Blood Marrow Disorder

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 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 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.

Myelodysplastic syndrome (MDS), also called bone marrow failure, is a condition where the bone marrow produces fewer blood cells due to abnormal cell development. This study evaluates a new approach to treating MDS by using an alternating low-dose schedule of two chemotherapy drugs, 5-azacitidine (5AZA) and decitabine (DEC), to overcome resistance that can occur when either drug is given alone. The study is an early phase 1 pilot trial focusing on this combined treatment for myeloid malignancies including MDS and related disorders. Participants will receive 5AZA and DEC in a weekly alternating schedule: 5AZA at 50 mg/m² on Day 1 and DEC at 5 mg/m² on Day 4 each week. The first 8 weeks serve as an induction phase, followed by a long-term treatment phase starting from week 9. Treatment will continue for at least 24 weeks unless the disease progresses. Those who respond to therapy may continue treatment until relapse or disease progression not responsive to dose escalation. During the trial, participants will be regularly monitored for response using criteria including complete or partial response and hematologic improvement. Safety will be assessed by tracking adverse events. The study also explores biological markers related to treatment response. Participants may remain in the study for up to 6 months after treatment to assess overall response, with some outcomes followed for up to 2 years. Careful evaluation of blood counts, disease status, and side effects will guide treatment continuation and study assessments.

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 whether a high-fiber plant-based diet (HFPBD) can improve quality of life for people with clonal hematopoiesis (CH). This diet mainly includes plant foods such as fruits, vegetables, nuts, beans, and whole grains. The study measures quality of life using questionnaires and surveys filled out by participants. Participants will be randomly assigned to one of two groups. One group will receive high-fiber plant-based meals delivered to their homes for 12 weeks and have regular phone or video calls with a dietitian every 2 weeks for the first 12 weeks, every 4 weeks from weeks 13 to 24, and every 8 weeks from weeks 25 to 52. The other group will receive usual care with phone calls every 4 weeks for the first 12 weeks and dietitian calls at weeks 13 and 53. During the study, participants will complete quality of life surveys using the EORTC QLQ C30 questionnaire. Assessments include phone or video visits with dietitians, dietary intake monitoring, and adherence to the assigned diet. The main outcome is improvement in quality of life within the first 12 weeks. The total study duration for participants is 52 weeks, with ongoing monitoring and support throughout this time.

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.

This research aims to monitor participants who previously received TSC-100 or TSC-101 T-cell receptor engineered T-cell (TCR-T) therapies in the TSCAN-001 study. The goal is to assess the long-term safety and efficacy of these investigational treatments over a 15-year period following their administration, focusing on people treated for conditions such as AML, ALL, and MDS. Participants will not receive any new study drug during this long-term follow-up study. Instead, they will be observed after completing the initial TSCAN-001 trial, while continuing any other cancer treatments as necessary. Monitoring will include a period lasting 15 years from the date of their original TCR-T cell therapy. During the study, participants will be evaluated for treatment-emergent adverse events to assess safety and tolerability. Researchers will also monitor overall survival, relapse-free survival, and progression-free survival over the 15 years. This observational study involves regular assessments to track long-term outcomes without administering additional investigational treatments.

This research follows patients with Hemoglobinopathy and Sickle Cell Disease who previously received BEAM-101, a gene-edited cell treatment, in an earlier study. The trial is a long-term observational follow-up to monitor safety and health outcomes over an extended period. It aims to gather important information about the long-term effects and risks after receiving BEAM-101 treatment. Participants in this study have received a single dose of BEAM-101 by intravenous infusion after a conditioning treatment with busulfan. This follow-up study will track their health for 13 years, adding up to 15 years from the initial treatment. Study visits will happen annually for the first 5 years, then every 3 years until year 11, with a final visit at year 15. Additionally, virtual or phone check-ins will occur every 6 months for the first 5 years, then annually thereafter. During the study, participants will undergo regular safety and efficacy assessments to monitor their health, including how well they remain free of severe sickle cell crises and their blood hemoglobin levels. Researchers will review long-term safety data and mortality over the 13 years. The study includes blood tests and other evaluations at scheduled visits and check-ins. Participation will last up to 15 years, with ongoing monitoring to understand the lasting impact of BEAM-101 treatment.

Researchers are investigating how cancer affects the health and well-being of both patients and their families throughout the cancer journey. This study focuses on family health, needs, perceived support, quality of life, self-efficacy, depression, stress, and resilience among patients with breast, prostate, colorectal cancer, or lymphoma and their caregivers. The goal is to identify families who are particularly burdened or vulnerable and understand the factors contributing to their vulnerability. Participants include 240 patients and their appointed adult caregivers recruited from six Danish hospital departments. The study uses a mixed-methods approach, combining a longitudinal survey and family interviews. Patients and caregivers complete questionnaires at baseline, 3, 6, 12, and 18 months, assessing various aspects such as family health, support, quality of life, self-efficacy, depression, distress, and resilience. A subset of families participates in interviews at 3 and 12 months to explore their experiences and needs more deeply. During the study, participants will fill out validated questionnaires including the Family Health Scale, ICE Family Perceived Support Questionnaires, EQ-5D-5L, General Self-Efficacy Scale, Patient Health Questionnaire-9, Distress Thermometer, and Connor-Davidson Resilience Scale. The interviews will be conducted in person, at home, or online, lasting about one hour. Researchers will analyze the data to better understand family dynamics and identify vulnerable families, monitoring family health outcomes over a one-year period.