Swidnik

Researchers are evaluating the long-term safety and effects of nerandomilast in people with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF) who have previously completed treatment with nerandomilast in earlier studies. The study aims to understand how well participants tolerate nerandomilast over time, and whether it helps improve lung function, delays symptom worsening, reduces hospital visits, or impacts survival. This is a Phase 3 open-label extension trial. Participants take nerandomilast tablets daily for up to 1 year and 10 months while continuing their usual pulmonary fibrosis treatments. The study follows an open-label design where all participants receive nerandomilast. There are no placebo or comparator groups in this extension phase. Throughout the study, participants regularly visit their doctors for health assessments and lung function tests. Doctors monitor any health problems or side effects experienced during treatment. The main outcome measured is whether participants experience any adverse events up to the final follow-up visit, which occurs at week 99. This close monitoring helps evaluate the long-term safety and potential benefits of nerandomilast in this patient group.

Researchers are evaluating the effectiveness and safety of BHV-7000 in adults with refractory focal onset epilepsy, a condition where seizures originate in one area of the brain and do not respond well to current treatments. This Phase 2/3 clinical trial aims to determine whether BHV-7000 can reduce seizure frequency in this population. The study is divided into two parts. In Part A, participants are randomly assigned to receive either 25 mg or 50 mg of BHV-7000, or a matching placebo, taken once daily. After completing Part A, participants move to Part B, where they are randomized to receive 75 mg of BHV-7000 or a matching placebo, also taken once daily. Both parts are randomized and double-blinded to ensure unbiased results. Participants will be monitored from Week 8 to Week 20 of each part for changes in average seizure frequency, serious adverse events, discontinuations due to side effects, and laboratory abnormalities. Researchers will track seizure diaries and assess safety and tolerability throughout the study. The total duration includes both study parts with regular evaluations to measure the drug’s impact and participant safety.

Researchers are evaluating the effectiveness and safety of a single inhaled dose of Staccato alprazolam compared to a placebo in quickly stopping prolonged seizure episodes in people aged 12 years and older with stereotypical prolonged seizures. This Phase 3 study aims to determine if the treatment can stop seizures within 90 seconds and prevent seizure recurrence for up to 2 hours after administration. Participants will receive one inhaled dose of either Staccato alprazolam or a placebo during the treatment period. The study is randomized, double-blind, and placebo-controlled, conducted at multiple outpatient centers. The intervention consists of a single administration designed to rapidly terminate seizure episodes. During the study, participants are closely monitored for seizure treatment success within 90 seconds and no seizure recurrence up to 2 hours post-treatment. Researchers will assess seizure control and safety outcomes throughout the treatment period. The study involves baseline assessments, caregiver involvement to observe seizures, and a focus on quick treatment response and safety over several hours following drug administration.

Researchers are evaluating the effectiveness and safety of the investigational drug omecamtiv mecarbil in patients with symptomatic heart failure and severely reduced ejection fraction. This study aims to see if the drug can lower the risk of serious heart-related events, such as cardiovascular death, heart failure events, need for heart device implantation, heart transplantation, or stroke. The study is a Phase 3, multi-center, double-blind, randomized, placebo-controlled trial involving adults aged 18 to 85 years. Participants will be randomly assigned to receive either omecamtiv mecarbil or a placebo, both given as oral tablets. The study is event-driven and will continue until at least 850 participants have experienced either a heart failure event or cardiovascular death. An interim analysis will be done after about 570 such events have occurred. The estimated participation period for each patient is up to 3 years. During the study, participants will be monitored for the time until the first occurrence of cardiovascular death or heart failure event. Researchers will collect various health data and monitor safety throughout the study. The study includes regular assessments to track heart function and adverse events, with follow-up until the study concludes when the required number of events is reached.

Researchers are evaluating whether the drug zilebesiran can reduce the risk of major cardiovascular events such as cardiovascular death, nonfatal heart attacks, strokes, or heart failure in adults who have hypertension that is not well controlled and who either have established cardiovascular disease or are at high risk for it. This Phase 3 global study is designed to continue until enough cardiovascular events have occurred to assess the treatment's effect. Participants will be randomly assigned to receive either zilebesiran or a placebo, both given as injections under the skin (subcutaneous administration). All participants will continue with their standard care, which includes treatment with at least two antihypertensive medications, one of which must be a diuretic such as a thiazide or loop diuretic. The study is double-blind, so neither participants nor researchers know who is receiving the active drug or placebo. During the study, participants will be closely monitored for cardiovascular events including heart attacks, strokes, heart failure hospitalizations, and cardiovascular deaths over approximately five years. Researchers will collect data on these events to determine the time until the first occurrence of any of these outcomes. Safety assessments and standard clinical evaluations will also be performed throughout the study period to ensure participant well-being.