Bornova

Researchers are investigating the safety and effectiveness of efruxifermin in people with non-cirrhotic nonalcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH) who have moderate to advanced liver fibrosis (stage 2 or 3). This Phase 3 study is randomized, double-blind, and placebo-controlled, enrolling a total of 1650 participants in two groups to evaluate treatment outcomes. Participants will receive either efruxifermin or a placebo by subcutaneous injection. The study involves two cohorts, with Cohort 1 including patients who have biopsy-confirmed NASH or MASH and specific liver fibrosis and activity scores. The treatment period and detailed dosing schedules are not provided but the study compares the effects of the active drug against placebo. During the study, participants will be monitored for improvement in liver disease status, including resolution of NASH/MASH and at least a one-stage improvement in liver fibrosis after 52 weeks for Cohort 1. Long-term outcomes such as event-free survival will be observed over 240 weeks. Safety and efficacy assessments will be conducted throughout the study period, including evaluations of liver histology and metabolic health.

Researchers are evaluating the safety and effectiveness of two doses of inhaled pirfenidone (called AP01) compared to a placebo in people with progressive pulmonary fibrosis (PPF). This Phase 2b study is randomized, double-blind, and placebo-controlled, involving up to 300 participants who will continue their standard care during the 52-week trial. The goal is to see how well AP01 works and how safe it is when added to usual treatments for PPF. Participants will be randomly assigned to one of three groups: high-dose AP01, low-dose AP01, or placebo. All treatments are given as an oral inhalation solution twice daily. The study will last for 52 weeks, during which researchers will monitor and compare the effects of these treatments on lung function and disease progression. During the study, participants will undergo various assessments including lung function tests and clinical evaluations to track their respiratory health. Researchers will check for changes in lung capacity and symptoms and monitor safety throughout the treatment period. The main outcome measured is the impact of AP01 doses compared to placebo after 52 weeks of treatment.

Researchers are studying an experimental combination of two drugs called pozelimab and cemdisiran for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The study aims to evaluate the long-term safety and effectiveness of this drug combination. It also investigates how the drugs behave in the body, possible side effects, and whether the body might develop antibodies against them that could affect treatment. Participants in this Phase 3 open-label extension study receive pozelimab and cemdisiran according to the study protocol. The study includes people who completed a parent study or those with a specific genetic variation (C5 polymorphism) making them resistant to certain other treatments. Treatments are administered as directed, and meningococcal vaccinations are required as part of the protocol. During the study, participants will attend clinic visits for various assessments, including safety monitoring for serious and special side effects up to week 108, and blood tests to measure changes in lactate dehydrogenase (LDH) levels up to week 36. Researchers will track adverse events, treatment tolerance, and collect data on drug levels and antibody formation. The total study duration covers long-term follow-up to better understand the treatment's effects and safety.

Researchers are evaluating the safety and effectiveness of bomedemstat (MK-3543) compared with the best available therapy (BAT) in adults with essential thrombocythemia (ET) who have not responded well to or cannot tolerate hydroxyurea. This phase 3 clinical trial aims to determine if bomedemstat provides a better durable clinicohematologic response in these participants. Participants will receive either bomedemstat as an oral capsule or one of the best available therapies, including anagrelide (oral capsule), busulfan (oral tablet), interferon alfa or its pegylated forms (subcutaneous solution), or ruxolitinib (oral tablet). The study involves a randomized, open-label design where treatments are compared directly. Throughout the study, participants will be monitored for their hematologic response up to about 52 weeks. Assessments include platelet and neutrophil counts before starting treatment to ensure eligibility. Safety and efficacy are tracked to evaluate the long-term impact of the treatments on ET.

Researchers are evaluating the safety, how the body processes the drug, and the effectiveness of calderasib alone or combined with other treatments in adults with advanced solid tumors that have a specific KRAS G12C mutation. This is a Phase 1, open-label, multicenter study focusing on participants with this genetic mutation in their tumors, aiming to understand how calderasib works alone and with other drugs. Participants receive calderasib as an oral dose, and some may also receive other medications such as pembrolizumab through intravenous infusion, or drugs like carboplatin, pemetrexed, cetuximab, oxaliplatin, leucovorin, and 5-fluorouracil according to standard guidelines. The treatments may be given alone or in combination depending on the study arm, with dosing schedules following label instructions or protocol specifications. During the study, participants will be closely monitored for any dose-limiting toxicities and adverse events, including reasons for stopping treatment. Researchers will assess these effects for up to about 21 days for dose-limiting toxicities and up to 56 months for adverse events and treatment discontinuation. The study involves regular evaluations to track safety, tolerability, and how well the treatment works over time.

