Tromso

This research investigates the long-term effects of mirikizumab in children and adolescents aged 2 to 19 years with moderate-to-severe ulcerative colitis or Crohn's disease. The study is designed as a Phase 3, multicenter, open-label extension trial aiming to assess the ongoing safety and efficacy of this treatment in pediatric participants. It includes those who have completed previous related studies and are expected to benefit from continued mirikizumab treatment. Participants will receive mirikizumab either by subcutaneous injection or intravenous infusion as part of this extended treatment. The study may last approximately 172 weeks and involve up to 44 visits over this period. There is also a possibility for participants to continue receiving treatment through a Continued Access Period after the main study. Throughout the study, participants will be regularly monitored with clinical assessments to determine remission status using the Modified Mayo Score for ulcerative colitis and the Pediatric Crohn's Disease Activity Index for Crohn's disease at week 52. Safety and efficacy will be closely followed, including the evaluation of any adverse events or changes in disease activity, ensuring comprehensive long-term observation.

Researchers are evaluating whether the drugs retatrutide and tirzepatide can prevent major adverse liver outcomes (MALO) in adults with metabolic dysfunction-associated steatotic liver disease (MASLD) who are at high risk. This Phase 3 trial enrolls about 4,500 adults with MASLD identified by non-invasive tests indicating an increased likelihood of developing serious liver problems. The study aims to understand how these treatments might affect liver health over time compared to a placebo. Participants will be randomly assigned to receive either retatrutide, tirzepatide, or a placebo, all given by subcutaneous injection. The study will last approximately 224 weeks, during which participants may attend 25 to 30 clinic visits for monitoring and assessment. After the main study, eligible participants can join an optional 2-year extension where all will receive either retatrutide or tirzepatide regardless of their original group. Throughout the trial, participants’ liver function and disease progression will be closely monitored through various health assessments. Researchers will track the time to the first major adverse liver event as the main outcome. Safety and health status will be evaluated regularly during clinic visits, ensuring thorough observation over the long study period.

Researchers are investigating a new clinical pathway for managing borderline resectable and locally advanced pancreatic cancer in Norway. This nationwide prospective study aims to evaluate how often surgery can successfully remove tumors and assess survival rates following primary chemotherapy. The goal is to achieve a 50% resection rate for borderline resectable pancreatic cancer and 15% for locally advanced cases, with targeted survival and complication rates after surgery based on national guidelines. Patients receive chemotherapy according to Norwegian standards, preferably with mFOLFIRINOX or gemcitabine-nab-paclitaxel. Surgery is planned within four weeks after completing chemotherapy, involving different types of pancreatic removal procedures, potentially including vascular resections. Diagnostic steps include endoscopic ultrasound biopsy for tumor confirmation and molecular analysis, with optional PET/CT scans at baseline and after at least two months of chemotherapy to assess treatment response. Participants undergo careful evaluations including histological diagnosis, imaging, and monitoring throughout treatment. The main measure of success is the rate of tumor removal by surgery from November 2024 to December 2027. Researchers also track survival, complications, and treatment outcomes to better understand the effectiveness of this personalized approach over time.

Researchers are evaluating the effects of guselkumab, a medication targeting the IL-23 pathway, in children aged 2 to 17 years with moderately to severely active Crohn's Disease. This Phase 3 study aims to assess both clinical and endoscopic improvement by the end of one year of treatment, focusing on participants who responded to guselkumab after 12 weeks. Participants receive guselkumab either as an injection under the skin or through an intravenous infusion. The study measures effectiveness at Week 52, following initial response at Week 12, to determine remission rates and healing observed via endoscopy. Throughout the study, children will be closely monitored with clinical assessments, endoscopic exams, and evaluation of disease activity scores. Researchers will track safety and treatment response over the 52-week period to understand guselkumab's role in managing pediatric Crohn's Disease.

Researchers are evaluating mirikizumab in children and adolescents aged 2 to 17 years with Crohn's disease, including those with fistulizing disease and active inflammation in the colon, ileum, or both. This Phase 3 study aims to assess how well mirikizumab works, its safety, tolerability, and how it is absorbed in the body for pediatric participants who have moderately to severely active Crohn's disease and a history of inadequate response or intolerance to other treatments. Participants will be randomly assigned to receive mirikizumab either intravenously or by injection under the skin. The study consists of a 12-week induction period to start treatment, followed by a maintenance period lasting up to Week 52, and a safety follow-up period continuing for up to 16 weeks after treatment. The entire study will last about 74 weeks and may include up to 19 visits for treatment and assessments. During the study, participants will undergo evaluations including endoscopy to assess disease activity, clinical assessments using the Pediatric Crohn's Disease Activity Index (PCDAI), and monitoring for treatment response and remission at Weeks 12 and 52. Safety and tolerability will be closely followed throughout the study and during the safety follow-up. The main outcomes measured are the percentage of participants achieving clinical and endoscopic response over the study period.

