Maebashi

Researchers are evaluating the effectiveness, safety, and tolerability of subcutaneous ianalumab in adults with diffuse cutaneous systemic sclerosis. This Phase 2 study compares ianalumab with a placebo in participants diagnosed according to established classification criteria, focusing on those with active disease and specific autoantibodies. The goal is to better understand ianalumab's impact on this condition over a long treatment period. The study includes several phases: up to 6 weeks for screening, followed by a 52-week initial treatment period where participants receive either ianalumab or placebo by subcutaneous injection. After this, there is a second 52-week open-label treatment period where all participants receive ianalumab. Finally, a post-treatment follow-up period lasts at least 20 weeks and can extend up to 2 years after the last dose. Participants will undergo various assessments throughout the study, including evaluations of their skin condition using the rCRISS25 response at week 52. Safety and tolerability will also be closely monitored. The study involves regular visits for clinical evaluations, laboratory tests, and monitoring of disease activity and antibody status, with the total participation potentially lasting over two years including follow-up.

Researchers are evaluating the efficacy, safety, pharmacokinetics, and pharmacodynamics of a new treatment called NXT007 compared to emicizumab prophylaxis in people aged 12 years and older with severe or moderate congenital hemophilia A without factor VIII inhibitors, as well as those with hemophilia A of any severity who have factor VIII inhibitors. This phase III clinical trial aims to better understand how well these treatments work and how safe they are for managing hemophilia A. Participants will receive either NXT007 or emicizumab as prophylactic treatments. NXT007 is given by subcutaneous injection using a combined drug-device product, while emicizumab is administered subcutaneously using a vial and syringe. The study compares these two methods of treatment over a main study treatment period starting from the second month until at least seven months after the first dose. During the study, researchers will monitor how often treated bleeding episodes occur, measured as the Annualized Bleed Rate (ABR). Participants' medical histories, including previous treatments and bleeding episodes, will be carefully documented. Safety and treatment effects will be closely observed throughout the study to evaluate the overall impact of both treatments on the participants' condition.

Researchers are evaluating whether the drugs retatrutide and tirzepatide can prevent major adverse liver outcomes (MALO) in adults with metabolic dysfunction-associated steatotic liver disease (MASLD) who are at high risk. This Phase 3 trial enrolls about 4,500 adults with MASLD identified by non-invasive tests indicating an increased likelihood of developing serious liver problems. The study aims to understand how these treatments might affect liver health over time compared to a placebo. Participants will be randomly assigned to receive either retatrutide, tirzepatide, or a placebo, all given by subcutaneous injection. The study will last approximately 224 weeks, during which participants may attend 25 to 30 clinic visits for monitoring and assessment. After the main study, eligible participants can join an optional 2-year extension where all will receive either retatrutide or tirzepatide regardless of their original group. Throughout the trial, participants’ liver function and disease progression will be closely monitored through various health assessments. Researchers will track the time to the first major adverse liver event as the main outcome. Safety and health status will be evaluated regularly during clinic visits, ensuring thorough observation over the long study period.

Researchers are studying bleximenib, an investigational drug taken orally, to find the best dose for treating acute leukemia and to evaluate its safety and effectiveness. In Phase 1, they aim to identify the recommended Phase 2 dose (RP2D) through a dose escalation and expansion process. Phase 2 will focus on assessing how well bleximenib works at the recommended dose in participants with relapsed or refractory acute leukemia, particularly those with specific genetic alterations in KMT2A, NPM1, or NUP98/NUP214. The study involves administering bleximenib orally and includes different participant groups based on age and disease status. Phase 1 includes pediatric participants aged 2 to less than 18 years and adults 18 years and older with relapsed or refractory acute leukemia who have limited treatment options. Phase 2 focuses on adults over 18 with relapsed or refractory acute myeloid leukemia harboring KMT2A or NPM1 mutations. The trial monitors participants for dose-limiting toxicities, adverse events, and treatment tolerability over periods lasting up to nearly five years. Participants will undergo evaluations of safety, including the number and severity of adverse events and dose-limiting toxicities during the first cycle. The effectiveness measure in Phase 2 is the rate of complete remission or remission with partial blood count recovery. Throughout the study, participants will be assessed using laboratory tests, performance status scales, and pregnancy tests as applicable. Safety monitoring and long-term follow-up will continue for up to 4 years and 9 months to fully evaluate treatment effects and tolerability.

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.

