Patna

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 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 enfortumab vedotin, a treatment for advanced or metastatic urothelial cancer cancer of the bladder lining, in Indian adults. This phase 4, open-label study aims to confirm the safety of enfortumab vedotin in participants whose cancer has progressed after previous treatments including checkpoint inhibitors and platinum-containing chemotherapy. Participants will receive enfortumab vedotin through intravenous infusion on days 1, 8, and 15 of each 28-day treatment cycle. This single-arm study involves repeated cycles of treatment, with all participants receiving the same study drug. The infusion schedule and dosage are designed to monitor treatment safety and tolerability. Throughout the study, participants will visit the clinic multiple times for health assessments including monitoring of adverse events, laboratory tests, vital signs, and electrocardiograms. Researchers will evaluate safety outcomes up to 8 months and also assess cancer response up to 34 months. The study involves careful tracking of participant health and treatment effects during and after the infusion cycles.

Researchers are evaluating the safety, immune response, and consistency between different vaccine lots of BBV87, an inactivated Chikungunya virus vaccine, in healthy individuals aged 12 to 65 years. This Phase IIIII clinical trial is randomized, observer-blind, and placebo-controlled, aiming to understand how well the vaccine works and its safety profile across multiple study centers. Participants will be randomly assigned to receive one of three lots of the BBV87 vaccine or a placebo. Each subject will receive two doses of 40 micrograms administered as injections into the deltoid muscle of the arm, with the second dose given about 28 days after the first. Safety data will be reviewed by an independent board during the study to ensure participant protection. During the study, participants will attend visits for vaccination and follow-up assessments, including blood tests to measure antibody levels against the virus 28 days after each dose. Researchers will monitor for any adverse events for up to 11 months post-vaccination. The main outcomes measured include immune response levels and the percentage of participants who develop antibodies, along with the safety and tolerability of the vaccine.

Researchers are evaluating the safety and effectiveness of guselkumab treatment in adults with psoriatic arthritis PsA, a chronic autoimmune condition causing joint inflammation. This phase IV, open-label study focuses on Indian participants who have not responded adequately to standard therapies, including conventional drugs and biologics. The goal is to understand how well guselkumab works and how safe it is in this specific patient population. Participants will receive subcutaneous injections of guselkumab at Weeks 0, 4, 12, and 20. The study monitors participants up to 32 weeks to assess safety outcomes such as adverse events and serious adverse events. Researchers will also measure treatment effects by evaluating responses using the American College of Rheumatology ACR 20 criteria at Weeks 12 and 24, along with changes in inflammation markers like C-reactive protein CRP. During the study, participants will undergo regular assessments including physical exams, medical history reviews, vital signs, ECGs, and tuberculosis screenings before treatment. The study team will monitor safety and efficacy through scheduled visits corresponding with treatment weeks and follow-up periods. Participants will be observed for up to 32 weeks to track any side effects and treatment responses, ensuring comprehensive evaluation of guselkumabs impact.

Researchers are evaluating mavorixafor, a drug being studied for people aged 12 and older who have congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders. These conditions cause low neutrophil levels and lead to recurrent or serious infections. The study aims to show if mavorixafor can improve clinical outcomes and increase neutrophil counts, while also assessing its safety and tolerability. Participants will continue their current treatment during the study, which may include therapies like granulocyte-colony stimulating factor G-CSF, immunoglobulin replacement, antibiotics, or no active treatment. They will be randomly assigned to receive either mavorixafor or a placebo orally once daily for up to 52 weeks. The study uses a quadruple-blind design to compare these groups in parallel. During the trial, participants will have regular assessments to monitor infection rates and severity, neutrophil counts, antibiotic use, oral ulcers, and fatigue levels using questionnaires. An independent committee will review infections to ensure accuracy. Safety and treatment effects will be followed throughout the 52-week treatment period. The total participation may last until the study ends in late 2027.

Researchers are evaluating orforglipron to measure its effects on cardiovascular outcomes in adults aged 50 and older who have atherosclerotic cardiovascular disease ASCVD andor chronic kidney disease CKD. This phase 3 study aims to compare orforglipron with a placebo to better understand its impact on major cardiovascular events over about five years. Participants will be randomly assigned to receive either orforglipron orally along with standard care or a placebo orally along with standard care. The study is double-blinded, meaning neither participants nor researchers will know who receives the active drug or placebo during the trial period. During the study, participants will be followed for around five years, with researchers monitoring the time to the first major cardiovascular event and additional outcomes such as cardiovascular and kidney events, changes in kidney function measured by eGFR, and the onset of type 2 diabetes. The study includes regular assessments to track these outcomes and ensure participant safety throughout the long-term follow-up.

This trial is evaluating the effectiveness and safety of two eye treatments, T1695 and Ciclosporin, in children and teenagers with moderate to severe vernal keratoconjunctivitis, an eye condition causing inflammation. The purpose is to compare these two treatments to see how well they control the disease and how safe they are for participants aged 4 to under 18 years. Participants will be randomly assigned to receive either T1695 ophthalmic suspension or Ciclosporin ophthalmic emulsion. Both treatments involve instilling the medication into the eyes. The study is designed as a single-blind Phase 2 trial to assess and compare these two eye drops over the course of the study. During the trial, participants will undergo eye exams including slit lamp examination and visual acuity testing. Researchers will measure the change in corneal fluorescein staining using a modified Oxford scale from the start of treatment to day 29 week 4 to assess treatment impact. Safety and effectiveness will be closely monitored throughout the study period ending in 2027.

Researchers are studying finerenone to evaluate its safety and effectiveness in patients hospitalized with acute decompensated heart failure who have mildly reduced or preserved left ventricular ejection fraction. This international trial is randomized, double-blind, and placebo-controlled, focusing on how finerenone compares to placebo in reducing heart failure events and cardiovascular death. Participants receive either oral finerenone or a matching placebo while hospitalized or recently discharged for heart failure. The study monitors patients over approximately 30 months to assess the total heart failure events, cardiovascular death, and adverse events related to the treatment. Throughout the study, participants undergo regular assessments including symptom scoring using the Kansas City Cardiomyopathy Questionnaire, monitoring for serious adverse events, and evaluation of heart failure outcomes. The study tracks safety and efficacy data over the long term, with follow-up visits scheduled to measure the impact of treatment on morbidity and mortality in heart failure patients.

This trial is for adults who have had an acute ischemic stroke caused by a blood clot blocking a brain vessel. It focuses on people whose stroke occurred or was discovered more than 4.5 hours ago, including those who woke up with stroke symptoms. The study aims to find out if the medicine tenecteplase helps recovery when given after this 4.5-hour window, compared to standard medical care. Tenecteplase is already used within 4.5 hours after stroke onset, but this study tests its effect when given later. Participants are randomly assigned to one of two groups one receives a single injection of tenecteplase into a vein, and the other receives the usual standard treatment. Both groups have an equal chance of receiving either treatment. The study lasts about three months, starting with approximately one week of hospital stay. During the study, participants have seven clinical examinations or visits, with the final two visits conducted remotely from home to allow for easier participation. Throughout the study, doctors regularly assess participants recovery using a scale that measures disability and dependence in daily activities. They also monitor overall health and record any side effects. The main outcome measured is the level of recovery 90 days after treatment, comparing the two groups. This includes neurological improvement, bleeding events, and survival over the study period.