Mao Ming Shi

Researchers are evaluating the safety and effectiveness of a drug called AND017 in adults aged 18 to 65 who have transfusion-dependent or non-transfusion-dependent beta-thalassemia. This phase II study compares different oral doses of AND017 with a placebo, alongside optimal supportive care such as blood transfusions and iron removal based on the clinicians judgment. The goal is to understand how well AND017 works and how safe it is for these patients. Participants will be randomly assigned to receive one of several doses of AND017 capsules 8 mg, 12 mg, or 16 mg or matching placebo capsules, taken once daily. The study treatment will continue for up to 24 weeks. Throughout the treatment period, patients will continue to receive their usual supportive care including blood transfusions as needed. During the study, participants will undergo regular assessments including blood tests to measure hemoglobin levels, red blood cell counts, iron-related markers, and changes in transfusion needs. These evaluations will occur at multiple visits from baseline through week 24. Researchers will closely monitor safety and tolerability and track changes from baseline in various blood parameters to assess the impact of AND017. The total participation time includes treatment and follow-up visits lasting up to 24 weeks or until early discontinuation.

Researchers are investigating how to accurately assess pseudoprogression a treatment-related effect that can look like tumor growth following immunotherapy in patients with rectal cancer. This observational study uses multiple MRI scans taken at different times and parameters, alongside blood biomarkers and clinical indicators, to evaluate changes after immunotherapy treatment. The goal is to improve the ability to distinguish true tumor progression from pseudoprogression in this patient group. Participants undergo multiparametric MRI scans before starting immunotherapy, and again at 6 and 12 weeks after treatment. Blood tests measuring markers such as IL-8, NLR, LDH, and S100, as well as clinical indicators like KPS score, CEA level, and T-stage, are collected at the same time points. Follow-up imaging and pathological assessments are used to confirm disease status. The study involves a single observational group without experimental interventions. Throughout the study, researchers collect imaging, blood, and clinical data to build predictive models for pseudoprogression. They will analyze accuracy, sensitivity, specificity, and predictive values of these models from baseline to preoperative MRI. Long-term outcomes including disease-free and overall survival will be monitored for up to 60 months. Participants will be followed to evaluate immunotherapy response and disease progression using standardized criteria and pathology when needed.

Researchers are studying how amide proton transfer-weighted imaging APTWI, a special MRI technique, can help predict how rectal cancer responds to treatment before surgery. The study focuses on patients with locally advanced rectal cancer who have received neoadjuvant therapy, aiming to see if this imaging method can foresee a complete pathological response. This observational study is sponsored by the Sixth Affiliated Hospital, Sun Yat-sen University and collects data as part of regular MRI exams. Participants undergo amide proton transfer-weighted imaging as part of their routine MRI scans before surgery. The study examines the imaging results between the baseline MRI and the preoperative MRI, which should be within 12 months. Researchers analyze how well APTWI predicts tumor response, measuring accuracy, sensitivity, specificity, and predictive values. They also monitor disease-free and overall survival for up to 60 months after enrollment. During the study, participants MRIs are reviewed to assess tumor response to prior therapy. Researchers evaluate the imagings ability to predict outcomes and compare it to pathological results after surgery. The primary outcome is the area under the curve AUC of prediction models within 12 months, with additional measures including survival outcomes. Participants are followed for up to 5 years, with data collected from regular MRI exams and clinical records.

