New Plymouth

Researchers are evaluating the effects of a medicine called BI 764198 in adults and adolescents who have a kidney condition known as focal segmental glomerulosclerosis FSGS. This Phase 3 clinical trial aims to determine whether BI 764198 helps improve kidney function in people with primary FSGS or genetic FSGS linked to TRPC6 gene variants. The study is randomized and placebo-controlled, meaning participants are randomly assigned to receive either the medicine or a placebo, and neither the participants nor the researchers know which treatment each person receives. Participants take either BI 764198 tablets or placebo tablets once a day for up to two years, while continuing their usual medication for FSGS. The study involves two groups running in parallel. The main treatment period lasts 104 weeks about two years, during which the participants regularly visit the study site approximately every three months. Both groups are compared to see if BI 764198 affects kidney protein levels and function. During the study, participants provide urine samples regularly to assess their kidney health. Researchers measure changes in urine protein-creatinine ratio and kidney filtration rate from the start to the end of the treatment period. Questionnaires about health-related quality of life are also completed. Doctors monitor participants health and note any side effects throughout the two years. This thorough follow-up helps understand how BI 764198 impacts kidney disease and overall well-being.

Researchers are evaluating the effects of a medicine called BI 690517 combined with empagliflozin in adults with chronic kidney disease CKD who are at risk of their kidney condition getting worse. The study includes people with or without type 2 diabetes and those who may already be taking medicines like angiotensin converting enzyme inhibitors ACEi, angiotensin receptor blockers ARB, or sodium-glucose cotransporter-2 inhibitors SGLT2i. The goal is to understand if adding BI 690517 can help delay worsening kidney function, hospitalizations due to heart failure, or cardiovascular death. After a run-in period where all participants take empagliflozin and other standard medications, participants are randomly assigned to receive either BI 690517 tablets or placebo tablets once daily alongside empagliflozin. The run-in period confirms that participants are stabilized on empagliflozin before randomization. The treatment phase continues for about three to four years until enough kidney or heart-related events have occurred to compare outcomes between the two groups. During the study, participants visit the study site about five times in the first six months and then every six months thereafter. At these visits, health is regularly checked through blood and urine tests, blood pressure and weight measurements, kidney function monitoring, and collection of any side effect information. The main outcome measured is the time until the first occurrence of kidney disease progression, hospitalization for heart failure, or cardiovascular death.

Researchers are evaluating whether metformin, a medication commonly used for diabetes, can be repurposed to slow kidney function decline in adults diagnosed with early-stage Autosomal Dominant Polycystic Kidney Disease ADPKD. ADPKD causes cyst growth in kidneys leading to pain, high blood pressure, chronic kidney disease, and reduced quality of life. This global Phase III randomized controlled trial aims to find effective, affordable treatments to reduce illness impact and improve life quality for ADPKD patients. Participants are randomly assigned to receive either extended-release metformin Metformin XR or a placebo, alongside standard care, for 104 weeks. The metformin dose ranges from 500 to 2000 mg daily based on individual tolerance and kidney function. The study includes a control group receiving identical placebo tablets for comparison. This design allows assessment of metformins effect on slowing disease progression. During the study, participants undergo regular monitoring including kidney function tests, assessment of kidney disease progression, quality of life questionnaires, and tracking of side effects and healthcare use over 24 months. The primary outcome is the change in estimated glomerular filtration rate eGFR. Secondary outcomes include kidney failure, mortality, medication dosing changes, and symptoms related to ADPKD. Safety and effectiveness are carefully followed throughout the trial period, which may last until 2030.

Researchers are evaluating new treatments for acute hypoxemic respiratory failure AHRF, a serious condition that affects millions worldwide each year and carries high risks of death and long-term health problems. This platform trial, called PRACTICAL, uses adaptive methods to test various interventions at different stages, including mechanical ventilation approaches, extracorporeal life support, drugs, and medical devices. The study aims to find better strategies to protect lungs and diaphragm function during ventilation and improve patient outcomes. The trial includes multiple domains studying different treatments or strategies. These include ultra-protective ventilation using extracorporeal carbon dioxide removal, various invasive mechanical ventilation strategies, corticosteroid use in early and extended phases, nebulized furosemide, positive end-expiratory pressure settings during extracorporeal support, fludrocortisone for airspace disease, and inspiratory muscle training. Some domains are observational, collecting data and biomarkers in immunocompromised patients or examining patient-ventilator interactions. Participants will be randomized to different treatment arms or observed, depending on the domain. Participants will undergo assessments including physiological and biological measurements, mechanical ventilation monitoring, and evaluation of clinical outcomes such as mortality, ventilator-free days, organ failure, and long-term cognitive and physical function. Data will be collected daily or at specified time points up to 180 days. The study uses Bayesian adaptive analysis to efficiently evaluate efficacy and safety of interventions. The total participation time varies by domain but includes active treatment periods, follow-up, and feasibility assessments over months to years.

Researchers are evaluating treatments for community-acquired pneumonia CAP, especially in patients admitted to intensive care units ICUs. This trial also adapts to study treatments for respiratory pandemics like COVID-19. The goal is to determine which treatment strategies improve outcomes for patients with severe pneumonia, using a flexible, ongoing approach that can test multiple therapies simultaneously and update as new information becomes available. Participants receive different treatment strategies based on random assignment to study groups. Treatments include various antibiotics, steroids, antivirals, immune modulators, anticoagulation methods, and ventilation strategies. Some treatment options have been closed to recruitment, reflecting the trials adaptive nature. The trial includes several domains targeting specific infections such as influenza and COVID-19, with dosing and duration guided by clinical practice and local guidelines. During the study, participants are monitored closely with assessments including survival up to 90 days, days alive without organ support in ICU, ICU and hospital length of stay, ventilator-free days, organ failure-free days, and quality of life up to 6 months. Researchers collect detailed data on patient outcomes, organ support needs, and hospital discharge status. The study runs until February 2028, with ongoing evaluation to improve pneumonia treatment strategies in ICU settings and during respiratory pandemics.

This research aims to find out whether an early three-day oral steroid treatment called dexamethasone can improve both physical and mental recovery in children with Sydenhams chorea. Sydenhams chorea is a movement disorder caused by brain inflammation after an abnormal immune response to certain bacterial infections, affecting childrens movements, mood, and concentration. The condition commonly affects children with acute rheumatic fever, and recovery can take months. The study is a phase 3, randomized, double-blinded, placebo-controlled trial involving children in New Zealand and Australia. Participants will be randomly assigned to receive either oral dexamethasone or a placebo for three days. The dexamethasone dose is 20 mg per square meter of body surface per day, divided into three doses, with a maximum of 24 mg per day. The placebo group will take matching capsules on the same schedule. This short course treatment is being tested to see if it reduces the severity of Sydenhams chorea and its psychiatric symptoms. Children in the study will be monitored through scheduled visits on days three, seven, one month, three months, and twelve months. Researchers will assess chorea severity, relapse rates, psychiatric symptoms, hospital stay length, and treatment failure. Safety will be checked for any side effects possibly related to dexamethasone. The trial plans to recruit 80 participants, and overall involvement may last up to 12 months to track recovery and treatment effects comprehensively.