Precision Medicine

Biliary tract carcinoma BTC includes cancers of the gallbladder, intrahepatic cholangiocarcinoma, and extrahepatic cholangiocarcinoma. It is a highly aggressive cancer with poor outcomes, ranking sixth in gastrointestinal cancer incidence and tenth in cancer-related deaths worldwide. Because early symptoms are often absent and the disease tends to recur and spread, only about 16.5% of patients can have curative surgery, and the overall 5-year survival rate is under 5%. Early and accurate detection is crucial to improve patient outcomes. This research aims to assess the use of cell-free DNA cfDNA methylation in blood as a liquid biopsy for diagnosing and managing BTC. The study involves several groups, including healthy individuals, patients with confirmed benign biliary lesions, other gastrointestinal cancers, and those with confirmed or suspected BTC. Researchers will analyze methylation patterns in circulating tumor DNA ctDNA, a small fraction of cfDNA that carries tumor genetic and epigenetic information. This approach is studied for its potential to detect BTC early, assist in differential diagnosis, monitor prognosis, and guide therapy. Different cohorts serve as internal training, validation sets, and independent validation groups. Participants will provide blood samples and undergo clinical evaluations to measure the accuracy of the ctDNA methylation test in diagnosing BTC. The study will assess diagnostic performance by cancer subtype and stage, and ability to distinguish BTC from other conditions. Vital signs, organ function, and other health markers will be monitored to ensure participant safety. The study welcomes adults aged 18 to 80 years, with follow-up and assessments continuing until May 2026 to evaluate the tests clinical utility.

Pancreatic neuroendocrine neoplasms pNENs are tumors with increasing incidence and limited treatment options, especially after disease progression or drug resistance from current therapies like somatostatin analogues, chemotherapy, and targeted drugs. Existing treatments such as sunitinib and everolimus have shown limited clinical efficacy with low objective response rates. Researchers are investigating new combinations, including sorafenib and gefitinib, based on evidence linking the EGFR pathway to tumor progression and poor survival in pNEN patients. This study evaluates a single treatment group receiving oral sorafenib 250mg and gefitinib 250mg daily, each dose taken once a day. Treatment cycles last 4 weeks, aiming to explore the effectiveness and safety of this combination for patients with pNENs who have progressed after prior treatments. Sorafenib is a tyrosine kinase inhibitor targeting VEGFR, FGFR1, and CSF1R, which may help inhibit tumor angiogenesis and regulate immune responses, while gefitinib targets EGFR. Participants will be assessed regularly with imaging every 8 weeks to measure tumor response and progression-free survival, with survival assessments every 12 weeks up to two years. Safety and tolerability of the drug combination will also be evaluated over two years. The study involves a screening process, treatment period, and follow-up evaluations to monitor outcomes and side effects, supporting comprehensive observation of the treatments impact on disease progression.

Researchers are studying a new treatment approach for patients with surgically removable head and neck squamous cell carcinoma HNSCC, a common and serious type of cancer in the head and neck region. The study evaluates the safety and tolerability of combining immune-targeted therapy with a lysogenic herpes simplex virus HSV as a neoadjuvant treatment to reduce tumor size before surgery. This innovative combination aims to improve surgery outcomes and lower the chances of cancer returning or spreading. Participants receive a combination of treatments including Tislelizumab given on days 1 and 22, and continuous daily Afatinib from days 1 to 42. They also receive injections of the lysogenic HSV virus directly into lymph nodes, with dosing based on lymph node size, given twice two weeks apart. After these treatments, patients undergo standard surgical removal of the tumor. The HSV virus injection includes an initial dose-escalation phase followed by a dose-expansion phase to find the most effective dose. During the study, patients are closely monitored for side effects and treatment tolerance, including checking for dose-limiting toxicities during surgery. Researchers will assess how well the tumor responds to treatment by examining major and complete pathological responses and overall tumor shrinkage up to eight weeks after treatment. They will also monitor adverse events for up to 12 weeks and follow disease-free survival for one year. The study duration and procedures are planned to provide detailed information on the safety and effects of this combined therapy.

Researchers are investigating the relationship between gut microbiota dysfunction and Alzheimers disease AD, focusing on how gut-derived short-chain fatty acids SCFAs may influence brain function through the gut microbiota-SCFAs-brain networks pathway. This observational study will explore differences in SCFAs among people across the AD spectrum, including cognitively normal individuals, those with subjective cognitive decline SCD, mild cognitive impairment MCI, and AD dementia. The project aims to clarify the interactions between gut microbiome, metabolites, and brain changes using high-throughput metabolomics and multi-modal MRI techniques. Participants will be grouped into four categories cognitively normal, SCD, MCI, and AD dementia. The study involves collecting multi-omics data, including gut microbiome analysis, metabolomics, and neuroimaging, to establish a diagnostic model for SCD due to preclinical AD using machine learning methods. The study spans five years, during which gut microbiome changes, SCFAs levels, and multi-omics biomarkers related to cognitive impairment conversion will be monitored. Participants will undergo cognitive tests and brain imaging scans, along with gut microbiome and metabolite sample collection. Researchers will assess the interaction mechanisms of the gut microbiota-SCFAs-brain networks over five years. The study includes both healthy volunteers and patients aged 60 to 80. Outcome measures focus on changes in gut microbiome, SCFAs, and biomarkers linked to cognitive decline, with long-term follow-up to better understand the disease progression and its early detection.

