Polycythemia Vera

Researchers are studying biomarkers from 129Xe gas exchange MRI to understand how they change with different medical interventions. The study focuses on interactions between 129Xe and red blood cells in the lungs. Participants include healthy volunteers and patients with conditions like interstitial lung disease, pulmonary hypertension, acute or chronic pulmonary embolism, anemia, polycythemia, and dyspnea. The study uses hyperpolarized xenon gas inhaled in multiple doses followed by breath holds, alongside oxygen administration. Participants are grouped by treatment: those undergoing transfusion or phlebotomy, patients receiving oxygen for lung-related conditions or healthy volunteers, and those recently diagnosed with acute or chronic pulmonary embolism. Treatments and responses are monitored at baseline and various follow-up points. Participants will undergo MRI scans to measure red blood cell transfer, chemical shifts after oxygen delivery, and changes in red blood cell signal oscillations before and after treatment. The study includes visits up to 3–6 months after interventions to track changes. Researchers also monitor safety and participant adherence throughout the study, which lasts until mid-2028.

Researchers are studying the use of 3'-deoxy-3'-[18F] fluorothymidine (FLT) positron emission tomography (PET) imaging in patients with cancer. This phase I trial aims to evaluate how well FLT PET imaging measures tumor growth and the activity of the DNA synthetic pathway in various cancers, including solid tumors and blood cancers. The study also seeks to determine how effective this imaging method is at detecting lesions and assessing response to treatment. Participants receive up to four FLT PET imaging procedures. During each procedure, a small amount of the FLT tracer compound is injected into the vein, followed by PET scan data collection for two hours to measure tumor growth. Blood samples may be taken during the scans, and urine samples collected afterward to analyze breakdown products of the tracer. Throughout the study, patients undergo assessments including PET or CT PET scans to measure tracer uptake and retention in tumors and normal organs. Researchers also evaluate changes in key enzymes related to DNA synthesis before and after therapy. These evaluations help monitor tumor activity and treatment response. The total time participants spend in the scanner during imaging is up to two hours per session, with a focus on capturing detailed tumor growth information.

Researchers are conducting the GENESIS clinical study to map the HLA genomic region in the Greek population and explore its possible links with various underlying diseases. This non-interventional, multicenter study aims to provide a pilot map of genetic variation in HLA that may be useful in medical research and clinical applications related to selected diseases. The study plans to include 12,000 participants over a total duration of 36 months. Each participant will attend one visit at a participating site during which they will provide demographic data, lifestyle information such as smoking and alcohol use, blood pressure measurements, details on diagnosed diseases and treatments, and recent laboratory test results if available. Buccal swab samples will be collected from each participant to extract DNA for HLA genotyping analysis. Selected samples will undergo further whole genome sequencing to investigate associations with autoimmune diseases. Participants will receive a personalized ancestry report after analysis completion. During the study visit, data collection includes demographic and health information, as well as laboratory and clinical test results from the past year. The genetic material from buccal swabs will be stored and processed for genetic analysis. Researchers will measure allele frequency of HLA alleles in the Greek population and assess the prevalence and risk associations of selected HLA-related diseases. The study's total duration is 36 months with results available at the end of this period.

Researchers are evaluating the safety, tolerability, and effectiveness of TQB3909 tablets combined with azacitidine in adults with myeloid malignancies, including acute myeloid leukemia and myelodysplastic syndromes. This open, multi-center clinical trial is designed as a Phase Ib/II study to better understand how this combination treatment works in these blood cancers. Participants receive TQB3909 tablets once daily in 28-day treatment cycles along with azacitidine. The study focuses on monitoring how well patients tolerate the treatment and its effects on their disease. The trial includes assessment of various response rates and survival outcomes over several weeks. Throughout the trial, participants undergo regular evaluations including monitoring for adverse events and laboratory tests for up to 24 weeks. Researchers measure remission rates, duration of remission, and survival outcomes up to 60 weeks. Participants' safety and response to treatment are closely tracked during the study.

Researchers are conducting an international multicenter retrospective observational study to understand the progression and outcomes of patients with Essential Thrombocythemia (ET) who carry the JAK2V617F mutation and have developed Polycythemia Vera (PV). The study includes two parts: a nested case-control study and a comparative retrospective cohort study. It aims to compare patients who progressed from ET to PV with those who did not, as well as patients diagnosed with PV without prior ET. The study groups consist of ET-to-PV patients, ET patients who did not progress to PV, and patients diagnosed with de novo PV. Matching is done to ensure comparable groups based on year and age at diagnosis, as well as disease duration. The ET-to-PV cases are identified by the date of PV diagnosis up to the end of 2020, with controls matched to these cases. The de novo PV patients must have been diagnosed by 2020 to allow at least 5 years of follow-up. Participants' medical records will be reviewed retrospectively to gather data up to the baseline and at the time of progression, if applicable. Researchers will analyze clinical outcomes and phenotypic evolution, using the date of PV diagnosis as a key reference point. The study does not involve any interventions or treatments and relies on existing patient data collected from multiple centers. Participation involves no active treatment or visits, focusing on data analysis for up to several years of follow-up.

