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Researchers are evaluating the long-term safety and effects of nerandomilast in people with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF) who have previously completed treatment with nerandomilast in earlier studies. The study aims to understand how well participants tolerate nerandomilast over time, and whether it helps improve lung function, delays symptom worsening, reduces hospital visits, or impacts survival. This is a Phase 3 open-label extension trial. Participants take nerandomilast tablets daily for up to 1 year and 10 months while continuing their usual pulmonary fibrosis treatments. The study follows an open-label design where all participants receive nerandomilast. There are no placebo or comparator groups in this extension phase. Throughout the study, participants regularly visit their doctors for health assessments and lung function tests. Doctors monitor any health problems or side effects experienced during treatment. The main outcome measured is whether participants experience any adverse events up to the final follow-up visit, which occurs at week 99. This close monitoring helps evaluate the long-term safety and potential benefits of nerandomilast in this patient group.

This research aims to collect long-term safety and effectiveness data for participants treated with ibrutinib, a medicine used for various blood cancers and conditions including Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Mantle Cell Lymphoma, Follicular Lymphoma, Diffuse Large B-cell Lymphoma, Waldenstrom Macroglobulinemia, and Chronic Graft Versus Host Disease. It also provides ongoing access to ibrutinib for participants who have completed previous ibrutinib studies, continue treatment, and benefit from it. This is an open-label Phase 3b study without formal hypothesis testing. Participants will continue their current ibrutinib dosing regimen from the prior study, taken orally once daily as capsules in doses of 560 mg, 420 mg, 280 mg, or 140 mg, around the same time each day. Treatment continues until the investigator decides the participant no longer benefits due to disease progression or side effects, the participant withdraws, alternative ibrutinib access becomes available, or the study ends. Participants not able to access ibrutinib elsewhere can keep receiving the single-agent ibrutinib until all transition or stop treatment, or until the study is stopped. During the study, safety is monitored throughout and summarized, and effectiveness may be analyzed together with previous study data. The main outcome measured is the number of participants experiencing any adverse events within 30 days after the last dose or until starting another cancer treatment. Participants will undergo assessments including pregnancy testing and investigator evaluations to ensure ongoing benefit and safety. The study duration depends on when participants stop treatment or transition to other access.

Researchers are evaluating the effects of pelacarsen (TQJ230), given as a monthly injection under the skin, in people with mild to moderate calcific aortic valve stenosis. This study aims to see if pelacarsen can safely slow the progression of this heart valve condition compared to a placebo. The trial is a phase 2, randomized, double-blind, placebo-controlled study conducted at multiple centers. Participants will receive either pelacarsen 80 mg or a matching placebo once a month. Before starting the treatment, they must have elevated lipoprotein(a) levels and be optimally treated for existing cardiovascular risk factors. The study focuses on those aged 50 to under 80 years with mild or moderate calcific aortic valve stenosis. During the 36 months of participation, researchers will monitor changes in peak aortic jet velocity and aortic valve calcium score to assess disease progression. Safety, tolerability, and the impact of the treatment will be evaluated. Participants will undergo regular assessments, including laboratory tests and clinical evaluations, to track heart valve condition and overall health throughout the study.

Researchers are evaluating the safety and effects of fosmanogepix, a study medicine, for treating candidemia and invasive candidiasis, which are serious fungal infections caused by Candida species. This Phase 3 clinical trial compares fosmanogepix to the standard treatment of caspofungin followed by fluconazole, aiming to show that fosmanogepix is not worse than the standard therapy by a margin of 15%. The study includes adult patients diagnosed with these infections. Participants will receive either fosmanogepix or caspofungin as an intravenous infusion daily at the study clinic. After the initial infusion phase, patients may switch to oral tablets of fosmanogepix or fluconazole capsules, which can be taken at the clinic or at home if discharged. Treatment duration varies by individual, lasting up to six weeks depending on infection clearance and symptom improvement. A follow-up visit will take place six weeks after stopping treatment. During the study, patients will undergo multiple visits to monitor their health and treatment response. Researchers will assess outcomes such as the proportion of patients alive at 30 days and the overall treatment success at the end of study treatment, up to day 42. Safety will be closely monitored throughout the study and during follow-up, ensuring comprehensive evaluation of the treatments over the entire participation period.

Researchers are evaluating the safety and effectiveness of fosmanogepix, given either intravenously or orally, for treating adults diagnosed with invasive mold infections. This Phase 3 study focuses on patients infected with molds such as Aspergillus species, Fusarium species, Lomentospora prolificans, Mucorales fungi, or other multidrug-resistant molds. The main goal is to compare the overall death rate at 42 days against a fixed threshold to assess treatment outcomes. Participants will be assigned to one of two groups: Cohort A includes patients receiving either the study drug fosmanogepix or the standard antifungal treatment based on institutional practice, while Cohort B includes patients receiving fosmanogepix as a salvage treatment after not responding to or not tolerating prior therapies. Fosmanogepix is administered through intravenous infusion or oral tablets. The study treatment period targets 84 days but can be extended up to 180 days depending on patient needs. Throughout the study, lasting up to approximately 8 months including follow-up, participants will undergo evaluations to monitor their response, safety, and overall health status. Researchers will track the all-cause mortality rate by Day 42 as the primary outcome. Safety and treatment effects will be assessed regularly during treatment and follow-up to ensure participant well-being and gather comprehensive data on fosmanogepix’s impact.

