Bari

This observational study aims to create a national registry in Italy for multiple myeloma to monitor routine clinical practice and describe the standard care used for diagnosis and treatment. Multiple myeloma accounts for a small percentage of tumor diagnoses in Italy, with incidence rates of 9.5 per 100,000 males and 8.1 per 100,000 females. The registry will help analyze current treatment patterns and patient demographics to address future changes in care. The study involves a non-interventional, multicenter registry collecting both retrospective and prospective data from patients diagnosed with multiple myeloma since January 1, 2019. Data will be gathered using an electronic platform, and patients will be enrolled consecutively during their regular appointments after providing informed consent. Patients participating in other studies can also be included, with baseline and survival data collected for those in interventional trials. Participants will have hospital visits every six months, where clinical data and patient-reported outcomes will be recorded. Researchers will assess overall survival and time to next treatment over three years, along with patient-reported outcomes and costs incurred by patients. The study provides long-term monitoring without altering patients usual care, and participation duration may vary depending on individual follow-up.

Researchers are investigating new treatments for advanced renal cell carcinoma RCC that has returned after prior therapy. The study aims to find out if the combination of belzutifan and zanzalintinib can help people with recurrent advanced RCC live longer without their cancer worsening compared to the drug cabozantinib. This is a phase 3 randomized trial evaluating these treatments in participants who have experienced recurrence during or after prior anti-PD-1L1 therapy. Participants are randomly assigned to receive either belzutifan plus zanzalintinib taken orally once daily or cabozantinib taken orally once daily. They continue their assigned treatment until certain reasons require stopping the study intervention. The study compares the effects of these treatments on cancer progression and survival among people with advanced RCC who have had disease recurrence after adjuvant therapy. During the study, participants will be regularly monitored for progression-free survival and overall survival for up to about 73 months. Researchers will also assess tumor response, duration of response, adverse events, and quality of life using questionnaires over approximately 25 months. The study involves ongoing evaluations to understand how these treatments affect symptoms, functioning, and overall health during long-term follow-up.

Researchers are evaluating the effectiveness and safety of an investigational drug called BNT323 also known as DB-1303 compared with standard chemotherapy in women with recurrent endometrial cancer. The study includes two groups based on the level of HER2 protein in the tumor Cohort 1 with HER2 levels 1 or 2 who have been previously treated with immune checkpoint inhibitors, and Cohort 2 with HER2 level 3. The study aims to understand how well BNT323 or chemotherapy controls cancer progression and how the drug affects patients immune response and quality of life. Participants in Cohort 1 will be randomly assigned to receive either BNT323 or chemotherapy drugs such as doxorubicin, paclitaxel, or docetaxel. In Cohort 2, participants will receive BNT323 alone. Treatments are given intravenously and continue until the cancer progresses, unacceptable side effects occur, or consent is withdrawn. The study includes screening, treatment, safety follow-up, efficacy follow-up, and a long-term survival follow-up lasting up to about 53 months. During the study, participants will undergo regular assessments including tumor evaluations, safety monitoring, and quality of life questionnaires. Researchers will measure progression-free survival in Cohort 1 and tumor response rate in Cohort 2. Safety is monitored by tracking adverse effects and drug levels in the body. Participants can expect to be followed for up to 53 months after treatment to assess long-term outcomes and survival.

This research is a global, multicenter, prospective observational registry studying patients with Pompe disease, including both late-onset LOPD and infantile-onset IOPD forms. It enrolls both untreated patients and those receiving approved therapies to better understand the long-term safety, real-world effectiveness, and quality of life impacts of treatments for Pompe disease. The study also aims to describe the natural history of untreated Pompe disease. Participants include groups receiving different enzyme replacement therapies such as Cipaglucosidase alfa with Miglustat, other approved ERTs like Alglucosidase alfa or Avalglucosidase alfa, as well as untreated individuals who are not on any medical therapy for Pompe disease. No experimental treatments are given as this is an observational study tracking real-world treatment use and outcomes. During the study, participant data on adverse events, treatment effectiveness, quality of life, and patient-reported outcomes will be collected over a period of at least five years. Researchers will monitor safety through the frequency of adverse events and serious adverse events. Participants health and treatment impacts will be regularly evaluated to provide long-term insights into Pompe disease management and outcomes.

Researchers are investigating the long-term effects of a multidomain program that includes physical activity, choir singing, learning, and horticulture on preventing cognitive decline in older adults who show signs of frailty. The study aims to see if engaging in these varied activities, especially music, can slow down cognitive and perceptual decline, encourage positive brain changes during aging, and reduce loneliness by promoting social interaction. Participants will be community-dwelling elderly individuals aged 65 and older. Participants will be randomly assigned to one of three groups one that takes part in the multidomain intervention including choir and other activities, an active control group that engages in several activities excluding music, and a passive control group that does little or sporadic activity. The program lasts for nine months, during which participants regularly attend their assigned activities. Researchers will collect saliva samples and perform various assessments before and after the program, as well as six months later. Throughout the study, participants will be tested three times to measure brain-derived neurotrophic factor BDNF levels, cognitive abilities, hearing function, frailty status, and psychosocial well-being. They will also keep weekly diaries to track the time spent on activities. The study includes detailed evaluations such as neuropsychological tests, audiometric exams, and brain response measurements to understand how these interventions impact brain health and overall function over time. The total study duration for each participant covers the nine-month intervention plus a six-month follow-up.

