Anemia

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 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.

This research aims to evaluate the long-term safety and effectiveness of CS-101 in participants who previously received CS-101 in an earlier study (CS-101-03). It focuses on individuals with beta-thalassemia who underwent treatment with this base-edited autologous hematopoietic stem cell transplantation. The study is led by the Children's Hospital of Fudan University and monitors participants up to two years after their infusion. Participants in this follow-up study have completed the initial CS-101-03 trial where they received the CS-101 infusion. This current study tracks their health and treatment outcomes for up to two years after the last six-month follow-up visit post-infusion. The study does not introduce new treatments but observes the long-term effects of the prior CS-101 therapy. During the study, participants will undergo evaluations for serious adverse events, death, new cancers or blood disorders, and whether they achieve transfusion independence for at least 12 consecutive months. Researchers will also monitor changes in fetal and total hemoglobin levels, and the genetic modification presence in blood and bone marrow cells. All assessments occur from the time of consent through two years post-infusion, with safety and efficacy carefully observed throughout this period.

Researchers are evaluating 9MW3011, a drug being studied in patients with non-transfusion-dependent beta-thalassemia, a condition affecting blood production. This phase Ib, randomized, double-blind, placebo-controlled study aims to assess the safety, tolerability, how the drug moves and acts in the body, and immune responses to 9MW3011. The study involves 40 patients divided into four dose groups to explore these effects carefully. Participants will receive either 9MW3011 or a placebo through intravenous infusion in a 4:1 ratio within each dose group. The study has multiple ascending dose cohorts to test increasing doses of 9MW3011. This design helps researchers understand the drug's impact at different dose levels and compare it to a placebo under blinded conditions. During the study, participants will be monitored up to day 169 for adverse events, vital signs, physical exams, ECG results, and lab tests to evaluate safety. Researchers will measure drug levels in the blood, effects on iron metabolism markers, immune responses, and liver iron concentration. Participants will be involved in scheduled visits for assessments, tests, and safety monitoring throughout the study duration.

Researchers are evaluating a new combination of evidence-based interventions (EBIs) aimed at improving diagnostic safety and efficiency in primary care for patients with anemia and decreased glomerular filtration rate. The study is designed as a stepped wedge cluster randomized control trial to measure how these interventions affect patient safety and healthcare efficiency, while also assessing factors influencing their implementation such as acceptability, cost, and sustainability. The study compares an enhanced diagnostic team approach to usual care. The enhanced approach includes automated detection and tracking of abnormal test results, expanding the primary care team to include clinical pharmacists to guide anemia evaluation, and engaging patients through nurse navigators to increase their activation in the diagnostic process. The study involves several clinic groups that receive the intervention at different times, ranging from 12 to 24 months, with some initial control periods. Participants will have their diagnostic accuracy for causes of low hemoglobin and decreased glomerular filtration rate assessed within six months. Researchers will track time to diagnosis, appropriate test usage, treatment costs, and primary care physicians' views on the intervention's acceptability and feasibility. The study also monitors how well the diagnostic process steps are followed, the intervention's reach among patients, and sustainability in clinics over 2.5 years. Patient activation and clinic-level facilitators and barriers are evaluated through surveys and measures during the study period.

Researchers are evaluating the safety, tolerability, and effectiveness of a gene therapy treatment called BD211 for patients aged 3 to 35 years with transfusion-dependent beta-thalassemia. This condition requires regular blood transfusions, and the study aims to assess how well the gene-modified stem cells work and how safely they can be used. The study is a Phase 1, open-label trial sponsored by Shanghai BDgene Co., Ltd., focusing on patients who need frequent transfusions and have limited treatment options. The treatment involves a single intravenous infusion of BD211, which consists of autologous CD34+ hematopoietic stem cells that have been genetically modified with a lentiviral vector to produce healthy beta-globin. Participants receive one dose of at least 5 million cells per kilogram of body weight. The study includes an 18-month follow-up period to monitor safety endpoints and effectiveness, including the ability to reduce or eliminate the need for transfusions. During the study, participants undergo regular assessments to track neutrophil and platelet engraftment, transfusion independence, blood hemoglobin levels, ferritin levels, and the presence of the beta-globin protein in blood. Researchers also monitor for adverse events, survival rates, hospitalizations, and any signs of complications related to the gene therapy. Participants are expected to comply with scheduled visits, laboratory tests, and long-term follow-up assessments to help determine the treatment's overall safety and efficacy.

