Cheng Du Shi

Researchers are evaluating remibrutinib (LOU064) in adolescents aged 12 to under 18 years who have chronic spontaneous urticaria (CSU) that is not well controlled by H1-antihistamines. This Phase 3 trial aims to assess the effectiveness, how the drug is processed in the body, and safety of remibrutinib compared to a placebo. The study also intends to gather long-term data on how well remibrutinib works and its safety over several years after treatment ends. The trial includes three periods. First, the core period is a 24-week double-blind phase where about two-thirds of participants receive remibrutinib and one-third receive placebo, with about 10 site visits over approximately 32 weeks. Next is an optional open-label extension lasting from one to three years, where participants who completed the core period may receive remibrutinib or enter an observational treatment-free phase depending on their symptoms. Participants may cycle through treatment and observational periods up to six times. Finally, an optional long-term treatment-free follow-up can last up to three years with one site visit and up to four phone calls. During the study, participants undergo assessments including changes in urticaria activity scores (UAS7), itching severity (ISS7), and hive severity (HSS7) measured from baseline to 12 weeks. Regular visits monitor safety, symptoms, and drug effects. The study tracks these measures to understand remibrutinib's impact on CSU symptoms and overall safety profile during and after treatment, with total participation potentially lasting several years.

Researchers are evaluating the effects of reduced-dose radiotherapy (40.2Gy) compared to conventional-dose radiotherapy (49.2Gy) on low-risk target volumes in patients with chemosensitive intermediate-stage nasopharyngeal carcinoma. This phase 3 trial includes patients who have responded well to induction chemotherapy and whose plasma EBV-DNA levels have dropped to zero or below detection limits. The goal is to see if lowering the radiation dose can maintain treatment effectiveness while reducing side effects and improving quality of life. Participants will be randomly assigned to receive either reduced-dose or conventional-dose radiotherapy targeting the CTV2 area, while both groups receive the full course of PD-1 monoclonal antibody immunotherapy. The immunotherapy consists of 12 courses given every three weeks, starting with induction chemotherapy and continuing through radiotherapy and post-radiotherapy maintenance. Induction chemotherapy includes three cycles of gemcitabine and cisplatin or alternative drugs, administered intravenously. Throughout the study, patients will be closely monitored for progression-free survival and the occurrence of significant adverse events over three years. Researchers will assess survival outcomes, side effects, and quality of life differences between the two groups. Regular evaluations include imaging, laboratory tests, and clinical assessments to ensure patient safety and treatment effectiveness during the entire follow-up period.

Researchers are studying early pathological changes in Alzheimer's disease using new molecular probes called [18F]AV45 (Aβ) and [18F]AV1451 (Tau) with PET/CT imaging. The goal is to identify the main brain areas involved in the disease and how positive lesions relate to mental state and cognitive assessment scores. This approach aims to provide detailed molecular imaging information to better understand Alzheimer's disease development and improve diagnosis. The study evaluates these novel imaging agents that can detect Alzheimer's changes before symptoms appear by visualizing the disease mechanism at a molecular level. Participants receive PET/CT scans using the [18F]AV45 and [18F]AV1451 molecular probes. This imaging method offers earlier and clearer detection of brain changes linked to Alzheimer's disease. Participants will undergo brain imaging and cognitive assessments including mental state and Montreal Cognitive Assessment scales. The researchers will establish objective and quantitative diagnostic criteria for the use of these probes in early Alzheimer's detection, aiming to reduce reliance on doctors' subjective judgment and lengthy diagnostic processes. The primary outcome is to define diagnostic criteria within 2 months using Aβ and Tau PET/CT imaging.

Researchers are evaluating the safety and effectiveness of rilzabrutinib compared to placebo in adults with active Immunoglobulin G4 Related Disease (IgG4-RD). This Phase 3, randomized, double-blind study aims to measure the time until the first IgG4-RD clinical disease flare during a 52-week treatment period. Additional goals include assessing disease control, flare-free rates, use of glucocorticoid rescue, and monitoring safety through adverse events, laboratory tests, and electrocardiograms. Participants will be randomly assigned to receive either oral rilzabrutinib tablets or placebo for 52 weeks. Glucocorticoids may be used as rescue medication if needed. The study includes a screening period lasting 4 to 6 weeks before treatment begins, followed by the 52-week double-blind treatment phase, and a 2-week follow-up after treatment. An optional open-label extension lasting up to 108 weeks is also available for participants. During the study, participants will attend 16 visits for assessments, which may include clinical evaluations, imaging tests such as CT, MRI, PET, or ultrasound to monitor disease activity, and laboratory tests. Researchers will track time to disease flare and collect data on flare-free rates, safety parameters, and medication use. Participants' vaccination status and contraceptive use will be monitored according to local guidelines, and overall study participation could last up to 60 weeks or longer if joining the extension phase.

This clinical trial focuses on patients with Mucopolysaccharidosis Type I (MPS I), a rare genetic disorder caused by a deficiency of the alpha-L-iduronidase gene. Current treatments, like enzyme replacement therapy and stem cell transplantation, have limitations such as inability to treat the brain or high risks. Researchers are studying a new gene therapy called JWK008 designed to cross the blood-brain barrier to potentially improve treatment outcomes. This is a Phase 1, single-center, single-arm, non-randomized dose-escalation study to evaluate safety, tolerability, and early effectiveness of JWK008 in adults with MPS I. Participants will receive a single intravenous infusion of JWK008 at one of two dose levels using a 3+3 dose escalation design. The low dose is 5.0 x 10^12 vector genomes per kilogram, and the high dose is 2.0 x 10^13 vector genomes per kilogram. Only one infusion is given per participant, and the study is not controlled or blinded. During the study, researchers will monitor participants closely for safety and side effects over a period of 5 years, including tracking any adverse events. Participants will undergo evaluations including liver MRI to ensure eligibility and will be observed regularly to assess how well they tolerate the treatment. The main outcome measured is the occurrence of adverse events for up to five years following treatment.

