Bhubaneswar

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 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 the safety and effectiveness of three different dose regimens of MORF-057, a small molecule drug, in adults with moderately to severely active Crohns disease CD. This Phase 2, randomized, double-blind, placebo-controlled, multicenter study aims to compare these doses with a matching placebo during an induction treatment period. The study includes adult participants who have active symptoms of CD and have not adequately responded to other treatments. Participants will first undergo a 14-week induction period where they receive either one of the three blinded MORF-057 dose regimens or a matching placebo, all taken orally. Following this, all participants enter a 38-week maintenance period receiving open-label MORF-057. Those who complete this 52-week treatment phase may have the chance to continue treatment for an additional 52 weeks during a long-term extension. MORF-057 is designed to selectively inhibit integrin 47. During the study, participants will have their disease activity monitored using endoscopic assessments and clinical symptom scores, such as the Simple Endoscopic Score for Crohns Disease SES-CD and the Crohns Disease Activity Index CDAI. Researchers will assess the proportion of participants showing endoscopic response and clinical remission at Week 14. Safety and adherence will be closely followed throughout the treatment and extension phases. The entire study spans up to 6 years, allowing for long-term evaluation.

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 the safety and effects of a new medicine called NNC0487-0111 in people who have Heart Failure with preserved Ejection Fraction HFpEF or Heart Failure with mildly reduced Ejection Fraction HFmrEF and excess body weight. This phase 3 clinical trial aims to find out if NNC0487-0111 is safe and effective for treating these conditions compared to a placebo. Participants have HFpEF or HFmrEF and a body mass index of 30 or above. The study is sponsored by Novo Nordisk AS and uses a randomized, quadruple-masked design. Participants will receive either NNC0487-0111 or a matching placebo by injection under the skin once a week. The NNC0487-0111 is given in increasing doses over time. The study is parallel in design, meaning participants are randomly assigned to one of the two groups and receive that treatment throughout the trial. This treatment period extends for up to about 165 weeks. The study evaluates the time to certain heart failure events, hospitalizations, cardiovascular deaths, and other major cardiovascular events. During the study, participants will be monitored regularly to assess heart failure outcomes and kidney function, as well as quality of life using questionnaires like the Kansas City Cardiomyopathy Questionnaire. Safety and effectiveness are assessed through hospital visits, heart failure event tracking, and blood tests including kidney function and blood sugar levels. The total participation spans over three years, with ongoing evaluations to measure the time to heart failure events and cardiovascular outcomes. Participants receive close medical monitoring throughout the study period.

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 efficacy and safety of two different dose regimens of pegozafermin compared to a placebo in adults with metabolic dysfunction-associated steatohepatitis MASH who have liver fibrosis stage F2 or F3. This Phase 3 study aims to better understand how pegozafermin may impact liver fibrosis and steatohepatitis in this population. Participants will receive subcutaneous injections of either one of two pegozafermin regimens or a matched placebo. These treatments are given in parallel groups, and participants are randomly assigned to one of the study groups. The study compares the effects of pegozafermin on liver fibrosis and steatohepatitis over a treatment period that includes evaluations up to 52 weeks and monitoring for disease progression up to 5 years. During the study, participants will be monitored through biopsies and blood tests to assess liver fibrosis improvement, resolution of steatohepatitis, changes in liver enzyme levels, and enhanced liver fibrosis scores. Safety and disease progression are also tracked throughout the study period. The total participation duration includes treatment and long-term observation to evaluate outcomes and any potential changes in liver health.

Researchers are conducting a Phase I open-label study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and optimal biological dose of AUR107 in adults with relapsed advanced malignancies. The study focuses on patients with selected advanced solid tumors, such as non-small cell lung cancer, gastric cancer, urothelial cancer, kidney cancer, colon cancer, and esophageal cancer, who have no curative or life-prolonging treatment options left. This first-in-human trial aims to find the best dose of AUR107 using a traditional dose-escalation design. Participants will receive oral AUR107 once daily at escalating doses ranging from 5 mg to 200 mg. The trial follows a 33 dose escalation design to evaluate safety and determine the optimal biological dose based on safety, pharmacokinetics, and pharmacodynamics data. The study is multicenter and includes dose expansion following dose escalation. During the study, participants will be closely monitored for dose-limiting toxicities and treatment-related adverse events over 28-day cycles. Researchers will measure pharmacokinetic parameters such as maximum concentration, time to maximum concentration, area under the curve, mean residence time, and elimination half-life at various time points under fasting and fed conditions. Safety assessments and clinical evaluations will be conducted throughout the study, which is expected to continue until June 2027.

Researchers are evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and optimal biological dose of an oral drug called AUR108 in adult patients with relapsed advanced lymphomas, including Non-Hodgkin and Hodgkin lymphoma. This Phase 1, open-label, first-in-human study focuses on patients who have no available curative or life-prolonging treatments left and have exhausted all effective local therapies. The study uses a traditional dose-escalation design to determine the best dose of AUR108 based on safety and biological data. Participants will receive AUR108 orally with doses planned at 50, 90, 150, 220, and 300 mg in a cycle of 3 days of dosing followed by 4 days off each week. The study follows a 33 dose-escalation method to evaluate safety and drug behavior in the body and to select the optimal biological dose. This is a multicenter trial with dose expansion planned after the initial dose-escalation phase. During the study, participants will be closely monitored for dose-limiting toxicities during the first 28-day cycle and treatment-related adverse events throughout the trial, which may last about a year. Researchers will collect blood samples to measure drug levels and effects at multiple time points, including days 1, 8, and 17. Participants will undergo safety assessments, organ function tests, and evaluations of their lymphoma status to track treatment impact and side effects over time.