Researchers are evaluating the treatment of anemia in patients with chronic kidney disease (CKD) who are on hemodialysis. This Phase III, investigator-blinded, randomized, multicenter study compares two drugs, efepoetin alfa and darbepoetin alfa, to see how well they maintain hemoglobin levels in these patients. The study aims to maintain hemoglobin between 10.0 g/dL and 12.0 g/dL, which is important for managing anemia in CKD. Participants will be randomly assigned in a 2:1 ratio to receive either efepoetin alfa or darbepoetin alfa. Both drugs are given by intravenous injection, typically after dialysis sessions. Efepoetin alfa is administered weekly from Day 1 to Week 28, with possible interval changes to one or two weeks from Week 29 to Week 52 based on investigator judgment. The study consists of three periods: screening (up to 28 days), treatment (about 52 weeks), and a 4-week follow-up with phone contacts up to Week 56 or the last visit. During the study, participants will undergo regular assessments including hemoglobin level monitoring to evaluate the mean change between Week 20 and Week 28. Safety and efficacy are closely observed, with dosages adjusted to maintain target hemoglobin levels. Follow-up will include phone contacts to monitor participants up to Week 56. The total participation duration is approximately one year, starting from screening through treatment and follow-up.

Researchers are evaluating the safety and effectiveness of emapalumab to treat prolonged severe cytopenia in participants with large B-cell lymphoma (LBCL) who have received CAR T-cell therapy (CART). This pilot study aims to assess how well emapalumab works and its safety, while also exploring biomarkers that might show how participants respond or resist the treatment. The study is a Phase 2 interventional trial sponsored by M.D. Anderson Cancer Center. Participants will receive emapalumab by infusion and be assigned to one of two dose levels. Around 16 to 32 participants will be enrolled across these doses. The trial follows a stepwise approach: if enough participants show improvement at a dose level, the dose is considered effective, and the study may test a higher dose following the same approach. Dosing and enrollment continue based on participants' blood test improvements after treatment. During the study, participants will undergo blood tests and safety monitoring for about one year. Researchers will check for adverse events and evaluate the treatment's impact on blood cell counts. Bone marrow biopsies will be performed before and after each treatment cycle and if severe cytopenia recurs. Participants will also be monitored for infections and organ function. The total involvement includes screening, treatment, and follow-up visits to assess treatment effects and safety.

This research aims to evaluate how human lactoferrin combined with low-dose iron affects iron levels, aerobic fitness, and lactate metabolism in women who exercise and have low iron stores. The study focuses on healthy menstruating women aged 18 to 45 years with serum ferritin below 35 g/L, aiming to understand if lactoferrin can improve iron regulation and exercise outcomes. Iron deficiency is common in active women and can reduce oxygen delivery and exercise performance, making this study important for finding better supplementation methods. Participants will be randomly assigned to one of three groups for an 8-week period: receiving 100 mg lactoferrin plus 5 mg iron daily, 300 mg lactoferrin plus 5 mg iron daily, or a placebo plus 5 mg iron daily. The study uses a triple-blind design where neither participants nor researchers know who receives which treatment. The intervention is oral supplementation taken daily, and participants will attend six visits including screening, baseline, and follow-ups at weeks 2, 4, 6, and 8. During the study, participants will undergo blood tests to measure iron biomarkers and blood parameters, along with assessments of aerobic capacity using treadmill tests measuring VO2peak, time to exhaustion, and blood lactate levels. Questionnaires will evaluate gastrointestinal symptoms, menstrual symptoms, quality of life, and recovery perception. Daily supplement adherence and any side effects will be monitored throughout. The primary outcome is the change in serum ferritin concentration over time, while secondary measures include changes in hemoglobin, hematocrit, red blood cell count, and exercise performance indicators.

Researchers are evaluating the long-term safety of Desidustat for treating anemia in people with chronic kidney disease (CKD). This Phase 4, single-arm study includes both participants who are dependent on dialysis and those who are not, aiming to gather real-world evidence on the treatment's safety over one year. Participants will take oral Desidustat tablets from the start of the study up to 52 weeks. The study group includes 1004 individuals, split evenly between those on dialysis and those who are not. The treatment is monitored closely to assess any adverse events and serious adverse events throughout the duration. During the year-long study, participants will undergo regular assessments including blood tests to monitor hemoglobin levels, lipid profiles, VEGF, and serum Hepcidin. Researchers will track side effects and serious adverse events to evaluate safety. The study ends at week 52, after which participants' treatment and health status will be reviewed.