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Researchers are investigating new treatments for people with high-risk, early-stage breast cancer, specifically targeting triple-negative breast cancer (TNBC) and hormone receptor (HR)-low positive/HER2-negative breast cancer. These types have little or no HER2 protein and involve hormones like estrogen or progesterone. The study aims to evaluate if the addition of sacituzumab tirumotecan (sac-TMT), a targeted therapy, combined with pembrolizumab and chemotherapy can improve outcomes compared to pembrolizumab with chemotherapy alone. Participants receive treatments including sacituzumab tirumotecan, pembrolizumab, and chemotherapy drugs such as carboplatin and paclitaxel, all given by intravenous infusion. Rescue medications like antihistamines, acetaminophen, dexamethasone, or steroid mouthwash may be used as needed. The study is randomized and open-label, comparing sac-TMT followed by chemotherapy plus pembrolizumab to chemotherapy and pembrolizumab without sac-TMT. During the study, researchers will monitor participants up to about 30 weeks to assess the percentage of people with no remaining cancer cells at surgery. They will also follow participants for up to approximately 92 months to track event-free survival, meaning time without cancer growth, spread, or return. Participants will undergo imaging, clinical assessments, and laboratory tests to evaluate treatment effects and safety throughout the study.

Researchers are evaluating the safety and effectiveness of combining durvalumab and domvanalimab compared to durvalumab plus placebo in adults with locally advanced (Stage III), unresectable non-small cell lung cancer (NSCLC) whose disease has not worsened after definitive platinum-based concurrent chemoradiation therapy. This Phase III, randomized, double-blind, placebo-controlled, international study involves multiple centers. Participants receive intravenous infusions of durvalumab and domvanalimab or durvalumab and placebo. The treatments are given after patients have completed concurrent platinum-based chemotherapy and radiation therapy with a total radiation dose of approximately 60 Gy. The study monitors patients over time to assess treatment effects and safety. During the study, participants undergo evaluations including tumor tissue analysis for PD-L1 status, performance status assessments, and monitoring of organ and marrow function. The main outcome measured is progression-free survival up to 8 years after randomization. Researchers also monitor for any adverse effects and disease progression throughout the study period.

Amyotrophic lateral sclerosis (ALS) is a serious, fast-progressing nervous system disease with an average survival of 2.5 years after diagnosis. Currently, effective treatments are limited to Riluzole. Research suggests that increasing cell access to Nicotinamide Adenine Dinucleotide (NAD) and stimulating enzymes called sirtuins may slow disease progression. This study aims to evaluate whether a combination of Nicotinamide Riboside (NR) and Pterostilbene, called EH301, can slow neurodegeneration, delay disease progression, improve survival, and enhance quality of life in ALS patients. The NO-ALS extension study follows patients who completed the original NO-ALS trial. All participants receive the active treatment EH301, which combines Nicotinamide Riboside and Pterostilbene, as an open-label extension. This study provides patients the option for compassionate use of the supplement while assessing its effects on motor symptoms, lung function, and survival. Participants will be monitored for adverse events throughout the study, which lasts up to 1 year. Researchers will track safety, progression of motor symptoms, changes in vital capacity, and overall survival. This extension allows long-term observation of EH301's impact on ALS progression and patient well-being.

Researchers are studying acute intermittent porphyria (AIP), a genetic disease causing buildup of a substance called porphobilinogen (PBG), which leads to severe symptoms like abdominal pain, paralysis, fatigue, and risks of kidney failure and liver cancer. The study focuses on how a carbohydrate-rich diet may affect these symptoms and biological markers such as tissue and serum glucose, plasma insulin, cytokines, amino acids, and gut microbiota. The trial also explores mitochondrial activity, genetic mutations, and new biological markers in people with AIP. Participants will follow two different diet plans in a crossover design: one with 60-65% carbohydrates (Diet Plan A) and another with 40-45% carbohydrates (Diet Plan B), each lasting 4 weeks and separated by a 4-week washout period. Half of the participants will start with Diet Plan A before switching to Diet Plan B, while the other half will do the reverse. Both diets meet recommended nutrient guidelines to maintain stable weight. During the study, researchers will measure PBG levels, glucose, insulin, cytokines, amino acids, gut microbiota, body composition, and physical activity with accelerometers before and after each diet period. They will also assess mitochondrial oxidative activity at the cellular level and analyze mitochondrial DNA and RNA for mutations. The study involves multiple hospitals and universities and includes detailed monitoring of biological and health outcomes over the intervention periods.

Researchers are evaluating whether D-serine, a modulator of the N-methyl-D-aspartate receptor (NMDAR), has therapeutic effects on Parkinson's disease (PD). This randomized, double-blind, placebo-controlled Phase 2 trial includes 100 participants diagnosed with PD within the past 5 years. The study aims to assess D-serine's impact on symptom severity using the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and also investigates effects on brain dopamine transporter levels and cognitive function. Participants will receive both placebo and D-serine during different periods of the 58-week treatment phase. D-serine dosing begins with 2 capsules of 500 mg twice daily in the first week and increases to 4 capsules twice daily for the remaining intervention period. Participants' existing dopaminergic medications will be optimized before study start and maintained stable for the first 32 weeks; adjustments may be made after this time. Following the treatment phase, participants will stop study drugs and enter a 12-week washout period with a final study visit. Throughout the study, participants will undergo clinical evaluations including rating scales and questionnaires, cognitive testing, blood sample collection, and dopamine transporter imaging using single-photon emission tomography (DaTscan). Researchers will monitor changes in clinical symptoms, brain dopamine transporter levels, and cognition, as well as safety aspects. The total study duration for each participant includes screening, 58 weeks of treatment, and 12 weeks of follow-up after stopping study drugs.

