Tromso

Researchers are evaluating the long-term effects of mirikizumab treatment in children and teenagers aged 2 to 19 years with moderate-to-severe ulcerative colitis or Crohns disease. This phase 3 study aims to assess the clinical remission rates and other health outcomes related to these conditions over an extended period. The study is sponsored by Eli Lilly and Company and follows a treatment focus for pediatric participants with these inflammatory bowel diseases. Participants receive mirikizumab administered by subcutaneous injections, with doses adjusted based on their weight. There are up to six planned doses, and if needed, intravenous rescue dosing is available if a participants condition worsens. The study may include a continued access period providing additional treatment beyond the main study duration. Participants are involved for about 172 weeks, attending up to 44 visits throughout the study. Regular assessments include evaluating clinical remission using the Modified Mayo Score for ulcerative colitis and the Pediatric Crohns Disease Activity Index for Crohns disease, along with other response and remission measures. Researchers monitor laboratory tests such as C-reactive protein levels and track corticosteroid use. Safety and health status are closely observed during the study and any continued treatment periods.

Researchers are evaluating whether retatrutide and tirzepatide can prevent major adverse liver outcomes in adults with metabolic dysfunction-associated steatotic liver disease MASLD who are at high risk based on non-invasive tests. This Phase 3 randomized controlled trial aims to assess these treatments compared to placebo in about 4,500 adults over approximately 224 weeks. The study is sponsored by Eli Lilly and Company and focuses on liver disease progression and related health measures. Participants will be randomly assigned to receive retatrutide, tirzepatide, or placebo, all administered by subcutaneous injection. The trial includes two placebo groups corresponding to each experimental drug. After completing the main study, eligible participants may join a 2-year extension where all will receive either retatrutide or tirzepatide regardless of their initial assignment. During the study, participants may attend around 25 to 30 clinic visits for health monitoring, study procedures, and assessments of liver function and disease status. Researchers will measure the time to major adverse liver outcomes, changes in liver fibrosis scores, liver stiffness, liver fat content, liver enzyme levels, body weight, and cardiovascular events. Monitoring will continue from baseline through study completion, with detailed evaluations at multiple timepoints including week 104.

Researchers are evaluating resectability rates and survival outcomes in patients with borderline resectable and locally advanced pancreatic cancer who receive primary chemotherapy. This nationwide Norwegian single-arm prospective study follows national guidelines for diagnosis, oncological treatment, surgery, and follow-up. The goal is to achieve a 50% resection rate in borderline resectable pancreatic cancer and 15% in locally advanced cases, aiming for adequate overall survival and controlled morbidity and mortality after surgery. Participants will receive primary chemotherapy according to national guidelines, preferably using mFOLFIRINOX or gemcitabine-nab-paclitaxel regimens. Surgical exploration and resection may follow chemotherapy, with surgery scheduled within four weeks after the last neoadjuvant infusion. Surgical procedures include pancreatoduodenectomy, distal pancreatectomy with splenectomy, or total pancreatectomy with splenectomy, possibly involving venous or arterial resection and reconstruction. Diagnostic procedures include endoscopic ultrasound fine-needle biopsy for diagnosis and molecular pathology and optional PETCT scans at baseline and after two months of chemotherapy. Participants will be assessed regularly with quality of life questionnaires at baseline, 3, 6, and 12 months from diagnosis, then yearly thereafter. Researchers will monitor resection rates, overall survival, mortality and morbidity after surgery, adverse events, and molecular pathology markers. The study collects data from November 2023 through December 2027, with a focus on surgical outcomes and patient well-being following treatment.

