Gui Yang Shi

Researchers are evaluating the effectiveness and safety of B001 injection in patients who have neuromyelitis optica spectrum disorder NMOSD and test positive for aquaporin-4 antibodies. This condition involves recurrent attacks affecting the nervous system. The study is a multicenter, randomized, double-blind, placebo-controlled trial conducted in phases II and III to understand how well B001 works and how safe it is for these patients. Participants will receive intravenous doses of either B001 or a placebo on Day 1 and Day 15 during the randomized controlled period. The study includes two groups one receiving B001 injections and the other receiving placebo injections matching B001s schedule. The trial will extend over several years, monitoring patients closely for disease relapse and treatment side effects. During the study, participants will be regularly assessed for the time to their first NMOSD attack, changes in disability status, vision acuity, and opticospinal function. Researchers will also observe the annual relapse rate and document any adverse events. The trial includes safety monitoring for about three years to ensure comprehensive data collection on treatment impact and participant health.

Researchers are conducting a Phase 1, open-label, multicenter clinical study to evaluate the safety, pharmacokinetics, and preliminary efficacy of HMPL-506 in patients with hematological malignancies. The study focuses on patients with specific genetic alterations in blood cancers, including relapsed or refractory Acute Myeloid Leukemia AML, Acute Lymphocytic Leukemia ALL, and multiple myeloma MM. The trial is sponsored by Hutchmed and aims to enroll between 60 and 132 patients across two phases dose escalation and dose expansion. In the dose escalation phase, approximately 30 to 38 patients with MLL-rearranged andor NPM1-mutant relapsedrefractory AML or ALL will receive escalating oral doses of HMPL-506 once daily. Starting at 50 mg, doses may increase based on safety, efficacy, and pharmacokinetic data, with adjustments guided by a Safety Monitoring Committee. The dose expansion phase will enroll about 30 to 60 patients divided into three cohorts based on specific genetic markers or disease type. Patients in this phase will receive the recommended phase 2 dose in 28-day cycles until disease progression, unacceptable toxicity, or other study endpoints. Participants will undergo regular assessments including bone marrow aspiration and biopsy, safety monitoring for dose-limiting toxicities and adverse events, and evaluations of anti-tumor efficacy every treatment cycle. Pharmacokinetic and pharmacodynamic analyses will be conducted, along with electrocardiograms and laboratory tests. Safety follow-up will continue up to 42 months after the last dose. The study includes monitoring for serious adverse events and overall survival, with participants remaining under medical supervision throughout the trial duration.

Researchers are evaluating the effectiveness and safety of TQB2102 for injection compared to a standard chemotherapy regimen called TCbHP in patients with HER2-positive breast cancer. This phase III, randomized, open-label, multi-center study focuses on neoadjuvant treatment, which is therapy given before surgery. The study aims to measure the total pathological complete response and other outcomes such as event-free survival and overall survival. Participants receive either TQB2102 for injection at 6 mgkg by intravenous infusion every 3 weeks for 8 cycles or a combination of Trastuzumab, Pertuzumab, Docetaxel, and Carboplatin given intravenously every 3 weeks for 6 cycles. The study monitors participants throughout the treatment period and collects data on tumor response and side effects. During the study, participants will undergo assessments including tumor response evaluations by independent review and investigators, safety monitoring for adverse events, and laboratory tests. Follow-up will continue for up to 50 months after the start of the study to observe long-term outcomes. Participants are expected to comply with contraceptive use requirements and attend all scheduled visits for treatment and evaluations.

Researchers are evaluating the safety and effects of marstacimab, a study medicine, for potential treatment of hemophilia in boys and male children aged 1 to 17 years. The trial focuses on those with severe Hemophilia A or moderately severe to severe Hemophilia B, including participants with or without inhibitors. The study aims to compare participants experiences with marstacimab to their historical bleeding episodes without the medicine. All participants will receive weekly subcutaneous injections of marstacimab. The first dose is administered at the study site by staff, and subsequent doses can be given at home or by study staff during the 12-month treatment period. Enrollment opens sequentially by age groups, starting with adolescents 12-17 years, followed by children 6-11 years, and finally children 1-5 years. The study includes about 1 month of screening, 12 months of treatment, and 1 month of follow-up. Participants will visit the study site at least 10 times during the study, with the option for two visits to occur at home if allowed. They will also receive six phone calls approximately every two months. Researchers will monitor bleeding rates, adverse events, injection site reactions, and immune responses. The overall participation lasts about 14 months, including screening, treatment, and follow-up periods.