Researchers are evaluating the safety and effectiveness of a drug called B001 injection in patients who have neuromyelitis optica spectrum disorder (NMOSD) and test positive for aquaporin-4 antibodies. This study is a multicenter, randomized, double-blind, placebo-controlled Phase II/III clinical trial designed to compare B001 with a placebo in this patient population. The goal is to assess whether B001 can reduce the time to the first NMOSD attack during the study period. Participants will receive either an intravenous dose of B001 or a matching placebo on Day 1 and Day 15 during the randomized controlled period (RCP). Both treatment groups follow the same dosing schedule to evaluate the effects of B001 compared to placebo over approximately 48 weeks. During the study, participants will be closely monitored through regular assessments to track any NMOSD attacks and overall health. Researchers will measure the time to the first NMOSD attack as the primary outcome. Safety and any side effects of the treatment will also be evaluated throughout the study period. Participants are expected to complete all required tests and follow study procedures as part of their involvement.

Researchers are evaluating the effectiveness and safety of a drug called B007 in people with generalized myasthenia gravis, a condition that affects muscle strength. This study is a Phase II/III clinical trial designed to compare B007 with a placebo to better understand its impact on daily living activities in affected patients. Participants receive either a high or low dose of B007 or a matching placebo, both given as subcutaneous injections on days 1 and 15. The study includes two groups: those treated with B007 and those given placebo, with treatment administered twice during the trial period. During the study, participants will be monitored for changes in their myasthenia gravis activities of daily living profile (MG-ADL). The main outcome measured is the proportion of participants whose MG-ADL score decreases by 2 or more after approximately 16 weeks. Safety and adherence are also tracked throughout the study.

Researchers are evaluating the safety and effectiveness of B007 in adult subjects diagnosed with pemphigus, a condition characterized by specific clinical signs. This Phase II/III clinical study aims to determine how well B007 works in treating this disease, including those newly diagnosed or experiencing a relapse. Participants receive B007 through subcutaneous injections given on day 1 and day 15. The study includes varying doses of B007 to assess treatment outcomes over time. This controlled dosing schedule allows researchers to monitor responses to the medication closely. During the study, researchers will track the proportion of subjects who achieve complete remission with minimal treatment by approximately 36 weeks. Participants are expected to follow the study protocol, which includes regular assessments to monitor treatment effects and safety. Long-term monitoring will help determine the success of B007 in managing pemphigus symptoms.

Researchers are studying whether combining calderasib, a targeted therapy for the KRAS G12C mutation, with subcutaneous pembrolizumab can treat non-small cell lung cancer (NSCLC). The study aims to determine if people receiving calderasib with pembrolizumab live longer without their cancer growing or spreading compared to those receiving pembrolizumab with chemotherapy. This is a phase 3, randomized, open-label, multicenter clinical trial focusing on participants with advanced or metastatic nonsquamous NSCLC carrying the KRAS G12C mutation. Participants will receive one of two treatment combinations. One group will take calderasib orally along with subcutaneous pembrolizumab and berahyaluronidase alfa injections. The other group will receive subcutaneous pembrolizumab combined with chemotherapy drugs pemetrexed and a platinum-based drug, either carboplatin or cisplatin, administered by intravenous infusion. These treatments are given as first-line therapy, and the study evaluates their safety and effectiveness. During the study, researchers will monitor participants for progression-free survival, especially focusing on those with at least 1% PD-L1 tumor proportion score, for up to approximately 48 months. Participants will undergo regular assessments to track cancer progression and response to treatment. Safety and efficacy data will be collected throughout the study to understand how well the treatments work and their side effects over time.

Researchers are evaluating new treatment options for adults with locally advanced or metastatic colorectal cancer that cannot be removed by surgery and has a specific KRAS G12C gene mutation. This study compares the safety and effectiveness of adding calderasib and cetuximab, both targeted therapies, to a standard chemotherapy regimen called mFOLFOX6. The goal is to see if this combination can help patients live longer without their cancer growing or spreading compared to current treatments that may include mFOLFOX6 with or without bevacizumab. The study has two parts. It involves treatment with calderasib taken as an oral tablet, cetuximab given according to standard procedures, and mFOLFOX6 chemotherapy combining oxaliplatin, leucovorin/levofolinate calcium, and 5-fluorouracil. Some participants may receive bevacizumab or a bevacizumab biosimilar as part of the comparison. The treatments are given following approved dosing schedules. This design allows researchers to assess the safety and tolerability of these drug combinations in treating this type of colorectal cancer with the KRAS G12C mutation. Participants will be monitored for side effects, treatment tolerability, and cancer progression over a period that may last up to about 44 months. Researchers will track outcomes such as how many participants experience dose-limiting toxicities or adverse events, how many stop treatment due to side effects, and progression-free survival time. Assessments include health evaluations, laboratory tests, and imaging to observe cancer status. This long-term follow-up aims to understand both safety and effectiveness of the treatment combinations.