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Researchers are investigating the drug bezuclastinib in an open-label, two-part Phase 2 study for patients with Advanced Systemic Mastocytosis (AdvSM), including Aggressive Systemic Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL). The study aims to evaluate the safety, effectiveness, and how the drug behaves in the body for these serious conditions. Bezuclastinib is given orally as tablets taken continuously in 28-day cycles. The study has two parts: Part I focuses on identifying safe and tolerable doses of bezuclastinib over 18 months, while Part II evaluates its effectiveness by measuring the objective response rate and confirming the relationship between drug exposure and response during another 18-month period. Participants will undergo assessments including clinical evaluations, laboratory tests, and monitoring of their disease status to determine treatment effects and safety. Researchers will track the drug's impact on the disease and patient health throughout the study, which involves continuous treatment and follow-up over the specified time frames.

Researchers are evaluating the safety and effectiveness of elenestinib (BLU-263) combined with symptom-directed therapy (SDT) compared to placebo plus SDT in people with indolent systemic mastocytosis (ISM) whose symptoms are not well controlled by SDT alone. This Phase 2/3 randomized, double-blind, placebo-controlled study includes participants with ISM and smoldering systemic mastocytosis, and also involves groups for pharmacokinetic studies and participants who previously received a selective KIT inhibitor. The study is divided into multiple parts. Parts 1 and 2 enroll participants with ISM who will receive either elenestinib oral tablets or placebo alongside their symptom-directed therapy. Participants from Part 2 may continue into Part 3, which is an open-label extension where all receive elenestinib. Part K enrolls participants with ISM who have prior experience with selective KIT inhibitors. The study tracks treatment effects and safety over time. Participants will be monitored for up to 5 years, with assessments including the number of treatment-emergent adverse events, changes in symptom scores measured by the ISM-Symptom in Assessment Form, and overall safety monitoring. Evaluations occur at baseline, 13 weeks, 49 weeks, and throughout the long-term follow-up. The study also includes detailed tracking of symptom control and adverse events to evaluate the impact of treatment on participants' health and quality of life.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.

Researchers are evaluating the use of benralizumab in adults with severe eosinophilic asthma to understand its effectiveness in achieving partial and complete clinical remission. This multicenter, observational, prospective study called the ATHENA study aims to add real-world evidence on benralizumab's role in clinical practice. The study also seeks to explore the immunological effects of benralizumab to better understand asthma biology and to collect long-term safety data. Participants will receive benralizumab, administered as a 30mg subcutaneous injection following the approved prescribing information. The study focuses on patients who have either recently started benralizumab treatment within 7 days before enrollment or plan to start within 7 days after enrollment. Treatment and monitoring will follow routine clinical practice without additional interventions. During the study, researchers will track the number and percentage of patients who achieve clinical remission according to the SANI definition over 24 months. Participants will be closely monitored for treatment effectiveness, immunological changes, and safety outcomes throughout this period. The study involves collecting relevant clinical data and ensuring participants adhere to treatment and follow-up visits as per standard care.

This research focuses on patients with Relapsed/Refractory Multiple Myeloma (RRMM) who have been treated with the T-cell redirectors teclistamab or talquetamab outside of clinical trials. The study aims to describe the clinical outcomes and safety management of these treatments in a real-world setting, analyzing how patients respond and survive after receiving these therapies. The study does not administer any new interventions but instead collects and analyzes retrospective data from medical records of patients who have received teclistamab or talquetamab. Participants are grouped based on when they received their first dose, covering different time periods up to the end of 2025. This allows the study to assess outcomes for patients treated with these drugs over several years. Throughout the study, researchers will monitor various outcomes, including overall response rates, time to response, duration of response, minimal residual disease status, overall survival, progression-free survival, and time to next treatment. Safety management during treatment is also described. Data will be collected from baseline (day 1) through up to 40 months following treatment initiation, using medical records to understand treatment effects and patient characteristics over time.

Researchers are evaluating the safety and effectiveness of KarXT in adults aged 55 to 90 who have mild to severe Alzheimer's Disease (AD) accompanied by moderate to severe psychosis related to AD. This phase 3 study aims to better understand how KarXT compares to a placebo in treating the psychotic symptoms associated with Alzheimer's Disease. Participants must have documented AD diagnosis and a history of psychotic symptoms lasting at least two months prior to starting the study. Participants will receive either KarXT or a placebo, with specified doses given on designated days. The study is designed as a randomized, double-blind, placebo-controlled trial with parallel groups to assess the treatment's effects. Details about dosing schedules and administration are planned but not specified here. During the study, researchers will measure changes from baseline in the Neuropsychiatric Inventory-Clinician: Hallucinations and Delusions (NPI-C: H+D) score up to week 14 to evaluate the impact on psychosis symptoms. Participants will undergo brain imaging (MRI or CT) if not already done within the past five years to rule out other conditions, and safety monitoring including laboratory tests will be conducted. The total participation duration covers screening through at least 14 weeks of treatment and assessment.