Researchers are evaluating the safety and effectiveness of calderasib combined with pembrolizumab as a first treatment in adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) that has a specific KRAS G12C mutation and a PD-L1 tumor proportion score of 50% or higher. This Phase 3 trial aims to test if the combination of calderasib and pembrolizumab improves progression-free survival and overall survival compared to pembrolizumab with a placebo. Participants receive oral calderasib tablets or placebo along with pembrolizumab given by intravenous infusion. The study compares these two treatment groups to see which provides better outcomes. Treatments continue during the study, and there are no additional interventions described beyond these drugs. During the trial, participants undergo regular assessments including scans and tests to monitor their cancer's progression and overall health. The main outcomes measured are progression-free survival for up to about 42 months and overall survival for up to about 56 months. Safety is monitored throughout, and participants are followed for several years to evaluate long-term effects of the treatments.

This research aims to compare intismeran autogene combined with pembrolizumab versus placebo with pembrolizumab as an additional treatment after surgery for people with stage II, IIIA, or IIIB (with nodal involvement) non-small cell lung cancer (NSCLC) that has been fully removed with clear margins. The study is a phase 3 trial investigating whether the combination including intismeran autogene improves disease-free survival compared to the placebo combination. Participants will receive either intismeran autogene by intramuscular injection plus pembrolizumab by intravenous infusion or a placebo injection plus pembrolizumab. The treatments are given after surgery and standard platinum-based chemotherapy. No more than 24 weeks can pass from surgery to the first pembrolizumab dose. The study evaluates these treatments as adjuvant therapy to reduce cancer recurrence. During the trial, researchers will monitor participants for disease-free survival for up to approximately 78 months. Participants undergo regular assessments including medical evaluations to track cancer status and treatment effects. The study excludes those with prior neoadjuvant therapy, certain infections, or other cancer treatments that might interfere. Safety and long-term outcomes are carefully observed throughout the study period.

Researchers are evaluating the safety, tolerability, and effectiveness of nemtabrutinib combined with venetoclax compared to venetoclax plus rituximab in adults with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) who have received at least one prior therapy. This Phase 3, open-label study aims to confirm the dose of nemtabrutinib in this combination and test the hypothesis that adding nemtabrutinib improves progression-free survival based on 2018 iwCLL criteria assessed by blinded independent central review. Participants receive treatment with tablets of nemtabrutinib (5, 20, 45, or 65 mg) and venetoclax (10, 50, or 100 mg) or with venetoclax plus intravenous rituximab infusions (100 mg/10 mL and 500 mg/50 mL doses). The study compares these two regimens to assess efficacy and safety. The treatment period lasts up to approximately 71 months for progression-free survival evaluation, with earlier assessments of dose-limiting toxicities within 12 weeks and adverse events monitored up to around 28 months. During the study, participants undergo evaluations of safety including adverse events and treatment tolerability, with monitoring for discontinuation due to side effects. Disease progression is assessed using iwCLL criteria by a blinded central review. Participants must have confirmed active disease and meet specific health and organ function requirements before starting treatment. The study includes long-term follow-up to measure progression-free survival and overall safety over several years.

Researchers are evaluating the effectiveness and safety of pirtobrutinib (LOXO-305) compared to ibrutinib in adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The study includes participants who may have had prior treatment as well as treatment-nave participants with a specific genetic deletion (17p deletion). The study is a Phase 3, open-label, randomized trial designed to assess these treatments in different patient groups over varying durations. Participants receive either oral pirtobrutinib or oral ibrutinib. Part 1 compares these two drugs in participants with or without prior therapy, and participation can last up to six years. Part 2 focuses on pirtobrutinib alone in treatment-nave participants with 17p deletions, with participation lasting up to two years. The study carefully monitors responses to treatment, including complete and partial remissions. Throughout the study, participants undergo regular assessments to track their response to therapy, including measuring overall response rates from the start of treatment until disease progression or new treatments begin. Safety and organ function are monitored, and laboratory tests help evaluate blood counts and kidney function. The study aims to provide detailed information on how well the treatments work and their safety over the long term.

Researchers are investigating whether sacituzumab tirumotecan alone or combined with pembrolizumab can treat triple-negative breast cancer (TNBC). This phase 3 study compares these treatments to chemotherapy chosen by the physician, aiming to see if participants live longer or have longer periods without cancer growth or spread. The study focuses on people with previously untreated locally recurrent unresectable or metastatic TNBC with low PD-L1 expression. Participants receive sacituzumab tirumotecan through intravenous infusion alone or with pembrolizumab, also given intravenously. The study compares these to treatment options including paclitaxel, nab-paclitaxel, or gemcitabine plus carboplatin. Pre-medications like antihistamines, acetaminophen, and steroids are given before sacituzumab tirumotecan infusions to help reduce side effects. The trial evaluates safety and effectiveness over several months. Throughout the study, researchers monitor participants up to about 39 months for progression-free survival and up to about 61 months for overall survival. Participants undergo regular assessments to track cancer status and side effects. The study includes careful safety monitoring, and participants must meet specific health criteria to join. The total time in the study and follow-up depends on each participant's response and health status.