Narita

Researchers are evaluating whether retatrutide and tirzepatide can prevent major adverse liver outcomes in adults with metabolic dysfunction-associated steatotic liver disease MASLD who are at high risk based on non-invasive tests. This Phase 3 randomized controlled trial aims to assess these treatments compared to placebo in about 4,500 adults over approximately 224 weeks. The study is sponsored by Eli Lilly and Company and focuses on liver disease progression and related health measures. Participants will be randomly assigned to receive retatrutide, tirzepatide, or placebo, all administered by subcutaneous injection. The trial includes two placebo groups corresponding to each experimental drug. After completing the main study, eligible participants may join a 2-year extension where all will receive either retatrutide or tirzepatide regardless of their initial assignment. During the study, participants may attend around 25 to 30 clinic visits for health monitoring, study procedures, and assessments of liver function and disease status. Researchers will measure the time to major adverse liver outcomes, changes in liver fibrosis scores, liver stiffness, liver fat content, liver enzyme levels, body weight, and cardiovascular events. Monitoring will continue from baseline through study completion, with detailed evaluations at multiple timepoints including week 104.

Researchers are evaluating the drug LP352 in a phase 3, randomized, double-blind, placebo-controlled trial to study its effects on seizures in children and adults with Dravet Syndrome DS. This serious condition involves various seizure types with onset between 1 and 20 months of age. The study aims to test the efficacy, safety, and tolerability of LP352 compared to placebo over a total duration of about 24 months. Participants will be randomly assigned to receive either LP352 or a matching placebo. LP352 or placebo will be given orally or through a feeding tube. The study includes three main phases a Screening phase, a Titration period where doses are gradually increased to the highest tolerated level, and a Maintenance period to assess ongoing treatment effects. Afterward, participants will undergo a Taper period to reduce dosing and a Follow-Up phase for observation. During the study, participants will be monitored for seizure frequency changes, safety, and tolerability. Researchers will track countable motor seizures and measure percent change compared to baseline over up to 15 weeks. Participants or caregivers will complete seizure diaries, and stable antiseizure medication use is required. Safety evaluations will continue up to 21 weeks, with study visits scheduled throughout these phases. Total participation lasts approximately two years.

Researchers are evaluating the combination of baxdrostat and dapagliflozin in people with chronic kidney disease CKD and high blood pressure hypertension. This Phase III, double-blind, placebo-controlled study aims to assess whether this combination reduces the risk of serious kidney damage, heart failure events, or cardiovascular death compared to dapagliflozin alone. The study includes participants with CKD and hypertension who meet specific kidney function and blood pressure criteria. Participants who are not already taking SGLT2 inhibitors will first complete a 4-week dapagliflozin run-in period. Then, they will be randomly assigned to receive either baxdrostat plus dapagliflozin or a placebo plus dapagliflozin. Baxdrostat dosing may start low and be increased if needed. Study visits will occur at 2, 4, 8, 16, 34, and 52 weeks after randomization, and then approximately every four months until the study ends, which is based on the number of key kidney or heart-related events. Throughout the study, participants will have regular assessments including blood tests to monitor kidney function and potassium levels, blood pressure measurements, and evaluations of heart and kidney health. If participants stop the blinded study drug early, they will continue dapagliflozin if possible and remain in the study for ongoing visits and monitoring. The main outcome is whether the combination treatment reduces the risk of a 50% sustained decline in kidney function, kidney failure, heart failure events, or cardiovascular death over up to 37 months.

Researchers are studying the effectiveness of prophylactic treatment with vonicog alfa, a recombinant von Willebrand factor rVWF, in children diagnosed with severe Von Willebrand Disease VWD. This Phase 3, open-label study aims to evaluate how well vonicog alfa works in preventing bleeding episodes in participants who have previously been treated with VWF or plasma-derived VWF products. The focus is on children under 18 years of age with severe VWD who require ongoing replacement therapy to control bleeding. Participants will receive intravenous infusions of vonicog alfa twice weekly for 12 months. The initial dose will range from 40 to 60 international units per kilogram, adjusted by age groups under 6 years, 6 to under 12 years, and 12 to under 18 years. Some participants may also receive ADVATE, another intravenous treatment, as needed to manage breakthrough bleeding episodes or bleeding related to surgery. Treatment is personalized and monitored throughout the study. During the 12-month treatment period, participants will visit the study clinic five times to assess their response and safety. Researchers will monitor the annualized bleeding rate ABR for spontaneous or traumatic bleeding events and record any adverse events. Blood samples will be taken to measure vonicog alfa levels and antibody development. Other assessments include vital signs, laboratory tests, and evaluation of breakthrough bleeding treatment efficacy. The study also collects data on infusion frequency and the amount of vonicog alfa used.

Researchers are evaluating the safety and effectiveness of brenipatide at different dose levels compared with placebo in adults with uncontrolled moderate to severe asthma. This Phase 2 study aims to better understand how brenipatide may affect asthma symptoms and exacerbations over one year of treatment. Participants in this study are adults aged 18 to 75 years with a history of asthma and recent severe exacerbations. Participants will be randomly assigned to receive either one of two doses of brenipatide or a placebo, all administered by subcutaneous injection. The treatment period lasts 52 weeks, during which participants receive their assigned injections and are monitored regularly. The study includes a screening period before treatment and a follow-up period after treatment to assess ongoing safety and effects. During the study, participants will attend visits to complete questionnaires, lung function tests such as forced expiratory volume in one second FEV1, and assessments of asthma control and medication use. Researchers will monitor asthma exacerbation rates, rescue medication use, and the presence of anti-drug antibodies. The total study duration including screening, treatment, and follow-up is approximately 65 weeks.

