Bayrut

Researchers are evaluating the long-term safety and effectiveness of etavopivat, a new oral medicine being developed to treat inherited blood disorders such as sickle cell disease and thalassemia. These disorders affect hemoglobin, the protein responsible for carrying oxygen in the body. This phase 3 study aims to monitor how well etavopivat works and its safety profile over an extended period. Participants will receive one of three forms of etavopivat (A, B, or C) as oral doses. The study is open-label and multicenter, involving adults, adolescents, and children who have previously completed treatment in an etavopivat parent study and continue to benefit clinically. The treatment period can last up to 264 weeks but may end earlier if etavopivat is approved in the participant's country. During the study, researchers will track the number of treatment-emergent adverse events and adverse reactions for each participant by indication and age group from baseline through the end of the study, which can last up to 316 weeks. Participants' safety and response to long-term treatment will be closely monitored throughout this period.

Researchers are evaluating how well etavopivat works to reduce the number of vaso-occlusive crises (painful blood vessel blockages) in adolescents and adults living with sickle cell disease. The study also aims to assess if etavopivat can decrease organ damage, improve exercise tolerance, and reduce fatigue. This is a global Phase 3 study involving participants aged 12 years and older with confirmed sickle cell disease. The study is randomized, double-blind, and placebo-controlled to ensure accurate evaluation of the treatment effects. Participants will receive either etavopivat or a matching placebo by mouth. Which treatment they receive is determined randomly. The study will last about two years, during which participants will take the assigned medication and be monitored closely. Etavopivat is an investigational drug currently under evaluation in multiple studies for sickle cell disease. During the study, participants will have regular assessments including documentation of vaso-occlusive crisis events, blood tests, and physical evaluations. Researchers will track the number of crises that require medical attention over a 52-week period, as well as measures of organ health, exercise ability, and fatigue. Safety and overall health will be monitored throughout the study, with the total participation time lasting approximately two years.

Researchers are evaluating etavopivat, a once-daily oral medicine, in children and adolescents with sickle cell disease. This phase 1/2 study aims to understand the safety of etavopivat and how it behaves in the bloodstream, while also exploring potential benefits for patients. The study focuses on pediatric patients aged from 6 months to under 18 years with confirmed sickle cell disease and severe symptoms. Participants will receive etavopivat tablets by mouth once daily for a continuous 96-week treatment period. After completing treatment, there will be a final study visit four weeks later to assess any lasting effects. The study includes monitoring drug levels in the blood at various points to measure how etavopivat is processed by the body. During the study, participants will have regular assessments to monitor safety and treatment effects, including lab tests to measure drug concentration, and tracking of any side effects or adverse events. Researchers will observe the number of dose changes, interruptions, and early discontinuations throughout the 24-week primary period and beyond. The total study duration includes the 96-week treatment and a 4-week follow-up, with comprehensive monitoring of health status and medication impact.

Researchers are evaluating the success of endotracheal intubation using the GlideScope video laryngoscope performed by either two operators or a single operator. This study aims to determine if using two operators affects the first attempt success rate of intubation. It also examines other factors such as the time taken to intubate, the need for adjustments during the procedure, the impact of difficult intubation predictors, and the occurrence of complications like low oxygen levels, airway injury, and post-operative sore throat. Participants will be assigned to one of two groups over a 9-month period. One group will undergo intubation performed by a single operator using the GlideScope and an angulated stylet. The other group will have a two-operator technique where a senior resident inserts the GlideScope until an optimal view is achieved, then an attending physician holds the device in place while the resident completes the intubation. If intubation fails, alternative methods will be used as decided by the attending anesthesiologist. During the study, researchers will record the first attempt success rate of intubation and measure the time taken to complete the procedure. They will monitor for oxygen desaturation below 90% and airway trauma. Additional assessments will track the severity of sore throat after surgery. The study involves adult patients requiring orotracheal intubation with a single-lumen tube under general anesthesia, with close observation throughout the procedure and data collection by both residents and attending physicians.

WAYFIND-R is a global registry focused on collecting high-quality real-world data from cancer patients diagnosed with solid tumors who have undergone next-generation sequencing (NGS) testing. The registry aims to support clinical and epidemiological research, generate evidence to better understand health outcomes and cancer care, and describe treatments and clinical courses for these patients. Participants must be adults diagnosed with any type of solid tumor at any disease stage and have had NGS testing within three months before enrollment. The study collects data without assigning specific treatments or interventions, instead tracking clinical characteristics and outcomes over time. During the study, researchers will gather information linking NGS results to treatments and patient outcomes, including overall survival for up to five years from enrollment. Participants provide informed consent, and data collected will help improve understanding of solid tumor cancers and their management in real-world settings.

