Bornova

Researchers are evaluating Mim8, a new medicine designed to help people with haemophilia A, including those with or without inhibitors. Mim8 aims to prevent bleeding episodes by replacing the function of the missing clotting factor VIII. This long-term study will last up to 5.5 years, ending either when Mim8 is approved in the participants country or by June 2028, whichever comes first. The study includes participants who have been involved in earlier related studies or are new infants with severe haemophilia A. Participants will receive Mim8 as a preventive treatment through subcutaneous injections. Depending on their entry point, participants may use an enhanced cartridge or a DV3407 pen-injector device for administering Mim8. The treatment is given regularly over the study period, with participants potentially receiving up to 262 injections. In the event of bleeding, additional haemostatic medications may be used as agreed with the study doctor. Female participants who are pregnant, breastfeeding, or planning pregnancy during the study are not eligible. During the study, participants will be monitored for any side effects, including injection site reactions and the development of antibodies against Mim8. Researchers will also track bleeding episodes, Mim8 blood levels, and device handling for some participants. Participants and their representatives will complete diaries and questionnaires about their treatment and health. Safety will be carefully followed throughout the study, which may last several years depending on individual enrollment and study progress.

Researchers are evaluating efruxifermin EFX in a phase 3, randomized, double-blind, placebo-controlled study involving adults with compensated cirrhosis caused by NASH Nonalcoholic Steatohepatitis or MASH Metabolic Dysfunction-Associated Steatohepatitis. This study aims to assess the safety and effectiveness of EFX in preventing significant clinical events such as disease progression and liver decompensation over a period of up to 5 years. Participants are randomly assigned to receive either efruxifermin 50 mg or a placebo, both given by subcutaneous injection. The study includes two cohorts one with biopsy-proven compensated cirrhosis and specific metabolic scores, and another with biopsy-proven or non-invasive diagnosis of compensated cirrhosis. The study treatment and monitoring extend up to 5 years, with evaluations at 96 weeks and long-term follow-up to track liver fibrosis, markers of liver injury, insulin sensitivity, glycemic control, body weight, and safety outcomes. During the trial, participants undergo regular assessments including laboratory tests, ECGs, ultrasounds, and vital sign monitoring. Researchers will measure changes in liver fibrosis, steatohepatitis resolution, and metabolic markers throughout the study. Safety and tolerability are closely tracked by documenting adverse events and exposure duration. The study duration allows for long-term observation of treatment effects and disease progression, with participant involvement lasting up to 5 years.

Researchers are evaluating efruxifermin EFX in adults with non-cirrhotic nonalcoholic steatohepatitis NASH or metabolic dysfunction-associated steatohepatitis MASH who have liver fibrosis stage 2 or 3. This Phase 3, multi-center, randomized, double-blind, placebo-controlled study aims to assess the safety and efficacy of EFX compared with placebo. The trial includes about 1,650 participants divided into two cohorts based on liver biopsy characteristics and fibrosis stage. Participants will be randomly assigned to one of three groups EFX 28 mg, EFX 50 mg, or placebo, each given as a weekly subcutaneous injection. Cohort 1 will be evaluated over 52 weeks for histologic efficacy endpoints, while Cohort 2 will have assessments over 96 weeks. After these periods, participants may continue long-term treatment and clinical follow-up for up to approximately 240 weeks total. A follow-up visit will occur about 30 days after the last dose. During the study, participants will undergo liver biopsies, blood tests, and non-invasive assessments such as FibroScan and Enhanced Liver Fibrosis ELF score to monitor liver health and fibrosis. Researchers will track liver-related clinical outcomes, including liver events and survival, as well as safety and tolerability of the treatment. Participants who stop the study drug may still continue with scheduled assessments to support long-term safety and efficacy evaluations.