Researchers are evaluating the clinical and endoscopic effects of guselkumab in children aged 2 to 17 years with moderately to severely active Crohns Disease who show clinical response at Week 12. This Phase 3 study focuses on assessing the treatments impact at the end of a one-year maintenance period in pediatric participants with this condition. The trial is sponsored by Janssen Research & Development, LLC. Participants first receive guselkumab either intravenously or subcutaneously based on their body weight during a 12-week open-label induction phase. Those who respond at Week 12 are then randomly assigned to one of two subcutaneous guselkumab dosing regimens for a double-blind maintenance phase lasting up to Week 48. Non-responders at Week 12 enter an open-label maintenance phase with guselkumab subcutaneous dosing up to Week 48. Throughout the study, participants undergo clinical and endoscopic evaluations to measure remission and response rates, including endoscopic scores and clinical remission at Week 52. Additional assessments include growth measurements such as weight and height changes, and plasma concentrations of guselkumab. Safety is monitored with adverse event tracking up to Week 64, with the total duration of participation spanning just over one year.

Researchers are evaluating mirikizumab for children and teenagers aged 2 to 17 years with moderately to severely active Crohns disease, including fistulizing Crohns disease with active inflammation in the colon or ileum. The study aims to assess the treatments effectiveness, safety, tolerability, and how well it is absorbed in the body. Participants must have a history of inadequate response or intolerance to other Crohns disease medications and will be randomly assigned to receive mirikizumab or another intervention. The treatment involves receiving mirikizumab either intravenously IV or subcutaneously SC, with dosing based on the participants weight in three different weight groups over 40 kg, 20 to 40 kg, and 9 to 20 kg. The study includes a 12-week induction period, followed by a maintenance phase lasting until Week 52. After treatment, there is a safety follow-up period lasting up to 16 weeks. The entire study spans about 74 weeks and may include up to 19 visits. Participants will undergo assessments including clinical response evaluations by the Pediatric Crohns Disease Activity Index PCDAI, endoscopic assessments using the Simple Endoscopic Score for Crohns Disease SES-CD, and laboratory tests such as C-reactive protein and fecal calprotectin levels. Pharmacokinetic studies will measure how the drug is cleared and distributed in the body. Safety monitoring continues during the follow-up period to track any adverse events or treatment tolerability throughout the study duration.

Researchers are studying nipocalimab to see if it can reduce the risk of severe fetal and neonatal alloimmune thrombocytopenia FNAIT compared to a placebo. This condition affects newborns and can lead to low platelet counts and serious bleeding. The trial is designed as a double-blind, randomized, placebo-controlled study focusing on pregnant women at risk of FNAIT. Participants will be randomly assigned to receive either nipocalimab or a placebo through intravenous infusions. The study includes pregnant women between 13 and 18 weeks of gestation who have a history of FNAIT in prior pregnancies and meet specific antibody and fetal genotype criteria. Treatment will continue with monitoring up to 24 weeks postpartum, with safety and effectiveness closely evaluated. During the study, participants and their babies will undergo various assessments including blood platelet counts at birth, bleeding events monitoring, and developmental evaluations up to 104 weeks. Maternal health will be monitored through physical exams, laboratory tests, and ECGs. Researchers will track adverse events and antibody responses to nipocalimab, aiming to understand its impact on reducing risks associated with FNAIT while ensuring maternal and infant safety.

Researchers are investigating new treatments for people with proficient mismatch repair pMMR endometrial cancer that is advanced or has returned after surgery. This cancer starts in the tissues inside the uterus and has spread locally or to other parts of the body, making it not removable by surgery. The study aims to learn if combining sacituzumab tirumotecan sac-TMT with pembrolizumab helps people live longer and without their cancer worsening compared to pembrolizumab alone. All participants first receive an Induction Phase of six cycles, each lasting three weeks, which includes pembrolizumab combined with carboplatin or cisplatin and paclitaxel or docetaxel. Those whose cancer does not progress then enter a Maintenance Treatment Phase and are randomly assigned to receive either pembrolizumab plus sac-TMT or pembrolizumab alone. If the cancer progresses, participants may enter a Subsequent Treatment Phase where they are randomly assigned to pembrolizumab plus sac-TMT or sac-TMT alone. During maintenance and subsequent phases, sac-TMT is given on Days 1, 15, and 29 of each 6-week cycle, and pembrolizumab is given on Day 1 of each 6-week cycle. Participants are involved throughout the study, receiving intravenous treatments over several months and up to 14 maintenance cycles lasting about 19 months. Researchers will monitor cancer progression and survival up to approximately 44 to 54 months. They also assess safety by tracking adverse events and quality of life using questionnaires. The study requires regular visits for treatment and evaluation over this period to understand how well the treatments work and their effects on participants.

