Ernakulam

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 oral icotrokinra as a treatment for adults and adolescents with moderately to severely active ulcerative colitis, a chronic inflammatory disease of the large intestine causing ulcers in the colon lining. The study aims to assess how well icotrokinra works, along with its safety and tolerability in this population. This is a Phase 3, randomized, double-blind, placebo-controlled trial with a parallel group design including both adults and adolescents. Adult participants will be randomly assigned to receive either icotrokinra or placebo daily by mouth during a 12-week induction phase. At Week 12, those showing clinical response will enter a maintenance phase where they will continue icotrokinra or placebo daily for 40 weeks. Adults who do not respond will also enter the maintenance phase and receive icotrokinra. Adolescents will receive open-label icotrokinra during induction and then continue on icotrokinra during maintenance regardless of response. After completing the 40-week maintenance phase, eligible participants may join a long-term extension study. Participants will be monitored regularly through clinical assessments at specified time points including Week 12 for induction and Week 40 for maintenance. Outcomes measured include rates of clinical remission, symptom improvement, endoscopic and histologic healing, and quality of life scores. Safety will be evaluated by tracking adverse and serious adverse events throughout the study. The total study duration may extend up to approximately 6 years, ending in 2032, allowing long-term evaluation of icotrokinra in ulcerative colitis management.

Researchers are evaluating elritercept TAK-226, KER-050, an investigational drug, for treating anemia in adults with very low, low, or intermediate risk myelodysplastic syndromes MDS who need regular red blood cell RBC transfusions. The study is a Phase 3, randomized, double-blind, placebo-controlled trial designed to assess how well elritercept reduces the need for RBC transfusions and to evaluate its safety and tolerability over time. Participants will be randomly assigned in a 21 ratio to receive either elritercept or a matching placebo, both given as subcutaneous injections every 4 weeks. The study includes a Primary Phase lasting 24 weeks and a Secondary Phase lasting an additional 24 weeks, during which participants continue their assigned treatments. Eligible participants may also enter an Extension Phase to continue treatment until individual discontinuation or study unblinding. After treatment ends, a Safety Follow-Up Period of 8 weeks and a long-term follow-up lasting up to 5 years will monitor participants. During the study, participants will have visits approximately every 2 weeks initially, then every 4 weeks, to assess treatment effects and safety. Researchers will evaluate the percentage of participants achieving transfusion independence and monitor adverse events, laboratory values, vital signs, and heart tests. Long-term follow-up will continue through regular check-ins for up to five years or until the participant withdraws or the study ends.

Researchers are conducting an 18-month Phase 3 study to evaluate the safety and effectiveness of oral nizubaglustat AZ-3102 in children and adolescents with late-infantile and juvenile forms of Niemann-Pick type C disease. This randomized, double-blind, placebo-controlled trial aims to show whether nizubaglustat improves ataxic symptoms compared with placebo. The study also assesses other neurological and behavioral effects, pharmacokinetics, pharmacodynamics, and safety of the treatment. Participants receive either daily oral dispersible tablets of nizubaglustat or matching placebo. The study follows a parallel design with random assignment to treatment groups. The treatment period lasts for 18 months, during which participants take the assigned medication once daily. The study monitors treatment effects and safety throughout this period. During the trial, participants undergo assessments including the Scale for the Assessment and Rating of Ataxia SARA, Vineland Adaptive Behavior Scale, Penetration-Aspiration Scale, 9-Hole Peg Test, and other neurological and functional tests at baseline and at months 6, 12, and 18. Blood samples are collected to study drug levels and biomarkers. Researchers also track seizure frequency and adverse events. The primary outcome measures focus on changes in total and functional SARA scores from baseline to month 18, with safety and tolerability evaluated throughout participation.