Researchers are evaluating the efficacy, safety, and pharmacokinetics of intravenous prasinezumab compared with placebo in people with early-stage Parkinson's disease. Participants must be on stable symptomatic monotherapy with levodopa and meet specific criteria including body weight and disease stage. This Phase III study aims to understand how prasinezumab affects motor progression in Parkinson's disease. Participants will receive either prasinezumab or a placebo through intravenous infusion following a schedule outlined in the study protocol. The study compares these two groups to assess the impact of prasinezumab on disease progression. The treatments are administered regularly over the course of the trial. During the study, participants will be monitored for motor progression using the Movement Disorder Society - Unified Parkinson's Disease Rating Scale Part III score, with assessments continuing up to at least week 104. Researchers will also evaluate safety and pharmacokinetics throughout the trial. Participants are required to adhere to contraception requirements and attend scheduled visits for evaluations and infusions.

Researchers are evaluating the effectiveness of TEV-56286, taken orally, for treating adults with Multiple System Atrophy (MSA), a rare neurological condition. This Phase 2 trial aims primarily to measure changes in patients' symptom severity using the Modified Unified Multiple System Atrophy Rating Scale over 48 weeks. The study also examines other specific effects of TEV-56286 and evaluates its safety and tolerability. Participants will be randomly assigned to receive either TEV-56286 capsules or a matching placebo, both given orally during a 48-week double-blind treatment period. The overall study duration for each participant is about 56 weeks, which includes a screening phase lasting up to 4 weeks and a follow-up visit approximately 4 weeks after treatment ends. The trial is conducted across multiple countries including the US, Israel, Italy, Spain, Germany, France, Japan, and Serbia. During the study, participants will undergo regular assessments to monitor their condition using rating scales specific to MSA symptoms. Researchers will track changes from baseline scores over time to evaluate treatment effects. Safety and tolerability will be closely watched throughout the study and during follow-up visits. The total planned duration of the study is around 27 months.

Researchers are evaluating ACP-204, a drug that blocks a specific serotonin receptor, in adults aged 55 to 95 with Alzheimer's Disease Psychosis (ADP). The study is designed as a master protocol with three independent, multicenter, randomized, double-blind, placebo-controlled trials. The trials include Phase 2 and Phase 3 studies to assess the drug's effectiveness and safety in treating psychotic symptoms associated with ADP. The research involves three substudies. Substudy 1 (Phase 2) tests two doses of ACP-204, 30 mg and 60 mg, against a placebo to evaluate dose response. Substudies 2A and 2B (both Phase 3) will independently confirm the effects of either both doses or a single dose from Part 1 compared to placebo. Each substudy includes a screening period of up to 49 days, a six-week double-blind treatment phase, and a 30-day safety follow-up for those not continuing into an open-label extension. Vital status follow-up is conducted for participants who end the study early. Participants will receive regular assessments, including evaluations of psychotic symptoms using the Scale for the Assessment of Positive Symptoms-Hallucinations and Delusions subscales from baseline to Week 6. Other study involvement includes brain imaging scans and biomarker tests to confirm Alzheimer's disease diagnosis, cognitive testing, and monitoring of safety and vital status throughout the study periods. Stable living arrangements and support from a caregiver are required to complete all study visits.

Researchers are evaluating whether AI-driven remote monitoring using a mini-invasive wearable device can improve clinical outcomes in adult patients aged 18 years and older with chronic heart failure (CHF). The study aims to determine if continuous remote monitoring can reduce hospital admissions by 20% compared to standard care and improve functional, biochemical, and instrumental parameters. This prospective, multicenter, observational study compares patients using the EmbracePlus wearable device integrated with AI analytics to those receiving standard clinical follow-up. Participants in the intervention group will wear the EmbracePlus device continuously for six months, which monitors physiological signals such as oxygen saturation, heart rate variability, electrodermal activity, respiratory rate, temperature, and sleep quality. The AI system analyzes data in real time, generating automated alerts for healthcare providers when abnormal changes are detected. These alerts may lead to teleconsultations, treatment adjustments, or in-person evaluations. The control group will receive standard care with scheduled in-person visits every three months, including laboratory tests and cardiac evaluations without wearable device use. Throughout the study, participants will attend follow-up visits both remotely and in person for clinical assessments and treatment modifications. Researchers will measure changes in hospital admissions over six months and assess cardiac function, neurohormonal biomarkers, exercise tolerance, quality of life, and therapy-related adverse events. Data collected will also be used to develop AI models for predicting CHF progression. All data are pseudonymized and the study adheres to ethical standards, with participants providing informed consent before enrollment.