Vellore

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 treatments for participants with KRASNRAS and BRAF wild-type recurrent, unresectable, or metastatic colorectal cancer who have previously received chemotherapy. The study compares how long participants remain free of disease progression and the overall survival time when treated with amivantamab plus chemotherapy versus cetuximab or bevacizumab plus chemotherapy. This Phase 3 trial focuses on patients who have already undergone prior chemotherapy for their cancer. Participants are randomly assigned to one of two groups. One group receives amivantamab combined with the chemotherapy regimen FOLFIRI, which includes 5-fluorouracil, leucovorin calcium or levoleucovorin, and irinotecan. The other group receives either cetuximab or bevacizumab combined with the same chemotherapy regimen FOLFIRI. Treatments are given in 28-day cycles and continue until the cancer progresses or other criteria require stopping treatment. During the study, participants undergo regular assessments to monitor disease status, including imaging scans reviewed by independent experts. Researchers measure progression-free survival and overall survival over periods up to several years. Additional outcomes include response rates, duration of response, quality of life assessments, and side effects. Safety and laboratory evaluations are conducted throughout the study, which spans up to four years and four months in total duration.

This research aims to evaluate the long-term safety and effectiveness of the drug baricitinib in treating juvenile idiopathic arthritis JIA in children and adolescents aged 1 to under 18 years. Participants in this study have previously taken part in other baricitinib studies for JIA. The purpose is to monitor how well baricitinib works and to assess any serious side effects over an extended period. Participants will receive baricitinib orally during the study. This is a single-group trial where all participants receive the same treatment. The study follows participants for a total duration of up to 264 weeks approximately 5 years, during which they will be monitored closely for safety and disease activity. Throughout the study, participants will have regular assessments including monitoring for serious adverse events and treatment discontinuations. Researchers will also evaluate disease activity, pain levels, and other health measures using various clinical tools at baseline and at the end of the study period. This long-term follow-up ensures thorough evaluation of baricitinibs impact and participant well-being.

Researchers are evaluating elritercept TAK-226, KER-050, an investigational drug, for treating anemia in adults with very low, low, or intermediate risk myelodysplastic syndromes MDS who need regular red blood cell RBC transfusions. The study is a Phase 3, randomized, double-blind, placebo-controlled trial designed to assess how well elritercept reduces the need for RBC transfusions and to evaluate its safety and tolerability over time. Participants will be randomly assigned in a 21 ratio to receive either elritercept or a matching placebo, both given as subcutaneous injections 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 their assigned treatments. Eligible participants may also enter an Extension Phase to continue treatment until individual discontinuation or study unblinding. After treatment ends, a Safety Follow-Up Period of 8 weeks and a long-term follow-up lasting up to 5 years will monitor participants. During the study, participants will have visits approximately every 2 weeks initially, then every 4 weeks, to assess treatment effects and safety. Researchers will evaluate the percentage of participants achieving transfusion independence and monitor adverse events, laboratory values, vital signs, and heart tests. Long-term follow-up will continue through regular check-ins for up to five years or until the participant withdraws or the study ends.

Researchers are evaluating clemizole hydrochloride EPX-100 as an additional treatment for children and adults with Dravet syndrome DS, a severe form of epilepsy. This Phase 3, multicenter, randomized, double-blind, placebo-controlled study aims to assess the safety and effectiveness of clemizole hydrochloride in reducing seizures. The study includes participants with confirmed DS who continue to have seizures despite other treatments and have a documented SCN1A genetic mutation. Participants first undergo a 4-week Observational Period to establish baseline seizure activity. Then, they enter a 16-week Double-Blind Period where they receive either clemizole hydrochloride or a placebo as an oral solution. Following this, eligible participants who complete the double-blind phase may continue to an Open-Label Extension Period, receiving clemizole hydrochloride for up to 3 years. During the study, participants are regularly monitored for changes in countable motor seizures per 28 days and other seizure-related outcomes. Assessments include clinical evaluations and seizure tracking, with safety monitored throughout the trial. The primary outcome focuses on seizure reduction over 16 weeks, while secondary outcomes evaluate seizure frequency, seizure-free days, and clinical impressions of improvement. Participants are followed for up to approximately 172 weeks, ensuring long-term safety and efficacy data are collected.