People with end stage kidney disease ESKD who require dialysis face a much higher risk of cardiovascular disease compared to the general population, with cardiac issues causing 58% of deaths in this group. However, while aspirin is known to reduce cardiovascular problems in the general population, there is limited evidence about its effects in dialysis patients. The ASPIrin to Reduce Event in Dialysis ASPIRED trial aims to study whether taking low-dose aspirin can safely improve cardiovascular outcomes in people with ESKD receiving dialysis. This study is a multi-center, double-blind, randomized controlled trial that will compare daily low-dose aspirin 100 mg to a placebo pill in patients undergoing dialysis. The trial uses an existing dialysis registry platform to screen, recruit, and collect data during routine clinical care, minimizing participant burden and study costs. Randomization is managed through a secure web-based system, and follow-up visits occur every six months as part of regular clinic visits. The trial is expected to last approximately five years and includes oversight by an independent safety and monitoring board. Participants will be involved in regular six-monthly clinic visits where their health will be monitored as part of their usual dialysis care. Researchers will track the occurrence of major cardiovascular events, including heart attacks, strokes, vascular complications, and deaths, throughout the study period. Data will be collected from routine clinical procedures and the dialysis registry to evaluate outcomes. Safety and efficacy will be closely monitored, and the study will follow participants for up to five years to assess the long-term effects of aspirin use in this population.

Researchers are evaluating the effects of using an amino acid 15 peritoneal dialysis solution in patients with diabetes who are undergoing peritoneal dialysis. This single-center, open, prospective, self-controlled clinical study aims to observe changes in glycosylated hemoglobin HbA1c 90 days after starting the amino acid dialysis solution. The study focuses on diabetic patients already receiving peritoneal dialysis to better understand how this treatment may influence blood sugar control and related health measures. Participants will use one bag 2 liters of amino acid 15 peritoneal dialysis solution daily after breakfast, followed by treatment with glucose peritoneal dialysis solution lactate. The study period involves monitoring patients from the start Day 0 through 90 days of treatment with this amino acid solution. This intervention is assessed without a comparison group, as each patient serves as their own control. During the study, participants will have regular assessments of blood markers including glycosylated hemoglobin, fasting blood glucose, serum albumin, hemoglobin, prealbumin, daily urine volume, ultrafiltration volume, Ktv, creatinine clearance rate Ccr, and hypoglycemic drug use. These measurements will be taken at baseline, 30 days, and 90 days to track changes over time. The total study duration for each participant is 90 days, with ongoing monitoring for safety and effectiveness of the amino acid dialysis solution.

Researchers are evaluating the effects of a medicine called BI 690517 combined with empagliflozin in adults with chronic kidney disease CKD who are at risk of their kidney condition getting worse. The study includes people with or without type 2 diabetes and those who may already be taking medicines like angiotensin converting enzyme inhibitors ACEi, angiotensin receptor blockers ARB, or sodium-glucose cotransporter-2 inhibitors SGLT2i. The goal is to understand if adding BI 690517 can help delay worsening kidney function, hospitalizations due to heart failure, or cardiovascular death. After a run-in period where all participants take empagliflozin and other standard medications, participants are randomly assigned to receive either BI 690517 tablets or placebo tablets once daily alongside empagliflozin. The run-in period confirms that participants are stabilized on empagliflozin before randomization. The treatment phase continues for about three to four years until enough kidney or heart-related events have occurred to compare outcomes between the two groups. During the study, participants visit the study site about five times in the first six months and then every six months thereafter. At these visits, health is regularly checked through blood and urine tests, blood pressure and weight measurements, kidney function monitoring, and collection of any side effect information. The main outcome measured is the time until the first occurrence of kidney disease progression, hospitalization for heart failure, or cardiovascular death.

