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Researchers are comparing how long participants with KRAS/NRAS and BRAF wild-type recurrent, unresectable, or metastatic colorectal cancer remain disease-free and their overall survival time when treated with two different regimens. This phase 3 study focuses on patients who have previously received chemotherapy. The study aims to evaluate progression-free survival and overall survival in participants receiving amivantamab plus FOLFIRI versus cetuximab or bevacizumab plus FOLFIRI. The study involves two treatment groups: one receiving amivantamab combined with chemotherapy drugs 5-fluorouracil, leucovorin calcium or levoleucovorin, and irinotecan (FOLFIRI), and the other receiving either cetuximab or bevacizumab with the same chemotherapy regimen. Participants will be randomly assigned to one of these treatment arms. The treatments will be administered according to protocol to assess their effects on the cancer. Participants will be monitored for up to 2 years and 1 month to measure progression-free survival through blinded independent central review and followed for overall survival for up to 4 years and 4 months. The study includes assessments of tumor response, safety, and other clinical evaluations. Tissue samples and detailed clinical data will also be collected. This comprehensive monitoring will help determine the comparative effectiveness of the treatment options over time.

Researchers are evaluating the long-term safety and effectiveness of baricitinib for treating Juvenile Idiopathic Arthritis (JIA) in children and teenagers aged 1 to less than 18 years. This phase 3 study focuses on participants who have previously taken part in baricitinib studies I4V-MC-JAHV or I4V-MC-JAHU. The goal is to monitor how well baricitinib works and how safe it is over an extended period in this young population. Participants will receive baricitinib orally as part of the study treatment. Since all participants have prior exposure to baricitinib in earlier studies, this trial continues to observe their response and any long-term effects. The study does not mention a separate comparator group or additional interventions beyond baricitinib administration. During the study, researchers will track serious adverse events and any permanent discontinuations of baricitinib from the start through week 264. Participants will be regularly monitored for safety and treatment effects throughout this long-term follow-up. This extended observation helps assess the ongoing impact of baricitinib on juvenile arthritis over several years.

Researchers are evaluating how well elritercept (TAK-226, KER-050) works in reducing the need for red blood cell (RBC) transfusions in adults with very low, low, or intermediate risk myelodysplastic syndromes (MDS) who require regular blood transfusions. The study is a Phase 3, double-blind, randomized, placebo-controlled trial that also aims to assess the safety and tolerability of elritercept over both short and longer periods, including in adults with high transfusion needs. Participants will be randomly assigned in a 2:1 ratio to receive either elritercept or a matching placebo by subcutaneous injection 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 the same treatment. Following these phases, an Extension Phase allows eligible participants to continue treatment until discontinuation or study unblinding. Study visits occur every 2 weeks during the first 6 cycles and every 4 weeks thereafter. Treatment continuation depends on meeting disease assessment criteria every 24 weeks. Participants will undergo various assessments including bone marrow aspirates, transfusion evaluations, and disease status checks throughout the study. Safety follow-up lasts for 8 weeks after the last dose, with visits every 4 weeks during this time. Afterward, long-term follow-up occurs quarterly for up to 5 years or until withdrawal, death, loss to follow-up, or study closure. The main outcome measured is the percentage of participants achieving transfusion independence for at least 8 weeks during the first 24 weeks of treatment.

Researchers are conducting a global Phase 3 study to evaluate the safety and effectiveness of clemizole hydrochloride (EPX-100) as an additional treatment for children and adults with Dravet syndrome, a severe form of epilepsy. The study focuses on participants with seizures that are not fully controlled by anti-epileptic drugs and includes those with a confirmed SCN1A gene mutation and other specific clinical features of Dravet syndrome. This randomized, double-blind, placebo-controlled trial aims to provide important data on this investigational therapy. Participants will receive either clemizole hydrochloride or a placebo, both given as oral solutions. The study includes a 4-week observational period without treatment changes, followed by a 16-week double-blind treatment phase where participants receive either the study drug or placebo. After this, there is an open-label extension period where all participants can receive clemizole hydrochloride. The treatment periods are designed to assess the drug's effect on seizure control over time. During the study, participants will be closely monitored for changes in the frequency of countable motor seizures compared to their baseline levels. Evaluations will include seizure tracking over 28-day periods during the titration, maintenance, and baseline phases. Safety and tolerability will also be assessed throughout the trial. Overall, the trial lasts about 20 weeks, with additional follow-up during the extension phase to gather long-term information about the treatment's effects.

Researchers are evaluating how well elritercept works compared to epoetin alfa in treating anemia in adults with very low, low, or intermediate risk myelodysplastic syndromes (MDS) who need regular red blood cell (RBC) transfusions. The study aims to see if elritercept can reduce the need for RBC transfusions, improve tiredness without transfusions, lower transfusion burden, and enhance quality of life. It also examines the immune response to elritercept and monitors its safety. Participants receive either elritercept or epoetin alfa as subcutaneous injections. The study is a phase 3, multicenter, randomized trial comparing the efficacy and safety of these two drugs. The treatment period lasts through 24 weeks, with each cycle lasting 28 days. Researchers monitor participants for RBC transfusion independence lasting at least 12 weeks and a significant increase in hemoglobin levels. During the study, participants undergo regular assessments including blood tests to measure hemoglobin and other blood counts. Researchers track transfusion needs and quality of life reports. Safety is carefully monitored throughout the trial. Participants are involved from screening through 24 weeks of treatment, with evaluations to measure the effectiveness of the treatments and any side effects.