Researchers are evaluating the effectiveness of a personalized prevention program PPP based on the Comprehensive Geriatric Assessment CGA for older adults with non-communicable chronic diseases NCDs. The study aims to reduce hospitalization rates and explore biological factors related to aging and frailty. This research involves 1216 participants recruited by General Practitioners GPs in Italy and focuses on the impact of the CGA in primary care settings. Participants are randomly assigned to two groups an intervention group that receives the PPP guided by the CGA and a control group receiving standard clinical care. The PPP includes assessments using the Brief-MPI scale covering motor, cognitive, nutritional, medication, vaccination, daily activities, and living situation domains. Participants in the intervention group receive brochures with tailored advice for 12 months, specialist referrals if needed, and provide saliva samples for biomarker analysis. The control group continues usual care without CGA evaluation or saliva collection. All participants will be monitored at baseline, 6 months, and 12 months with follow-up contacts to assess adherence, satisfaction, and health outcomes. Researchers will measure hospitalization rates as the primary outcome and secondary outcomes including combined health events, unplanned GP visits, and mortality rates. The study also collects data on psychological wellbeing and resilience. Statistical analyses will compare outcomes between groups over 12 months to assess the programs impact.

Researchers are studying sonrotoclax alone and in combination with dexamethasone plus carfilzomib, daratumumab, or pomalidomide to evaluate its safety, tolerability, and effectiveness in adults with relapsed or refractory multiple myeloma who have the chromosomal translocation t1114. This phase 1b2 trial aims to find the best doses and assess how well these treatments work, focusing on patients whose disease has progressed after previous therapies. Participants receive sonrotoclax as an oral daily medication, with dexamethasone given once weekly either orally or intravenously. Additional drugs include carfilzomib administered intravenously weekly, daratumumab given subcutaneously weekly, and pomalidomide taken orally daily. The study includes a dose-escalation phase to identify the maximum tolerated dose and up to seven expansion groups to further evaluate safety and response in different treatment combinations. During the study, participants are closely monitored for side effects and treatment responses through regular assessments including blood tests and bone marrow analysis. Key outcomes measured include dose-limiting toxicities, adverse events, and various levels of treatment response over approximately four years. This comprehensive follow-up helps researchers understand how the treatments affect disease progression and overall survival.

Researchers are comparing INCA033989 with the best available therapy for adults who have essential thrombocythemia ET with a CALR mutation and have previously received cytoreductive treatment. The study aims to evaluate the effects of these treatments on this specific patient group. It is a Phase 3 clinical trial sponsored by Incyte Corporation to assess treatment responses and safety. Participants will be randomly assigned to receive either INCA033989 administered intravenously or the best available therapy chosen by their doctor. The treatments are given according to the study protocol. The study focuses on treatment outcomes over a period of weeks, including response durability and symptom changes, with assessments at specified timepoints. During the study, participants will have regular visits to monitor their clinical and hematologic responses, symptoms, and any side effects. Researchers will collect data on mutation levels, symptom questionnaires, and fatigue assessments up to 48 weeks. Safety monitoring will continue for 60 days following the last dose. The total duration of participation may extend up to several months as outlined by the trial schedule.

Researchers are investigating how plant-based diets affect the gut microbiome and related metabolites in patients with smoldering multiple myeloma sMM. The study aims to show that diets rich in fiber can promote beneficial gut bacteria that produce short-chain fatty acids SCFAs, potentially preventing progression to multiple myeloma. This national, multicenter study involves up to 62 sMM patients and focuses on the impact of dietary changes on gut health and immune balance. Participants will be encouraged to follow a balanced, high-fiber diet for 12 weeks, choosing from various diet plans based on their preferences. Medical visits will take place at enrollment and at week 12, with nutritional counseling provided initially, at one month, and as needed. Stool and blood samples will be collected before the diet starts, and again at 4 and 12 weeks to monitor changes in the gut microbiome and SCFA levels. Dietary adherence will be tracked daily through questionnaires and food diaries. During the study, participants will have routine medical visits and sample collections aligned with standard sMM follow-up care. Researchers will compare biological data from baseline and follow-up samples to assess diet effects on SCFA concentration and gut microbiome composition. Evaluation of diet adherence and safety monitoring will also be ongoing throughout the trial, lasting 12 weeks with encouragement to continue plant-based eating beyond this period.

Researchers are conducting a large prospective, observational cohort study to evaluate the clinical impact of new Monoclonal Antibodies MAB in patients with B-cell Non-Hodgkin Lymphoma NHL treated in Italian clinical practice. The study focuses on collecting information about the use, feasibility, effectiveness, and both short- and long-term side effects of novel MABs that have been approved by the European Medicines Agency since 2020 and prescribed according to authorized indications in Italy. Participants will be divided into groups based on treatment indication, antibody type, and lymphoma subtype to allow detailed analysis. The study observes patients with B-cell NHL who have received at least one dose of a novel MAB either alone or in combination, as authorized for use in Italy. Both patients receiving first-line treatment and those with relapsed or refractory disease are included. Various cohorts and sub-cohorts will be analyzed to understand outcomes by indication, antibody type, and histological subtype, providing a comprehensive overview of these treatments in real-life settings. Participants will be followed for at least five years to assess outcomes such as overall response rate, complete response rate, progression-free survival, overall survival, event-free survival, time to next treatment, non-relapse mortality, duration of response, and the incidence of early and late adverse events. Clinical data collection will include treatment details, safety monitoring, and long-term follow-up to evaluate both effectiveness and toxicity. The study spans several years, allowing researchers to capture extensive real-world evidence on novel MAB use in B-cell NHL.