Concepcion

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.

Researchers are evaluating tulisokibart as a potential treatment for radiographic axial spondyloarthritis (r-axSpA), a type of arthritis causing pain, stiffness, and inflammation in the spine and pelvis joints, visible on X-rays. This Phase 2b study aims to determine if different doses of tulisokibart improve symptoms better than a placebo, which looks like the study medicine but contains no active drug. The study has two main parts: a 16-week placebo-controlled period where participants receive either tulisokibart or placebo through subcutaneous injections, followed by a 124-week long-term extension divided into a 40-week main extension and an 84-week optional extension. This allows researchers to assess both the short-term and longer-term effects and safety of tulisokibart. Participants will be monitored for their response using the Assessment of Spondyloarthritis International Society (ASAS) 40 response at week 16 as the primary outcome. Throughout the study, researchers will evaluate disease activity and safety while tracking symptoms and any side effects. The total involvement spans up to 140 weeks, including both initial treatment and extension phases.

Researchers are evaluating the efficacy, safety, and tolerability of two dosing regimens of itepekimab compared to placebo as an add-on treatment to intranasal corticosteroids in adult men and women with chronic rhinosinusitis with nasal polyps (CRSwNP). This multinational, randomized, double-blind, placebo-controlled Phase 3 study includes participants aged 18 years and older who have inadequately controlled CRSwNP. The study aims to better understand how these treatments impact nasal polyp symptoms and disease control over a one-year period. Participants will be randomly assigned to receive one of two dosing regimens of itepekimab or a placebo, all administered by subcutaneous injection. All participants will continue using mometasone furoate nasal spray as standard intranasal corticosteroid therapy. Treatment will last up to 52 weeks, followed by a 20-week safety follow-up period. The study includes a total of 9 site visits and 20 phone or home visits during the participant's involvement. Participants will be involved in regular assessments including endoscopic nasal polyp scoring and nasal congestion symptom evaluations at baseline and throughout the 24 weeks, among other time points. Researchers will monitor changes in nasal polyp scores and nasal congestion scores to measure the treatment effects. Safety and tolerability will be closely followed during the treatment and safety follow-up periods, with total participation lasting up to 76 weeks for most participants, or 56 weeks for those transitioning to an extension study.

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 effectiveness of camizestrant compared to standard endocrine therapy in patients with early breast cancer that is estrogen receptor positive (ER+) and human epidermal growth factor receptor 2 negative (HER2-). These patients have an intermediate or high risk of cancer recurrence and have already completed local treatments such as surgery and possibly chemotherapy, alongside at least 2 years and up to 5 years of standard adjuvant endocrine therapy. The study is a Phase III, open-label trial designed to assess outcomes over a long term. Participants will be randomly assigned to receive either camizestrant, an oral selective estrogen receptor degrader, or one of several standard endocrine therapies including tamoxifen, anastrozole, letrozole, or exemestane, administered according to local approved guidelines. The treatment duration for both groups is planned to last 60 months. Eligible patients may have previously used CDK4/6 inhibitors, and the study will specifically include those with intermediate or high risk of recurrence as determined by clinical and biological markers. During the study, participants will be monitored for up to 10 years from the last patient's randomization to evaluate invasive breast cancer-free survival. Additional outcomes include invasive disease-free survival, distant relapse-free survival, overall survival, safety, and clinical outcome assessments. The study involves ongoing assessments of health status, treatment effects, and safety to determine the long-term benefits and risks of camizestrant compared to standard therapies.

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 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 the safety, tolerability, and effectiveness of nemtabrutinib combined with venetoclax compared to venetoclax plus rituximab in adults with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) who have received at least one prior therapy. This Phase 3, open-label study aims to confirm the dose of nemtabrutinib in this combination and test the hypothesis that adding nemtabrutinib improves progression-free survival based on 2018 iwCLL criteria assessed by blinded independent central review. Participants receive treatment with tablets of nemtabrutinib (5, 20, 45, or 65 mg) and venetoclax (10, 50, or 100 mg) or with venetoclax plus intravenous rituximab infusions (100 mg/10 mL and 500 mg/50 mL doses). The study compares these two regimens to assess efficacy and safety. The treatment period lasts up to approximately 71 months for progression-free survival evaluation, with earlier assessments of dose-limiting toxicities within 12 weeks and adverse events monitored up to around 28 months. During the study, participants undergo evaluations of safety including adverse events and treatment tolerability, with monitoring for discontinuation due to side effects. Disease progression is assessed using iwCLL criteria by a blinded central review. Participants must have confirmed active disease and meet specific health and organ function requirements before starting treatment. The study includes long-term follow-up to measure progression-free survival and overall safety over several years.

Researchers are investigating whether sacituzumab tirumotecan alone or combined with pembrolizumab can treat triple-negative breast cancer (TNBC). This phase 3 study compares these treatments to chemotherapy chosen by the physician, aiming to see if participants live longer or have longer periods without cancer growth or spread. The study focuses on people with previously untreated locally recurrent unresectable or metastatic TNBC with low PD-L1 expression. Participants receive sacituzumab tirumotecan through intravenous infusion alone or with pembrolizumab, also given intravenously. The study compares these to treatment options including paclitaxel, nab-paclitaxel, or gemcitabine plus carboplatin. Pre-medications like antihistamines, acetaminophen, and steroids are given before sacituzumab tirumotecan infusions to help reduce side effects. The trial evaluates safety and effectiveness over several months. Throughout the study, researchers monitor participants up to about 39 months for progression-free survival and up to about 61 months for overall survival. Participants undergo regular assessments to track cancer status and side effects. The study includes careful safety monitoring, and participants must meet specific health criteria to join. The total time in the study and follow-up depends on each participant's response and health status.

Researchers are evaluating whether tucatinib combined with trastuzumab and mFOLFOX6 works better than the standard treatments for people with HER2 positive metastatic colorectal cancer, which is cancer that has spread or cannot be removed by surgery. This phase 3 study also aims to identify the side effects that may occur with this drug combination. Participants must have HER2 positive disease confirmed by testing and measurable cancer according to specific criteria. Participants will be randomly assigned to one of two groups. One group will receive tucatinib taken orally twice daily along with intravenous trastuzumab and the mFOLFOX6 chemotherapy regimen, which includes oxaliplatin, leucovorin or levoleucovorin, and fluorouracil given by IV every two weeks. The other group will receive standard care, which could be mFOLFOX6 alone or combined with either bevacizumab or cetuximab, both given by IV on specific schedules. Treatment continues as per the study protocol. During the study, participants will be monitored for progression-free survival up to about three years using imaging reviewed by independent experts. Researchers will assess side effects and disease response. Participants must be able to provide tumor tissue samples for testing and have a good performance status. The study includes brain imaging to check for metastases and monitors safety closely throughout the treatment period.