Dysplasia

Researchers are evaluating the safety, side effects, best dose, and effectiveness of a combination conditioning treatment for donor stem cell transplantation in patients with high-risk acute myeloid leukemia AML, acute lymphoblastic leukemia ALL, and myelodysplastic syndrome MDS. This phase I trial studies a targeted radioimmunotherapy drug, 225Ac-DOTA-Anti-CD38 daratumumab, combined with chemotherapy drugs fludarabine and melphalan, and total marrow and lymphoid irradiation TMLI. Daratumumab targets CD38 on cancer and immune cells, potentially helping the immune system to attack cancer cells. Participants receive daratumumab intravenously followed by indium In 111-DOTA-daratumumab and actinium Ac 225-DOTA-daratumumab on day -15. Total marrow and lymphoid irradiation is given twice daily from days -8 to -5, fludarabine is given intravenously on days -4 to -2, and melphalan on day -2. On day 0, participants undergo hematopoietic cell transplantation HCT. Graft-versus-host disease GVHD prevention with sirolimus and tacrolimus starts on day -1. Throughout the study, participants undergo various scans, biopsies, and blood tests to monitor treatment effects and safety. Participants are monitored closely after transplantation with visits twice weekly for the first 100 days, then twice monthly up to six months, and monthly thereafter until immunosuppressive therapy is stopped without GVHD signs. Yearly follow-up continues for two years. Assessments include adverse events, survival rates, relapse, graft-versus-host disease incidence, infections, blood cell recovery, organ function, and drug distribution. The study aims to determine the maximum tolerated dose and evaluate overall safety and effectiveness of the treatment combination.

Researchers are evaluating a new AI-assisted Mobile Capsule Gastroscopy MCG system designed to detect various gastric diseases, including gastric cancer and precancerous lesions, in elderly and high-risk individuals. Gastric diseases pose a significant health challenge worldwide, especially in China, where gastric cancer has high incidence and mortality rates. Early detection through screening is crucial, as prompt treatment can greatly improve survival rates. This study aims to assess the effectiveness and practicality of MCG in community health centers for these populations. The MCG system includes a disposable capsule and a wireless portable receiver, allowing participants to complete the examination independently by following video instructions on a smartphone app and adjusting their body position to guide the capsule. An AI algorithm monitors gastric coverage in real time, and the video is accessible on the smartphone and can be uploaded for remote diagnosis. This non-invasive and user-friendly system does not require expensive magnetic navigation, making it suitable for widespread use in community settings. The study is part of a public health screening program targeting 10,000 elderly and high-risk residents. Participants will provide demographic and medical history data, undergo laboratory tests and MCG examination, and may receive esophagogastroduodenoscopy EGD and biopsies if needed. Short-term follow-up will monitor capsule excretion and any adverse events up to 14 days after MCG, while long-term follow-up will continue annually for five years to track gastric disease incidence and survival. Researchers will evaluate detection rates, safety, examination quality, patient satisfaction, and cost-effectiveness of the MCG system to inform future gastric disease prevention strategies.

Researchers are studying children with type II collagen disorders who have short stature to better understand how these conditions progress over time. This observational natural history study aims to gather clinical, imaging, and laboratory data to identify possible predictors of disease progression and outcomes. The study will help provide important information that could guide future clinical trials on treatments for these disorders. The study will follow up to 60 children diagnosed with a type II collagen disorder for up to 3 years. During this time, participants will have visits every 3 months in the first year and then every 6 months thereafter. Assessments include physical exams, height measurements, vision and breathing tests, x-rays, and blood samples collected once or twice a year. The study activities closely follow the recommended care for children with these disorders. Participants will undergo regular evaluations to track changes in motor function, pulmonary health, eye assessments, skeletal abnormalities, and bone growth biomarkers. Questionnaires will assess quality of life, pain, and fatigue over time. Researchers will collect data retrospectively and prospectively for up to 3 years to understand the natural course and burden of the disease. This comprehensive monitoring will support safety and outcome assessments throughout the study.

Researchers are evaluating rezatapopt combined with azacitidine in patients with TP53Y220C-mutant myeloid malignancies, including acute myeloid leukemia AML and myelodysplastic syndrome MDS. This phase Ib study aims to assess the safety and tolerability of rezatapopt and also looks at its clinical effectiveness, event-free survival, overall survival, and duration of response in both newly diagnosed and relapsed or refractory patients. The study also explores genetic changes related to TP53Y220C mutations and immunologic profiles. Participants receive oral rezatapopt daily along with intravenous azacitidine for 7 days in each treatment cycle. Treatment is administered either in an inpatient or outpatient setting. The study focuses on adults with confirmed TP53Y220C mutations and specific classifications of MDS. The design is a single-group, non-randomized treatment study. Throughout the study, participants are closely monitored for safety and adverse events over about one year. Assessments include genetic sequencing of bone marrow samples, evaluation of mutation changes, and immunologic markers. Participants must adhere to visit schedules and undergo laboratory tests to evaluate organ function and treatment response. The total study duration extends up to August 2029, ensuring long-term follow-up and data collection.

Researchers are evaluating canakinumab, a monoclonal antibody, for treating patients with low or intermediate-1 risk myelodysplastic syndrome MDS or chronic myelomonocytic leukemia CMML. The study is a phase II trial focusing on patients with these blood disorders to assess the drugs clinical activity, safety, and effects on transfusion independence and response duration. Participants receive canakinumab through a subcutaneous injection on the first day of each 28-day cycle. Treatment continues in repeated cycles unless there is disease progression or unacceptable side effects. After completing treatment, patients will be followed up at 30 days and then every six months to monitor their health. Throughout the trial, researchers will assess hematological improvement after two cycles, monitor adverse events for four weeks, and evaluate longer-term outcomes such as transfusion independence, duration of response, progression-free survival, leukemia-free survival, and overall survival for up to two years. Participants will undergo regular assessments and safety monitoring during the study period, which lasts until disease progression or unacceptable toxicity occurs.