This research aims to evaluate the effectiveness and safety of two different dose schedules of pegozafermin compared to a placebo in adults with metabolic dysfunction-associated steatohepatitis (MASH) who have liver fibrosis at stage F2 or F3. This phase 3 study focuses on improving liver fibrosis and steatohepatitis in this patient group, which involves chronic liver disease associated with metabolic dysfunction. Participants will receive either pegozafermin or a placebo through subcutaneous injections. The study compares two doses of pegozafermin to assess their impact on liver fibrosis and steatohepatitis. The treatment period lasts up to 52 weeks, with outcomes measured at this time point. During the study, participants will be monitored for improvements in liver fibrosis and resolution of steatohepatitis without worsening fibrosis by week 52. Researchers will also track the time until any disease progression occurs, up to 5 years. Throughout the trial, safety and efficacy will be carefully assessed through clinical evaluations and laboratory tests to ensure participant well-being.

This research focuses on men with prostate cancer who have previously participated in an enzalutamide clinical study sponsored by Astellas or Medivation. It aims to gather long-term safety information from participants who continue to benefit from enzalutamide treatment. This is a Phase 2 open-label extension study designed to monitor ongoing treatment effects after the initial study has completed its primary analysis or evaluation period. Participants will continue their previous treatment regimens, which may include enzalutamide taken orally once daily. Some may also receive abiraterone acetate with prednisone or leuprolide acetate depending on their prior study enrollment. Dose adjustments are allowed with medical monitor approval. The first visit of this study should occur within seven days of the last visit of the prior study unless treatment is temporarily paused. Participants are asked to return to their study site every 24 weeks for safety reviews, including adverse event monitoring and medication checks. At visits every 12 weeks, participants return unused study drugs and receive new supplies if needed. Safety data, including all adverse events and serious adverse events, are collected from consent until study completion, which may last up to 96 months. The study follows local standard care guidelines and includes a post-marketing phase in South Korea.

Researchers are evaluating the combination of bleximenib, venetoclax (VEN), and azacitidine (AZA) compared to placebo with venetoclax and azacitidine alone in treating adults newly diagnosed with acute myeloid leukemia (AML) who have specific gene mutations (NPM1 or KMT2A) and are not eligible for intensive chemotherapy. This is a phase 3 randomized, double-blind, placebo-controlled study focusing on participants with AML harboring these genetic abnormalities. The study aims to assess treatment effectiveness by measuring complete remission rates and overall survival. Bleximenib and venetoclax are given orally, while azacitidine is administered either intravenously or under the skin. Participants will receive either the combination of bleximenib, venetoclax, and azacitidine or placebo with venetoclax and azacitidine, following a rigorous treatment schedule. The study includes an initial treatment period where the effects of these drugs are compared to determine their impact on AML with the given mutations. Participants will be closely monitored through regular assessments, including evaluations of remission status and survival over a period of up to 4 years and 1 month. Safety and treatment responses will be tracked throughout the study. Participants must consent to follow the study procedures and agree to contraception requirements during and after treatment. The trial involves continuous observation to gather comprehensive data on treatment outcomes and participant health.

Researchers are evaluating the effectiveness of Saruparib (AZD5305) compared to placebo when added to a standard radiation therapy (RT) and androgen deprivation therapy (ADT) regimen in men with high-risk and very high-risk localized or locally advanced prostate cancer who have a BRCA gene mutation. This phase III study aims to assess whether Saruparib can improve metastasis-free survival in this population. About 700 adult male participants will be randomly assigned to receive either Saruparib or placebo along with ADT. There are two groups: Cohort A includes 400 participants with newly diagnosed high-risk or very high-risk prostate cancer treated with primary RT or with high-risk biochemical recurrence after radical prostatectomy receiving salvage RT. Cohort B includes 300 participants with very high-risk locally advanced prostate cancer receiving primary RT combined with ADT and abiraterone. Saruparib and placebo will be given orally, and standard ADT and abiraterone with prednisone/prednisolone will be administered as per the regimen. Participants will be followed for up to about 93 months to monitor metastasis-free survival and overall safety. Assessments include imaging scans like CT, MRI, bone scans, and PSMA-PET to confirm disease status. The study also monitors organ function, performance status, and treatment adherence. An independent committee will review safety and efficacy data throughout the trial to ensure participant well-being and study integrity.

Researchers are evaluating nemtabrutinib compared with the investigator's choice of ibrutinib or acalabrutinib in adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have not received any prior therapy. This Phase 3 study aims to determine if nemtabrutinib is not worse than ibrutinib or acalabrutinib in terms of objective response rate and if it is better regarding progression-free survival, both assessed using standardized disease criteria by independent review. Participants will be randomly assigned to receive one of the three oral treatments: nemtabrutinib, ibrutinib, or acalabrutinib. The study compares the effectiveness of nemtabrutinib against the other two drugs chosen by the investigator to treat first-line CLL/SLL. Treatment continues with monitoring over months to assess response and disease progression. During the study, participants will undergo evaluations based on the International Workshop on Chronic Lymphocytic Leukemia criteria, including blinded independent central reviews of their disease status. Researchers will track objective response rates up to about 33 months and progression-free survival up to around 104 months. Participants will also be monitored for safety and treatment adherence throughout the trial period.