Researchers are conducting an 18-month Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat AZ-3102 in children aged 4 years and older diagnosed with late-infantile or juvenile forms of Niemann-Pick type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. This randomized, double-blind, placebo-controlled, multicenter trial uses a Master Protocol Research Program to study these related conditions in separate subprotocols based on disease type. Participants are randomly assigned in a 21 ratio to receive either oral nizubaglustat tablets or a matching placebo. The treatment and procedures are specific to each disease subprotocol, detailing different eligibility requirements, safety assessments, and efficacy endpoints. The study includes subprotocols for Niemann-Pick type C disease and for GM1 or GM2 gangliosidosis, each with tailored treatment plans and monitoring. Throughout the study, participants will undergo evaluations as described in their respective subprotocols, which may include clinical assessments, safety monitoring, and outcome measurements over the 18-month period. Researchers will track participant allocation to each subprotocol as the primary outcome. The trial is designed to monitor safety and treatment effects closely until the study completes in 2028, with detailed procedures provided within each disease-specific subprotocol.

Researchers are conducting a Phase 1 study to evaluate the safety and tolerability of SBO-154 in adults with advanced solid tumors who have either not responded to or cannot tolerate standard treatments. The study aims to find the highest dose of SBO-154 that patients can safely tolerate and then further assess its safety in specific advanced cancers. The study is divided into two parts. In Part 1, participants receive escalating doses of SBO-154 administered intravenously every three weeks to determine the maximum tolerated dose. In Part 2, the safety of the selected doses from Part 1 is further evaluated in patients with particular types of advanced solid tumors. Treatment continues with dosing every three weeks during the study period. Participants will be closely monitored through scheduled visits involving laboratory tests and assessments to track any dose-limiting toxicities and treatment-related adverse events up to 30 days after the last dose. Researchers will also evaluate tumor response using imaging every six weeks for up to 12 months and monitor the presence of anti-drug antibodies for up to one year. The overall study duration extends through 2030, with ongoing safety and response evaluations.

Researchers are studying how the medicine dazukibart works in people with active idiopathic inflammatory myopathies, specifically dermatomyositis DM or polymyositis PM. These conditions cause inflammation and weakness in muscles near the bodys center, affecting activities like climbing stairs or lifting objects. DM also involves a skin rash, and these disorders can impact lung and heart function, reducing quality of life. This trial is designed as a Phase 3, multi-center, open-label extension to assess long-term safety, tolerability, and effects of dazukibart. Participants who took part in a prior dazukibart study and completed up to Week 52 may join this extension. Those receiving dazukibart will get an intravenous IV infusion lasting about one hour every 4 weeks for up to 48 weeks about 12 months. After the treatment period, there is a safety follow-up lasting about 4 months. Participants who choose not to receive the study medicine or are ineligible will only participate in safety follow-up visits, which happen every 4 weeks for up to 4 visits. During the study, participants receiving dazukibart will attend about 18 visits over roughly 16 months, while those on safety follow-up alone will have up to 4 visits. The trial monitors treatment-emergent side effects, lab and vital sign changes, lung function Forced Vital Capacity and lung gas exchange, and mental health using the Columbia-Suicide Severity Rating Scale. Secondary measures include muscle strength, disease activity assessments, patient-reported physical function, fatigue, quality of life, and medication use. This comprehensive monitoring helps evaluate the long-term impact of dazukibart in managing idiopathic inflammatory myopathies.

Researchers are evaluating two strategies for complete revascularization in patients with acute myocardial infarction MI specifically ST-segment elevation MI STEMI or non-ST-segment elevation MI NSTEMI who also have multivessel coronary artery disease CAD and have undergone successful treatment of the culprit lesion with Percutaneous Coronary Intervention PCI. The study aims to compare physiology-guided complete revascularization to angiography-guided complete revascularization, focusing on both efficacy and safety outcomes. Participants are randomly assigned to one of two groups. One group receives physiology-guided PCI where the function of non-culprit lesions NCLs is assessed using resting full-cycle ratio RFR or fractional flow reserve FFR, with PCI performed on lesions meeting specific thresholds. The other group undergoes angiography-guided PCI, receiving routine staged PCI on all qualifying NCLs identified before randomization. Both approaches follow local practice standards for PCI. During the study, researchers monitor participants for a minimum of two years to assess the time to first occurrence of major cardiovascular events, including cardiovascular death, new MI, or ischemia-driven revascularization, as well as safety outcomes like significant bleeding, stroke, stent thrombosis, or kidney injury related to contrast use. The trial also includes an observational imaging study using optical coherence tomography in a subset of patients. Follow-up assessments will enable evaluation of both efficacy and safety over the study period.