Researchers are studying an experimental treatment combining two drugs, pozelimab and cemdisiran, to evaluate their long-term safety and effectiveness for adults with paroxysmal nocturnal hemoglobinuria PNH. This Phase 3 study aims to answer key questions about how well this combination works, potential side effects, drug levels in the blood, and whether the body develops antibodies against the drugs that could affect treatment. Participants include those who have completed treatment in a related parent study and those with a specific C5 genetic variation making them resistant to other treatments. The study involves administering the study drugs per protocol, including a loading dose of pozelimab given intravenously on Day 1 for some participants. The study is open-label and non-randomized, with two groups based on prior treatment history or genetic markers. During the study, participants will attend clinic visits to receive treatments and undergo various assessments such as blood tests to monitor hemolysis and hemoglobin levels, measure drug concentrations, and check for antibodies. Researchers will track serious and other adverse events, treatment discontinuation, and changes in quality of life. The study lasts up to around 108 weeks, with ongoing safety and effectiveness monitoring throughout this period.

Researchers are evaluating the safety, how the body processes the drug, and the effects of calderasib alone and in combination with other therapies in adults with advanced solid tumors that have a specific genetic mutation called KRAS G12C. This study focuses on participants with measurable disease and adequate organ function who have this mutation confirmed by tissue or blood testing. It is a Phase 1 trial aiming to understand treatment tolerability and effectiveness in this patient group. Participants receive different study treatments depending on their assigned group. Some receive daily oral escalating doses of calderasib up to 800 mg until the disease progresses or treatment stops. Others receive calderasib combined with pembrolizumab infusions every 21 days for up to about 24 months, sometimes along with other chemotherapy drugs like carboplatin, pemetrexed, cetuximab, oxaliplatin, leucovorin, and 5-fluorouracil, given according to standard dosing schedules. Dosing may be adjusted based on safety. The study includes several treatment arms with different combinations. During the study, participants undergo regular safety monitoring, including tracking dose-limiting toxicities and adverse events over approximately 56 months. Researchers measure treatment effects, such as tumor response and duration, and study how calderasib behaves in the body through blood tests at designated times during treatment cycles. Participants will be followed through multiple cycles lasting 3 or 4 weeks depending on the arm, with assessments continuing for up to about 56 months to capture long-term effects and safety.

Researchers are evaluating the effectiveness and safety of combining calderasib with pembrolizumab as the first treatment for people 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. The study compares this combination to pembrolizumab with a placebo to see if it improves the time participants live without the cancer worsening and overall survival. Participants receive pembrolizumab through an intravenous infusion every 21 days for up to 35 cycles. They also take calderasib or a matching placebo by mouth daily until they meet criteria to stop treatment. The study is randomized and double-blind, meaning neither participants nor researchers know who receives calderasib or placebo. During the study, participants will have regular assessments to monitor cancer progression, overall survival, response rate, and quality of life measures through questionnaires. Safety will be closely monitored by tracking adverse events and treatment discontinuations. The study may last up to about 56 months, including follow-up to observe long-term outcomes and quality of life changes.

Researchers are evaluating intismeran autogene combined with pembrolizumab compared to placebo plus pembrolizumab as an additional treatment after surgery for participants with certain stages of non-small cell lung cancer NSCLC. The study focuses on participants with margin-negative, completely resected Stage II, IIIA, or IIIB with nodal involvement NSCLC. The main question is whether the combination including intismeran autogene improves disease-free survival compared to pembrolizumab with placebo. Participants are randomly assigned to two groups. One group receives 1 mg of intismeran autogene by intramuscular injection every 3 weeks for nine doses plus 400 mg of pembrolizumab by intravenous infusion every 6 weeks for up to nine doses. The other group receives a placebo injection on the same schedule plus pembrolizumab on the same infusion schedule. Treatment continues until disease recurrence, unacceptable side effects, or approximately one year, whichever comes first. During the study, participants are monitored through regular assessments up to about 78 months for disease-free survival and up to 12 years for overall survival and other health outcomes. Quality of life questionnaires and adverse event monitoring are conducted at baseline and throughout the study. The research team tracks lung cancer-specific survival and distant metastasis-free survival, as well as changes in symptoms like coughing and chest pain. Safety is closely observed throughout treatment and follow-up periods.

