Plzen Mesto

Researchers are studying adults with confirmed Primary Biliary Cholangitis (PBC) and cirrhosis, a scarring of the liver caused by damage to bile ducts. PBC is a slowly progressing disease that causes bile acid buildup and further liver damage, which can lead to cirrhosis. This study aims to evaluate if elafibranor, a daily medication, can prevent worsening clinical outcomes such as the need for liver transplant or death, compared to a placebo. It also looks at the safety of long-term elafibranor use and its effect on symptoms like itching and tiredness. Participants will take either an 80 mg tablet of elafibranor or a matching placebo once daily for up to 3.5 years in a double-blind setup, meaning neither the participants nor researchers know who receives which treatment. This long-term treatment period is designed to monitor the drug's impact over time. The study includes two groups: one receiving elafibranor and the other receiving placebo, with treatment lasting up to approximately 42 months. During the study, participants will be regularly assessed from the start until 4 weeks after treatment ends, with a maximum involvement of 3.5 years. Researchers will measure event-free survival, tracking if participants avoid clinical events indicating disease worsening. Safety monitoring will include tracking side effects and overall health, while symptom impact will be evaluated. Participants will provide informed consent and follow the study protocol throughout this extended observation period.

Researchers are evaluating the effect of vipoglanstat on reducing non-menstrual pelvic pain related to endometriosis in women. This phase 2 trial focuses on women who have moderate to severe pain caused by endometriosis, aiming to see how well vipoglanstat works compared to a placebo. The study is designed to measure changes in pain over a period of about four months. Participants in this trial will receive either vipoglanstat capsules or matching placebo capsules taken orally for approximately four menstrual cycles during the treatment period. The study is randomized and double-blind, meaning neither participants nor researchers know who receives the active drug or placebo until the study ends. Two different doses of vipoglanstat are being tested to assess safety and effectiveness. During the study, women will be monitored for changes in their endometriosis-related non-menstrual pelvic pain, with the primary measure being the percentage of participants who meet a specific pain response criterion from the start of the study to the fourth month of treatment. The trial includes careful tracking of symptoms and safety over the treatment duration to evaluate how well vipoglanstat manages pain in this population.

Researchers are evaluating the safety and effectiveness of belzupacap sarotalocan (bel-sar) compared to a sham control in patients with primary indeterminate lesions or small choroidal melanoma. This is a Phase 3 randomized, masked, controlled trial aiming to provide clear evidence on bel-sar's use in these eye conditions. The study focuses on patients without metastatic disease who have not previously been treated for their lesions, except possibly with photodynamic therapy. Participants receive bel-sar through a suprachoroidal injection using a specialized microinjector, followed by activation with an infrared laser. The control group undergoes similar procedures with sham devices and sham laser application to maintain masking. The trial is carefully designed to keep patients, assessors, and sponsors unaware of the treatment assignments to ensure unbiased results. Throughout the study, researchers will monitor the time it takes for tumors to progress, up to 65 weeks. Participants will undergo imaging and clinical evaluations to track safety and tumor status. The trial's structured follow-up includes assessments for any adverse effects and effectiveness of the treatment, helping to determine bel-sar's role in managing these eye tumors.

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 the safety and effectiveness of KarXT combined with KarX-EC in adults aged 55 to 90 who have agitation related to Alzheimer's Disease. This phase 3 study aims to better understand how these treatments impact agitation symptoms in this population by comparing them to a placebo group. Participants must have a confirmed Alzheimer's diagnosis and meet specific criteria for agitation severity to join the study. Participants will receive either the Xanomeline/Trospium Chloride Capsule, Xanomeline Enteric Capsule, or a placebo, each given at specified doses on designated days. The study is randomized, double-blind, and placebo-controlled to ensure reliable comparison of treatment effects. The treatment period lasts through Week 14, during which dosing schedules are closely followed. Throughout the study, participants will be regularly assessed using the Cohen-Mansfield Agitation Inventory-International Psychogeriatric Association (CMAI-IPA) to measure changes in agitation levels from baseline to Week 14. Caregivers will provide reports on participant status and help ensure medication compliance. Safety and symptom changes will be carefully monitored to evaluate the treatments' effects during this period.

Researchers are investigating the effectiveness, safety, and tolerability of combining baxdrostat with dapagliflozin compared to dapagliflozin alone in people with chronic kidney disease (CKD) and high blood pressure. This Phase III, international, multicenter, double-blind, placebo-controlled study aims to see if this combination reduces risks such as significant kidney function decline, kidney failure, heart failure events, or cardiovascular death. The study includes a 4-week run-in period where participants not previously treated with SGLT2 inhibitors receive dapagliflozin alone. After this, participants are randomly assigned to receive either baxdrostat plus dapagliflozin or placebo plus dapagliflozin in a double-blinded manner. Study visits occur frequently initially (at 2, 4, 8, 16, 34, and 52 weeks after randomization) and then approximately every 4 months. If participants stop the blinded treatment early, they continue dapagliflozin alone unless specific criteria require its discontinuation. Participants will undergo regular assessments including blood pressure monitoring and laboratory tests related to kidney function and cardiovascular health. The primary outcome measures the reduction in risk of major kidney and heart events over up to 37 months. Even if participants stop the study treatment, they will continue follow-up visits and data collection to ensure comprehensive safety and efficacy evaluation throughout the study duration.

Researchers are conducting a global study to understand the impact of moderate to severe alopecia areata (AA), non-segmental vitiligo (NSV), and hidradenitis suppurativa (HS) on adolescents and adults. This study aims to assess the burden these conditions place on patients' quality of life and daily functioning in a large real-world population. The study involves participants diagnosed by a physician with one of the three conditions: AA, NSV, or HS. There are no interventional treatments or medications being tested in this study, as it is observational in nature. Data collection focuses on patient-reported outcomes and measures that evaluate disease severity and its effects. Participants will complete various questionnaires and assessments related to their condition, such as the Alopecia Areata Symptom Impact Scale (AASIS) for AA, the Severity of Alopecia Tool (SALT) for scalp hair loss in AA, the Facial Vitiligo Area Scoring Index (F-VASI) and Vitiligo Quality of Life Score (VitiQoL) for vitiligo, and the Dermatology Life Quality Index (DLQI) and International Hidradenitis Suppurativa Severity Scoring System (IHS4) for HS. These tools help researchers understand how symptoms affect quality of life and disease severity. The study collects information up to the day of the study visit.

Researchers are conducting a Phase 3 study to compare two front-line treatments for adults with nonsquamous non-small cell lung cancer (NSCLC) that is stage IV or advanced stage IIIB/C. The study focuses on patients whose tumors have a KRAS p.G12C mutation and are negative for PD-L1 expression. The main goal is to evaluate how each treatment affects progression-free survival and overall survival over about 2.5 years. Participants will be randomly assigned to receive either sotorasib combined with platinum doublet chemotherapy or pembrolizumab combined with platinum doublet chemotherapy. Sotorasib is given orally, while pembrolizumab is given intravenously. Both groups will receive the combination therapies as their initial treatment for advanced NSCLC. During the study, participants will be monitored regularly to assess treatment effects and safety. Researchers will track how long patients live without the cancer worsening and overall survival over approximately 2.5 years. The study includes evaluations to determine eligibility and ongoing assessments to monitor health and treatment response throughout the trial period.

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.

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.