Reggio Calabria

This research aims to observe the real-world use and effects of pegcetacoplan in adults diagnosed with Paroxysmal Nocturnal Hemoglobinuria PNH. As a new treatment with a unique mechanism of action, pegcetacoplans effectiveness and safety in routine medical practice are being studied to provide valuable information for patients, healthcare providers, and payers. The study will also gather data on blood transfusions and healthcare resource use before and after starting pegcetacoplan. Patients who have started pegcetacoplan treatment within the past 12 months or are prescribed the drug at enrollment will be included. Data collection includes retrospective information from up to 12 months before treatment start and prospective monitoring for approximately 36 months, with the total data period extending up to about 48 months. After stopping pegcetacoplan, patients remain in the study for 8 weeks to record any adverse events. Patients continue regular clinic visits, where data from each visit will be gathered. Participants will have data collected on various health measures such as hemoglobin levels, blood markers, transfusion needs, and patient and physician treatment satisfaction at regular intervals up to 36 months. Safety and adverse events will be monitored throughout. The main outcome measured is the change in hemoglobin level from treatment start to 6 months. This long-term observational study allows for comprehensive tracking of pegcetacoplans use and effects over time in usual care settings.

Researchers are evaluating the combination of bleximenib, venetoclax VEN, and azacitidine AZA compared to placebo with VEN and AZA in treating adults with newly diagnosed Acute Myeloid Leukemia AML who have mutations in the NPM1 or KMT2A genes. This Phase 3 study focuses on participants who are not eligible for intensive chemotherapy due to age or other health conditions. The goal is to understand how these treatments work in this specific AML population. Participants receive treatment in 28-day cycles, either with bleximenib plus VEN and AZA or placebo plus VEN and AZA. Bleximenib, VEN, and placebo are taken orally, while AZA is given intravenously or under the skin. Treatment continues until disease progression or unacceptable side effects occur. During the study, participants will be monitored for response to treatment including complete remission and overall survival for up to over four years. Researchers will track event-free survival, duration and timing of remission, transfusion independence, and other health outcomes. Safety is also closely observed through adverse events and lab tests. Participation involves regular visits for treatment and assessments over the study period.

Researchers are evaluating elritercept TAK-226, KER-050, an investigational drug, for treating anemia in adults with very low, low, or intermediate risk myelodysplastic syndromes MDS who need regular red blood cell RBC transfusions. The study is a Phase 3, randomized, double-blind, placebo-controlled trial designed to assess how well elritercept reduces the need for RBC transfusions and to evaluate its safety and tolerability over time. Participants will be randomly assigned in a 21 ratio to receive either elritercept or a matching placebo, both given as subcutaneous injections every 4 weeks. The study includes a Primary Phase lasting 24 weeks and a Secondary Phase lasting an additional 24 weeks, during which participants continue their assigned treatments. Eligible participants may also enter an Extension Phase to continue treatment until individual discontinuation or study unblinding. After treatment ends, a Safety Follow-Up Period of 8 weeks and a long-term follow-up lasting up to 5 years will monitor participants. During the study, participants will have visits approximately every 2 weeks initially, then every 4 weeks, to assess treatment effects and safety. Researchers will evaluate the percentage of participants achieving transfusion independence and monitor adverse events, laboratory values, vital signs, and heart tests. Long-term follow-up will continue through regular check-ins for up to five years or until the participant withdraws or the study ends.

This research aims to observe the real-life effectiveness, safety, and patient-reported outcomes of standard antimyeloma treatments in adults with previously treated relapsed andor refractory multiple myeloma over a 24-month period. The study focuses on participants receiving routine care for this condition as part of their regular medical treatment. Participants with relapsed or refractory multiple myeloma undergoing standard antimyeloma treatments in routine clinical practice will be observed. There is no investigational treatment administered by the study instead, medical records will be the primary data source to track treatments and outcomes during the study. During the study, participants medical records will be reviewed to assess response rates, survival, disease progression, quality of life, and adverse events over up to 52 months. Patient health and well-being will be monitored using standardized questionnaires, and safety will be evaluated through reported side effects. The overall involvement includes observation and data collection without changing the participants treatment plans.

Researchers are evaluating the effectiveness of axatilimab combined with corticosteroids compared to a placebo with corticosteroids as the initial treatment for moderate or severe chronic graft-versus-host disease cGVHD. This Phase 3 randomized, double-blind, placebo-controlled study aims to improve treatment outcomes for patients experiencing cGVHD after allogeneic hematopoietic cell transplantation allo-HCT. Participants will receive either axatilimab or a matching placebo, both administered by intravenous infusion, alongside corticosteroids given orally or by infusion at protocol-defined doses. The study randomly assigns participants to one of these two treatment groups to compare their effects as initial therapy for cGVHD. During the study, participants will be monitored over a period of up to three years to assess event-free survival and other outcomes such as response rates, corticosteroid dose reduction, survival, and safety. Researchers will collect data including laboratory tests, monocyte phenotype changes, and bone health markers. This comprehensive monitoring is designed to evaluate the benefits and risks of the treatments throughout and following the active treatment period.