Researchers are evaluating the safety, tolerability, and dosing of nemtabrutinib combined with venetoclax compared to venetoclax plus rituximab in participants with relapsed or refractory chronic lymphocytic leukemia CLL or small lymphocytic lymphoma SLL. The study aims to determine if the combination of nemtabrutinib and venetoclax improves progression-free survival based on established criteria assessed by blinded independent review. Participants in one group will take daily oral nemtabrutinib tablets starting from the first treatment cycle and begin venetoclax tablets from the second cycle, continuing up to two years or until disease progression or discontinuation. The other group will receive daily venetoclax tablets from the first cycle and intravenous rituximab infusions once per 28-day cycle for six cycles, also continuing up to two years or until disease progression or discontinuation. A treatment cycle lasts four weeks. Throughout the study, participants will undergo assessments of dose-limiting toxicities, adverse events, and treatment discontinuations due to side effects, with monitoring periods ranging from approximately 12 weeks to over five years depending on the outcome measured. Researchers will also evaluate progression-free survival, minimal residual disease, overall survival, response rates, and duration of response. Participants need to meet specific health criteria and will be monitored carefully during and after treatment to track safety and effectiveness measures.

Researchers are evaluating the effectiveness and safety of pirtobrutinib LOXO-305 compared to ibrutinib in participants with chronic lymphocytic leukemia CLL or small lymphocytic lymphoma SLL. The study includes participants who may or may not have received prior treatment for their cancer. Part 1 of the trial lasts up to six years, while Part 2 focuses on treatment-nafve participants with a specific genetic deletion 17p deletion and lasts up to two years. Participants will receive pirtobrutinib or ibrutinib orally, depending on their assigned study group. Part 1 compares pirtobrutinib to ibrutinib in a randomized, open-label design. Part 2 evaluates pirtobrutinib alone in participants with the 17p deletion who have not yet been treated. Treatment continues until disease progression, unacceptable side effects, or other study-defined reasons. During the study, participants undergo regular assessments including clinical evaluations and monitoring of their response to treatment using established criteria. Researchers measure overall response rates, progression-free survival, event-free survival, duration of response, overall survival, time to next treatment, symptom worsening, and treatment tolerability. Participation involves ongoing monitoring for up to six years in Part 1 and two years in Part 2 to evaluate long-term outcomes and safety.

Researchers are evaluating whether sacituzumab tirumotecan alone or combined with pembrolizumab can treat people with triple-negative breast cancer TNBC that is locally recurrent, unresectable, or metastatic. The study aims to determine if these treatments help participants live longer overall or without their cancer growing or spreading compared to chemotherapy chosen by their physician. This is a phase 3, randomized, open-label trial focusing on patients whose tumors express PD-L1 at less than 10 combined positive score CPS. Participants are assigned to one of three groups. One group receives sacituzumab tirumotecan intravenously every two weeks until disease progression, toxicity, or stopping treatment. Another group gets the same sacituzumab tirumotecan schedule plus pembrolizumab intravenously every six weeks for up to about two years. The third group receives the physicians choice of chemotherapy, which may include paclitaxel, nab-paclitaxel, or gemcitabine plus carboplatin, given intravenously on various schedules until disease progression, toxicity, or discontinuation. Pre-medications are given before sacituzumab tirumotecan to help manage side effects. Participants will be monitored for how long they live without their cancer worsening and overall survival, with follow-up lasting up to around 61 months. Researchers will assess treatment response, quality of life using questionnaires, and physical and emotional functioning. Safety will be closely tracked by recording adverse events and treatment discontinuations. The total participation time depends on treatment duration and follow-up assessments.