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Researchers are investigating CGT9486, also known as bezuclastinib, in an open-label Phase 2 study for patients with Advanced Systemic Mastocytosis AdvSM. This includes those diagnosed with 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, pharmacokinetics, and pharmacodynamics of bezuclastinib in this patient population. Participants will receive bezuclastinib tablets orally, taken continuously in 28-day cycles. The study is divided into two parts Part I focuses on identifying effective and tolerable dosing exposures over 18 months, while Part II evaluates the drugs efficacy by measuring objective response rates and confirming the exposure-response relationship, also over 18 months. Additional assessments include effects on mutation allele burden, serum tryptase levels, histopathologic changes, spleen and liver volume, and safety monitoring. During the study, participants will undergo various clinical evaluations, including laboratory tests, imaging to monitor organ size changes, and assessments of disease response and progression. Researchers will track adverse events and pharmacokinetic profiles throughout the 18 months. The study involves continuous monitoring of participants to understand the treatments impact on survival and disease progression over this period.

Researchers are evaluating the safety and effectiveness of elenestinib BLU-263 combined with symptom directed therapy SDT compared to placebo plus SDT in adults with indolent systemic mastocytosis ISM whose symptoms are not well controlled by SDT alone. This randomized, double-blind, placebo-controlled Phase 23 study includes multiple parts to assess different doses and durations of elenestinib treatment, including an open-label extension for participants finishing earlier phases. The study also enrolls participants who have previously received an approved selective KIT inhibitor and includes pharmacokinetic groups. Participants receive oral elenestinib or placebo once daily alongside SDT, which is personalized based on individual symptom management needs. Part 1 focuses on short-term treatment lasting up to 12 weeks, while Part 2 extends treatment to approximately 48 weeks. Part 3 and other parts allow treatment for up to about 5 years. The study monitors participants through these phases to evaluate how elenestinib affects symptoms, disease markers, and safety over time. During the study, participants undergo regular assessments of symptoms using the ISM-Symptom Assessment Form ISM-SAF, laboratory tests including serum tryptase and KIT D816V allele levels, bone marrow evaluations, and quality of life measures. Researchers track adverse events and changes in disease-related factors at various points up to 5 years. This thorough monitoring helps measure treatment effects and safety over both short and long-term periods, with total participation lasting several years depending on the study part.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study. This trial focuses on patients with established atherosclerotic cardiovascular disease ASCVD or those at high risk for a first ASCVD event. The study aims to assess how AZD0780 compares to placebo in reducing the risk of major adverse cardiovascular events, also known as MACE-PLUS, over the course of the trial. Participants are randomly assigned to receive either oral AZD0780 once daily or a matching placebo once daily. The study continues until a primary analysis censoring date, which may be up to approximately 54 months from randomization. After this, a study closure visit will be conducted as the final visit for each participant. During the study, participants will be regularly monitored for cardiovascular events including heart attacks, strokes, urgent coronary revascularizations, and other related outcomes. Researchers will track the time to first occurrence of these events as the primary outcome. Safety and other secondary outcomes like all-cause mortality will also be assessed. The total participation time can last up to about 54 months, with ongoing evaluations throughout this period.

Researchers are conducting an observational, multicenter, prospective study involving adults with severe eosinophilic asthma who are treated with benralizumab. The study aims to evaluate how often patients achieve partial or complete clinical remission, as defined by SANI criteria. Additionally, it seeks to gather more evidence on benralizumabs immunological effects and its role in asthma treatment, while also consolidating long-term safety data. Participants will be treated with benralizumab 30mg administered subcutaneously according to the approved label and local reimbursement rules. The study will observe patients who have started or plan to start benralizumab within 7 days of enrollment. This real-life study does not involve experimental dosing but follows patients receiving benralizumab in routine clinical practice to collect important data over time. During the study, participants will undergo regular assessments for up to 24 months. Researchers will measure clinical remission rates at multiple time points and monitor asthma control using questionnaires like the Asthma Control Test ACT and Asthma Control Questionnaire ACQ. Lung function tests such as FEV1 and FVC will be evaluated before and after bronchodilator use. Medication use and exacerbation rates will also be tracked. The study focuses on understanding benralizumabs effects and safety in a real-world setting over a long period.

This observational study aims to describe the use of teclistamab and talquetamab in treating patients with relapsed or refractory multiple myeloma RRMM outside of clinical trial settings. It collects real-world data to better understand how these treatments are used and their outcomes in typical healthcare environments. The study enrolls participants who have received at least one dose of teclistamab or talquetamab outside clinical trials during specified time periods from before December 2022 through December 2025. No study interventions are administered instead, researchers analyze existing medical records to document treatment details and patient experiences with these therapies. Participants medical records are reviewed to capture baseline characteristics, response rates, time to response, duration of response, survival outcomes, safety management, and treatment use up to 40 months. The study focuses on collecting comprehensive retrospective data without requiring active treatment or additional procedures, providing insight into real-world treatment patterns and outcomes for RRMM patients.