Researchers are evaluating the safety and effectiveness of nipocalimab, compared with a placebo, in lowering the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT). This condition involves a low platelet count in newborns caused by maternal antibodies, and the study focuses on pregnant women who have had previous pregnancies affected by FNAIT but without severe bleeding or brain hemorrhage. This is a Phase 3, double-blind, randomized, placebo-controlled trial targeting pregnant women at risk for this condition. Participants will receive either nipocalimab or a placebo intravenously during their current pregnancy. Nipocalimab is the drug being tested, and both the drug and placebo are given by intravenous infusion. The study enrolls women between 13 and 18 weeks of pregnancy and will monitor their pregnancies closely to assess the effects of the treatments on the fetus and newborn. Throughout the study, researchers will monitor the health of both the mothers and their babies. This includes physical exams, medical history reviews, vital signs, ECGs, and lab tests. The main outcome being measured is whether the fetus or newborn experiences death, severe bleeding, or a very low platelet count within one week after birth. Participants and their babies will be followed until the last visit to ensure safety and evaluate treatment impact, with the total involvement lasting through pregnancy and shortly after birth.

Hidradenitis suppurativa (HS) is a chronic and often painful skin disease that causes lumps, abscesses, and scars in areas like under the breasts, armpits, inner thighs, groin, and buttocks. Researchers are evaluating the investigational drug lutikizumab compared to placebo in adults and adolescents with moderate to severe HS. This study aims to assess the disease activity and safety of lutikizumab in a Phase 3 clinical trial involving about 1280 participants worldwide.

Researchers are evaluating new treatment options for people with proficient mismatch repair (pMMR) endometrial cancer (EC) that is advanced or has come back after prior treatment. This type of cancer starts in the lining of the uterus and is considered advanced when it has spread locally or to other body parts and cannot be removed by surgery. The study aims to compare the effectiveness of sacituzumab tirumotecan (sac-TMT), an antibody drug conjugate, combined with pembrolizumab versus pembrolizumab alone, to see which approach helps people live longer without the cancer worsening. Participants first receive an induction phase of six cycles, each lasting three weeks, of pembrolizumab combined with carboplatin and either paclitaxel or docetaxel through intravenous infusions. Those whose cancer does not progress after this phase enter the maintenance treatment phase, where they are randomly assigned to receive either pembrolizumab plus sac-TMT or pembrolizumab alone. If the cancer does progress, participants may enter a subsequent treatment phase and be randomly assigned to pembrolizumab plus sac-TMT or sac-TMT alone. During the study, researchers monitor participants for progression-free survival and overall survival for up to approximately 44 and 54 months, respectively. Participants undergo regular imaging, assessments, and laboratory tests to evaluate cancer status and treatment effects. The study also tracks safety and tolerability throughout all phases, providing a comprehensive follow-up to understand treatment impact over time.

Researchers are evaluating the effects of a medicine called BI 764198 in adults and adolescents aged 12 years and older who have a kidney condition known as focal segmental glomerulosclerosis (FSGS). This Phase 3 study aims to understand whether BI 764198 can improve kidney health in people with primary FSGS or genetic FSGS related to TRPC6 gene variants by comparing changes in urine protein levels over 104 weeks. Participants are randomly assigned to one of two groups: one group takes BI 764198 tablets once daily, and the other group takes placebo tablets that look like the medicine but contain no active drug. All participants continue their usual FSGS treatments during the study, which lasts up to two years. During the study, participants visit the study site approximately every three months. They regularly provide urine samples to monitor kidney function, and doctors check their overall health and note any side effects. The main outcome measured is the change in the 24-hour urine protein-to-creatinine ratio from the start of the study to week 104, helping researchers understand the treatment's impact on kidney disease.

Researchers are evaluating a new comprehensive treatment protocol for infants, children, and young adults aged 0 to 45 years with acute lymphoblastic leukemia (ALL). This pilot study collects data from successful treatments used by various well-known study groups and aims to improve survival and quality of life by using a personalized risk-based approach. The study involves countries and groups preparing to join the ALLTogether1 collaboration and focuses on optimizing diagnostic, registration, and treatment systems before the main study begins. The study is observational and does not include any experimental interventions; it follows the standard of care outlined in the master protocol. Treatment decisions are based on a novel personalized algorithm considering clinical features, genetic changes in leukemia, and therapy response. High-risk patients may be offered Chimeric Antigen Receptor T-cell (CAR-T) therapy as an alternative to intensive treatments to reduce side effects. The protocol also supports adding randomized and non-randomized interventions and translational research. Participants will be diagnosed and treated at specialized pediatric or adult hematology centers in participating countries. The study involves collecting diagnostic and treatment data, with follow-up over five years to measure event-free survival and overall survival compared to historical controls. Safety and quality of survival will be monitored through this long-term observation, helping to refine treatment approaches and support future research collaborations.