Vila Real

Researchers are evaluating the use of elafibranor in adults with Primary Biliary Cholangitis PBC who also have cirrhosis, a serious liver condition involving scarring. PBC is a slowly progressing disease that damages bile ducts, causing bile acids to build up and harm the liver further. This study aims to see if elafibranor can better prevent worsening of the disease, including the need for liver transplant or death, compared to a placebo. The safety of long-term use and effects on symptoms like itching and tiredness will also be assessed. Participants will be randomly assigned to take either one 80 mg tablet of elafibranor or a matching placebo tablet once daily, taken orally with or without food, at about the same time each morning. The treatment period can last up to 3.5 years in a double-blind setting, meaning neither participants nor researchers know who receives the drug or placebo. This design allows a direct comparison of elafibranors impact on disease progression and safety over a long term. Throughout the study, participants will undergo regular assessments including blood tests, physical exams, vital signs, ECGs, and liver imaging to monitor liver function and stiffness. Researchers will track a range of outcomes such as survival without clinical events, changes in liver and blood markers, symptom severity, and quality of life measures. Safety monitoring will continue until 4 weeks after the last dose. Each participant may be involved for up to 3.5 years from baseline to final evaluation.

Researchers are investigating the effectiveness and safety of combining petosemtamab with pembrolizumab compared to pembrolizumab alone as first-line treatment for recurrent or metastatic PD-L1 positive head and neck squamous cell carcinoma HNSCC. This Phase 3 randomized, open-label study focuses on patients who have not received previous systemic therapy for incurable recurrent or metastatic disease and aims to improve treatment outcomes in this population. Participants will be randomly assigned to receive either the combination of petosemtamab plus pembrolizumab or pembrolizumab alone. The study excludes patients previously treated with anti PD-L1 or anti-EGFR therapies, with some exceptions for local treatments like cetuximab under specific conditions. The treatments will be administered as part of the trial, with researchers comparing the two approaches over the treatment period. During the study, participants will undergo regular evaluations including tumor assessments based on RECIST v1.1 criteria, health-related quality of life questionnaires, and safety monitoring for adverse events. The main outcomes measured include overall survival and objective response rate up to approximately three years. Additional assessments include progression-free survival, duration of response, and pharmacokinetics. These activities aim to provide comprehensive data on how well the treatments work and their safety profiles throughout the study period.

Researchers are evaluating the clinical efficacy, safety, and tolerability of XEN1101 as an additional treatment for people with focal-onset seizures in a Phase 3 randomized, double-blind, placebo-controlled study. This trial aims to compare two doses of XEN1101 with a placebo to see how well the medication can reduce seizure frequency in patients who continue their current antiseizure medications. The study involves adults diagnosed with focal epilepsy who have tried at least two antiseizure medicines without achieving seizure freedom. About 360 participants will be randomly assigned to receive either 25 mg or 15 mg of XEN1101 or a placebo once daily with an evening meal. The study includes up to 9.5 weeks of baseline monitoring to track seizure frequency followed by 12 weeks of blinded treatment. Participants maintaining the study drug can then join an open-label extension to continue treatment or enter an 8-week follow-up after treatment ends. Throughout the study, participants will keep accurate seizure diaries and continue their stable antiseizure medications. Researchers will measure the median percentage change in seizure frequency from baseline through the 12-week treatment period, along with secondary outcomes like the proportion of participants with at least a 50 reduction in seizures and patient-reported improvement. Safety will be monitored from screening until 56 days after the last dose. Overall, participants are involved for the baseline, treatment, and follow-up phases lasting several months.

This trial investigates the effectiveness of elacestrant compared to standard endocrine therapy for adults with node-positive, estrogen receptor-positive ER, HER2-negative early breast cancer who are at high risk of recurrence. The study aims to understand if elacestrant can improve outcomes in this group over standard treatments. Participants will be randomly assigned to receive either 345 mg of elacestrant once daily for five years or continue with their previous standard endocrine therapy, which may include anastrozole, letrozole, exemestane, or tamoxifen. Both treatments are taken orally, and the study is open-label, meaning participants and researchers know which treatment is given. During the study, participants will be monitored for up to five years for outcomes such as invasive breast cancer-free survival, distant relapse-free survival, overall survival, and quality of life changes. Assessments include questionnaires on health status and physical functioning, symptom evaluation, and blood tests to measure elacestrant levels. Safety and adverse events will be tracked throughout and for 28 days after treatment ends.

Researchers are evaluating trastuzumab deruxtecan T-DXd as a treatment for adult patients with advanced HER2-positive gastric or gastroesophageal junction GEJ adenocarcinoma who have previously received a trastuzumab-based regimen. This study aims to assess the real-world effectiveness of T-DXd, patient characteristics, treatment patterns, and safety in this population. It also includes data collection on patients receiving conventional therapies for exploratory comparison. This is a non-interventional observational study where patients receive T-DXd or conventional therapies as part of routine clinical care according to approved guidelines SmPC. No investigational drugs are given. The study tracks patients starting T-DXd as a second-line or later treatment and collects data on other therapies such as chemotherapy and immunotherapy used in clinical practice. Participants will be followed for up to approximately 2 years from baseline to monitor outcomes including time to next treatment, changes in treatment, physician-reported safety events, use of prophylactic treatments, and quality of life using validated questionnaires. Data on physician visits, treatment discontinuation, and safety events will also be collected to understand treatment tolerability and patient experience in a real-world setting.

