Carshalton

Researchers are evaluating DMX-200 repagermanium, a drug that blocks a receptor involved in inflammation, in patients with focal segmental glomerulosclerosis FSGS who are also receiving an angiotensin II receptor blocker ARB. This Phase 3 study aims to assess the safety and effectiveness of DMX-200 compared to placebo over two years in adults and adolescents aged 12 to 17 years. The study is led by Dimerix Bioscience Pty Ltd and includes a double-blind period followed by an open-label extension to observe long-term effects. Participants receive either 120 mg of DMX-200 or a matching placebo capsule twice daily for 104 weeks during the double-blind treatment phase. Afterward, those who complete this phase may enter a two-year open-label extension where all participants receive DMX-200 twice daily. The study includes a screening and qualification period lasting 6 to 14 weeks, a possible titration phase, a stabilization phase, and a follow-up period after treatments. Throughout the trial, patients will undergo assessments including urine proteincreatinine ratio and kidney function tests like estimated glomerular filtration rate eGFR at multiple time points up to week 104 and during the extension. Safety and tolerability are closely monitored through regular evaluations, adverse event tracking, and follow-up visits. Total participation may last about 230 weeks, covering all study phases and follow-up periods.

This research aims to collect data from patients with active severe ANCA-associated vasculitis AAV, focusing on those treated with avacopan and those treated with cyclophosphamide or rituximab-based induction regimens without avacopan. The study is non-interventional, multinational, and prospective, lasting up to 7 years, including about 3 years for recruitment. Its main goal is to evaluate the incidence of specific safety events in patients starting avacopan treatment. The study will include two groups one with approximately 250 adults treated with avacopan, and another with about 250 adults receiving standard care using cyclophosphamide or rituximab without avacopan. Patients who began treatment within 6 months prior to enrollment or those starting treatment at enrollment will be included. Follow-up visits will occur periodically during routine clinic visits until 4 years after the last patient enrolls. Participants will be monitored through routine clinical visits, where data on safety events, adverse events, and various laboratory markers will be collected. The study will assess changes over time in kidney function, immune markers, and other blood tests. Researchers will also track flare occurrences, medication use, and safety outcomes over the entire study period, which may last up to 7 years.

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 the efficacy, safety, tolerability, and pharmacokinetics of VX-147 in adults and children aged 10 to 65 with apolipoprotein L1 APOL1-mediated proteinuric kidney disease. This condition involves kidney damage characterized by protein in the urine. The study is a combined Phase 23 adaptive, double-blind, placebo-controlled clinical trial sponsored by Vertex Pharmaceuticals Incorporated. Participants will be randomly assigned to receive different doses of VX-147 tablets or matching placebo tablets. They will take the assigned treatment for at least 96 weeks in Part A. Those who complete Part A will continue receiving VX-147 for an additional 96 weeks in Part B. The study uses a quadruple masking design to keep treatment assignments confidential. Throughout the study, participants will undergo regular assessments including urine protein to creatinine ratio UPCR and estimated glomerular filtration rate eGFR slopes to monitor kidney function. Safety is monitored by tracking adverse events and serious adverse events over several years. Blood samples will measure VX-147 levels, and treatment acceptability will be assessed in pediatric participants. Total participation may last up to approximately four years, with ongoing safety and efficacy evaluations.

This research aims to evaluate the effects of lowering blood phosphate levels in adults with end-stage kidney disease ESKD who are receiving dialysis. Elevated phosphate levels are common in ESKD and linked to higher risk of death and heart problems, but it is unclear if reducing phosphate improves important patient outcomes. The study will compare intensive lowering of phosphate to a more liberal phosphate target to see if this reduces heart-related deaths and events, improves physical health, and is cost-effective. Participants will be randomly assigned to one of two groups one aiming for a liberal serum phosphate target of 2.0 to 2.5 mmolL, and the other aiming for an intensive target of 1.50 mmolL or lower. Doctors will decide the type and dose of phosphate-lowering medications to help participants reach their assigned phosphate levels, following usual local practices. The study will last up to five years and is conducted internationally with 3600 adults on dialysis. During the study, researchers will monitor participants for major heart-related events and deaths, physical health, fatigue, quality of life, patient satisfaction, and itching. Regular assessments will include measuring time to cardiovascular death or major cardiovascular events and evaluating overall survival and quality of life using the EQ5D-5L tool. The study will also assess cost-effectiveness and continue to observe participants for five years to collect comprehensive data on these outcomes.

