Torino

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 metastatic cervical cancer, which is cancer that has spread beyond the cervix, the lower part of the uterus. This study evaluates the safety and effectiveness of the antibody drug conjugate sacituzumab tirumotecan sac-TMT combined with pembrolizumab and bevacizumab. The goal is to find out if these treatments, given together or with some variations, help patients live longer or delay cancer progression compared to standard care. The study has two parts. In Part 1, participants receive sac-TMT, pembrolizumab, and bevacizumab together to assess safety. In Part 2, all participants first get standard induction treatment with pembrolizumab, paclitaxel, and cisplatin or carboplatin, possibly with bevacizumab. Those whose cancer does not worsen then enter maintenance treatment, where they are randomly assigned to receive either pembrolizumab alone or sac-TMT plus pembrolizumab, with optional bevacizumab. Participants are involved for up to about 20 months during maintenance treatment after up to 4 months of induction. The study monitors safety by tracking side effects and treatment discontinuations. Effectiveness is measured by progression-free survival and overall survival up to several years. Quality of life and physical functioning are also assessed through questionnaires. Treatments and evaluations occur through regular intravenous infusions and periodic monitoring visits.

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.

Researchers are evaluating the safety and effects of marstacimab, a study medicine, for potential treatment of hemophilia in boys and male children aged 1 to 17 years. The trial focuses on those with severe Hemophilia A or moderately severe to severe Hemophilia B, including participants with or without inhibitors. The study aims to compare participants experiences with marstacimab to their historical bleeding episodes without the medicine. All participants will receive weekly subcutaneous injections of marstacimab. The first dose is administered at the study site by staff, and subsequent doses can be given at home or by study staff during the 12-month treatment period. Enrollment opens sequentially by age groups, starting with adolescents 12-17 years, followed by children 6-11 years, and finally children 1-5 years. The study includes about 1 month of screening, 12 months of treatment, and 1 month of follow-up. Participants will visit the study site at least 10 times during the study, with the option for two visits to occur at home if allowed. They will also receive six phone calls approximately every two months. Researchers will monitor bleeding rates, adverse events, injection site reactions, and immune responses. The overall participation lasts about 14 months, including screening, treatment, and follow-up periods.

This research aims to assess the effectiveness of a combination of rituximab and golcadomide as a chemotherapy-free treatment for older, frail patients newly diagnosed with Diffuse Large B Cell Non-Hodgkin Lymphoma DLBCL. The study focuses on patients considered frail based on a simplified geriatric assessment sGA and who are not suitable candidates for standard anthracycline-based chemotherapy treatments like R-CHOP. It is a prospective, multicenter, phase II trial designed to explore this targeted approach in a vulnerable population. Participants will undergo an induction phase receiving rituximab, golcadomide, and dexamethasone during the first cycle, with a total of up to six 28-day cycles. Response evaluations occur after the fourth and sixth cycles to determine treatment continuation or discontinuation. Patients achieving at least a partial response proceed to a consolidation phase involving golcadomide alone for up to six additional cycles, possibly alongside involved site radiotherapy on PET-positive areas. Those with stable or progressive disease at interim assessments will stop the protocol treatment and receive alternative therapies. Throughout the study, participants are closely monitored with imaging scans such as PETCT or CT for disease and sarcopenia assessment, and quality of life evaluations at multiple points including baseline, six months, and twelve months. Follow-up continues for 24 months with regular visits to track progression-free survival and overall health. Treatment failures are followed for survival until study completion. The study also records treatment discontinuation rates and quality of life changes to comprehensively understand the treatment impact.

Researchers are studying how different surgical methods affect nipple and skin sensation after nipple-sparing mastectomy NSM in women. This study compares three types of NSM conventional open surgery, minimally invasive endoscopic surgery, and robotic-assisted surgery. The goal is to understand how much nipple sensation remains after each type and to evaluate surgery-related complications, patient-reported quality of life, body image, and tissue nerve structure. Participants will have one of the three NSM surgeries, followed by immediate breast reconstruction with an implant during the same operation. The conventional method involves a visible breast incision, the endoscopic method uses a small armpit incision with a camera and instruments, and the robotic method is performed through a small armpit incision with robotic assistance. Each participants nipple sensation will be tested before surgery and again at 1, 3, and 6 months after surgery using a device called the Semmes-Weinstein esthesiometer. During the study, participants will complete sensation tests and quality of life surveys at scheduled times. Tissue samples from surgery will be analyzed to explore the nerve structures related to sensation. Researchers will monitor surgical complications up to 3 months post-surgery. The study provides important data on physical and emotional outcomes to help improve care for women undergoing mastectomy and reconstruction.

Primary immune thrombocytopenia ITP is a condition where the immune system mistakenly destroys platelets, leading to a lower number of platelets and increased risk of bruising or bleeding. This Phase 3 study evaluates the long-term safety, tolerability, and effectiveness of mezagitamab in adults with chronic primary ITP. The study also investigates how the body processes mezagitamab over an extended period. Participants who completed previous mezagitamab studies TAK-079-3002 or TAK-079-1004 will be invited to join this continuation trial. Eligible participants may receive mezagitamab injections on demand, with treatment courses repeated as needed based on specific criteria and the investigators clinical judgment. The treatment is administered subcutaneously. During the study, participants will visit the clinic several times for assessments. Researchers will monitor safety by tracking treatment-emergent adverse events, and evaluate effectiveness through platelet response and remission rates. Measurements of drug levels and antibodies will also be taken. The study may last up to approximately 108 weeks, allowing detailed long-term follow-up.

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 evaluating the long-term safety and tolerability of dazodalibep in people with Sjgrens Syndrome. This phase 3 open-label study extends previous trials by continuing to monitor participants who completed 48 weeks of treatment with dazodalibep or placebo. The study is sponsored by Amgen and aims to better understand the safety profile of dazodalibep over an extended period. Participants who finished the initial 48-week trials HZNP-DAZ-301 or HZNP-DAZ-303 will receive an assigned dose of dazodalibep intravenously for an additional 132 weeks. This extension study involves a single treatment group receiving dazodalibep without placebo, focusing on ongoing treatment effects and participant safety. During the study, participants will be monitored for treatment-emergent adverse events for up to 152 weeks. Researchers will also measure the presence of anti-drug antibodies and plasma concentrations of dazodalibep for up to 132 weeks. Participants need to be available for all study visits and procedures, with safety assessments conducted regularly throughout the long-term extension period.

This research aims to gather long-term safety and effectiveness information for people treated with ibrutinib, a medicine taken by mouth that blocks a specific enzyme called brutons tyrosine kinase. The study focuses on participants who previously took part in ibrutinib studies that have finished and are still receiving ibrutinib treatment, continuing to benefit from it. It is an open-label study, meaning both participants and researchers know the treatment being given. Participants will continue taking ibrutinib capsules daily at the dose they were given in their prior study until the doctor decides the treatment is no longer helpful due to disease progression or side effects, the participant chooses to stop, other treatment options become available, or the study ends. Safety will be monitored throughout, and effectiveness data may be combined with previous study results. No formal testing of hypotheses is planned in this extension. During the study, participants will be regularly monitored for safety and disease status. The main outcome is the number of participants experiencing side effects within 30 days after the last ibrutinib dose or before starting another cancer therapy. Participants may continue treatment until alternative access to ibrutinib is arranged or the study ends, which is planned for December 2029. Researchers will collect ongoing data to understand the long-term effects of ibrutinib treatment.