Shizuoka

Researchers are evaluating the efficacy, safety, and tolerability of zorevunersen in patients with Dravet syndrome, a condition marked by reduced levels of the Nav1.1 protein due to mutations in the SCN1A gene. Zorevunersen is an investigational antisense oligonucleotide designed to increase the expression of the sodium channel Nav1.1 protein by boosting the production of its messenger RNA. This Phase 3, multicenter, randomized, double-blind, sham-controlled study aims to assess the potential of zorevunersen for disease modification by measuring changes in major motor seizure frequency and other health outcomes. The study has two treatment periods. In Treatment Period 1, participants assigned to zorevunersen receive the drug by intrathecal administration on Day 1, Day 57, Day 169, and Day 281 with doses of 70 mg initially and then 45 mg later. The sham group undergoes a procedure mimicking drug administration without receiving the drug. In Treatment Period 2, those initially on zorevunersen receive 45 mg doses on Day 393, Day 477, and Day 589. Participants initially in the sham group are then given zorevunersen doses of 70 mg on Day 393 and Day 477, and 45 mg on Day 589. Participants will be closely monitored throughout the study with a primary focus on seizure changes measured at Week 28. Secondary assessments include behavior, cognition, clinical status, and quality of life. The study includes an initial 8-week baseline period and a 6-week observation period to confirm seizure frequency and stability of other treatments. Patients may continue to an open-label extension study to receive zorevunersen if eligible. The study involves children aged 2 to under 18 years and tracks safety and tolerability alongside efficacy outcomes.

Researchers are evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of a drug called S230815 in children aged 2 to 12 years who have Developmental Epileptic Encephalopathy caused by specific genetic changes in the KCNT1 gene. This is a Phase Ib/II, first-in-human, multicenter, open-label study focusing on this rare and severe form of epilepsy. The study includes two main parts. Part 1 involves giving participants increasing doses of S230815 through injection to determine the best dose. After completing Part 1, participants may enter Part 2, a long-term extension where they continue receiving their assigned dose of S230815 for up to 72 weeks. Participants will be closely monitored throughout the study for side effects and drug levels. Researchers will collect safety information, including any adverse events, for up to 116 weeks. Genetic testing confirms eligibility, and participants will undergo various assessments during the screening and treatment periods to evaluate the drug's effects and safety over time.

Researchers are evaluating the safety, tolerability, and effectiveness of LP352 in reducing seizures among children and adults diagnosed with Dravet Syndrome (DS). This phase 3, double-blind, randomized, placebo-controlled study aims to compare LP352 with a placebo to better understand its impact on seizure frequency in this population. The study involves participants aged 2 to 65 years and addresses the challenges patients with DS face due to various seizure types and treatment responses. Participants will receive either LP352 or a matching placebo, administered orally or through a feeding tube (G-tube or PEG tube). The study includes several phases: an initial screening period, followed by a titration period to adjust doses, then a maintenance period where treatment continues, and finally a taper period to gradually reduce treatment before a follow-up phase. The entire study duration is approximately 24 months. During the study, participants will be monitored for changes in the frequency of countable motor seizures compared to their baseline seizure activity over up to 15 weeks. They will be required to complete diaries throughout the study to track seizures and treatment adherence. Safety and tolerability will also be assessed throughout all study phases. The researchers will collect data on seizure counts and monitor participants' health to evaluate LP352's effects comprehensively.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.

Researchers are evaluating the safety and effectiveness of HLX22 combined with trastuzumab and chemotherapy as the first treatment for patients with HER2-positive locally advanced or metastatic adenocarcinoma of the gastric or gastroesophageal junction. This phase 2, double-blind, randomized, and multiregional study compares this combination against trastuzumab and chemotherapy with or without pembrolizumab. The study aims to measure how well the treatments work in controlling the disease and improving survival for up to five years. Participants will be randomly assigned to one of two groups. One group receives HLX22 at 15 mg/kg every three weeks along with trastuzumab, chemotherapy (XELOX regimen), and possibly a placebo for pembrolizumab. The other group receives a placebo for HLX22 plus trastuzumab, chemotherapy (XELOX), and possibly pembrolizumab every three weeks. Treatment continues until the disease worsens, unacceptable side effects occur, withdrawal of consent, or other protocol-specified reasons. Throughout the study, participants will undergo regular assessments including tumor scans reviewed by an independent committee to evaluate progression-free survival and overall survival over up to five years. Other evaluations include safety monitoring and organ function tests. The study tracks how long patients live without disease progression and overall survival, aiming to better understand the benefits and risks of HLX22 combined with current standard treatments.

