Ernakulam

Researchers are evaluating whether the drugs retatrutide and tirzepatide can prevent major adverse liver outcomes (MALO) in adults with metabolic dysfunction-associated steatotic liver disease (MASLD) who are at high risk. This Phase 3 trial enrolls about 4,500 adults with MASLD identified by non-invasive tests indicating an increased likelihood of developing serious liver problems. The study aims to understand how these treatments might affect liver health over time compared to a placebo. Participants will be randomly assigned to receive either retatrutide, tirzepatide, or a placebo, all given by subcutaneous injection. The study will last approximately 224 weeks, during which participants may attend 25 to 30 clinic visits for monitoring and assessment. After the main study, eligible participants can join an optional 2-year extension where all will receive either retatrutide or tirzepatide regardless of their original group. Throughout the trial, participants’ liver function and disease progression will be closely monitored through various health assessments. Researchers will track the time to the first major adverse liver event as the main outcome. Safety and health status will be evaluated regularly during clinic visits, ensuring thorough observation over the long study period.

Researchers are evaluating how well oral icotrokinra works, its safety, and how well patients tolerate it in adults and adolescents with moderately to severely active ulcerative colitis, a chronic condition where the colon lining becomes inflamed and develops ulcers. This is a Phase 3 study aimed at finding effective treatments for this condition using a rigorous comparison. Participants will receive either icotrokinra tablets or placebo tablets taken by mouth. The study includes an induction phase and a maintenance phase, with adults participating in a randomized, double-blind, placebo-controlled design, while adolescents join an open-label maintenance study. Throughout the study, researchers will monitor clinical remission rates at 12 weeks during induction and at 40 weeks during maintenance. Participants will undergo assessments including endoscopic evaluations and pregnancy tests for females of childbearing potential. Safety and tolerability will be closely observed, with the total study duration covering both induction and maintenance periods.

Researchers are evaluating how well elritercept (TAK-226, KER-050) works in reducing the need for red blood cell (RBC) transfusions in adults with very low, low, or intermediate risk myelodysplastic syndromes (MDS) who require regular blood transfusions. The study is a Phase 3, double-blind, randomized, placebo-controlled trial that also aims to assess the safety and tolerability of elritercept over both short and longer periods, including in adults with high transfusion needs. Participants will be randomly assigned in a 2:1 ratio to receive either elritercept or a matching placebo by subcutaneous injection 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 the same treatment. Following these phases, an Extension Phase allows eligible participants to continue treatment until discontinuation or study unblinding. Study visits occur every 2 weeks during the first 6 cycles and every 4 weeks thereafter. Treatment continuation depends on meeting disease assessment criteria every 24 weeks. Participants will undergo various assessments including bone marrow aspirates, transfusion evaluations, and disease status checks throughout the study. Safety follow-up lasts for 8 weeks after the last dose, with visits every 4 weeks during this time. Afterward, long-term follow-up occurs quarterly for up to 5 years or until withdrawal, death, loss to follow-up, or study closure. The main outcome measured is the percentage of participants achieving transfusion independence for at least 8 weeks during the first 24 weeks of treatment.

Researchers are evaluating the safety and effectiveness of oral nizubaglustat (AZ-3102) in treating children and young people with late-infantile and juvenile forms of GM1 or GM2 gangliosidosis, rare inherited metabolic disorders. This Phase 3 study is designed as a double-blind, randomized, placebo-controlled trial lasting 18 months. It aims primarily to show improvements in ataxic symptoms, with additional goals to assess other neurological effects, the drug's behavior in the body, and its safety and tolerability. Participants will receive either oral nizubaglustat or a placebo daily over the 18-month treatment period. The study is conducted at multiple centers and includes careful monitoring of the drug's levels in the body and its impact on disease manifestations. The design ensures that neither the participants nor the researchers know which treatment is being given until the study concludes. During the study, participants will undergo assessments to measure changes in ataxia scores from baseline to month 18, along with evaluations of other functional symptoms. Safety will be closely monitored throughout. The total duration of participation is 18 months, during which researchers will collect data on neurological function, drug effects, and any side effects experienced by participants.

