Mangaluru

Glycogen storage disorders GSD are inherited metabolic diseases affecting glycogen production or breakdown, mainly involving the liver and muscles. These disorders vary in severity from mild to fatal in infancy. This study focuses on hepatic GSD types 0a, I, III, IV, VI, IX, and XI in Indian children. It aims to establish a comprehensive Indian GSD registry to better understand the spectrum of genetic defects, natural progression, and how genetic variations relate to disease symptoms in this population. The study is a multicenter observational effort collecting both retrospective and ongoing prospective data from genetically confirmed pediatric hepatic GSD cases. It involves analyzing clinical presentations, outcomes, and genetic variations across multiple centers in India. Retrospective data collection and analysis are planned between May 2024 and April 2025, with continued data submission from new centers and periodic follow-up every 6 months to 1 year. The registry will help guide individualized treatment decisions, including medical therapy or liver transplantation. Participants are children diagnosed genetically with hepatic GSD. The research team reviews clinical data, genetic testing results, and long-term outcomes such as native liver survival and post-transplant complications. The study measures the association between specific gene variants and clinical disease expression over a 5-year period. This ongoing project aims to improve understanding of GSD in Indian children to support better diagnosis, management, and health policies.

Researchers are evaluating the effects of a triple therapy inhaler combining budesonide, glycopyrronium, and formoterol fumarate BGF MDI 32014.49.6 g compared to a dual therapy inhaler with glycopyrronium and formoterol fumarate GFF MDI 14.49.6 g on heart and lung outcomes in adults with Chronic Obstructive Pulmonary Disease COPD who have a higher risk for heart and lung events. This Phase III study is randomized, double-blind, and conducted at multiple centers, focusing on participants with COPD and elevated cardiopulmonary risk. Participants will receive either the triple therapy inhaler or the dual therapy inhaler, both administered twice daily. The study compares these two inhalers over a period of up to three years, monitoring for serious cardiac or COPD events. The trial includes careful evaluation of various heart and lung-related health events during this period. During the study, participants will be closely monitored through regular visits, assessments, and tests to measure lung function, heart events, and COPD exacerbations. Researchers will track the time until the first severe cardiac or COPD event and evaluate other cardiovascular and respiratory outcomes over up to three years. Participants will also be assessed for their ability to properly use the inhaler and adherence to the study protocol throughout the trial.

Researchers are conducting a phase 3, open-label extension study to assess the long-term safety and tolerability of KarXT for treating mania or mania with mixed features in adults with Bipolar-I disorder. The study focuses on evaluating how participants respond to KarXT over an extended period, emphasizing safety measurements such as adverse events and symptom changes. Participants will receive KarXT at specified doses over a treatment period lasting up to 54 weeks. This study includes participants previously involved in related placebo-controlled studies as well as new participants diagnosed with Bipolar-I disorder with manic symptoms. The treatment may be given alongside standard therapeutic doses of lithium, valproate, or lamotrigine as applicable. Throughout the study, participants will undergo regular assessments including monitoring of treatment emergent adverse events, serious adverse events, and psychiatric symptom scales like the Columbia-Suicide Severity Rating Scale, Young Mania Rating Scale, and others. Safety and tolerability will be closely tracked, with evaluations occurring up to week 54. The entire participation may last until the study end date in June 2028, ensuring comprehensive long-term follow-up.

This research aims to evaluate the effectiveness and safety of adding KarXT to current treatment for mania in adults with Bipolar-I Disorder. Participants must be experiencing an acute manic episode, with or without mixed features, and currently taking lithium, valproate, or lamotrigine. The study is a Phase 3, randomized, double-blind, placebo-controlled trial assessing KarXT as an adjunctive therapy. Participants will be randomly assigned to receive either KarXT combined with lithium, valproate, or lamotrigine, or a placebo combined with these mood stabilizers. The study drug or placebo will be administered at specified doses on designated days. The trial focuses on treatment during an acute manic episode with monitoring over several weeks to assess changes in mania symptoms and other clinical outcomes. Participants will be monitored through scheduled visits where researchers will measure changes in mania severity using the Young Mania Rating Scale YMRS and other clinical scales. Safety assessments will include tracking adverse events and evaluating other symptom scales related to bipolar disorder. The total study duration includes treatment and follow-up periods lasting up to seven weeks, during which participants health and responses to the study drug are carefully observed.

Researchers are evaluating the efficacy and safety of remibrutinib in patients with secondary progressive multiple sclerosis SPMS. This is a Phase III, randomized, double-blind, placebo-controlled, multi-center study involving approximately 1275 participants. The study aims to provide important data on remibrutinibs effect on disability progression in SPMS and includes both a Core Part and an Extension Part for further assessment. Participants are randomly assigned to receive either remibrutinib or a matching placebo as oral film-coated tablets during the Core Part. The Core Part includes double-blind treatment, followed by an Extension Part where all participants receive open-label remibrutinib tablets. Treatment is taken orally, and the study is event-driven, continuing until required endpoints are met. During the study, participants undergo regular assessments of disability progression using the Expanded Disability Status Scale EDSS, Timed 25-Foot Walk, 9-Hole Peg Test, and Symbol Digit Modalities Test, among others. Brain imaging and safety monitoring for adverse events are performed throughout up to approximately five years. Researchers track changes in brain lesions and atrophy, and follow participants for safety and treatment effects over time.