Researchers are evaluating ziltivekimab as a treatment for people living with heart failure and inflammation. This Phase 3 study compares ziltivekimab to a placebo in participants with heart failure who have mild to preserved ejection fraction and systemic inflammation. The study aims to assess the effect of ziltivekimab on cardiovascular death, heart failure hospitalization, or urgent heart failure visits over a period of up to 4 years. Participants will receive monthly injections of either ziltivekimab or a placebo using a pre-filled syringe or a pen-injector. The study medication is administered subcutaneously once a month for up to 4 years. The trial includes up to 20 clinic visits during which participants will be monitored and assessed. During the study, participants will use a study app on their phone to record all injections and complete questionnaires. Researchers will monitor participants for key outcomes like cardiovascular events and heart failure episodes from the time of randomization until the end of the study. Safety and health status will be regularly evaluated throughout the study period, which may last up to 48 months.

Researchers are evaluating the effectiveness of anitocabtagene autoleucel compared to standard of care therapy in adults with relapsed or refractory multiple myeloma who have previously received one to three treatments, including an anti-CD38 monoclonal antibody and an immunomodulatory drug. The study is a Phase 3, randomized, open-label trial aiming to assess how well anitocabtagene autoleucel works versus existing therapies in this patient group. Participants will receive either a single infusion of anitocabtagene autoleucel, which is a CAR+ transduced autologous T cell therapy, or one of several standard of care treatments. The standard treatments include combinations involving drugs such as pomalidomide, bortezomib, dexamethasone, daratumumab, carfilzomib, cyclophosphamide, and fludarabine, administered either orally or intravenously/subcutaneously. After the treatment period, those receiving anitocabtagene autoleucel will enter a follow-up phase and then transition to a long-term follow-up study lasting up to 15 years. During the study, participants will be monitored for progression-free survival for up to four years and for minimal residual disease complete response rate at nine months. Researchers will assess disease progression, treatment safety, and other health markers. Follow-up includes regular evaluations to track the participant's response and overall health status, with continued long-term monitoring planned for those treated with anitocabtagene autoleucel.

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.

This trial is focused on adults with KRAS/NRAS and BRAF wild-type unresectable or metastatic left-sided colorectal cancer. It compares the length of time participants remain free from disease progression when treated with amivantamab combined with chemotherapy regimens (mFOLFOX6 or FOLFIRI) versus cetuximab combined with the same chemotherapy regimens. The study is a randomized, open-label Phase 3 clinical trial designed to evaluate progression-free survival over a period of up to 4 years and 2 months. Participants receive either amivantamab with chemotherapy drugs including 5-fluorouracil, leucovorin calcium or levoleucovorin, oxaliplatin, or irinotecan hydrochloride, or cetuximab with the same chemotherapy regimens (mFOLFOX6 or FOLFIRI). Treatments are administered as first-line therapy for their colorectal cancer. The trial assesses how these treatments affect disease progression and survival. During the study, participants will be monitored regularly through assessments and evaluations to measure progression-free survival. Researchers will gather data via blinded independent central review to ensure unbiased assessment of disease status. Participants are followed up for safety and treatment efficacy over the study duration, which may last over four years.

The purpose of this study is to assess the anti-tumor activity and safety of amivantamab which will be administered as a co-formulation with recombinant human hyaluronidase PH20 (rHuPH20) (subcutaneous co-formulation \[SC-CF\]) in combination treatment (all cohorts except Cohort 4) and to characterize the safety of amivantamab SC-CF (Cohort 4).