Researchers are evaluating the use of epigenome-guided treatment selection compared to the usual standard-of-care (SOC) treatment in adults with active Crohn's Disease (CD) who are starting biologic therapy. This multicenter, prospective, randomized, controlled, open-label study aims to assess the efficacy, safety, and cost-effectiveness of this approach by comparing clinical remission and endoscopic response at Week 26. About 378 participants with active CD, defined by specific clinical and endoscopic criteria, will be included, with roughly half being biologic-naive and the other half exposed to no more than one prior biologic treatment. Participants will be randomly assigned to either receive biologic therapy guided by an epigenetic biomarker assay and the EpiPredict software, which predicts response to two biologics (Vedolizumab or Ustekinumab) or to receive treatment selected according to usual SOC without epigenome guidance. Biologic therapies will be administered following product labels and local SOC recommendations, with dose adjustments allowed as needed. Study assessments will follow the SOC schedule for each biologic during the 26-week treatment period, with different visit weeks depending on the biologic used. Participants will undergo blood sample collection for epigenetic testing during screening. Study visits will include clinical and endoscopic assessments at specified weeks, with long-term follow-up every six months up to 24 months after Week 26 using medical records and questionnaires. Researchers will measure outcomes related to clinical remission and endoscopic response, safety, and cost-effectiveness. Participants' adherence and ability to comply with protocol requirements will be monitored throughout the study.

Researchers are evaluating treatments for people with extensive stage small-cell lung cancer (ES-SCLC). This phase 3 study compares the effectiveness of adding tarlatamab to a combination of durvalumab, carboplatin, and etoposide against the combination without tarlatamab. The main goal is to see which treatment better prolongs overall survival and progression-free survival over about 3.5 years. Participants receive intravenous infusions of the study drugs. One group gets tarlatamab combined with durvalumab, carboplatin, and etoposide, while the other group receives durvalumab, carboplatin, and etoposide alone. All treatments are given as first-line therapy for their lung cancer. During the study, participants will be monitored regularly to assess their response to treatment and overall health. Researchers will measure overall survival and progression-free survival to evaluate treatment benefit. The study also involves ongoing safety monitoring, and participants will be followed for up to approximately 3.5 years to collect these outcomes.

The primary purpose of the study is to assess how well amivantamab in combination with lazertinib or in combination with chemotherapy works (antitumor activity) in participants with epidermal growth factor receptor mutated (EGFRm) non-small cell lung cancer (NSCLC; that is one of the major types of lung cancer).

Researchers are studying whether baricitinib can help preserve beta-cell function in children and adults newly diagnosed with type 1 diabetes. This Phase 3 trial focuses on participants aged 1 to less than 36 years who have recently been diagnosed with this condition. The goal is to understand if baricitinib, compared to a placebo, can maintain insulin-producing cell activity. Participants will be randomly assigned to receive either baricitinib or a placebo, both given orally. The study is double-blind, meaning neither participants nor researchers know who receives the active drug or placebo. Treatment and observation will continue for about 60 weeks. During the study, participants will undergo evaluations including measuring C-peptide levels to assess beta-cell function at the start and after 52 weeks. Researchers will monitor health status, collect laboratory tests, and track any side effects or changes in diabetes-related markers to determine the effects of baricitinib over the study period.

Researchers are evaluating the effectiveness and safety of a combination treatment called triple therapy, which includes bempedoic acid, ezetimibe, and either atorvastatin or rosuvastatin. This study focuses on patients with primary hypercholesterolemia or mixed dyslipidemia who are at high or very high cardiovascular risk. The goal is to understand how well this combination lowers LDL cholesterol (LDL-C) in a real-world clinical setting. The study observes patients who have already started triple therapy within the last four weeks. No drugs are administered as part of this study; instead, it monitors the ongoing treatment with bempedoic acid combined with ezetimibe and either rosuvastatin or atorvastatin. The study measures LDL-C changes from baseline to eight weeks after starting triple therapy and continues follow-up for one year to assess lipid goal achievement, adherence to therapy, treatment changes, laboratory value shifts, and occurrence of cardiovascular events. Participants will have their LDL-C levels and other lab values assessed at baseline, eight weeks, and one year after starting triple therapy. Researchers will collect data on adverse events, adherence to treatment, and cardiovascular outcomes such as heart attack, stroke, death from cardiovascular causes, and coronary procedures during the follow-up year. The study also tracks treatment pathways and changes over this period to better understand real-world use and effectiveness of this triple therapy approach.