Researchers are evaluating the efficacy, safety, and tolerability of remibrutinib compared to a placebo in adult patients with Generalized Myasthenia Gravis gMG who are already on stable standard-of-care treatments. This multicenter Phase III study is randomized, double-blind, and placebo-controlled, aiming to provide important information about remibrutinibs impact on gMG symptoms. Participants will be randomly assigned to one of two groups one receiving remibrutinib tablets orally, and the other receiving matching placebo tablets, both during a 6-month core treatment period. Following this, participants can join an extension phase lasting up to 60 months, where all will receive open-label remibrutinib tablets orally. During the study, participants will undergo various assessments to monitor their symptoms and quality of life, including the Myasthenia Gravis Activity of Daily Living MG-ADL score and other clinical scales. Safety and tolerability will also be closely observed throughout the core and extension periods. The total duration of participation may extend up to nearly six years, including both study phases.

Researchers are evaluating the use of rimegepant for intermittent prevention of menstrual migraine in women aged 18 to 45 who experience migraine attacks related to their menstrual cycle. This Phase 3, double-blind, randomized study compares rimegepant with placebo to assess its efficacy and safety during the peri-menstrual period. The study focuses on migraine frequency and severity in women with a history of menstrual migraine and regular menstrual cycles. Participants receive either rimegepant 75 mg orally disintegrating tablets for 7 days during each peri-menstrual period or a matching placebo, along with standard care for acute migraine treatment as needed. The study treatment cycles are repeated over 5 menstrual cycles in the double-blind treatment phase. The study also includes an acute treatment dosing option with rimegepant as needed. During the study, participants will be monitored for changes in the number of migraine and headache days, use of migraine medications, and functional disability related to migraine. Cognitive function is also assessed. Researchers will conduct evaluations over 5 months, covering 5 menstrual cycles, and measure outcomes related to migraine frequency and severity during the peri-menstrual period. Safety and tolerability will be closely monitored throughout the study.

This study evaluates the long-term safety and tolerability of pelacarsen TQJ230 in people with established cardiovascular disease and elevated Lipoproteina who completed a previous related study. It is an open-label extension trial, meaning all participants receive the study drug without placebo comparison. The trial is sponsored by Novartis Pharmaceuticals and focuses on continued treatment after the completion of the parent study. Participants receive monthly injections of pelacarsen 80 mg subcutaneously for up to 36 months during this extension phase. This phase is designed to provide access to the study drug after the initial trial and to monitor participants closely. The study does not involve randomization or blinding, and all enrolled participants receive the active drug. During the study, participants will undergo regular assessments including monitoring for adverse events and cardiovascular events, as well as measuring Lipoproteina levels at baseline and several time points over 36 months. Safety and tolerability will be closely tracked throughout the treatment period. The total duration of participation corresponds to the length of the extension phase, up to three years.

Researchers are evaluating ziltivekimab, a new medicine not yet approved anywhere, to see if it can help people who were hospitalized due to a heart attack. The study aims to find out if ziltivekimab can reduce the development of heart disease and prevent future heart attacks or strokes. This is a Phase 3 clinical trial comparing ziltivekimab to a placebo in patients with acute myocardial infarction. Participants will receive an initial loading dose of ziltivekimab or matching placebo by injection under the skin as soon as possible after an invasive heart procedure, within 36 hours for STEMI or 48 hours for NSTEMI patients. After the loading dose, they will get monthly injections of the same study medicine for up to two years, in addition to their standard care. During the study, participants will be monitored for major cardiovascular events such as heart attack, stroke, and cardiovascular death. Researchers will also track other heart-related outcomes and safety measures over a period of up to 25 months. The study involves regular visits for injections, assessments, and laboratory tests to evaluate the medicines effects and patient health throughout the trial.

Hypoxic-ischemic encephalopathy HIE is a serious brain condition in newborns caused by lack of oxygen and blood flow around the time of birth. This condition can lead to severe brain injury and long-term disabilities. Researchers are studying the use of sovateltide, a drug that targets brain cell receptors to support nerve cell growth and repair, as a new treatment option for neonates with HIE. Current treatments like therapeutic hypothermia have limited success and must be started quickly after birth, highlighting the need for additional therapies. In this phase II clinical trial, newborns with HIE will receive either sovateltide plus standard care or a placebo normal saline plus standard care. The sovateltide or placebo will be given intravenously in a bolus dose every 3 hours on days 1, 3, and 6 after randomization. Both groups will also receive the best available supportive treatments for perinatal asphyxia. Participants will be monitored over 24 months for outcomes such as death or moderate to severe disability, brain injury, seizures, cerebral palsy, blindness or hearing loss, and overall developmental progress using standardized assessments. Safety and tolerance of the treatment will be evaluated, with follow-up visits including clinical evaluations and neurological assessments. The study aims to determine if sovateltide can improve neurological outcomes and survival in newborns affected by HIE.