Bengaluru

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 efficacy and safety of volrustomig compared to observation in participants with unresected locally advanced head and neck squamous cell carcinoma LA-HNSCC who have not progressed after receiving definitive concurrent chemoradiotherapy cCRT. This phase III, randomized, open-label global study aims to assess whether volrustomig can improve outcomes in this patient population. Participants are randomly assigned to one of two groups those who receive volrustomig as sequential therapy, and those who undergo observation without additional treatment. The study compares these two approaches following prior curative concurrent chemoradiotherapy. The trial includes long-term follow-up to monitor patient outcomes. During the study, participants will be regularly assessed for progression-free survival, overall survival, physical functioning, and quality of life. Researchers will also monitor for the presence of anti-drug antibodies and adverse events related to volrustomig. Follow-up evaluations may continue for up to approximately eight years to fully understand the treatment impact and safety profile.

Researchers are evaluating the efficacy and safety of combining durvalumab and domvanalimab compared to durvalumab plus placebo in adults with locally advanced Stage III, unresectable non-small cell lung cancer NSCLC whose disease has not progressed after definitive platinum-based concurrent chemoradiotherapy cCRT. This Phase III, randomized, double-blind, placebo-controlled, international study aims to provide new insights into treatment options for this patient population. Participants will receive either durvalumab and domvanalimab or durvalumab plus placebo as intravenous infusions every four weeks, beginning on Day 1 and continuing for up to 12 months. The study includes two groups one receiving the combination of durvalumab and domvanalimab, and the other receiving durvalumab with a placebo. Both treatments are given through infusion to assess their effects on disease progression and safety. During the trial, participants will undergo regular assessments including monitoring progression-free survival for up to 8 years after randomization. Other measures include overall survival, response rates, duration of response, and various time-to-event outcomes related to disease progression and symptom deterioration. Researchers will also evaluate drug concentrations and immune responses approximately 12 weeks after the last dose. Participants can expect scheduled visits for infusions and evaluations as part of this long-term study.

Researchers are conducting an observational study to better understand fucosidosis, a rare disease, its symptoms, and how it changes over time. The study aims to gather detailed information about the diseases natural history and progression, including patients who have or have not undergone stem cell transplant. There is currently no approved treatment for fucosidosis, so this study focuses on observing the condition without introducing new medications. The study is divided into two parts Part A involves collecting past medical data from up to 57 participants, and Part B involves following up with up to 31 participants prospectively, many of whom may also be in Part A. Participants will continue to receive their usual medical care throughout the study, with no additional treatments or interventions provided by the study team. Participants will provide medical history and health information that will be reviewed over time. The main focus is to track disease progression in individuals untreated by investigational products for up to four years. This includes gathering retrospective data and ongoing prospective observations to aid future research and potential treatment development. The study is expected to run until January 2031.

Researchers are evaluating the efficacy and safety of elecoglipron, an oral tablet taken once daily, for weight management in adults with obesity or overweight. This Phase III global, randomized, double-blind, placebo-controlled trial includes two independent pivotal studies one in adults without type 2 diabetes T2DM and the other in adults with T2DM, all having at least one weight-related health condition. The goal is to understand how elecoglipron compares to placebo when combined with diet and exercise. Participants will be randomly assigned to receive either one of two doses of elecoglipron or a matching placebo daily. Study 1 involves about 3000 adults living with obesity or overweight without T2DM, while Study 2 involves about 1500 adults with obesity or overweight and T2DM. Both studies last 72 weeks, during which changes in body weight and other health measures will be monitored. During the trial, participants will undergo regular health assessments including measurements of body weight, waist circumference, blood sugar control, blood pressure, and other related health indicators. Researchers will track percent change in body weight from baseline at 72 weeks as the primary outcome. Participants will be monitored closely throughout the study to assess safety and effectiveness of the treatment in managing weight and associated health conditions.

Researchers are evaluating the safety and performance of the Polymer Free Sirolimus Eluting Coronary Stent Vivo ISAR in patients with coronary artery disease CAD. This observational registry focuses on individuals treated with this specific stent and planned for a short dual antiplatelet therapy DAPT of up to 3 months. The study aims to collect real-world data on clinical outcomes including safety and effectiveness over a 12-month period. Participants in this single-arm registry have undergone percutaneous coronary intervention PCI using the Vivo ISAR stent and will receive standard care short DAPT treatment for no more than 3 months. The study does not affect treatment choices or standard care procedures. After the PCI, eligible patients will be invited to join the registry and followed up at 1 month, 3 months, and 12 months. During the study, researchers will collect baseline medical data and conduct telephonic follow-ups at 30 days, 3 months, and 12 months. These follow-ups will check on medication use, laboratory assessments, adverse events, and any further interventions. The main outcomes measured include ischemic and bleeding events at 12 months, along with secondary outcomes such as mortality, heart attacks, strokes, stent thrombosis, and need for additional vessel treatments. The total participation duration is one year from the PCI procedure.