This research aims to assess the safety and effectiveness of oral nizubaglustat (AZ-3102) in treating late-infantile and juvenile forms of Niemann-Pick type C disease. It is an 18-month, double-blind, randomized, placebo-controlled, multicenter Phase 3 study focused on improving ataxic symptoms compared to placebo. Additional goals include evaluating other neurological symptoms, pharmacokinetics, pharmacodynamics, and overall safety and tolerability of the treatment. Participants will receive either oral nizubaglustat or a matching placebo once daily for 18 months. The study compares these two groups to measure the impact on ataxic manifestations and other disease symptoms. Pharmacokinetic assessments occur after the first dose and at steady state during multiple dosing. Safety monitoring continues throughout the treatment period. During the study, participants will undergo various assessments including the Scale for the Assessment and Rating of Ataxia (SARA) to measure changes in ataxia severity from baseline to month 18. Additional evaluations include functional assessments, safety monitoring, and tolerability checks. Researchers will also collect data on drug effects and side effects to understand how the treatment influences the disease over time.

Researchers are conducting an 18-month, Phase 3, randomized, double-blind, placebo-controlled, multicenter study to assess 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, GM1 gangliosidosis, or GM2 gangliosidosis. The study uses a Master Protocol Research Program where participants are assigned to different subprotocols according to their specific disease type. Additional details about eligibility, safety, and efficacy assessments are provided in disease-specific subprotocols. Participants will receive either oral nizubaglustat tablets or a matching placebo following the assigned subprotocol regimen. The study includes separate subprotocols for Niemann-Pick type C disease and for GM1 or GM2 gangliosidosis, each with its own specific procedures and endpoints. The treatment period lasts for 18 months, during which safety and efficacy are evaluated. Participants will be monitored and assessed throughout the 18-month treatment period, with evaluations tailored to the specific subprotocol. Researchers will measure the number of participants assigned to each subprotocol and track safety and efficacy outcomes. For detailed procedures and endpoints, participants and caregivers are referred to the corresponding clinical trial records for each disease subtype.

Researchers are evaluating how the study medicine dazukibart works in people with active idiopathic inflammatory myopathies, specifically dermatomyositis (DM) and polymyositis (PM). These conditions cause inflammation and weakness in muscles near the center of the body, making everyday activities like climbing stairs or lifting objects difficult. DM can also cause a skin rash, and both disorders can affect lung and heart function, impacting patients' quality of life and ability to function. This is a Phase 3, multi-center, open-label extension study focused on the long-term safety, tolerability, and efficacy of dazukibart in participants who completed a previous qualifying study. Dazukibart, an anti-interferon beta therapy, is given as an intravenous infusion lasting about one hour every four weeks from Day 1 through Week 48, approximately 12 months. Following this treatment period, participants enter a safety follow-up phase lasting around 4 months after the last infusion. Participants receiving the study medicine will have about 18 study visits over about 16 months. Some participants who do not receive the study medicine, either due to ineligibility or choice, will take part only in safety follow-up visits, up to 4 visits every 4 weeks. Participants' involvement includes regular study visits for treatment or safety follow-up. Researchers will monitor treatment-emergent adverse events, serious adverse events, special interest adverse events, and those leading to treatment stopping over 52 weeks. They will also evaluate clinically significant laboratory abnormalities, vital signs, ECG abnormalities, lung function changes (Forced Vital Capacity and Diffusing Capacity for Carbon Monoxide), and changes in suicide risk assessed by the Columbia-Suicide Severity Rating Scale. This comprehensive monitoring aims to understand the long-term safety and effects of dazukibart in this patient population.

Researchers are evaluating two strategies for complete revascularization in patients with acute myocardial infarction (MI), including both ST-segment elevation MI (STEMI) and non-ST-segment elevation MI (NSTEMI), who also have multivessel coronary artery disease (CAD). The trial compares physiology-guided revascularization, which uses specific measurements to decide treatment, with angiography-guided revascularization, which relies on imaging. The study aims to determine if the physiology-guided approach is not worse than the angiography-guided method in preventing cardiovascular death, new MI, or ischemia-driven revascularization, and whether it is better at reducing safety issues like bleeding, stroke, or kidney injury. Participants undergo procedures to treat non-culprit lesions (NCLs) using either physiology guidance or angiography guidance. In the physiology-guided group, percutaneous coronary intervention (PCI) is performed on lesions with resting full-cycle ratio (RFR) of 0.89 or less or fractional flow reserve (FFR) of 0.80 or less, according to local practice. The angiography-guided group receives PCI based on imaging assessments following local practice. The study includes an observational imaging sub-study using optical coherence tomography (OCT) for a subset of patients. Participants are involved for a minimum of two years, during which researchers monitor the time to first cardiovascular events such as death, new MI, or additional revascularization, along with safety events like bleeding or stroke. The study includes regular evaluations and follow-up to assess these outcomes, ensuring comprehensive safety and efficacy data collection in this patient population.