Researchers are evaluating the effects of a medicine called BI 764198 in adults and adolescents who have a kidney condition known as focal segmental glomerulosclerosis FSGS. This Phase 3 clinical trial aims to determine whether BI 764198 helps improve kidney function in people with primary FSGS or genetic FSGS linked to TRPC6 gene variants. The study is randomized and placebo-controlled, meaning participants are randomly assigned to receive either the medicine or a placebo, and neither the participants nor the researchers know which treatment each person receives. Participants take either BI 764198 tablets or placebo tablets once a day for up to two years, while continuing their usual medication for FSGS. The study involves two groups running in parallel. The main treatment period lasts 104 weeks about two years, during which the participants regularly visit the study site approximately every three months. Both groups are compared to see if BI 764198 affects kidney protein levels and function. During the study, participants provide urine samples regularly to assess their kidney health. Researchers measure changes in urine protein-creatinine ratio and kidney filtration rate from the start to the end of the treatment period. Questionnaires about health-related quality of life are also completed. Doctors monitor participants health and note any side effects throughout the two years. This thorough follow-up helps understand how BI 764198 impacts kidney disease and overall well-being.

Researchers are evaluating a master protocol designed to improve survival and quality of life for infants, children, and young adults aged 0 to 45 years who have been newly diagnosed with acute lymphoblastic leukemia ALL. This observational pilot study collects data from multiple well-known study groups, combining their experience into a comprehensive system for stratifying and treating ALL within this age group. The study aims to optimize diagnostics, registration systems, and collaborative logistics before the main study begins, focusing on standard of care treatments included in the master protocol. The study does not involve specific experimental interventions but serves as a platform for personalized risk-based diagnosis and treatment approaches. It includes standard treatments delivered at participating pediatric oncology or adult hematology centers in the involved countries. High-risk patients may be considered for specialized therapies such as Chimeric Antigen Receptor T-cell CAR-T therapy as an alternative to more intensive treatments to reduce side effects. The platform supports randomized and non-randomized interventions and translational research within the collaboration. Participants will be monitored through regular clinical assessments, with data collected on event-free survival and overall survival compared to historical controls over five years. The study involves confirmation of diagnosis by accredited laboratories, ongoing treatment at participating centers, and adherence to protocol procedures. This pilot phase focuses on collecting data and preparing the infrastructure for future randomized trials and extended research. Participants remain under their usual care throughout the study period, which spans several years for outcome evaluation.

This research aims to improve treatment and survival quality for infants, children, and young adults aged 0 to 45 years with acute lymphoblastic leukaemia ALL. It combines experiences from multiple European study groups into a master protocol that offers comprehensive risk stratification and treatment plans. The study includes randomized and interventional trials to identify therapies that may be less toxic and more effective, particularly focusing on reducing relapse and treatment-related side effects. Participants receive treatment based on a master protocol considered standard care, with additional randomized interventions testing modifications like reducing or omitting specific chemotherapy drugs such as Doxorubicin, Vincristine, and Dexamethasone. Some patients receive experimental treatments including Inotuzumab ozogamicin, Imatinib, 6-tioguanine, and Blinatumomab. Specific sub-studies also examine pharmacokinetics, neurocognitive outcomes, and cerebrospinal fluid diagnostics. The study design allows modular addition or stopping of sub-protocols and interventions. Throughout the study, participants undergo various assessments including monitoring of disease progression, side effects, and drug activity. Researchers evaluate event-free survival, disease-free survival, minimal residual disease response, and overall survival over multiple years, with follow-up periods extending up to 8 years. Participants cognitive functions and treatment toxicities are also assessed using specialized tests and questionnaires. Safety and adverse events are closely monitored to better understand treatment impacts over time.