Researchers are evaluating the safety and immune response of the RSVpreF vaccine in adults living in India. This phase 3, multicenter, double-blinded, placebo-controlled study aims to learn how the vaccine affects adults, including those with certain stable chronic medical conditions that may increase their risk of respiratory syncytial virus RSV disease. The study is sponsored by Pfizer and focuses on prevention of RSV infection. Participants will be randomly assigned to receive either the RSVpreF vaccine or a placebo. The vaccine is given as an injection, and the study will monitor participants for local and systemic reactions within 7 days after vaccination, as well as adverse events through 1 month and serious adverse events for 3 months post-vaccination. The immune response will be measured by neutralizing antibody levels against RSV A and RSV B before vaccination and 1 month afterward. During the study, participants will be observed for safety and immune response through scheduled visits. Researchers will collect data on side effects, serious health events, and antibody levels. The study includes adults aged 18 and older who live independently and are ambulatory. Participation involves medical history review and physical exams as needed. The trial will continue until January 25, 2027, with assessments focused on vaccine safety and immune response over several months.

Researchers are evaluating the safety and effectiveness of GIA632 in adults aged 18 to 99 years with non-segmental vitiligo NSV. This randomized, double-blind, placebo-controlled Phase 2b study aims to understand the dose-response relationship of GIA632 and determine the best dose to advance to a Phase 3 study. Participants have NSV affecting specific body surface areas confirmed by physical examination. Participants are randomly assigned to receive one of four different doses of GIA632 or a placebo. The assigned treatment is administered over a 48-week core period. After this period, an extension phase assesses longer-term safety and efficacy of the study drug. The study compares changes in facial and total body vitiligo scores at various time points up to 48 weeks. Throughout the study, participants undergo assessments including Vitiligo Area Scoring Index VASI measurements on the face and body, and the Vitiligo Noticeability Scale VNS. These assessments occur at baseline and multiple follow-up visits up to week 48. Researchers monitor participants for treatment effects and safety during the entire study duration, which runs until 2030, ensuring detailed evaluation of GIA632 over time.

Researchers are evaluating the combination of baxdrostat and dapagliflozin in people with chronic kidney disease CKD and high blood pressure hypertension. This Phase III, double-blind, placebo-controlled study aims to assess whether this combination reduces the risk of serious kidney damage, heart failure events, or cardiovascular death compared to dapagliflozin alone. The study includes participants with CKD and hypertension who meet specific kidney function and blood pressure criteria. Participants who are not already taking SGLT2 inhibitors will first complete a 4-week dapagliflozin run-in period. Then, they will be randomly assigned to receive either baxdrostat plus dapagliflozin or a placebo plus dapagliflozin. Baxdrostat dosing may start low and be increased if needed. Study visits will occur at 2, 4, 8, 16, 34, and 52 weeks after randomization, and then approximately every four months until the study ends, which is based on the number of key kidney or heart-related events. Throughout the study, participants will have regular assessments including blood tests to monitor kidney function and potassium levels, blood pressure measurements, and evaluations of heart and kidney health. If participants stop the blinded study drug early, they will continue dapagliflozin if possible and remain in the study for ongoing visits and monitoring. The main outcome is whether the combination treatment reduces the risk of a 50% sustained decline in kidney function, kidney failure, heart failure events, or cardiovascular death over up to 37 months.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study. This trial focuses on patients with established atherosclerotic cardiovascular disease ASCVD or those at high risk for a first ASCVD event. The study aims to assess how AZD0780 compares to placebo in reducing the risk of major adverse cardiovascular events, also known as MACE-PLUS, over the course of the trial. Participants are randomly assigned to receive either oral AZD0780 once daily or a matching placebo once daily. The study continues until a primary analysis censoring date, which may be up to approximately 54 months from randomization. After this, a study closure visit will be conducted as the final visit for each participant. During the study, participants will be regularly monitored for cardiovascular events including heart attacks, strokes, urgent coronary revascularizations, and other related outcomes. Researchers will track the time to first occurrence of these events as the primary outcome. Safety and other secondary outcomes like all-cause mortality will also be assessed. The total participation time can last up to about 54 months, with ongoing evaluations throughout this period.