Vila Franca De Xira

Researchers are evaluating the safety, tolerability, and effectiveness of two treatments, inebilizumab and blinatumomab, in adults with active and difficult-to-treat autoimmune diseases. This includes systemic lupus erythematosus (SLE) with nephritis, SLE with and without nephritis, and active refractory rheumatoid arthritis (RA). The study is a Phase 2, open-label, multicenter platform trial designed to assess these treatments across different subprotocols based on the specific condition and disease activity. Participants receive inebilizumab through intravenous infusion or blinatumomab via subcutaneous injection, depending on their assigned subprotocol. The study includes several parts: Subprotocol A focuses on SLE with nephritis treated with inebilizumab; Subprotocol B Part A and Part B assess blinatumomab in SLE with and without nephritis; and Subprotocol C Parts A and B evaluate blinatumomab in rheumatoid arthritis. The treatments are administered over specified periods, with some groups receiving treatment for up to 52 weeks. During the trial, participants undergo various assessments to monitor safety and disease response, including evaluation of treatment-emergent adverse events, serious adverse events, and measures of disease remission or activity. For example, kidney response and remission in SLE and disease activity scores in RA are measured at specific time points. Safety monitoring continues through the treatment period, with data collected on adverse events from Day 1 to Week 52. Participants' health status, laboratory tests, and disease activity are regularly evaluated to understand the treatments' effects and tolerability.

Researchers are evaluating how well elacestrant works compared to standard endocrine therapy in adults with node-positive, Estrogen Receptor-positive (ER+), Human Epidermal Growth Factor-2 negative (HER2-) early breast cancer who are at high risk of the cancer returning. This is a Phase 3 global, multicenter, randomized, open-label study focusing on participants who have had early invasive breast cancer removed and meet specific receptor and risk criteria. The study aims to understand which treatment better prevents invasive breast cancer over up to five years. Participants will receive either elacestrant or one of several standard endocrine therapies, including anastrozole, letrozole, exemestane, or tamoxifen, all given as oral tablets. Treatments will be administered according to the study plan, with careful monitoring throughout the trial. The study includes adults who have already received between 24 and 60 months of prior endocrine therapy, with or without certain inhibitors, and who have completed or stopped these treatments as required. During the study, participants will be monitored for invasive breast cancer-free survival for up to five years. Researchers will perform regular assessments to track treatment effects, side effects, and cancer recurrence. The study also includes safety monitoring and may involve additional tests or evaluations as needed to ensure participant well-being throughout the trial.

Researchers are studying the effects of DMX-200 (repagermanium), a drug that blocks a receptor involved in inflammation, in people with focal segmental glomerulosclerosis (FSGS) who are also taking an angiotensin II receptor blocker (ARB). This Phase 3 trial aims to assess the safety and effectiveness of DMX-200 compared to placebo over 104 weeks in adults and adolescents aged 12 to 17 years. Following the initial study, an open-label extension will evaluate long-term safety and benefits for up to two more years. Participants will be randomly assigned to receive either DMX-200 at 120 mg twice daily or a placebo, while continuing their ARB treatment. The study includes a screening and qualification period lasting 6 to 14 weeks, a 104-week double-blind treatment phase, and a 4-week follow-up after treatment. Those completing this phase may enter the open-label extension for an additional minimum of 104 weeks, with another 4-week follow-up period, making the total study duration about 230 weeks. During the trial, participants will undergo regular assessments including urine protein and creatinine testing, kidney function monitoring by estimated glomerular filtration rate (eGFR), and safety evaluations. The main outcomes measured are changes in proteinuria, kidney function slope up to week 104, and long-term safety through week 216. Safety will be closely monitored throughout both the double-blind and extension periods to understand the drug's effects over time.

Researchers are conducting a two-part, phase 2b/3 study to evaluate CSL300 (Clazakizumab) in adults with end stage kidney disease (ESKD) undergoing dialysis who have systemic inflammation and either atherosclerotic cardiovascular disease (ASCVD) or diabetes. The study aims to determine the best dose of CSL300 and assess its effects on cardiovascular outcomes and safety in this population. This multicenter, randomized, double-blind, placebo-controlled trial targets patients with elevated inflammation markers and significant health risks due to their conditions. In the first part (phase 2b), the study focuses on finding the appropriate dose of CSL300 compared to placebo. CSL300 is given through intravenous (IV) administration. The second part (phase 3) evaluates the impact of CSL300 on cardiovascular events such as heart attack or cardiovascular death over approximately 5 years, continuing to compare CSL300 to placebo for safety and efficacy. The placebo matches CSL300's excipient content but lacks the active drug. Participants will undergo baseline and regular assessments for inflammation markers like high-sensitivity C-reactive protein (hs-CRP) up to 12 weeks in phase 2b, and long-term monitoring for cardiovascular outcomes in phase 3. The study involves ongoing safety evaluations and efficacy measurements during the entire follow-up period. This comprehensive approach helps researchers understand how CSL300 affects inflammation and cardiovascular health in patients with ESKD on dialysis.

Researchers are investigating whether the medicine vicadrostat, when taken together with empagliflozin, can lower the risk of heart-related problems in adults who have type 2 diabetes, high blood pressure, and cardiovascular disease but no history of heart failure. This study is a Phase III trial that compares the effects of vicadrostat plus empagliflozin to a placebo plus empagliflozin in people with these conditions. Participants are randomly assigned to one of two groups: one group takes vicadrostat and empagliflozin tablets, and the other group takes placebo tablets that look like vicadrostat along with empagliflozin. All participants take one tablet daily for a period ranging from two and a half years up to four years and three months. Throughout the study, participants continue their usual medications for diabetes, high blood pressure, and cardiovascular disease. During up to 51 months of participation, participants visit the study site regularly where doctors collect health information and blood samples. Researchers track when participants experience cardiovascular events such as heart-related deaths or heart failure events. The study also monitors participants’ overall health and any side effects they may experience to assess the safety and effects of the treatments.

Researchers are studying the effects of cancer diagnosis and treatment during pregnancy on both mothers and their children. The aim is to understand the overall survival of mothers diagnosed with cancer during pregnancy and to test whether children exposed to cancer treatments like chemotherapy or radiation in the womb develop normally, particularly focusing on neurological and heart health. The study includes several parts addressing maternal health during and after pregnancy and long-term follow-up of children exposed to cancer treatments before birth. The study involves registering mothers diagnosed with cancer during pregnancy and collecting maternal blood samples, umbilical cord blood, and tissue samples from the placenta and umbilical cord. Mothers complete questionnaires about anxiety and emotional needs related to their diagnosis. Children who were exposed to cancer treatments in the womb will have regular health check-ups at ages 6 months, 18 months, 3, 6, 9, 12, 15, and 18 years. After age 18, cardiologic assessments and questionnaires will continue every five years, with optional MRI scans at several ages during childhood and adolescence. Participants will be monitored through neurological and cardiological exams performed by specialists to assess the children's development. Mothers’ outcomes will be tracked prospectively from diagnosis through treatment and delivery. The study includes long-term follow-up for both mothers and children, with comprehensive assessments to evaluate health effects over many years. Participants provide informed consent, and children older than 12 provide assent with consent from parents or themselves once they reach adulthood.