Researchers are evaluating Trastuzumab deruxtecan (T-DXd) in adult patients with unresectable or metastatic HER2-low expressing breast cancer. This non-interventional study aims to assess the effectiveness of T-DXd, patients' demographic and clinical characteristics, treatment patterns, tolerability, management of adverse drug reactions, and patient experience. The study also collects data on conventional chemotherapy treatments in a disease registry to better understand treatment outcomes in this population. Participants will receive treatment with Trastuzumab deruxtecan or conventional chemotherapy drugs such as capecitabine, eribulin, gemcitabine, paclitaxel, or nab-paclitaxel according to the Summary of Product Characteristics and routine clinical practice. No study drug will be administered by the researchers, as treatments follow physicians' standard care decisions. This approach allows observation of real-world treatment use and outcomes. During the study, patients' treatment timelines and responses will be followed, focusing on the time to next treatment up to 31 months. Researchers will monitor tolerability, adverse drug reactions, and patient-reported experiences. Data collection includes clinical and demographic information, treatment patterns, and outcomes to provide a comprehensive understanding of T-DXd and conventional chemotherapy use in this patient group.

Researchers are investigating whether the medicine vicadrostat, when taken together with empagliflozin, can lower the risk of heart-related problems in adults who have type 2 diabetes, high blood pressure, and cardiovascular disease but no history of heart failure. This study is a Phase III trial that compares the effects of vicadrostat plus empagliflozin to a placebo plus empagliflozin in people with these conditions. Participants are randomly assigned to one of two groups: one group takes vicadrostat and empagliflozin tablets, and the other group takes placebo tablets that look like vicadrostat along with empagliflozin. All participants take one tablet daily for a period ranging from two and a half years up to four years and three months. Throughout the study, participants continue their usual medications for diabetes, high blood pressure, and cardiovascular disease. During up to 51 months of participation, participants visit the study site regularly where doctors collect health information and blood samples. Researchers track when participants experience cardiovascular events such as heart-related deaths or heart failure events. The study also monitors participants’ overall health and any side effects they may experience to assess the safety and effects of the treatments.

Researchers are evaluating whether proactive therapeutic drug monitoring (TDM) is better than standard care for maintaining steady disease control in adults with rheumatoid arthritis (RA) who are treated with a subcutaneous tumor necrosis factor inhibitor (adalimumab). This Phase 4 study aims to determine if adjusting drug doses based on regular blood tests for drug levels and anti-drug antibodies can prevent disease flare-ups more effectively than standard dosing without such monitoring. Participants will be randomly assigned to one of two groups. The TDM group will have their adalimumab doses adjusted following specific rules based on blood test results to keep drug levels within a therapeutic range. Dose intervals may be shortened, lengthened, or therapy switched depending on antibody levels and drug concentration. The standard care group will continue treatment without these blood test-based adjustments. The study lasts 18 months with visits at baseline, 4, 8, 12, and 18 months, along with digital visits at 2, 6, 10, 14, and 16 months, including blood sampling at each visit. Participants will have regular blood tests to measure drug levels and antibodies every two months. They will attend on-site and digital visits for assessments of disease control and safety. The primary outcome is sustained disease control without flare over the 18-month follow-up. Researchers will monitor adherence, safety, and treatment effectiveness throughout the study period to compare the two treatment approaches.

Amyotrophic lateral sclerosis (ALS) is a serious and rapidly progressing nervous system disease with an average survival of two to three years after diagnosis. Patients experience significant physical and psychological suffering, and apart from the drug Riluzole, no effective treatment exists. The care for advanced ALS is costly, and one major challenge is deciding whether to extend ventilation support as the disease progresses, which can maintain quality of life but also imposes a heavy caregiving burden on family and healthcare workers. This study evaluates the impact of long-term mechanical ventilation support on the quality of life of ALS patients and their families. Patients either choose life-prolonging treatment with long-term mechanical ventilation support or decline it. The study involves using questionnaires to measure overall quality of life, health-related quality of life, and disease-specific quality of life before and after starting ventilation support. Participants include ALS patients, their partners, and children aged 8 and older. The study collects data at inclusion and at 3, 9, 15, and 21 months after inclusion using the Quality of Life Scale. Researchers aim to provide valuable information to help patients and clinicians make shared decisions about ventilation support by understanding its effects on patients and their families over time.

Researchers are investigating the genetic factors that may contribute to the development of Amyotrophic Lateral Sclerosis (ALS) in Norway. The study aims to better understand genetic causes relevant to ALS by analyzing gene frequencies, new ALS genes, and genetic risk factors from 2020 through 2030. Participants will provide written informed consent and complete a brief questionnaire about their family history. A blood sample will be collected for genetic analysis, which is carried out at the Department of Medical Genetics, Telemark Hospital Trust throughout the recruitment period. Participants may opt to receive their genetic results in a diagnostic setting. During the study, participants will be involved in providing clinical information, completing questionnaires, and submitting blood samples. Researchers will monitor genetic characteristics and analyze data related to gene frequency and new ALS genes over time. The study includes adults aged 16 to 100 years and involves continuous genetic analysis from 2020 to 2030.