Umraniye

This research aims to evaluate the effectiveness and safety of two different dose schedules of pegozafermin compared to a placebo in adults with metabolic dysfunction-associated steatohepatitis (MASH) who have liver fibrosis at stage F2 or F3. This phase 3 study focuses on improving liver fibrosis and steatohepatitis in this patient group, which involves chronic liver disease associated with metabolic dysfunction. Participants will receive either pegozafermin or a placebo through subcutaneous injections. The study compares two doses of pegozafermin to assess their impact on liver fibrosis and steatohepatitis. The treatment period lasts up to 52 weeks, with outcomes measured at this time point. During the study, participants will be monitored for improvements in liver fibrosis and resolution of steatohepatitis without worsening fibrosis by week 52. Researchers will also track the time until any disease progression occurs, up to 5 years. Throughout the trial, safety and efficacy will be carefully assessed through clinical evaluations and laboratory tests to ensure participant well-being.

Researchers are evaluating the effectiveness and safety of pegozafermin in adults aged 18 to 75 years who have compensated cirrhosis caused by metabolic dysfunction-associated steatohepatitis (MASH), previously known as nonalcoholic steatohepatitis (NASH). Participants in this phase 3 study must have biopsy-confirmed advanced liver fibrosis (stage F4) due to MASH and meet specific metabolic health criteria. The study aims to understand how well pegozafermin can help improve liver fibrosis and delay disease progression over time. Participants will receive either pegozafermin or a placebo through subcutaneous injections. The study will monitor participants over a long period, up to five years, to observe changes in liver fibrosis and any clinical events related to disease progression. The treatment is given to those with compensated cirrhosis, meaning their liver is damaged but still functioning, and the study carefully evaluates the safety and potential benefits of pegozafermin in this group. Throughout the study, participants will undergo regular assessments to track liver health, including fibrosis regression and timing of disease progression. Researchers will use clinical events and laboratory tests to measure outcomes from the start of the study through 24 months and up to five years. Safety and health will be monitored closely, ensuring any side effects or complications are identified promptly. This comprehensive follow-up helps provide detailed information on the long-term effects of the treatment and participants' liver condition.

Researchers are studying the pharmacokinetics, pharmacodynamics, efficacy, and safety of anifrolumab given by intravenous infusion compared with placebo in children aged 5 to under 18 years with moderate to severe active systemic lupus erythematosus (SLE) who are also receiving standard care treatments. This Phase III trial aims to better understand how anifrolumab works and its effects in this pediatric population. The study lasts about 116 weeks and includes several parts: a screening period of up to 30 days; Part A, a four-week double-blind, placebo-controlled, randomized phase focusing on pharmacokinetics; Part B, a double-blind, placebo-controlled, randomized safety and efficacy phase lasting 48 to 52 weeks depending on prior participation; Part C, a 52-week open-label extension where all participants receive anifrolumab; and Part D, a safety follow-up visit 12 weeks after the last dose. Participants will have regular evaluations including blood tests to measure drug levels and response, assessments of lupus activity, and monitoring for side effects. Key outcomes include drug concentration levels at various times and the number of participants responding to treatment at week 52. The study carefully tracks safety and effectiveness throughout all phases, with total participation lasting nearly two and a half years.