Researchers are evaluating elritercept for its ability to reduce the need for red blood cell RBC transfusions and its safety compared to epoetin alfa in adults with very low, low, or intermediate risk myelodysplastic syndromes MDS who require regular blood transfusions. The study aims to understand if elritercept improves tiredness, lowers transfusion burden, enhances quality of life, and elicits an immune response. Participants receive either elritercept or epoetin alfa injections. Elritercept is given as a subcutaneous injection starting at 3.75 mgkg every 4 weeks, with possible dose increases to 5.0 mgkg. Epoetin alfa is administered as a subcutaneous injection starting at 450 IUkg once weekly, with possible dose escalation up to 1050 IUkg. The study follows participants for approximately 5 years to monitor treatment effects and safety. During the trial, participants are regularly assessed for transfusion independence, hemoglobin levels, fatigue, quality of life, and hematological improvements. Researchers monitor blood samples for drug concentration and immune response. Safety is evaluated through medical history, lab tests, and adverse event tracking. The main outcome is the proportion of participants achieving RBC transfusion independence for at least 12 weeks during the first 24 weeks. The study uses questionnaires and clinical assessments to measure fatigue and quality of life.

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 safety and effectiveness of a single dose of RABI-767 administered by endoscopic ultrasound EUS guided peripancreatic injection in adults with predicted severe acute pancreatitis. This phase 2a clinical trial compares RABI-767 plus supportive care to supportive care alone to better understand if this treatment is safe and beneficial. The study is sponsored by Panafina, Inc. and focuses on adults aged 18 to 85 years with specific imaging and clinical criteria. Participants are randomly assigned to receive either a single 125 mg dose of RABI-767 by EUS-guided injection alongside standard care or standard care alone without the investigational drug. The trial uses a parallel design to compare these two groups. The single-dose RABI-767 administration occurs early in the treatment phase, with follow-up assessments extending up to 60 days after dosing. Throughout the study, participants will be monitored closely for adverse events and changes in clinical chemistry, hematology, vital signs, oxygen measurements, and pancreatitis severity scores. Imaging studies such as contrast-enhanced CT or MRI will assess pancreatic changes. Researchers will track hospital stay duration, intensive care use, and rehospitalizations. Safety and efficacy outcomes are collected up to 60 days following treatment, with the overall participation lasting several weeks to months depending on individual recovery and follow-up timing.

Researchers are conducting an 18-month Phase 3 study to evaluate 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. This randomized, double-blind, placebo-controlled trial aims to show whether nizubaglustat improves ataxic symptoms compared with placebo. The study also assesses other neurological and behavioral effects, pharmacokinetics, pharmacodynamics, and safety of the treatment. Participants receive either daily oral dispersible tablets of nizubaglustat or matching placebo. The study follows a parallel design with random assignment to treatment groups. The treatment period lasts for 18 months, during which participants take the assigned medication once daily. The study monitors treatment effects and safety throughout this period. During the trial, participants undergo assessments including the Scale for the Assessment and Rating of Ataxia SARA, Vineland Adaptive Behavior Scale, Penetration-Aspiration Scale, 9-Hole Peg Test, and other neurological and functional tests at baseline and at months 6, 12, and 18. Blood samples are collected to study drug levels and biomarkers. Researchers also track seizure frequency and adverse events. The primary outcome measures focus on changes in total and functional SARA scores from baseline to month 18, with safety and tolerability evaluated throughout participation.

Researchers are conducting an 18-month Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat AZ-3102 in children aged 4 years and older diagnosed with late-infantile or juvenile forms of Niemann-Pick type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. This randomized, double-blind, placebo-controlled, multicenter trial uses a Master Protocol Research Program to study these related conditions in separate subprotocols based on disease type. Participants are randomly assigned in a 21 ratio to receive either oral nizubaglustat tablets or a matching placebo. The treatment and procedures are specific to each disease subprotocol, detailing different eligibility requirements, safety assessments, and efficacy endpoints. The study includes subprotocols for Niemann-Pick type C disease and for GM1 or GM2 gangliosidosis, each with tailored treatment plans and monitoring. Throughout the study, participants will undergo evaluations as described in their respective subprotocols, which may include clinical assessments, safety monitoring, and outcome measurements over the 18-month period. Researchers will track participant allocation to each subprotocol as the primary outcome. The trial is designed to monitor safety and treatment effects closely until the study completes in 2028, with detailed procedures provided within each disease-specific subprotocol.