This research aims to improve the accuracy of MRI scans in detecting early-stage rectal cancer and significant rectal polyps. Many patients with early rectal cancer currently undergo major surgery or unnecessary treatments due to inaccurate staging. The study evaluates a new MRI reading method called PRESERVE, which has been shown to identify early rectal cancers more accurately and may allow more patients to have less invasive local excision surgery that preserves the rectum and quality of life. The study compares two phases a control phase where patients receive standard care, and an intervention phase following training of radiologists in the PRESERVE MRI staging method. During the intervention phase, MRI scans are recommended for rectal polyps 20mm or larger or with suspicious features before removal, alongside radiologist training. Each hospital transitions from control to intervention at a randomized time, with no crossover of patients between phases. Participants will be monitored through clinical investigations before, during, and after treatment as per standard care. Researchers will assess the accuracy of tumor staging on MRI, the number of patients identified for rectal preservation surgery, and quality of life outcomes over up to 5 years. Additional measures include scan quality, tumor characteristics, treatment costs, and histopathological biomarkers. The primary outcome is the impact of radiologist training on staging accuracy measured after one year.

The trial investigates the role of ixazomib in patients with relapsed multiple myeloma who have previously undergone autologous stem cell transplant ASCT. It is a phase III, randomized, controlled, multi-center, open-label study aiming to assess whether adding a proteasome inhibitor to salvage ASCT conditioning improves depth of response and how consolidation and maintenance therapy influence the durability of response. The study also evaluates overall survival, progression-free survival, response rates, minimal residual disease, toxicity, and quality of life. Participants first receive re-induction therapy with 4 to 6 cycles of ixazomib, thalidomide, and dexamethasone ITD, each cycle lasting 28 days. Those achieving at least stable disease are randomly assigned to receive either conventional ASCT using melphalan alone or augmented ASCT combining melphalan with ixazomib. After ASCT, participants with minimal response or better are further randomized to either receive consolidation with 2 cycles of ITD followed by ixazomib maintenance until disease progression or no further treatment. During the study, participants undergo regular assessments including response evaluation 100 days post-ASCT, monitoring of progression-free survival for up to 120 months, and measurement of minimal residual disease at several time points. Safety and toxicity are closely monitored throughout re-induction, post-ASCT, consolidation, and maintenance phases. Quality of life questionnaires are completed at baseline, post-re-induction, post-ASCT, and during follow-up visits up to 24 months. The study plans to register 406 participants, with 284 randomized at the first stage and 248 at the second stage.

This research aims to evaluate the clinical and cost effectiveness of different methods to prevent blood clots in people who have a leg injury treated with a plaster cast or splint. The study focuses on comparing oral tablets to injections in people at high risk of clots, and also whether any medication is better than advice alone for people at low risk. This is a phase III, multicenter study involving two linked trials called TiLLI-High and TiLLI-Low.

Researchers are studying patients with newly diagnosed stage I, II, and III colorectal cancer CRC to understand how circulating tumor DNA ctDNA in the blood can predict disease relapse. The study evaluates whether using ctDNA to guide adjuvant chemotherapy decisions after surgery is as effective as standard chemotherapy, aiming to reduce unnecessary treatments and side effects. This multi-center, prospective research includes both observational and randomized components to better manage early-stage CRC. The study has two parts Part B focuses on collecting tumor tissue, serial blood samples, and clinical data to detect minimal residual disease MRD using ctDNA after curative surgery. Part C is a randomized trial comparing ctDNA-guided adjuvant chemotherapy versus standard care in patients with high-risk stage II or III CRC. Patients are randomized post-surgery to either standard chemotherapy or a ctDNA-guided approach where those testing negative for ctDNA may receive less chemotherapy. Participants will undergo regular blood sampling and clinical assessments to monitor ctDNA levels and disease status. Researchers will measure outcomes such as 3-year disease-free survival and the relationship between ctDNA detection and treatment response over several years. The study includes follow-up periods of 4 to 8 years to evaluate long-term outcomes and safety. Participants need to consent, adhere to follow-up schedules, and be suitable for chemotherapy if randomized to Part C.