Busto Arsizio

Researchers are evaluating a phase 1/2 open-label study to investigate the safety, pharmacokinetics, pharmacodynamics, and clinical effects of an oral drug called Enzomenib (DSP-5336) in patients with acute leukemia, including relapsed or refractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), ambiguous lineage acute leukemia, and in certain sites, high-risk myelodysplastic syndromes (MDS) or relapsed multiple myeloma (MM). The study also examines Enzomenib combined with standard AML treatments such as venetoclax plus azacitidine and the intensive chemotherapy 7+3 regimen in patients newly diagnosed with AML who have specific genetic mutations (MLL rearrangement or NPM1 mutation). Participants receive oral Enzomenib either alone or combined with other drugs: venetoclax and azacitidine for a nonintensive treatment group, gilteritinib for a certain relapsed AML group, or intensive chemotherapy with cytarabine and daunorubicin (7+3) for newly diagnosed AML patients. The study includes dose escalation and expansion phases to determine recommended doses for phase 2. Treatment schedules and doses are adjusted based on response and safety, with some patients enrolled in specialized cohorts according to their genetic markers. Throughout the study, participants undergo regular assessments including clinical exams, laboratory tests, bone marrow samples for genetic analysis, and monitoring for adverse events. Researchers measure safety outcomes such as adverse and serious adverse events, determine optimal dosing for phase 2, and evaluate treatment effectiveness by tracking complete response rates. Safety is monitored up to 30 days after the last dose, with dose recommendations made within four months of treatment start and response assessed around six months. The total participation time varies based on individual treatment and study phase.

Researchers are evaluating the effects and safety of AZD6793 tablets in adults aged 40 years and older who have moderate to very severe chronic obstructive pulmonary disease (COPD). This is a Phase IIb, multicenter, randomized, double-blind, placebo-controlled study involving approximately 1160 participants at around 400 sites worldwide. The study aims to compare three different doses of AZD6793 against placebo tablets over 24 weeks to assess how well the treatment works and its safety profile in this population. Participants will be randomly assigned to one of four groups receiving either one of three doses of AZD6793 or a placebo in equal proportions. The treatment involves oral administration of AZD6793 tablets or placebo tablets daily for 24 weeks. The study is designed with parallel groups and includes careful dose-ranging to evaluate different levels of the investigational drug. During the study, participants will be monitored for the annualized rate of moderate or severe COPD exacerbations from baseline up to 24 weeks. Assessments include lung function tests such as pre- and post-bronchodilator FEV1/FVC ratios, symptom questionnaires like the COPD Assessment Test (CAT), and documentation of COPD exacerbation history. Safety will be continually evaluated through clinical assessments and laboratory tests throughout the treatment period.

Metastatic breast cancer (mBC) is a common and serious condition affecting many patients worldwide. Emotional distress (ED) is reported by about half of breast cancer patients and can negatively influence treatment adherence, symptom management, and quality of life. This research investigates how baseline emotional distress impacts the outcomes of patients with mBC receiving their first line of treatment, aiming to better understand its effect on therapy effectiveness. Participants will receive first-line treatments based on their breast cancer subtype, including endocrine therapy, chemotherapy, immunotherapy, and targeted therapy. They will be grouped into cohorts depending on their disease characteristics and treatment plans, such as immunotherapy combined with chemotherapy or targeted therapies like CDK4/6 inhibitors and trastuzumab. During the study, patients will complete questionnaires to assess emotional distress and quality of life. Throughout the study, researchers will monitor participants' progression-free survival over two years, focusing on differences related to emotional distress at baseline. Patients will undergo evaluations through specific questionnaires about their emotional and quality of life status. The study includes careful observation of treatment outcomes and safety, providing valuable information on how emotional health relates to treatment effectiveness in metastatic breast cancer.