Researchers are evaluating camizestrant, a new oral drug, compared to standard adjuvant endocrine therapies for patients with early breast cancer that is estrogen receptor positive and HER2 negative. This trial focuses on patients at intermediate-high or high risk for the cancer returning who have completed local treatments like surgery, with or without chemotherapy. The study is a Phase III open-label trial sponsored by AstraZeneca, aiming to see if camizestrant improves invasive breast cancer-free survival over a planned treatment duration of seven years. Participants will be randomly assigned to one of two treatment groups one receiving standard endocrine therapy chosen by the doctor including aromatase inhibitors such as exemestane, letrozole, or anastrozole, or tamoxifen with or without abemaciclib, and the other receiving camizestrant with or without abemaciclib. Treatments are taken orally, and both groups are followed for up to 10 years from the last patients randomization to monitor outcomes and safety. During the study, participants will have regular assessments to monitor invasive breast cancer-free survival, overall survival, and other outcomes like distant relapse-free survival and quality of life. Safety evaluations include tracking side effects using established criteria and patient-reported measures. Pharmacokinetics of camizestrant will be studied for six months, with adverse events monitored up to 28 days after the last treatment dose. The total involvement can last up to 14 years including treatment and follow-up periods.

This research aims to observe the use of trastuzumab deruxtecan T-DXd in adults with HER2-low unresectable or metastatic breast cancer who have previously received chemotherapy in the metastatic setting or experienced disease recurrence within 6 months after adjuvant chemotherapy. The study will collect information on patient characteristics, how treatments are used, tolerability, management of side effects, and patient experiences with T-DXd, as well as data on conventional chemotherapy treatments in a registry. Participants will receive T-DXd or conventional chemotherapy as chosen by their doctors according to approved guidelines in routine clinical practice. No study drug is given by the research team since this is a non-interventional study. The conventional chemotherapy group will be analyzed separately to better understand treatment patterns and outcomes. During the study, researchers will gather data for up to 31 months, monitoring real-world time to next treatment, treatment patterns, safety events, use of treatments to manage side effects, and patient-reported outcomes including tolerability and quality of life. They will also track occurrences of nausea and vomiting through patient diaries. This information will help evaluate treatment use and patient experiences outside of clinical trial settings.

People with psychotic disorders like schizophrenia often face challenges in daily functioning, physical health, and quality of life. This study evaluates the feasibility and impact of a structured, multimodal physical exercise program for adults with psychosis who are treated with long-acting injectable antipsychotics. The goal is to see if supervised exercise can improve physical function, psychological well-being, and biological markers related to brain health and metabolism. Participants will be divided into two groups one receiving usual care and the other participating in a 24-week supervised exercise program. The exercise includes aerobic activities such as walking and cycling, strength training with exercises targeting different muscle groups, and mobility, balance, and flexibility exercises. Sessions are conducted by qualified professionals in an outpatient psychiatric setting in Portugal. Throughout the study, participants will undergo assessments at baseline, during the program, after completion, and at an 8-week follow-up. These assessments measure physical function, body composition, psychological well-being, quality of life, and blood markers like brain-derived neurotrophic factor, dopamine, serotonin, and metabolic indicators. The study aims to provide evidence for including exercise as part of routine psychiatric care for people with psychosis.

Peripheral arterial disease PAD is a condition that mainly affects blood flow in the lower limbs and causes symptoms like pain and cramps during activity, known as intermittent claudication. Researchers are evaluating a personalised exercise program combining cardiovascular and resistance training to improve how patients with PAD function. This study focuses on using wearable technology and artificial intelligence to monitor patients real-time responses and tailor exercise doses individually. The study involves two groups one receives a supervised multicomponent exercise program three times a week for 12 weeks, including aerobic walking and resistance exercises with progressive intensity based on pain levels. The other group follows usual care with lifestyle advice but no specific exercise program. The exercise sessions last about 60 to 80 minutes, with detailed monitoring using near-infrared spectroscopy and accelerometers to track muscle oxygen levels and movements. Participants will be assessed before and after the 12-week program to measure pain-free and maximum walking distances, muscle oxygenation, muscle strength and power, blood pressure, quality of life, daily walking ability, blood glucose, and physical activity levels. The study uses random assignment to groups and collects data to understand how personalised exercise affects PAD symptoms and function. The total participation time is 12 weeks, with evaluations at the start and end of the study.

Researchers are evaluating the effectiveness of teclistamab combined with lenalidomide and teclistamab alone compared to lenalidomide alone as maintenance therapy in participants with newly diagnosed multiple myeloma. This Phase 3, multicenter, randomized, open-label study focuses on patients who have undergone autologous stem cell transplant. The goal is to assess the benefits of these treatments in preventing disease progression after transplant. Participants will be randomly assigned to one of three groups teclistamab with lenalidomide, teclistamab alone, or lenalidomide alone. Teclistamab is given as a subcutaneous injection, while lenalidomide is taken orally. The treatments are administered as maintenance therapy following stem cell transplantation. During the study, participants will be monitored for disease progression and survival for up to approximately eight years. Researchers will assess progression-free survival and the rate of complete response with no detectable minimal residual disease at 12 months. Quality of life, symptom impact, and patient-reported toxicities will also be evaluated through questionnaires. Safety and overall survival will be tracked throughout the study period.