Pessac

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 sotatercept as a potential treatment for pulmonary arterial hypertension (PAH), a condition where blood vessels in the lungs thicken and narrow, causing high blood pressure in the lungs and overworking the heart. PAH symptoms include difficulty breathing and reduced ability to be active. Current standard treatments address symptoms but do not stop disease progression. This Phase 3 study focuses on the long-term safety and tolerability of sotatercept when added to standard PAH therapy. Participants in this long-term follow-up study receive sotatercept through subcutaneous injections every three weeks. Only individuals who completed prior sotatercept PAH studies without early discontinuation may join. This study continues the observation and assessment of participants over an extended period to learn about the effects and safety of sotatercept combined with background PAH treatments. During the study, participants will be regularly monitored for adverse events, treatment discontinuations, and the presence of anti-drug antibodies for up to approximately 90 months. Laboratory tests will evaluate blood components such as platelets, hemoglobin, creatinine, bilirubin, and liver enzymes. Changes from baseline in body weight, blood pressure, and electrocardiogram readings will also be tracked. The study involves adherence to visit schedules and compliance with study procedures to ensure comprehensive long-term safety data collection.

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.

Researchers are conducting a French multicenter retrospective study to describe the clinical, histological, and radiological features of rare primary liver cancers. The study aims to collect biological tumor and blood samples and evaluate the effectiveness of treatments used in clinical practice to determine the best therapeutic sequences. This research will serve as the foundation for future translational studies to identify new molecular, histological, circulating, and radiological tumor biomarkers useful for diagnosis, prognosis, and treatment guidance. This study involves collecting data from patients diagnosed with rare liver cancers such as hepatocholangiocarcinoma, fibrolamellar hepatocellular carcinoma, epithelioid hemangioendothelioma, and hepatic angiosarcoma since January 1, 2018. Both living patients who agree to participate and deceased patients are included. Biological samples and tumor blocks are collected for analysis. Treatments received by patients in routine practice are reviewed to assess their efficacy and help define optimal treatment sequences. Participants provide consent for biological studies if living, and their medical records and tumor characteristics are reviewed. Researchers will describe the clinical, histological, and radiological tumor features and monitor outcomes up to five years from diagnosis. This detailed data collection supports long-term evaluation of rare liver cancers and aids in developing future biomarkers and therapeutic strategies.

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 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 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.

The PERFECT study is an observational research project that follows patients who were previously randomized in the EXCELLENT clinical trial for acute myocardial infarction. Its purpose is to evaluate the long-term clinical outcomes over an average of 10 years for patients assigned either to the Standard of Care treatment or the ProtheraCytes treatment group. This study aims to provide insight into the safety and effectiveness of these treatments over a decade. As an observational study, it involves no new treatments or interventions but focuses on collecting data from participants who completed the EXCELLENT trial. The study monitors patients from both the ProtheraCytes arm and the Standard of Care arm without altering their previous treatment plans. Participation must occur within 10 years of their initial randomization in the EXCELLENT trial. Participants are involved through ongoing data collection during the study period, with researchers assessing safety and efficacy outcomes up to study completion, around 10 years later. The study includes patients who completed their last visit in the EXCELLENT trial and excludes those who were assigned to the ProtheraCytes group but did not receive the product. Safety and the ability to give informed consent are monitored throughout the study.

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.

This research aims to evaluate the clinical benefit and safety of tabelecleucel for treating Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV+ PTLD) in patients who have undergone solid organ transplant (SOT) or allogeneic hematopoietic cell transplant (HCT) and have failed prior rituximab or rituximab plus chemotherapy treatments. The study focuses on participants with EBV+ PTLD after failure of rituximab alone or with chemotherapy, including subgroups based on chemotherapy eligibility and transplant type. Tabelecleucel, an off-the-shelf allogeneic T-cell immunotherapy, is given intravenously in 5-week cycles, with doses administered on Days 1, 8, and 15, followed by observation through Day 35. Treatment continues until maximal response, unacceptable side effects, new non-protocol therapy starts, or treatment failure. The study includes cohorts for solid organ transplant recipients (with or without chemotherapy) and hematopoietic cell transplant recipients, with up to 2 or 4 different HLA restrictions allowed, respectively. Participants undergo disease assessments including PET-CT or MRI scans to measure response, and safety is monitored throughout treatment. Follow-up lasts up to 5 years for some participants, with more frequent monitoring every 3 months for others, depending on enrollment timing and response. The primary outcome measured is the objective response rate over 2 years in different participant groups receiving commercial or comparable tabelecleucel products.