Researchers are studying the use of unlicensed cryopreserved cord blood units (CBUs) for transplantation in both pediatric and adult patients with various blood-related cancers and other disorders affecting the blood-forming system. This observational study aims to evaluate outcomes such as the recovery of a certain level of white blood cells after transplantation, as well as the incidence of infections, infusion reactions, survival rates, and graft-versus-host disease over time. The study involves patients receiving unlicensed CBUs at multiple U.S. transplant centers. These CBUs are used for patients with hematologic malignancies and other blood disorders. The protocol collects data on patients who receive these unlicensed transplant units, without administering a new treatment but observing the outcomes after transplantation. Participants will be monitored for neutrophil recovery at 60 and 100 days post-transplant, along with assessments of infection transmission, infusion reactions, survival one year after transplant, and occurrences of acute and chronic graft-versus-host disease. Platelet engraftment levels will also be tracked. The study includes patients of any age and follows them through the transplantation and recovery process to gather information on these key outcomes.

Researchers are evaluating AJ1-11095, an oral type II JAK2 inhibitor, in adults with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF) who have not responded to or relapsed after prior treatment with at least one type I JAK2 inhibitor. This phase 1, non-randomized, open-label trial aims to assess the safety, tolerability, pharmacokinetics, clinical activity, and biomarker changes of AJ1-11095 in this patient group. The study includes a dose escalation phase followed by an expansion phase to identify the best dose for future studies.

Researchers are evaluating IM-1021, an antibody-drug conjugate, in participants with advanced cancers including B-cell lymphomas and solid tumors. This Phase 1 open-label study aims to assess the safety, tolerability, pharmacokinetics, and early anti-tumor effects of IM-1021. The study includes a dose escalation phase to find safe doses and schedules, followed by an expansion phase to further assess these doses in specific cancer types. IM-1021 is given intravenously on a 21-day cycle, starting at 2 mg/kg, with alternative dosing schedules possible. The study has two parts: Part A focuses on escalating doses to evaluate safety and determine recommended doses, while Part B expands treatment in groups with specific cancer types to further evaluate safety and preliminary activity. Participants will undergo regular safety assessments including monitoring for treatment-related side effects from the first dose through 37 days after the last dose. Researchers will also measure drug levels in the body and evaluate anti-tumor activity starting at week 6 until disease progression or study discontinuation. The total study duration varies per participant. Safety, tolerability, and pharmacokinetic data will guide future development of IM-1021.

Researchers are studying the safety, tolerability, how the body processes it, and early signs of effectiveness of an investigational drug called PRT12396 in people with high-risk polycythemia vera (PV) and myelofibrosis (MF). These conditions are types of myeloproliferative neoplasms that affect blood cell production. The study aims to find the highest dose that can be tolerated and to recommend doses for further testing. It is a first-in-human, open-label Phase 1 trial conducted at multiple centers. The trial has two parts: first, a dose-escalation phase where participants receive increasing oral doses of PRT12396 twice daily to assess safety and determine the recommended dose. Second, a dose-expansion phase enrolls more participants to further evaluate the selected dose's safety, tolerability, and preliminary effects. PRT12396 capsules are taken by mouth with water, either an hour before or two hours after meals. Participants will attend scheduled visits and undergo laboratory tests to monitor their health and response to treatment. The research team will assess dose-limiting toxicities, adverse events, and various blood-related measures such as hematologic response and symptom scores over about two years. The study plans to enroll up to 100 participants and includes careful monitoring of safety and drug levels in the body throughout the trial period.

Researchers are evaluating cytokine induced memory-like natural killer (CIML NK) cells combined with IL-2 in adults aged 18 and older with Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), and Myeloproliferative Neoplasms (MPN) who have relapsed after haploidentical or HLA matched stem cell transplantation. The study also includes pediatric patients aged 12 and older with AML, MDS, and Juvenile Myelomonocytic Leukemia (JMML) who relapse after stem cell transplantation. This is a Phase I clinical trial focused on testing the safety and determining the appropriate dose of these investigational CIML NK cells, which have not yet been approved by the FDA for relapsed disease treatment. The treatment involves intravenous infusion of CIML NK cells on day 0. Prior to this, patients receive chemotherapy with fludarabine administered once daily for three doses starting on day -5, and cyclophosphamide given on days -5 and -4. This regimen is designed to prepare the body for CIML NK cell infusion. Both adult and pediatric patients undergo this treatment schedule. Participants will be closely monitored for safety over 6 weeks, with additional evaluations including objective response rate at 28 days, and assessments for leukemia-free survival and overall survival at 100 days and one year. Researchers will also track the incidence and severity of acute and chronic graft-versus-host disease over time. The study involves various tests such as bone marrow examinations, blood tests, and pregnancy tests when applicable. Participants must meet eligibility criteria and provide informed consent to join this study, which continues until the end date in December 2026.