Researchers are evaluating the effectiveness and safety of remibrutinib in adults aged 18 to 65 years with secondary progressive multiple sclerosis (SPMS). This Phase III study is randomized, double-blind, and placebo-controlled, designed to better understand how remibrutinib affects disability progression in SPMS patients over time. Participants will be randomly assigned to receive either oral remibrutinib tablets or matching placebo tablets during the Core Part of the study, which is event-driven and double-blinded. After this period, all participants may enter an Extension Part where they receive open-label remibrutinib treatment. This design allows researchers to compare remibrutinib against placebo and then monitor long-term effects when all participants receive the active drug. Throughout the study, participants will undergo regular assessments including MRI scans and clinical evaluations to track changes in disability using the Expanded Disability Status Scale (EDSS). The primary outcome measured is the time to confirmed disability progression over six months, with follow-up lasting up to approximately five years. Safety, tolerability, and other health parameters will also be closely monitored during both study phases.

Researchers are investigating 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. The study is a Phase 2a, multi-center, randomized, open-label trial aiming to determine if this treatment is safe and helps improve outcomes compared to standard supportive care alone. Participants have a diagnosis of acute pancreatitis and meet specific criteria indicating predicted severe disease. Participants will be randomly assigned to receive either a single 125 mg dose of RABI-767 via EUS-guided peripancreatic injection plus standard care, or standard care alone. The study compares the two groups to evaluate differences in safety and effectiveness. The treatment is given once, and the procedure involves specialized imaging to guide the injection. During the study, participants will be monitored from enrollment through Day 35. Researchers will track adverse events, serious adverse events, and changes from baseline in clinical chemistry, hematology, vital signs, pulse oximetry, and oxygen delivery measurements over specified timeframes up to Day 7 or Day 28. This monitoring helps assess safety and the body's response to treatment during the study period.

Researchers are evaluating the safety and effectiveness of oral nizubaglustat (AZ-3102) in treating children and young people with late-infantile and juvenile forms of GM1 or GM2 gangliosidosis, rare inherited metabolic disorders. This Phase 3 study is designed as a double-blind, randomized, placebo-controlled trial lasting 18 months. It aims primarily to show improvements in ataxic symptoms, with additional goals to assess other neurological effects, the drug's behavior in the body, and its safety and tolerability. Participants will receive either oral nizubaglustat or a placebo daily over the 18-month treatment period. The study is conducted at multiple centers and includes careful monitoring of the drug's levels in the body and its impact on disease manifestations. The design ensures that neither the participants nor the researchers know which treatment is being given until the study concludes. During the study, participants will undergo assessments to measure changes in ataxia scores from baseline to month 18, along with evaluations of other functional symptoms. Safety will be closely monitored throughout. The total duration of participation is 18 months, during which researchers will collect data on neurological function, drug effects, and any side effects experienced by participants.

This research aims to assess the safety and effectiveness of oral nizubaglustat (AZ-3102) in treating late-infantile and juvenile forms of Niemann-Pick type C disease. It is an 18-month, double-blind, randomized, placebo-controlled, multicenter Phase 3 study focused on improving ataxic symptoms compared to placebo. Additional goals include evaluating other neurological symptoms, pharmacokinetics, pharmacodynamics, and overall safety and tolerability of the treatment. Participants will receive either oral nizubaglustat or a matching placebo once daily for 18 months. The study compares these two groups to measure the impact on ataxic manifestations and other disease symptoms. Pharmacokinetic assessments occur after the first dose and at steady state during multiple dosing. Safety monitoring continues throughout the treatment period. During the study, participants will undergo various assessments including the Scale for the Assessment and Rating of Ataxia (SARA) to measure changes in ataxia severity from baseline to month 18. Additional evaluations include functional assessments, safety monitoring, and tolerability checks. Researchers will also collect data on drug effects and side effects to understand how the treatment influences the disease over time.

Researchers are conducting an 18-month, Phase 3, randomized, double-blind, placebo-controlled, multicenter study to assess 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, GM1 gangliosidosis, or GM2 gangliosidosis. The study uses a Master Protocol Research Program where participants are assigned to different subprotocols according to their specific disease type. Additional details about eligibility, safety, and efficacy assessments are provided in disease-specific subprotocols. Participants will receive either oral nizubaglustat tablets or a matching placebo following the assigned subprotocol regimen. The study includes separate subprotocols for Niemann-Pick type C disease and for GM1 or GM2 gangliosidosis, each with its own specific procedures and endpoints. The treatment period lasts for 18 months, during which safety and efficacy are evaluated. Participants will be monitored and assessed throughout the 18-month treatment period, with evaluations tailored to the specific subprotocol. Researchers will measure the number of participants assigned to each subprotocol and track safety and efficacy outcomes. For detailed procedures and endpoints, participants and caregivers are referred to the corresponding clinical trial records for each disease subtype.