Al Ahsa

Researchers are evaluating the efficacy and safety of luspatercept combined with best supportive care compared to placebo with best supportive care in adults with alpha-thalassemia hemoglobin H disease who experience anemia. The study also assesses the safety and drug levels of luspatercept in adolescents with this condition. This phase 2 trial aims to understand how luspatercept affects anemia and transfusion needs in this population. Participants are randomly assigned to one of four groups based on their transfusion dependence status adults who receive luspatercept plus best supportive care, adults who receive placebo plus best supportive care, adolescents who are transfusion dependent receiving luspatercept plus best supportive care, or adolescents who are non-transfusion dependent receiving luspatercept plus best supportive care. The treatments are given on specified days, and participants are monitored for up to 108 weeks, including assessments of drug levels, transfusion burden, hemoglobin changes, and adverse events. Throughout the study, participants undergo regular evaluations including blood tests to measure hemoglobin and transfusion needs, monitoring for side effects, and quality of life assessments. Researchers also track long-term safety outcomes and pharmacokinetics of the drug. The main outcomes include reductions in red blood cell transfusions over 12 to 48 weeks and increases in hemoglobin levels without transfusions. Participants may be followed for up to five years to monitor safety and treatment effects.

Researchers are evaluating the combined use of vicadrostat and empagliflozin in adults with chronic heart failure who have a reduced left ventricular ejection fraction LVEF below 40%. Participants must have had chronic heart failure diagnosed at least three months before starting the study. The trial aims to find out if this combination helps people with symptomatic heart failure classified as New York Heart Association classes II to IV. Participants are randomly assigned to one of two groups, with an equal chance of receiving either vicadrostat plus empagliflozin tablets or placebo plus empagliflozin tablets. The study medicines are taken once daily for approximately six months up to about 3.5 years. During this time, participants may continue their usual heart failure treatments, excluding certain medications. The trial includes a double-blind design, meaning neither participants nor study staff know who receives the active drug or placebo. Throughout the study, participants visit the study site regularly, with the number of visits depending on how long they stay enrolled. Some visits may occur by phone. They answer questions about their well-being, and doctors monitor health status, record any heart failure worsening, hospitalizations, or deaths. The main outcome is the time until cardiovascular death, heart failure hospitalization, or urgent heart failure visit, which is compared between groups. Safety and side effects are also closely followed during the trial.