This trial studies adults aged 55 to 90 who have psychosis linked to Alzheimers Disease. It is a Phase 3, 38-week, randomized, double-blind, placebo-controlled outpatient study. Its main goal is to assess how well KarXT capsules prevent relapse of psychosis compared to placebo. Additional goals include evaluating time to treatment discontinuation or relapse, and monitoring safety and tolerability. Participants receive either KarXT capsules at various doses or placebo capsules. The study involves a randomized assignment and is conducted under quadruple masking. The treatment period lasts 38 weeks during which KarXT or placebo is taken three times daily. Assessments continue up to approximately 42 weeks to monitor adverse events and other safety measures. Throughout the study, participants attend outpatient visits for evaluations including cognitive tests, assessments of psychosis severity, caregiver reports, lab tests, vital signs, and safety monitoring. Researchers measure relapse timing, treatment discontinuation, neuropsychiatric symptoms, movement scales, weight, and signs related to heart and urinary health. Safety is closely tracked with various assessments until about week 42.

Researchers are evaluating the safety and effectiveness of KarXT in adults aged 55 to 90 with mild to severe Alzheimers Disease who experience moderate to severe psychosis related to this condition. This Phase 3 trial aims to study KarXT compared to a placebo to better understand its impact on psychotic symptoms associated with Alzheimers. Participants will be randomly assigned to receive either KarXT or a placebo at specified doses on certain days. The study lasts up to 14 weeks, during which changes in psychosis symptoms, as measured by the Neuropsychiatric Inventory-Clinician Hallucinations and Delusions score, will be closely monitored. Additional assessments include cognitive tests and monitoring for side effects. During the trial, participants will undergo regular evaluations including symptom ratings, cognitive tests such as the Mini-Mental State Examination, laboratory tests, and safety monitoring. Researchers will track any adverse events and changes in mental and physical health. The study aims to provide detailed information about how KarXT affects psychosis and cognition in Alzheimers disease over the treatment period.

Researchers are evaluating the efficacy, safety, and how the body processes pharmacokinetics prasinezumab compared with a placebo in people with early-stage Parkinsons disease PD who are on stable levodopa monotherapy. This Phase III study aims to understand if prasinezumab can affect the progression of motor symptoms in this population. Participants will receive either prasinezumab or a placebo as an intravenous IV infusion during the double-blind treatment period. After completing this phase, eligible participants may enter an open-label extension where they can receive prasinezumab. Infusions are given according to a schedule detailed in the study protocol. Throughout the study, participants will be regularly assessed using the Movement Disorder Society - Unified Parkinsons Disease Rating Scale MDS-UPDRS Part III to monitor motor progression, along with other clinical measures and safety evaluations. Researchers will also track adverse events, antibody development against the drug, and drug levels in the blood. The study includes monitoring up to at least 104 weeks, with safety follow-up extending 70 days after the final dose.

Researchers are studying adult patients with generalized myasthenia gravis gMG who are positive for acetylcholine receptor AChR antibodies. The study aims to gather real-world evidence from Italian clinical practice on how efgartigimod alfa affects clinical outcomes and patient-reported experiences in those treated for gMG. This observational study will follow patients over a total duration of up to 23 months. The study involves collecting primary data at a baseline visit and follow-up visits at 6 and 12 months after baseline. Researchers will also gather secondary data from medical charts covering the 12 months before baseline, including details on MG-related therapies, exacerbations, hospitalizations, and the patients medical history. The treatment with efgartigimod alfa is decided independently by the treating physician as part of routine care, not influenced by study participation. Participants will be monitored through clinical assessments and questionnaires measuring changes in MG-Activities of Daily Living MG-ADL scores and the percentage achieving minimal symptom expression MSE up to 12 months. Additional measures include quality of life, fatigue, anxiety and depression, sleep quality, and caregiver burden scores tracked over the same period. The study collects comprehensive data to understand disease experience and treatment impact, with safety and outcomes evaluated throughout the 23-month period.

Researchers are evaluating RO7268489 as an add-on therapy to ocrelizumab in adults with progressive multiple sclerosis PMS. This phase II study aims to assess the safety, pharmacokinetics, pharmacodynamics, and effectiveness of RO7268489 in people with PMS, focusing on its impact on disability progression. Eligible participants have PMS and an Expanded Disability Status Scale EDSS score between 3.0 and 6.0. Participants are randomly assigned to receive one of three doses of RO7268489 or a placebo, all given alongside ocrelizumab following a predefined regimen. After the double-blind treatment phase, eligible participants may join an open-label extension to receive RO7268489 openly. The study uses a quadruple-blind design and compares these groups over approximately 110 weeks. During the study, participants will be regularly monitored for disability progression, brain volume changes, cognitive function, walking ability, hand function, and plasma levels of RO7268489 and its metabolites. Safety is tracked through adverse events and suicidal ideation assessments over up to five years. The study involves scheduled visits for treatment administration, assessments, and monitoring to thoroughly evaluate the impact of adding RO7268489 to ocrelizumab therapy.