Busto Arsizio

Researchers are evaluating Enzomenib DSP-5336, an oral drug, in patients with various types of acute leukemia, including relapsed or refractory acute myeloid leukemia AML, acute lymphocytic leukemia ALL, and acute leukemia of ambiguous lineage. The study also includes patients with high-risk myelodysplastic syndromes MDS and relapsed multiple myeloma MM in selected sites. This phase 12 trial aims to assess the safety, pharmacokinetics, pharmacodynamics, and clinical activity of DSP-5336 alone or combined with standard AML treatments, particularly in patients with specific genetic mutations like MLL rearrangement or NPM1 mutation. The study involves dose escalation and dose expansion of DSP-5336 administered orally. Participants may receive DSP-5336 alone or combined with standard AML regimens such as venetoclax plus azacitidine or intensive chemotherapy with cytarabine and daunorubicin 73. Different study arms include patients with or without certain medications like CYP3A4 inhibitor azoles, and those with specific genetic profiles. The trial evaluates recommended phase 2 doses for various patient groups and combination treatments. Participants will undergo assessments including monitoring for adverse events within 30 days after the last dose and evaluation of clinical responses approximately six months after treatment begins. Researchers will collect blood and bone marrow samples for genomic analysis and track drug levels in the body. Safety labs, ECGs, physical exams, and patient questionnaires will be performed throughout the study. The trial includes long-term follow-up of overall survival up to two years after treatment ends, with visits and tests scheduled to monitor health and treatment effects.

Researchers are evaluating the effect of AZD6793, an oral medication, in adults with moderate to very severe chronic obstructive pulmonary disease COPD. This Phase IIb, randomized, double-blind, placebo-controlled study involves approximately 970 participants across about 350 global sites. The trial aims to compare the efficacy and safety of two different doses of AZD6793 against placebo over a 24-week period. Participants will be randomly assigned to one of three groups receiving either dose 1 of AZD6793, dose 2 of AZD6793, or a matching placebo tablet. The study medication is taken orally and the trial lasts for 24 weeks. The study is designed as a parallel-group format with a 111 allocation ratio among the three arms. During the study, participants will be monitored through various assessments including lung function tests measuring forced expiratory volume FEV1, questionnaires evaluating breathlessness, cough, sputum, and quality of life, and tracking of COPD exacerbation events. Blood samples will be collected to measure plasma concentrations of AZD6793. Safety and efficacy outcomes will be evaluated up to 24 weeks, with the main outcome being the rate of moderate or severe COPD exacerbations.

Researchers are investigating how emotional distress ED affects treatment outcomes in patients with metastatic breast cancer mBC who are receiving first-line therapies. This study focuses on the impact of baseline emotional distress on the effectiveness of various treatments according to breast cancer subtypes. Emotional distress is common among breast cancer patients and has been linked to poorer treatment adherence and quality of life, but its role in metastatic breast cancer treatment success is not well understood. Participants will be grouped based on their breast cancer subtype and receive standard first-line treatments accordingly triple-negative PD-L1 positive patients receive chemotherapy with immune checkpoint inhibitors, triple-negative PD-L1 negative patients receive chemotherapy alone, HR-positiveHER2-negative patients receive a CDK46 inhibitor plus endocrine therapy, and HER2-positive patients receive chemotherapy combined with trastuzumab and pertuzumab. Emotional distress and quality of life will be assessed through questionnaires at baseline and during treatment. During the study, patients will complete specific questionnaires to measure emotional distress, quality of life, and fear of cancer progression. Researchers will monitor progression-free survival and objective response rates over two years, comparing outcomes based on emotional distress levels at the start and early in treatment. Quality of life and psychological factors will also be evaluated over three months. All participants must provide informed consent and meet eligibility criteria before joining.

