Dysplasia

Researchers are evaluating a new combination treatment for patients with high-risk acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndrome (MDS). This phase I trial studies the safety, side effects, best dose, and potential effectiveness of a radioactive antibody called 225Ac-DOTA-Anti-CD38 daratumumab combined with chemotherapy drugs and targeted radiation as a conditioning treatment before donor stem cell transplant. The treatments aim to prepare the body to accept donor cells and target cancer cells more precisely. Participants receive daratumumab intravenously followed by related radioactive compounds early in the treatment process. They then undergo total marrow and lymphoid irradiation (TMLI) twice daily for several days, combined with chemotherapy drugs fludarabine and melphalan given intravenously. On day 0, patients receive a stem cell transplant, and preventive medications for graft-versus-host disease (GVHD) start before transplant. The study includes detailed imaging scans and biopsies before and during treatment to monitor effects. Throughout the trial, participants have regular evaluations including blood tests, bone marrow biopsies, heart and lung function tests, and various scans. After transplant, they are followed closely with frequent visits for the first 100 days, then less often up to two years to track side effects, transplant success, survival, and disease status. Researchers monitor adverse events, transplant-related complications, and overall outcomes to determine the best dose and safety of this new conditioning approach.

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 the safety and possible benefits of a drug called rezatapopt combined with azacitidine in patients who have myeloid malignancies caused by a specific TP53Y220C gene mutation. These conditions include acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). This phase Ib clinical trial aims to assess how well patients tolerate the treatment and to explore its effects on disease progression and survival. Participants will take rezatapopt orally every day along with azacitidine given by intravenous infusion for seven days in each treatment cycle. Treatment may occur in either an inpatient or outpatient setting. The study also includes detailed assessments of genetic changes in the cancer cells, immune system responses, and how the drugs behave in the body. During the study, participants will be closely monitored for safety and side effects, with evaluations lasting about one year. Researchers will collect bone marrow samples and perform genetic sequencing to track changes in the cancer. Patients will have regular visits for treatment, testing, and assessments to understand the impact of the combination therapy on their condition.

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 drug's 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 (CMG+Ven) 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 treatment's 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 investigating the use of hyperbaric oxygen (HBO) therapy to improve umbilical cord blood (UCB) stem cell transplant outcomes for blood and lymph gland cancers such as AML, NHL, Hodgkin Disease, and myelodysplastic syndrome. UCB transplant has advantages like easier access and lower risk of infections and graft versus host disease, but challenges include limited stem cell doses and problems with homing, where stem cells lodge in the bone marrow. This study aims to see if HBO given before transplant can improve homing and engraftment by reducing a hormone that may impair this process. Participants in this randomized phase II trial will be assigned to either receive hyperbaric oxygen therapy or no additional intervention before their UCB transplant. HBO therapy involves breathing 100% oxygen in a pressurized chamber at 2.5 times normal atmospheric pressure. The study compares time to neutrophil recovery and other blood count and transplant success measures within 100 days after transplant between the two groups. Participants will be monitored closely with blood tests to track recovery and engraftment, including neutrophil and platelet counts and donor cell presence. Safety assessments and evaluations of transplant outcomes will occur regularly during the first 100 days post-transplant. The study runs until June 2027 and aims to provide important information on whether HBO therapy improves transplant success and immune recovery.

Researchers are evaluating a drug called SEA-CD70, alone and combined with azacitidine, to assess its safety and potential effects in adults with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). This phase 1, open-label study aims to find appropriate dosing and understand side effects and antitumor activity in participants with relapsed or refractory disease as well as previously untreated higher-risk cases. The trial is sponsored by Seagen, a subsidiary of Pfizer, and includes several parts to address different patient groups and treatment combinations. The study consists of seven parts: dose escalation and expansion of SEA-CD70 monotherapy for relapsed/refractory MDS and AML; dose-finding and expansion of SEA-CD70 combined with azacitidine for relapsed/refractory and untreated higher-risk MDS or MDS/AML; and dose-finding of SEA-CD70 with azacitidine and venetoclax in untreated AML patients unfit for standard chemotherapy. SEA-CD70 is given intravenously on Days 1 and 15 of each treatment cycle, azacitidine is given either subcutaneously or intravenously on Days 1 through 7, and venetoclax is taken orally daily with dose ramping. Participants will undergo safety and tolerability assessments, laboratory tests, and pharmacokinetic analyses through approximately two years after the last dose. Researchers will monitor adverse events, dose-limiting toxicities, and laboratory abnormalities. Secondary measures include response rates, remission durations, survival rates, and drug concentration levels. The study evaluates how well participants tolerate the treatments and examines the drugs' effects on disease progression over up to four years.