Researchers are evaluating the use of absorbable and moldable skull base support plates in patients undergoing extended endoscopic endonasal transsphenoidal surgery for sellar region tumors. The study aims to compare the incidence of cerebrospinal fluid CSF rhinorrhea within one month after surgery between patients receiving standard sellar floor repair combined with the support plate and those receiving standard repair alone. This phase II, randomized, controlled trial also assesses intracranial infection rates, surgery duration, hospital stay length, and safety, including nasal complications. Participants will be randomly assigned to one of two groups. The experimental group will receive a skull base repair that includes the use of a shaped and trimmed absorbable and moldable support plate placed during surgery, covered with nasal septal mucosa and stabilized with gelatin sponge. The control group will undergo the standard multi-layer sellar floor repair without the support plate but with similar packing and mucosal coverage. The trial follows a 11 parallel design and plans to enroll approximately 126 patients. During the study, participants will be monitored for CSF rhinorrhea and intracranial infections within the first month after surgery using clinical symptoms, imaging, and nasal endoscopy. Researchers will also record surgery duration and hospital stay length. Safety will be assessed by tracking treatment-related adverse events, especially nasal complications. Follow-up includes face-to-face visits for patients returning for reexamination and telephone follow-ups for those who cannot. Data will be analyzed using established statistical methods to evaluate the outcomes and safety over the study period.

Researchers are studying the effects and safety of SR1375 in hospitalized adults with community-acquired pneumonia CAP. This phase 2, randomized, double-blind, placebo-controlled study aims to evaluate SR1375 compared to placebo in patients who have not improved after initial standard pneumonia treatment. The study includes adults aged 18 to 85 years and is sponsored by Shanghai SIMR Biotechnology Co., Ltd. Participants will be randomly assigned to one of four groups to receive either 0.3 mg, 1 mg, or 3 mg of SR1375 capsules or a placebo daily by mouth for 8 weeks, alongside their usual pneumonia treatments. The study includes a screening period, an 8-week double-blind treatment period, and a 14-day safety follow-up after treatment ends. During the study, participants will undergo assessments including the NIAID-OS 8-point scale to monitor pneumonia severity, oxygen levels, and other health measures up to 56 days. Researchers will track outcomes such as oxygenation, mortality, hospitalization duration, and readmission rates. The total participation time for each patient is up to 10 weeks, with close monitoring of safety and health status throughout the study.

Researchers are evaluating the use of recombinant humanized anti-CD25 monoclonal antibody to prevent graft-versus-host disease GVHD in patients with transfusion-dependent thalassemia undergoing haploidentical or matched unrelated donor hematopoietic stem cell transplantation. GVHD is a major complication that affects the success and quality of life after transplantation. This Phase 2 trial aims to determine whether this antibody reduces GVHD incidence and to assess its safety and impact on quality of life over a two-year follow-up. Participants will be randomly assigned in a 21 ratio to either receive the recombinant humanized anti-CD25 monoclonal antibody combined with standard GVHD prevention or standard prevention alone. The antibody is given in four doses at 1 mgkg on days 7, 14, 28, and 42 after transplantation. The control group will not receive the antibody but will follow the usual GVHD prophylaxis. The study compares outcomes between these two groups. During the study, participants will be monitored for acute GVHD within 100 days and chronic GVHD, overall survival, thalassemia-free survival, transplant-related mortality, complications, infections, immune recovery, quality of life, and adverse events over two years. Regular assessments and follow-ups will evaluate the clinical efficacy and safety of the antibody treatment. The total participation time covers at least two years of follow-up after transplantation.

Recurrent respiratory tract infection RRTI is a common condition in children under six years old that affects their growth, development, and quality of life. This trial evaluates the clinical effects of Antrrix probiotics on children diagnosed with RRTI. The study aims to understand how probiotics might help improve respiratory symptoms and overall health by restoring gut microbiota balance and enhancing immune function. Participants are randomly assigned to receive either a daily dose of probiotic containing Lactobacillus rhamnosus CRL1505 and Bifidobacterium brevis M-16V strains in MCT oil or a placebo of MCT oil without probiotics. The treatment lasts for 8 weeks. Stool samples are collected before, during, and after treatment to analyze changes in gut bacteria through gene sequencing. During the study, researchers monitor respiratory symptoms and quality of life using questionnaires at multiple time points day 1, day 7, day 14, week 4, and week 8. They also assess immune biomarkers and gut microbiota profiles. The total participation period includes these assessments and sample collections to evaluate the probiotics impact and safety in young children with RRTI.