This observational study aims to assess the real-world safety of luspatercept in Korean adults diagnosed with myelodysplastic syndrome MDS or beta thalassemia. The research focuses on participants who will start treatment with luspatercept to better understand its safety profile in routine clinical use. Participants will receive luspatercept according to the approved label in the Republic of Korea. The study involves enrolling adults aged 19 years or older who are prescribed luspatercept for approved indications. No experimental treatments or placebos are involved, as this study observes standard clinical practice. During the study, researchers will monitor participants for adverse events up to six months after starting luspatercept. They will collect safety data while participants continue their prescribed treatment. This study helps provide important safety information while participants receive regular medical care under supervision.

Researchers are evaluating the effects of APG-157 in adults with oral dysplasia, including moderate to severe oral dysplasia or carcinoma in situ CIS. The study aims to determine whether APG-157 can reduce tumor size and affect certain tumor markers and oral rinses in participants with these oral conditions. This is a Phase II proof of concept trial focused on the safety and efficacy of APG-157. Participants will receive oral APG-157 therapy, taking 200mg two 100mg pastilles three times daily during each 4-week cycle for up to three cycles, with the third cycle being optional. The treatment period lasts up to 12 weeks. The study will measure the pathologic response rate during this time and monitor clinical response, changes in lesion appearance, and treatment-related adverse events over a follow-up period extending up to 28 months. During the study, participants will undergo blood tests, oral rinses, and tissue biopsies for evaluation. Researchers will track tumor size and lesion changes through clinical and pathological assessments. Safety will be monitored by recording adverse events for up to 16 weeks. Participants will be involved for the full treatment period and follow-up visits as scheduled to assess outcomes and monitor health.

Researchers are evaluating a combination treatment involving mitoxantrone hydrochloride liposomes, subcutaneous cytarabine, G-CSF, and Venetoclax CMGVen for adults with secondary acute myeloid leukemia AML or myelodysplastic syndrome with increased primordial cells type 2 MDS-IB2, as well as elderly AML patients. The study aims to assess the treatments effectiveness and safety in these recurrent or difficult-to-treat cases. Mitoxantrone is a chemotherapy drug that interferes with DNA and RNA, and this study explores a new liposomal form developed in China. The treatment includes an induction phase with mitoxantrone liposomes given intravenously, cytarabine injected subcutaneously twice daily for seven days, G-CSF injections starting one day before treatment, and oral Venetoclax given on days 4 to 10. Each treatment cycle lasts four weeks, with two cycles planned initially. Patients who respond well may receive an additional consolidation cycle or proceed to stem cell transplantation if eligible, while others continue consolidation therapy for several more cycles. Participants will undergo regular evaluations including clinical assessments, laboratory tests, and monitoring for side effects. The main outcome measured is the complete remission rate within one year. Additional outcomes monitored include overall response rate, survival, relapse-free survival, minimal residual disease levels, and adverse events over one to two years. The total study duration covers initial treatment, follow-up, and monitoring until the planned end date in 2027.

Researchers are evaluating the safety and dosing of a drug called SEA-CD70 alone and in combination with azacitidine in adults with myelodysplastic syndrome MDS and acute myeloid leukemia AML. This phase 1, open-label study aims to understand the side effects, tolerability, pharmacokinetics, and antitumor activity of SEA-CD70 in patients with these myeloid malignancies. The study includes several parts to address different patient groups and treatment combinations based on disease status and prior therapies. The study has seven parts Part A will determine the appropriate SEA-CD70 dose for relapsed or refractory MDS Parts B and C will evaluate safety and effectiveness of SEA-CD70 monotherapy in relapsedrefractory MDS and AML, respectively. Parts D, E, and F will explore SEA-CD70 combined with azacitidine for various MDS or MDSAML patient groups, including untreated higher-risk and relapsed cases. Part G will assess SEA-CD70 with azacitidine and venetoclax in untreated AML patients unfit for standard chemotherapy. Treatments include intravenous SEA-CD70 on Days 1 and 15 of each cycle, azacitidine given subcutaneously or intravenously on Days 1 to 7, and continuous oral venetoclax when applicable. Participants will undergo regular assessments of side effects, laboratory tests, and dose-limiting toxicities during treatment cycles lasting up to approximately two years, with some outcomes followed for up to four years. The study tracks pharmacokinetics and antitumor responses, including remission rates and survival measures. Safety is closely monitored up to 30-37 days after the last dose. The total participation duration varies depending on the study part and treatment response, with multiple visits scheduled for treatment administration and monitoring.

Researchers are creating a registry to study people with early changes in the stomach lining that may lead to gastric cancer. This includes those with gastric atrophy, intestinal metaplasia, and dysplasia. The study also enrolls healthy individuals and people with gastric cancer to compare baseline characteristics and better understand risk factors for disease progression. Participants fall into three groups those with specific stomach lesions, healthy controls, and people with gastric cancer. The study collects information through questionnaires about clinical history, environmental exposures, lifestyle, and diet. This observational study does not involve any treatment but gathers health data to support future research. Participants will complete questionnaires and provide health information over a period that may last up to 10 years. The main goal is to build a detailed participant registry. Researchers will monitor and analyze the collected data to identify factors related to the progression of stomach lesions to cancer. Participation involves regular data collection but no active treatment, with safety and privacy monitored throughout the study.