Researchers are evaluating the dose-effect relationship of TQH3906 capsules compared to placebo in treating active Psoriatic Arthritis PsA. This Phase II, randomized, double-blind, placebo- and active drug-controlled clinical trial aims to measure the proportion of participants achieving a 20% improvement in arthritis symptoms by Week 12, using the American College of Rheumatology ACR20 criteria as the primary endpoint. Participants are randomly assigned to receive one of several oral treatments daily from Day 1 to Day 85 either 24 mg or 16 mg of TQH3906 capsules, placebo capsules matching TQH3906, or 5 mg tofacitinib citrate tablets. The treatments are administered in the morning while fasting, with tofacitinib also taken at bedtime. This study evaluates efficacy and safety across these groups over 12 weeks of treatment. During the study, participants are assessed at multiple timepoints for improvements in arthritis symptoms ACR20, ACR50, ACR70 and psoriasis severity PASI 75 and PASI 90. Blood samples are collected to evaluate drug levels and immune markers at baseline and Weeks 2, 4, 8, and 12. Safety is monitored continuously through adverse event reporting up to 28 days after the last dose. The total study duration per participant is approximately 12 weeks of treatment plus follow-up.

Researchers are evaluating the efficacy and safety of volrustomig compared to observation in participants with unresected locally advanced head and neck squamous cell carcinoma LA-HNSCC who have not progressed after receiving definitive concurrent chemoradiotherapy cCRT. This phase III, randomized, open-label global study aims to assess whether volrustomig can improve outcomes in this patient population. Participants are randomly assigned to one of two groups those who receive volrustomig as sequential therapy, and those who undergo observation without additional treatment. The study compares these two approaches following prior curative concurrent chemoradiotherapy. The trial includes long-term follow-up to monitor patient outcomes. During the study, participants will be regularly assessed for progression-free survival, overall survival, physical functioning, and quality of life. Researchers will also monitor for the presence of anti-drug antibodies and adverse events related to volrustomig. Follow-up evaluations may continue for up to approximately eight years to fully understand the treatment impact and safety profile.

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 a new tuberculosis preventive treatment among adolescents and school staff who have been in close contact with active TB cases. This study compares a novel 1-month ultra-short regimen called 1H3P3 with the standard 3-month 3HR regimen, aiming to prevent latent TB infection from progressing to active tuberculosis. The trial is a phase 3, multi-center, open-label, cluster randomized controlled study conducted in school settings in China, with the goal of improving TB prevention strategies for adolescents. Participants are randomly assigned to receive either the 12-dose 1H3P3 regimen of isoniazid and rifapentine taken three times a week for one month, or the 3HR regimen of isoniazid and rifampicin taken once daily for three months. Close contacts of TB cases are screened using blood tests, chest X-rays, sputum testing, and symptom checks to rule out active TB before enrollment. The treatment period lasts either one or three months depending on the group, followed by two years of follow-up to monitor for TB development. During the study, participants will undergo regular assessments including monitoring for active tuberculosis through clinical and laboratory tests, adverse events, treatment adherence, and drug resistance if active TB occurs. The main measurement is the cumulative incidence of active TB over 24 months after treatment begins. Safety and treatment completion rates are tracked throughout. Participation involves screening, treatment administration, and periodic follow-up visits over two years to evaluate outcomes and overall TB prevention effectiveness.

Researchers are evaluating the anti-tumor effects of two drugs, ABSK061 and ABSK043, in patients with metastatic or unresectable solid tumors that have specific FGFR2 or FGFR3 gene changes. This Phase 2 study focuses on tumors driven by FGFR alterations regardless of the tumor type. The study includes a screening phase, a treatment phase, and a follow-up period lasting until the participant dies, withdraws consent, is lost to follow-up, or the study ends, whichever comes first. Participants receive oral capsules of ABSK061 and ABSK043. Some patients with HER2-negative gastric or gastroesophageal junction cancer with FGFR2 changes will also receive chemotherapy called CAPOX along with the study drugs. Treatment continues until the disease worsens, unacceptable side effects occur, consent is withdrawn, or the investigator decides to stop treatment. The study follows patients for up to five years to monitor effects. During the study, participants undergo evaluations including tumor assessments by RECIST criteria, safety monitoring for dose-limiting toxicities, and pharmacokinetic tests measuring drug levels over time. Researchers will track outcomes such as overall response rate, progression-free survival, duration of response, disease control rate, and overall survival. Follow-up continues after treatment ends to collect long-term data and ensure patient safety throughout the study duration.

Researchers are conducting a phase III clinical study to assess the safety, tolerability, pharmacokinetics, immunogenicity, and effectiveness of LBL-034 in patients with relapsed or refractory multiple myeloma, including plasma cell leukemia. The study includes a dose-escalation phase to find the recommended dose and a dose-expansion phase to evaluate treatment effects. The trial is open-label and involves multiple centers. The treatment involves intravenous infusions of LBL-034 at an initial dose defined as the maximum tolerated dose MTD, given every two weeks. The study has two parts the phase I dose escalation and expansion to assess safety and dosage, followed by phase IIa which evaluates efficacy in four patient cohorts. Biological samples will be collected from all participants for testing. Participants will undergo various assessments including monitoring for objective response rate, dose-limiting toxicities, and maximum tolerated dose during treatment and up to 30 days after stopping the drug. Additional evaluations include drug concentration levels, immunogenicity, minimal residual disease, and duration of response. The total trial duration extends until May 2027, with patients followed closely throughout treatment and after drug withdrawal.