Boise

Researchers are evaluating remibrutinib (LOU064) in adolescents aged 12 to under 18 years who have chronic spontaneous urticaria (CSU) that is not well controlled by H1-antihistamines. This Phase 3 trial aims to assess the effectiveness, how the drug is processed in the body, and safety of remibrutinib compared to a placebo. The study also intends to gather long-term data on how well remibrutinib works and its safety over several years after treatment ends. The trial includes three periods. First, the core period is a 24-week double-blind phase where about two-thirds of participants receive remibrutinib and one-third receive placebo, with about 10 site visits over approximately 32 weeks. Next is an optional open-label extension lasting from one to three years, where participants who completed the core period may receive remibrutinib or enter an observational treatment-free phase depending on their symptoms. Participants may cycle through treatment and observational periods up to six times. Finally, an optional long-term treatment-free follow-up can last up to three years with one site visit and up to four phone calls. During the study, participants undergo assessments including changes in urticaria activity scores (UAS7), itching severity (ISS7), and hive severity (HSS7) measured from baseline to 12 weeks. Regular visits monitor safety, symptoms, and drug effects. The study tracks these measures to understand remibrutinib's impact on CSU symptoms and overall safety profile during and after treatment, with total participation potentially lasting several years.

Researchers are evaluating molnupiravir, a study medicine designed to stop the COVID-19 virus from multiplying, to see if it can prevent severe illness from COVID-19 more effectively than a placebo. This Phase 3 randomized, placebo-controlled, double-blind study focuses on non-hospitalized adults at high risk of severe disease progression due to COVID-19. The study addresses the need for alternative treatments for people who cannot take certain COVID-19 medications due to availability or potential drug interactions. Participants will receive either molnupiravir or a placebo, both given orally as two 400 mg film-coated tablets every 12 hours for 5 days, totaling 10 doses. Some participants may also receive remdesivir as part of standard care if clinically appropriate and available. The study compares the effects of molnupiravir with placebo in preventing severe illness outcomes. Throughout the study, participants will be monitored for outcomes such as hospitalization, death, or medically attended visits related to COVID-19 up to 29 days. Safety is assessed by tracking adverse events for up to about 5 months and discontinuation of study treatment due to adverse events for about 5 days. The study involves laboratory tests, symptom assessments, and safety evaluations to understand molnupiravir's impact on disease progression and participant health.

Researchers are evaluating the safety and effectiveness of rilvegostomig compared to pembrolizumab, both combined with platinum-based doublet chemotherapy, as initial treatments for patients with metastatic non-squamous non-small cell lung cancer (mNSCLC) whose tumors express PD-L1. This Phase III, randomized, double-blind, global study focuses on patients whose tumors meet the PD-L1 expression threshold of 1% or higher and do not have certain genetic mutations or rearrangements that would require other targeted therapies. Participants receive either rilvegostomig or pembrolizumab intravenously on the first day of each 21-day treatment cycle. Both groups also receive platinum-based chemotherapy drugs such as carboplatin or cisplatin, administered intravenously up to four cycles, along with pemetrexed given intravenously on Day 1 of each cycle. The study monitors these treatments as first-line therapy for metastatic non-squamous NSCLC. During the study, participants undergo regular assessments including imaging scans to measure tumor size and response, as well as evaluations of organ and bone marrow function. Researchers track overall survival and progression-free survival for up to approximately five years. Safety is closely monitored throughout, and patients are followed long-term to assess outcomes related to treatment effectiveness and tolerability.

This is a Phase III, two-arm, randomized, double-blind, global, multicenter study assessing the efficacy and safety of rilvegostomig compared to pembrolizumab, both in combination with platinum-based doublet chemotherapy, as a first-line (1L) treatment for patients with squamous metastatic non-small cell lung cancer (mNSCLC) whose tumors express PD-L1 (tumor cells (TC) ≥ 1%).

Researchers are evaluating the safety and effectiveness of pulsed field ablation (PFA) therapy for treating persistent atrial fibrillation (PersAF) that does not respond to medication. The study compares PFA targeting pulmonary veins plus extra-PV sources identified by electrographic flow (EGF) mapping against PFA targeting pulmonary veins plus the left atrial posterior wall (PVI + PWA). The goal is to establish safety and to show that the new method is not less effective than the current approach in patients with symptomatic, drug-refractory PersAF. Participants will receive treatment using several devices, including the FARAPOINT Pulsed Field Ablation System, the OptiMap System for electrogram analysis, and the FARAWAVE NAV Catheter combined with the Opal HDx Mapping System for detailed heart mapping. All subjects will undergo electroanatomical mapping of the entire left atrium, followed by pulsed field ablation of the pulmonary veins, with additional ablation of either EGF-identified sources or the posterior wall, depending on the assigned treatment group. During the study, participants will be monitored for safety outcomes at 60 days and effectiveness outcomes at 365 days. They will receive a LUX-Dx insertable cardiac monitor to track heart rhythm continuously. Researchers will collect clinical data, imaging, and follow-up assessments to evaluate treatment impact and safety. The study includes ongoing follow-up visits to ensure thorough monitoring of heart function and study outcomes over one year.