Researchers are evaluating different treatment approaches for patients with relapsed or refractory Acute Myeloid Leukemia AML. This multicenter, randomized, open-label clinical trial compares high intensity reinduction chemotherapy to low intensity therapies for patients experiencing their first or second relapse. The study addresses the need to better understand and compare these therapies as new personalized treatments emerge but have not yet been fully tested against standard chemotherapy in this setting. Participants will be randomly assigned to receive either low intensity treatments or high intensity chemotherapy. Low intensity therapies involve innovative targeted drugs alone or combined with other agents, while high intensity treatments use high doses of chemotherapy drugs, possibly combined with antibodies or targeted agents. Both treatment options will be provided according to local practices, and the study follows a pragmatic approach to reflect real-world clinical use. During the study, participants will be monitored for clinical outcomes including event-free survival over 36 months. Researchers will assess overall survival, response rates, quality of life reported by patients, and safety of the therapies. Participants health will be regularly evaluated while receiving treatment to gather data on these outcomes. The total study duration extends up to three years to capture these long-term results and better understand the benefits and risks of each therapy.

Researchers are conducting an observational, prospective cohort study to collect data from patients diagnosed with relapsed or refractory acute myeloid leukemia RR AML. This hard-to-treat condition has a poor survival rate despite advances in intensive treatments. The study aims to gather real-world data on outcomes and the effectiveness of new therapies, including in diverse populations such as children, elderly, and those with rare mutations across Europe. Participants will be registered into the STREAM platform where baseline and follow-up data will be recorded according to their normal clinical care. Patients will be followed for up to 4 years, with no experimental treatments assigned by the study. The study facilitates data collection across multiple sites using a GDPR-compliant system to allow cross-country participation and to support future international trials. Participants will provide informed consent and their clinical information, diagnosis methods, treatment options, and outcomes will be monitored. Researchers will measure overall survival, remission rates, event-free survival, relapse-free survival, transplant rates, and associations between clinical and biological factors over 8 years. This study supports understanding RR AML in routine practice and helps identify populations for future research.

Researchers are studying children with Autism Spectrum Disorders ASD aged 0 to 11 years to evaluate the impact of ongoing real-life treatments provided in three different National Health Service settings. This observational study aims to explore multidimensional and comprehensive outcome measures that reflect global functioning, quality of life, and shared decision making. The study addresses the need for more integrated and long-term evaluation tools in ASD care, following Italian national guidelines. Participants will continue their usual treatments in line with national autism guidelines at different health facilities, representing diverse service organizations. New outcome tools, including the Child and Adolescent Needs and Strengths CANS scale, the Developmental Disabilities-Child Global Assessment Scale DD-CGAS, and the Pediatric Quality of Life Inventory PedsQL, will be used to assess changes pre- and post-intervention. The study will investigate correlations between these tools and identify clusters related to needs and strengths. During the study, children will undergo assessments using these scales from March 2025 to August 2026 to measure global functioning, quality of life, and evolving needs. Researchers will evaluate the acceptability and usefulness of the outcome tools for clinicians, patients, and families, as well as their sustainability in daily practice. The primary outcome focuses on identifying specific CANS item clusters that differentiate user profiles at baseline, with several secondary outcomes measuring changes over time.

Researchers are evaluating surovatamig AZD0486 monotherapy in adults with relapsed or refractory B-cell non-Hodgkin lymphoma NHL who have previously received at least two different treatments. This Phase 2, global, multi-center, open-label study includes two modules one for follicular lymphoma FL and another for large B-cell lymphoma LBCL. The goal is to assess the efficacy, safety, and tolerability of surovatamig at the recommended Phase 2 dose RP2D. Participants receive surovatamig as an intravenous infusion in both modules. Module 1 focuses on relapsedrefractory FL, and Module 2 on relapsedrefractory LBCL. The study is single-arm and open-label, meaning all participants receive the study drug. Treatment is given in repeated 28-day cycles, with dosing and safety monitored throughout the treatment period. During the study, participants undergo regular assessments including scans to measure tumor response, blood tests to monitor drug levels and safety, and evaluations for side effects. The primary outcome is the overall response rate assessed up to approximately 24 months for Module 1 and 12 months for Module 2. Secondary outcomes include duration of response, survival measures, and quality of life assessments, with follow-up lasting up to about 5 years to monitor long-term effects and safety.