Researchers are evaluating treatments for adults with relapsed or refractory Acute Myeloid Leukemia (AML) in this randomized, open-label clinical trial. The study compares the effects of low-intensity therapies versus high-intensity reinduction chemotherapy in patients experiencing their first or second relapse. Funded by the European Commission, the trial aims to determine whether low-intensity treatments provide similar clinical benefits as high-intensity chemotherapy over a 36-month period. Participants will receive either high-intensity chemotherapy regimens, such as combinations including Cytarabine, Mitoxantrone, or Fludarabine, or low-intensity therapies like Venetoclax with hypomethylating agents, Gilteritinib, or other targeted drug combinations. Both treatment options will be selected based on local availability and clinical suitability. The study follows a pragmatic approach, focusing on real-world treatment settings and personalized options for each patient. Throughout the trial, researchers will monitor patient outcomes, including event-free survival over three years. Assessments will include clinical evaluations, treatment adherence, and safety monitoring. Patients must be able to provide informed consent and will be followed closely to compare the effectiveness and tolerability of the different therapy intensities in managing relapsed or refractory AML.

Researchers are studying patients diagnosed with relapsed or refractory acute myeloid leukemia (AML), a challenging form of AML where the disease returns or does not respond to treatment. Despite improvements in treatment, many patients still face poor survival rates, and no standard care exists for these difficult cases. This study aims to collect detailed real-world data from patients of all ages and AML subtypes across Europe to better understand treatment outcomes and support future clinical trials. Participants who meet eligibility requirements will be registered in the STREAM platform, where their baseline health information and ongoing treatment details will be recorded. Patients will be followed according to standard medical care practices for up to four years, with no specific treatment interventions mandated by the study itself. This observational approach allows for broad participation, including patients with rare mutations, post-transplant relapse, and those from diverse geographic and clinical backgrounds. During the study, researchers will gather and monitor data related to patient outcomes, including overall survival over an eight-year period. Information will be collected through existing clinical practices and registries, ensuring patient privacy with specialized data protection tools. The study's findings aim to improve knowledge about rare patient groups and contribute to the development of new treatment strategies worldwide.

This research focuses on individuals with Autism Spectrum Disorders (ASD), a complex and lifelong condition influenced by various factors including treatments and life environments. The study aims to evaluate multidimensional outcome measures that consider global functioning, development, and long-term changes in real-world settings. It involves patients from three different NHS centers in Italy, grouped into two age categories: 0-5 years and 6-11 years. The study will explore correlations between new and existing outcome tools, assess their acceptability for patients, families, and operators, and investigate their usefulness and sustainability in daily clinical practice. Participants will undergo assessments using multiple tools such as the DD-CGAS, a clinician-rated scale of global child functioning; the Pediatric Quality of Life Inventory (PedsQL), which measures physical, emotional, social, and academic functioning; and the Child and Adolescent Needs and Strengths (CANS) scale, assessing life functioning, behavioral needs, risks, strengths, and caregiver factors. These instruments will be applied pre- and post-intervention over the study timeline. During the study, researchers will analyze specific clusters of CANS items to differentiate user profiles and understand their needs and strengths from March 2025 to August 2026. Data collection includes quality of life measures, global functioning assessments, and multidimensional evaluations. The study also monitors shared decision making and the quality of care improvement. Participant involvement includes completing questionnaires, clinician assessments, and ongoing monitoring of treatment outcomes to support comprehensive care evaluation.

Researchers are evaluating the safety, tolerability, and effectiveness of Surovatamig (AZD0486) given alone in adults with relapsed or refractory B-cell non-Hodgkin lymphoma (B-NHL), including follicular lymphoma (FL) and large B-cell lymphoma (LBCL). This Phase 2, global, multicenter, open-label study focuses on participants who have received at least two previous treatments. The study is divided into two modules: Module 1 for FL and Module 2 for LBCL. Participants receive Surovatamig as an intravenous infusion at the recommended Phase 2 dose (RP2D). The study is single-arm and open-label, meaning all participants receive the drug and both researchers and participants know the treatment being given. The study evaluates how well the drug works and its safety profile in this patient population. Participants will be monitored from the first dose until the end of treatment or data cutoff, which is up to about 24 months for Module 1 and up to about 12 months for Module 2. Researchers will measure overall response rate through central review. Throughout the study, participants undergo assessments including imaging to measure lymphoma lesions, blood tests for organ function, and performance status evaluations. Safety and tolerability are carefully tracked during treatment.