughout the study.

This research aims to evaluate YL217, an antibody-drug conjugate targeting the CDH17 protein, in patients with advanced solid tumors. YL217 combines a monoclonal antibody, a topoisomerase I inhibitor, and a special drug linker. Preclinical tests in mice showed that YL217 was well tolerated and slowed tumor growth, supporting its study in humans to address unmet needs in gastrointestinal cancer treatment. Participants will receive intravenous infusions of YL217 in a phase 1 clinical trial divided into three parts dose escalation, backfill stage, and dose expansion. Initially, patients will be enrolled at increasing dose levels to find safe and tolerable doses. After determining the maximum tolerated dose and recommended dose for expansion, the dose-expansion part will further support the selection of the optimal dose for future studies. Throughout the trial, researchers will monitor safety by tracking adverse events and dose-limiting toxicities over approximately three years. They will also assess tumor response rates and various pharmacokinetic parameters. Participants will undergo regular evaluations including tumor measurements, organ function tests, and performance status assessments to understand YL217s effects and safety profile.

Researchers are studying people with idiopathic pulmonary fibrosis IPF or progressive pulmonary fibrosis PPF who have previously completed treatment with the drug nerandomilast. The study aims to understand how well participants tolerate long-term treatment with nerandomilast and whether it helps improve lung function or delays worsening symptoms, hospitalizations, or death. This is a phase 3 open-label extension trial sponsored by Boehringer Ingelheim. Participants will take nerandomilast tablets daily for up to 1 year and 10 months. They may continue their usual treatments for pulmonary fibrosis alongside the study treatment. There is no placebo group, and all participants receive nerandomilast during this extension period. Throughout the study, participants will visit their doctors regularly for health check-ups and lung function tests. Researchers will closely monitor any side effects and changes in lung function, particularly forced vital capacity FVC, over nearly two years. The study will track time until symptom worsening, hospital visits, or death to assess the treatments long-term safety and effects.

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.

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 elafibranors 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.

Researchers are evaluating the efficacy and safety of duvakitug in people with moderately to severely active Ulcerative Colitis UC in a multicenter, randomized, double-blind, placebo-controlled Phase 3 maintenance study. The study is sponsored by Sanofi and aims to assess how well duvakitug maintains clinical remission and improves symptoms in participants who have responded to prior treatment. This investigation includes a long-term follow-up to understand the treatments effects over several years. Participants receive subcutaneous injections of duvakitug or placebo according to protocol. The study includes a 40-week pivotal maintenance phase followed by a 240-week open-label extension OLE phase for those who continue treatment. Participants not entering the extension phase will have a 45-day follow-up after the maintenance period. The total treatment duration may be up to 280 weeks, with up to 32 on-site visits during the entire study. During the study, participants will undergo regular assessments including clinical remission measured by the modified Mayo Score, endoscopic and histologic evaluations, symptom tracking like bowel urgency and abdominal pain, and quality of life questionnaires. Safety will be monitored through adverse event reporting and serum drug concentration measurements. The primary outcome is the proportion of participants achieving clinical remission at week 40, and the study will also track long-term safety and efficacy outcomes through the extension phase.

Researchers are evaluating a 12-week home-based mobile health mHealth intervention called the Shining Star study, aimed at improving adherence to the 24-Hour Movement Guidelines among preschool-aged children 3-4 years old who currently meet zero or one of the guidelines for physical activity, screen time, and sleep. The trial seeks to determine if the intervention increases the number of children meeting all three guidelines and if the program is feasible for parents to implement. This randomized controlled trial compares an intervention group using the app to a waitlist control group. Participants in the intervention group will use the Shining Star mobile app, which delivers weekly lessons and behavior-related goals through concise messages, gamification features, behavior trackers, and a parent forum. The control group will receive no intervention during the initial 12 weeks but will complete the same assessments as the intervention group. After 12 weeks, control group participants will gain access to the app and resources. Physical activity and sleep will be tracked with accelerometers, and parents will complete questionnaires. Childrens physical activity, sedentary behavior, and sleep patterns will be assessed at baseline, 6 weeks, and 12 weeks using accelerometers. Parents will provide feedback on their childs movement behaviors, development, and app usability through questionnaires and weekly surveys. Additional measures include motor skills tests, cognition assessments, behavioral problems, executive function, and BMI. Data will be securely stored and analyzed to evaluate the interventions impact and app feasibility over the 12-week period.

This research aims to observe the long-term safety of efgartigimod treatment in adults with generalized myasthenia gravis gMG. It compares patients who are starting or have recently started efgartigimod treatment with those who have not been exposed to efgartigimod and are receiving other standard treatments. The study is non-interventional and prospective, focusing on real-world safety outcomes over an extended period. Participants are divided into two groups one receiving efgartigimod and another receiving other medications for myasthenia gravis. This study does not involve any new treatments being administered as part of the trial but monitors patients during their routine care. The study will continue for up to 10 years to gather long-term safety data. During the study, researchers will track the occurrence of serious infections and other safety concerns in both groups. Patients will be monitored through their regular clinical visits without additional interventions from the study. Participants may be followed for up to 10 years to assess safety outcomes associated with their treatments in everyday medical practice.