Researchers are evaluating ibuzatrelvir, an oral medication, to determine its effectiveness and safety in adults and adolescents aged 12 years and older with COVID-19 who are not hospitalized but are at high risk for severe illness. The study is a phase 3, randomized, double-blind trial comparing ibuzatrelvir with a placebo. Participants must have confirmed SARS-CoV-2 infection with symptoms starting within 5 days and meet specific risk factor criteria based on age. Eligible participants will be randomly assigned to receive either ibuzatrelvir or a matching placebo twice daily by mouth for 5 days. The study allows co-administration of standard care treatments available locally. The total study duration is about 6 months, including follow-up. Participants will be monitored for emergency department visits related to COVID-19, hospitalizations, and mortality up to 28 days after starting treatment. Additional evaluations include symptom resolution, occurrence of long COVID symptoms, viral RNA levels, and safety measures such as adverse events through 24 weeks. The study involves regular assessments, including clinical visits and laboratory tests, to track outcomes and safety over time.

Researchers are evaluating the effects of a study medicine called ibuzatrelvir, alone and in combination with remdesivir, for treating symptomatic COVID-19 in adults who are severely immunocompromised. This Phase 3 clinical trial compares ibuzatrelvir with remdesivir to remdesivir alone to assess safety and effectiveness in non-hospitalized or hospitalized patients who do not require supplemental oxygen. Immunocompromised patients often have difficulty fighting infections and may benefit from extended or combination antiviral treatments. Participants are randomly assigned to one of three groups one receiving both ibuzatrelvir taken orally twice daily and intravenous remdesivir, one receiving ibuzatrelvir with a placebo infusion, and one receiving remdesivir with a placebo pill. Placebos that look like the study medicines are used to keep the groups similar in appearance. This design helps researchers determine if adding ibuzatrelvir improves treatment compared to remdesivir alone. Participants will attend about 10 study visits over 24 weeks, including clinic visits for blood tests, nasal swabs collected both at the clinic and at home, and questionnaires. Researchers will measure outcomes such as symptom improvement, viral levels, COVID-19-related healthcare visits, and safety events. The study carefully monitors participants throughout to understand the treatment effects and safety over time.

Researchers are evaluating the safety and effectiveness of different drug treatments in people with myasthenia gravis, a condition that affects muscle strength. This platform study uses a single master protocol to test multiple treatment regimens in separate groups called intervention-specific appendixes ISAs. The goal is to find the best treatments that reduce side effects and improve quality of life for patients with this disease. The study includes various treatment groups, such as those receiving intravenous infusions of efgartigimod, empasiprubart, or placebo, and subcutaneous administration of efgartigimod PH20 via pre-filled syringe. Each ISA has its own screening, treatment, and safety follow-up periods, which vary in length and design. Two ISAs are included one evaluating empasiprubart as add-on therapy to efgartigimod in people with partial responses, and another assessing empasiprubart monotherapy. Participants are involved in screening and treatment phases, with assessments of safety, tolerability, and effectiveness lasting up to about seven years depending on the ISA. Researchers will monitor clinical symptoms, side effects, and quality of life during and after treatment. The study aims to gather detailed information on how well these drug regimens work and their impact on patients living with myasthenia gravis.

This research aims to evaluate the safety and therapeutic importance of empasiprubart as an add-on treatment to efgartigimod in adults with AChR-Ab seropositive generalized myasthenia gravis who have a partial clinical response to efgartigimod. It is part of the ADAPT Forward platform study, which looks at the safety and effectiveness of different drugs to find the best ways to reduce side effects and improve quality of life for people with myasthenia gravis. Participants first complete screening and then enter a run-in period part A receiving efgartigimod intravenously. Those eligible move on to the add-on period part B, receiving both efgartigimod and empasiprubart intravenously. Participants not eligible for part B continue to a safety follow-up period part C where they receive efgartigimod only. The total study duration is approximately 54 weeks per participant. Throughout the study, participants undergo assessments including monitoring for adverse events, measuring changes in MG-ADL and QMG scores, and evaluating clinical responses. Researchers track safety and efficacy up to 21 weeks during parts A and B. Participants have scheduled visits for treatment and safety evaluations, with ongoing observation through the safety follow-up period to monitor treatment effects and side effects.

Researchers are evaluating the effect of muvalaplin in lowering cardiovascular risks among adults with elevated lipoproteina who either have atherosclerotic cardiovascular disease or are at risk of a first heart attack or stroke. This phase 3, randomized, double-blind study aims to investigate whether muvalaplin can reduce major adverse cardiovascular events compared to placebo in this high-risk population. Participants are randomly assigned to receive either muvalaplin or a placebo, both given orally. The study is designed with parallel groups and will last about 5.25 years, during which the occurrence of cardiovascular events and changes in lipoproteina levels will be closely monitored. Throughout the study, participants will undergo regular assessments including measurement of lipoproteina levels, monitoring of cardiovascular events such as heart attacks or strokes, and evaluation of healthcare resource use. The primary outcome is the time to first major adverse cardiac event, tracked from baseline until the study ends. Safety and pharmacokinetics of muvalaplin will also be evaluated during the trial period.