The trial investigates anxiety and fear in children aged 5 to 7 years undergoing local anesthesia for dental treatment. It compares two behavior guidance methods to reduce anxiety: the traditional Tell-Show-Do technique and a new approach using an artificial intelligence (AI)-driven avatar-based video called "Mini Dentist." The goal is to assess whether the AI video improves children's cooperation and lowers anxiety compared to the conventional method. Children eligible for the study will be randomly assigned to one of two groups. One group will experience the standard Tell-Show-Do method, where the pediatric dentist explains and demonstrates the procedure. The other group will watch a short animated AI avatar video that explains the injection process in a child-friendly way before local anesthesia is administered. All injections will be performed by the same dentist following a consistent protocol. During the single visit, researchers will monitor heart rate changes to measure anxiety, observe behavior using the FLACC scale, and collect self-reported pain scores with the Wong-Baker FACES scale. Behavioral responses will also be video recorded for evaluation. Participation is voluntary with informed consent from parents or guardians, and the study follows ethical research standards.

Researchers are evaluating the long-term safety of asciminib treatment in patients who have completed a previous Novartis-sponsored asciminib study and are considered by their doctors to benefit from continued treatment. This open-label, multi-center, global roll-over study focuses on patients with Philadelphia chromosome-positive Chronic Myelogenous Leukemia (CML) or Acute Lymphoblastic Leukemia (ALL) who need ongoing therapy but cannot access it outside the study. The study is part of a phase 4 trial to monitor safety during extended treatment. Participants receive asciminib either as a single agent or in combination with drugs such as imatinib, nilotinib, bosutinib, or dasatinib. Asciminib is taken orally twice daily or once daily in fasting conditions, with a special pediatric formulation dosed by body weight. Other drugs are taken once or twice daily with specified meal conditions. The study continues treatment similarly to the participants' parent study but offers access when treatment is otherwise unavailable. During the study, participants are monitored for adverse events over an eight-year period. Researchers track safety outcomes and participants' compliance with treatment plans and scheduled visits. The study includes various assessments to ensure ongoing treatment benefits and to observe any side effects. Participation involves long-term follow-up to evaluate the safety of continued asciminib use in this patient population.

This clinical trial investigates whether fiberoptic intubation causes less change in blood pressure and heart rate compared to direct oral intubation when opioid free anesthesia is used. The study focuses on adults aged 18 to 55 years who are scheduled for elective surgery under general anesthesia. It aims to assess hemodynamic stability and also compares the time needed for intubation along with the rates of postoperative sore throat, hoarseness, and upper airway injury. Participants will be randomly assigned to one of two groups: one undergoing direct conventional laryngoscopy using a Macintosh laryngoscope blade (size N3 for females and N4 for males), and the other undergoing fiberoptic guided intubation. Both groups will receive opioid free anesthesia induction. The study plans to recruit 90 adults, with 45 in each group, over one year. During the study, researchers will measure heart rate and blood pressure from baseline up to 5 minutes after intubation. They will also record the time taken to complete the intubation and monitor for postoperative symptoms such as sore throat, hoarseness, and signs of airway trauma. The trial focuses on ensuring patient safety and understanding the effects of these two intubation methods on hemodynamic stability in adults with normal airways.

Researchers are studying how electrical resistance, called impedance, affects nerve blocks used in local anesthesia, especially when guided by nerve stimulation or ultrasound. This research aims to measure impedance in different tissue types such as skin, fat, fascia, muscle, and nerve during an axillary peripheral nerve block. Understanding impedance can help improve the accuracy of needle placement, potentially increasing the success rate of nerve blocks and reducing complications like injections into nerves or blood vessels. The study focuses on ultrasound-guided nerve blocks including axillary, interscalene, and popliteal sciatic nerve blocks. Participants will undergo nerve block procedures where impedance measurements are taken across tissues to evaluate how impedance varies. This observational study does not involve experimental treatments but collects data during routine nerve block procedures. Participants will be monitored to assess impedance over a period of 3 months. Researchers will evaluate the electrical resistance across different tissues during the nerve block to understand its impact. The study requires patients to communicate effectively and have no contraindications or allergies to the drugs used. Safety and precision in needle placement are key outcomes being measured to improve nerve block procedures.

Benign prostatic hyperplasia (BPH) is a common condition affecting men, especially as they age, with up to 90% of men experiencing it by age 80. This research aims to create an ongoing international registry to collect and analyze demographic and clinical data from men with BPH who receive either medical therapy or surgical treatments. The registry helps track treatment patterns and outcomes worldwide to better understand the effectiveness and complications related to various BPH treatments. The registry collects detailed baseline information including patient-reported symptoms, sexual health, quality of life, urinary flow, and laboratory values such as prostate-specific antigen and testosterone. It also records any complications like bleeding, infections, incontinence, strictures, ejaculation issues, and erectile dysfunction. This data is gathered over a three-year period with no set endpoint, allowing for long-term follow-up and analysis of real-world treatment results. Participants provide medical records which are securely stored and accessed only by authorized users. The study monitors symptoms using standardized scores and quality of life measures, along with clinical tests such as post-void residual urine volume. Regular audits ensure data accuracy, and the registry’s technology supports future integration with patient portals and electronic medical records. The study duration is planned for at least three years, with possible extensions to continue follow-up and research.