This research aims to evaluate the long-term safety of luspatercept in participants who have previously taken part in other luspatercept clinical trials for conditions such as Myelodysplastic Syndromes MDS, Beta-thalassemia, and Myeloproliferative Neoplasm-associated Myelofibrosis. It is a Phase 3b, open-label, single-arm rollover study designed to continue monitoring participants who tolerated previous luspatercept treatment and may benefit from ongoing therapy, as well as those in post-treatment follow-up phases. Participants transitioning from prior luspatercept studies will enter a Transition Phase defined by an enrollment visit. Those continuing treatment will receive luspatercept injections subcutaneously at the same dose and schedule as their parent trial, administered by study staff at clinical sites. The study includes a Treatment Phase for ongoing luspatercept administration and a Follow-up Phase comprising a 42-day safety follow-up after the last dose and a long-term post-treatment follow-up phase lasting at least 5 years to monitor overall survival and progression to malignancies. Throughout the study, participants will be regularly assessed for adverse events, progression to high-risk MDS or AML, and development of other malignancies or treatment-related masses. Safety parameters are evaluated during the 42-day follow-up, while long-term survival and disease progression are monitored every six months for at least five years. The study will conclude when all participants have completed five years of combined treatment and follow-up.

Researchers are conducting a combined Phase 2b and Phase 3 clinical trial to study CSL300 Clazakizumab in adults with end stage kidney disease ESKD who are undergoing maintenance dialysis. The study aims to find the right dose of CSL300 and then evaluate its effect on cardiovascular outcomes and safety in people with systemic inflammation and either atherosclerotic cardiovascular disease ASCVD or diabetes. This is a randomized, double-blind, placebo-controlled study involving multiple centers. Participants will receive intravenous IV administration of either CSL300 or a placebo. The Phase 2b part focuses on determining the appropriate dose of CSL300 compared to placebo over about 12 weeks, while the Phase 3 part examines CSL300s effect on cardiovascular events over approximately five years. The study includes different dosing groups in Phase 2b and a larger comparison of CSL300 versus placebo in Phase 3. During the study, participants will be monitored regularly with blood tests that measure inflammation markers such as high-sensitivity C-reactive protein hs-CRP, cardiovascular events, and safety outcomes. Researchers will track changes in various blood components and adverse events up to 32 weeks in Phase 2b and follow cardiovascular outcomes for up to five years in Phase 3. The total participation lasts through these periods with scheduled assessments to evaluate treatment effects and safety.

Researchers are studying acute myeloid leukemia AML, a fast-growing cancer mainly affecting bone marrow cells, especially in older adults. This study focuses on how the inflammasome, a part of the immune system that controls inflammation, affects AML development and the response to chemotherapy. Understanding this connection may help explain why some patients respond differently to treatment and could guide the creation of personalized therapies. Patients with newly diagnosed AML will have biological samples, including peripheral blood and bone marrow, collected and analyzed before and after their first-line chemotherapy. The study includes both prospective and retrospective parts, with patients undergoing standard treatments following clinical guidelines. Common chemotherapy regimens include intensive protocols like 37 with daunorubicin and cytarabine, sometimes combined with other drugs, and non-intensive treatments such as azacitidine with venetoclax. During the study, participants will follow their usual diagnostic and treatment plans while additional blood and bone marrow samples are collected for research purposes. Researchers will perform tests like inflammasome activity assays, gene expression analysis, and immunophenotyping to evaluate treatment response and disease progression. The main outcome is to measure inflammasome activity at diagnosis, in case of treatment failure, and six months after starting chemotherapy. The observation period lasts up to six months, with data collected to monitor disease status and treatment effects.

Researchers are investigating the use of Trastuzumab deruxtecan T-DXd in adults with unresectable or metastatic HER2-low and HER2-ultralow breast cancer. This includes patients who have previously received chemotherapy for metastatic breast cancer or have hormone receptor-positive disease treated with endocrine therapy but are unsuitable for further endocrine treatment. The study aims to understand treatment effectiveness, patient characteristics, and experiences in a real-world setting through a non-interventional approach. Participants will be observed while receiving either T-DXd or conventional chemotherapy as part of their routine care, without any drug administration by the study itself. The study includes two groups one with patients having HER2-low breast cancer treated with T-DXd after prior chemotherapy, and another with hormone receptor-positive, HER2-low or HER2-ultralow breast cancer patients treated with either T-DXd or conventional chemotherapy but not prior chemotherapy for metastatic disease. Data will be collected on treatments, side effects, and management of adverse drug reactions. During the study, participants demographic and clinical data, treatment patterns, tolerability, and quality of life will be monitored over approximately 37 months. Assessments include the time to next treatment, treatment discontinuation, physician-reported safety events, patient-reported tolerability, quality of life questionnaires, and symptom diaries. This long-term observation will help evaluate real-world outcomes and patient experiences with T-DXd and conventional chemotherapy in this population.

Myelodysplastic syndromes MDS primarily affect older adults and involve problems with blood cell production, leading to anemia, low white blood cells, and low platelets. These conditions often cause patients to depend on red blood cell transfusions and carry risks like infection and bleeding. This research evaluates luspatercept, a protein designed to help mature red blood cells, in adults with MDS who have a specific chromosome deletion del5q and who are resistant or intolerant to prior treatments like lenalidomide. The study focuses on patients with low to intermediate risk MDS and aims to assess whether luspatercept can reduce the need for transfusions. Participants will receive luspatercept injections under the skin every three weeks, starting at a dose of 1.0 mgkg, with possible increases up to 1.75 mgkg. The study has a screening period, followed by a two-year treatment phase where patients receive the drug and are closely monitored. After treatment, there is a three-year follow-up to observe longer-term effects. Researchers will track responses such as transfusion independence, hemoglobin levels, quality of life, and safety during and after treatment. Throughout the study, participants will have regular visits for assessments including blood tests, quality of life questionnaires, and monitoring for side effects. The main outcome is whether patients achieve at least eight weeks without needing red blood cell transfusions within the first 24 weeks. Additional measures include long-term transfusion independence, duration of response, reduction in transfusions, changes in iron levels, and overall safety over five years. The total participation lasts up to five years, ensuring comprehensive evaluation of luspatercepts effects.