Researchers are evaluating RAP-219, an investigational drug, in adults with refractory focal epilepsy to study its long-term safety and antiseizure effects. This phase 2, multi-center, open-label study focuses on adults aged 18 to 70 years with refractory focal seizures who have completed a prior parent study treatment period. The trial aims to understand RAP-219's tolerability, pharmacokinetics, pharmacodynamics, and antiseizure activity over an extended period. Participants will start with a daily RAP-219 capsule of 0.125 mg for 3 days, followed by a 0.25 mg tablet daily for 28 days, then increase to a 0.75 mg tablet daily for the rest of the treatment period. The study does not mention a comparator group, focusing on continuous RAP-219 treatment and monitoring. The treatment period is long-term, with ongoing dosing and assessment. During the study, participants will be regularly assessed for safety, tolerability, and seizure activity. Researchers will monitor for any treatment-emergent adverse events from the start of RAP-219 through eight weeks after the last dose, up to week 112. Participant health, compliance with device data upload, and overall seizure control will be closely observed to evaluate the drug's long-term effects and safety profile.

Researchers are investigating the effects of multiple doses of vosoritide and comparing its therapeutic dose to human growth hormone (hGH) in children with idiopathic short stature (ISS). This Phase 2 study aims to understand how these treatments influence growth in this population. After an initial observation period of at least 6 months to measure baseline growth, participants are randomly assigned to receive either vosoritide, placebo, or hGH (the latter only in the United States). Those in the vosoritide and placebo groups undergo up to 6 months of randomized treatment, followed by open-label vosoritide until they reach near-final adult height or at least age 16 for females or 18 for males. Participants assigned to hGH receive open-label treatment for a minimum of 4 years. Throughout the study, safety is carefully monitored with clinical and imaging assessments focused on hips and lower extremities, as well as watching for hypotension, fractures, and slipped capital femoral epiphysis. An independent Data Monitoring Committee reviews safety data regularly. Study visits include a treatment completion visit about 4 weeks after the last dose, and follow-up assessments may continue annually through the end of the study. Key outcome measures include changes in annualized growth velocity at 6 months and changes in height and height Z-score after 4 years.

Researchers are evaluating the safety, tolerability, and immune response duration of a second dose of the RSVpreF vaccine given during later pregnancies. The study also examines how long immunity lasts from a single dose given during a previous pregnancy by analyzing blood samples from nonpregnant participants who had the vaccine before. This is a Phase 3 trial focused on pregnant women and their babies as well as nonpregnant women previously vaccinated. Pregnant participants are grouped into Cohort 1, who previously received RSVpreF in a Pfizer trial and will get a second dose, and Cohort 2, who received RSVpreF previously via commercial or trial means and will be randomly given either RSVpreF or placebo. Both groups will be monitored for safety and immune response. Cohort 3 includes nonpregnant women who had RSVpreF before and will provide blood samples to check how long protection lasts, without receiving further vaccination. Participants will undergo safety monitoring during pregnancy and after birth. Infants will be followed for six months to assess safety and antibody levels. Blood tests will measure immune response, including neutralizing antibodies at birth. The study tracks local and systemic reactions, adverse events, serious adverse events, and new medical conditions in both pregnant participants and their infants over various timeframes throughout the study.

Researchers are investigating the safety, tolerability, and effectiveness of two dosing regimens of itepekimab compared to placebo as an add-on to intranasal corticosteroids in adults with chronic rhinosinusitis with nasal polyps (CRSwNP) that is not well controlled. This multinational Phase 3, randomized, double-blind, placebo-controlled trial involves male and female participants aged 18 years and older living with CRSwNP. Participants are randomly assigned to one of three groups receiving either itepekimab injections or placebo injections, both administered subcutaneously, alongside mometasone furoate nasal spray delivered intranasally. The study includes a 4-week screening period, followed by a 52-week treatment phase, and a 20-week safety follow-up, totaling up to 76 weeks. Participants transitioning to an extension study (LTS18420) will have a total duration of 56 weeks. Study visits include nine site visits and 20 phone or home visits. During the trial, participants will undergo assessments including endoscopic Nasal Polyp Scores (NPS) and Nasal Congestion Scores (NCS) measured from baseline to week 24 to evaluate changes. Researchers will monitor safety and tolerability throughout, with regular evaluations involving symptom severity, treatment adherence, and adverse events. The study aims to understand how well itepekimab works and is tolerated as an additional treatment for CRSwNP over the study duration.

Researchers are evaluating the effectiveness of pemigatinib in adults with advanced or metastatic pancreatic cancer that has spread to nearby tissues, lymph nodes, or distant body parts, and that have specific genetic changes in the FGFR gene. The study focuses on patients whose cancer has FGFR2 gene fusions or other FGFR alterations, aiming to see if pemigatinib can block these abnormal gene functions to stop tumor growth and possibly improve quality of life. This is a phase II trial conducted nationwide using a fully decentralized telemedicine approach to reach participants. Participants receive pemigatinib as an oral medication once daily for 14 days within each 21-day cycle. Treatment continues unless the disease progresses or unacceptable side effects occur. Alongside the drug treatment, patients undergo various imaging tests including CT scans, MRI, optical coherence tomography (OCT), and when needed, whole body bone scans and dilated eye exams (ophthalmoscopy). After finishing treatment, patients are followed up at 30 days and then every four months for one year to monitor their condition. Throughout the study, patients provide blood samples and undergo scans to evaluate treatment response and detect resistance mutations. Researchers track the overall response rate for up to 24 months and assess safety and tolerability. Patients must comply with scheduled visits, tests, and oral medication intake. The total study participation includes treatment cycles and a follow-up period lasting up to approximately 16 months after treatment completion.