Esophageal squamous cell carcinoma ESCC is a common and deadly cancer in China, with many patients diagnosed at advanced stages. This study evaluates a combined treatment approach using induction immunochemotherapy followed by concurrent chemoradiotherapy, aiming to improve outcomes for patients with locally advanced, unresectable ESCC. Researchers also focus on using circulating tumor DNA ctDNA to monitor treatment response and predict tumor progression, as ctDNA changes can appear before imaging detects recurrence. Participants receive induction immunochemotherapy consisting of toripalimab combined with paclitaxel and cisplatin every three weeks for two cycles. This is followed by radical concurrent chemoradiotherapy with weekly paclitaxel and cisplatin for five cycles along with radiotherapy delivered five days per week. The study dynamically monitors ctDNA levels at several points before treatment, before chemoradiotherapy, after 20 radiotherapy fractions, and every three months after treatment completion. During the study, participants undergo regular assessments including blood tests for ctDNA analysis and monitoring of tumor status. The main outcome measured is progression-free survival at one year. Safety and efficacy are tracked throughout the treatment and follow-up periods. The total participation duration and timing of assessments are carefully planned to evaluate the treatment strategy and its correlation with patient prognosis.

Childhood obesity is a growing global health concern linked to serious health problems such as type 2 diabetes, high blood pressure, certain cancers, and mental health challenges. Researchers are evaluating a digital support system called Evira to help improve treatment outcomes for children with obesity. This randomized controlled study aims to assess the effects of adding Evira to the usual lifestyle treatment for childhood obesity in children aged 4 to 17 years. The study compares two groups one receiving Evira Care combined with standard lifestyle treatment, and the other receiving standard lifestyle care alone. Evira Care involves daily weight monitoring at home using a special scale connected to a mobile app and website, allowing parents and clinicians to track weight changes and communicate easily. The intervention group will receive guidance on how to use the system and make feasible lifestyle changes, with weight targets set for the first three months. The control group continues with standard care without restrictions on visits or clinical support. Participants will be involved for 12 months, undergoing clinical exams including puberty assessment, cardio-respiratory, thyroid, skin, and abdominal checks, along with measurements of weight, height, and blood pressure. Blood tests may be done as needed. All participants will complete questionnaires about quality of life, eating disorders, and treatment satisfaction. Researchers will monitor weight change as the primary outcome, along with treatment compliance and psychosocial health measures, while collecting information on any side effects.

Researchers are studying the effects of a low-protein diet on cancer patients receiving immunotherapy treatments. The study aims to see if reducing protein intake can change the tumor environment and boost the bodys immune response, potentially enhancing the effects of immune checkpoint inhibitors. This is a randomized, open-label study conducted at a single center to explore the feasibility of this dietary approach. Participants will be randomly assigned to one of two groups one receiving immunotherapy plus a control diet containing about 20% protein, and the other receiving immunotherapy combined with a low-protein diet containing 10% protein. The study intervention lasts for 4 months, during which the dietary impact on immune response and treatment effects will be evaluated. Throughout the 4-month study period, participants will be monitored for their ability to follow the dietary plan and for any changes in immune response to immunotherapy. Researchers will assess safety, tolerability, clinical efficacy using RECIST v1.1 criteria, and enrollment and drop-out rates. Laboratory tests and imaging scans will also be conducted to evaluate disease progression and treatment impact.

Researchers are studying the effects of intermittent caloric restriction combined with a plant-based diet in cancer patients receiving chemotherapy. This pilot trial aims to assess whether this dietary approach is feasible and safe alongside standard chemotherapy, and whether it may reduce side effects while potentially enhancing the chemotherapys antitumor effects. Participants will follow a protocol involving alternate-day intermittent caloric restriction along with a plant-based protein diet during their chemotherapy treatment. The study evaluates how this combined dietary intervention interacts with standard chemotherapy regimens over the course of treatment. During the study, participants will be monitored for adherence to the dietary plan and chemotherapy treatment. Researchers will assess feasibility over 12 months, including safety and preliminary evidence of clinical efficacy. Evaluations may include health assessments, laboratory tests, and monitoring of side effects throughout the study period.

This research investigates adjuvant chemotherapy decision-making for patients with high-risk stage III colorectal cancer, specifically those with T4N or T1-3N2 disease. It evaluates the use of plasma circulating tumor DNA ctDNA methylation to guide treatment choices. The study is a randomized controlled trial sponsored by Fudan University, focusing on how adding bevacizumab to standard chemotherapy affects patient outcomes. Participants will be randomly assigned to one of two groups the control group will receive standard chemotherapy with FOLFOX or CAPOX for six months, while the intervention group will receive the same chemotherapy combined with bevacizumab for six months. Blood samples will be collected at one, three, and six months after surgery to monitor plasma ctDNA dynamically. During the study, participants will undergo regular assessments including blood draws for ctDNA analysis. Researchers will measure outcomes such as the patients two-year progression-free survival and ctDNA clearance rate at six months. The study aims to follow participants over a two-year period to evaluate treatment effects and safety.

Researchers are evaluating the safety and tolerability of single and multiple increasing doses of GenSci098 given under the skin in patients with active thyroid eye disease TED related to Graves disease. This Phase 1 clinical trial aims to understand how the body processes this drug and to monitor for any side effects. The study is sponsored by Changchun GeneScience Pharmaceutical Co., Ltd. Participants will receive subcutaneous injections of GenSci098 at one of five dose levels 15mg, 45mg, 90mg, 180mg, or 270mg or a matching placebo. The study includes two parts a single ascending dose SAD phase where participants receive one dose, and a multiple ascending dose MAD phase where participants receive repeated doses. The study uses a randomized and quadruple-blind design to compare GenSci098 and placebo. During the study, participants will be monitored for adverse events through physical exams, vital signs, laboratory tests, and ECGs over periods of up to 169 days in the SAD part and 281 days in the MAD part. Researchers will also measure how GenSci098 moves through and acts in the body, including antibody responses. Participants will be asked to comply with study treatments and assessments until the study ends.