Leczna

Researchers are evaluating the use of baloxavir marboxil in children under 12 years old with influenza. This study has two parts: Part A focuses on checking for resistance-related changes in the virus before and after treatment, while Part B looks at how influenza might spread from young children treated with baloxavir marboxil to their household contacts. Enrollment for Part B has stopped as per the latest protocol. Baloxavir marboxil is given as an oral suspension, with the dose based on the child's body weight: 80 mg for those 80 kg or more, 40 mg for 20 to less than 80 kg, and 2 mg per kg for those under 20 kg. Part A involves monitoring these children for resistance changes at baseline and during treatment on specific days. Part B included participants from Part A who lived with household contacts, assessing transmission, but no new participants are being enrolled in this part. Participants will be involved in screening to confirm influenza and absence of COVID-19, with symptom onset within 48 hours before starting treatment. Researchers will measure resistance-associated viral changes at baseline and during treatment days 4, 6, and 10. Household contacts in Part B were also assessed for influenza transmission risk and monitored through scheduled visits, but Part B enrollment is closed. The total study duration varies depending on participation in Parts A and B.

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 evaluating the effectiveness and safety of obefazimod compared to a placebo in adults with moderately to severely active Crohn's Disease who have not responded well or are intolerant to conventional or advanced treatments. The study is a Phase 2b trial and includes three treatment phases: a 12-week Induction Phase, a 40-week Maintenance Phase, and a 48-week Extension Phase. The main goals are to assess how well obefazimod controls disease activity and its safety over these periods. Participants will receive either obefazimod or a matching placebo once daily, preferably in the morning with food. The trial includes an initial 12-week treatment to induce response, followed by a 40-week maintenance period to sustain results. Those who complete these phases may enter a 48-week Extension Phase to further evaluate the long-term safety and tolerability of obefazimod compared to placebo. During the study, participants will undergo regular assessments including clinical evaluations of disease activity using the Crohn's Disease Activity Index and endoscopic scoring at various time points up to week 52. Safety is monitored throughout, especially during the Extension Phase with checks for adverse events, blood tests, and other laboratory evaluations at scheduled visits. Overall, participation may last over a year, with careful monitoring of treatment effects and safety.

Researchers are evaluating the effect of tozorakimab, added to standard care, in adults hospitalized with viral lung infection who need supplemental oxygen. The study focuses on preventing death or progression to invasive mechanical ventilation or extracorporeal membrane oxygenation by day 28. This is a Phase III, multicenter, randomized, double-blind trial comparing tozorakimab to placebo in patients with viral lung infection causing acute respiratory failure. Participants will receive a single intravenous dose of either tozorakimab or a matching placebo on the first day of the study. Both groups continue to receive standard care for their viral lung infection. The study is designed to assess the safety and efficacy of tozorakimab as an add-on therapy in this patient population. Throughout the study, researchers will monitor participants for survival and the need for invasive mechanical ventilation or ECMO up to 28 days after treatment. The main outcome measured is the proportion of patients who die or require mechanical ventilation or ECMO by day 28. Participants will be closely observed during hospitalization, with data collected on their respiratory status and treatment outcomes to evaluate the study drug's impact and safety.

The trial investigates the safety and effectiveness of a medication called OCTAPLEX, a four-factor prothrombin complex concentrate, in patients experiencing acute major bleeding while on direct oral anticoagulant (DOAC) therapy with a factor Xa inhibitor. This phase 3, multicenter, prospective, randomized, double-blinded study compares two doses of OCTAPLEX, low-dose and high-dose, to assess hemostatic efficacy in this patient group. Participants will be randomly assigned in a 1:1 ratio to receive either the low-dose or high-dose OCTAPLEX. The study focuses on patients who have acute major bleeding related to their use of oral factor Xa inhibitors. Treatment administration and the comparison of dosing strategies are designed to evaluate how well OCTAPLEX controls bleeding in these patients. During the study, researchers monitor the effectiveness of the treatment by measuring hemostatic efficacy within 24 hours after starting management. Patients are closely observed for safety and treatment response. The duration of participation involves initial treatment and monitoring of bleeding control, with assessments based on clinical signs and laboratory tests related to bleeding and clotting function.

Researchers are investigating transmural healing (TMH) as a treatment target for patients with moderately to severely active Crohn's disease (CD). TMH, assessed by intestinal ultrasound (IUS), may be linked to better long-term outcomes but is not yet a formal treatment goal. This phase 4 study aims to find out if targeting corticosteroid-free IUS outcomes along with clinical symptoms and biomarkers improves corticosteroid-free endoscopic remission compared to targeting clinical symptoms and biomarkers alone, measured by the Simple Endoscopic Score for Crohn's Disease (SES-CD). Participants will be randomly assigned to one of two groups for 48 weeks of treatment with vedolizumab. All start with an induction regimen of 300 mg IV at weeks 0, 2, 6, and 10, followed by 300 mg IV every 8 weeks beginning at week 14. Group 1's treatment targets corticosteroid-free IUS-based outcomes plus clinical and biomarker remission, with IUS response assessments at weeks 22 and 30 and transmural healing as the final target at week 38. Group 2 aims for corticosteroid-free clinical and biomarker remission without the IUS component. Treatment adjustments may occur at weeks 22, 30, and 38 based on target assessments. During the study, participants will undergo regular evaluations to monitor disease activity and treatment response, including IUS assessments, clinical symptom reviews, and biomarker testing. Researchers will measure the percentage of participants achieving corticosteroid-free endoscopic remission at week 48. Safety and adherence will be monitored throughout the 48-week treatment period. The study includes adults aged 18 to 80 with moderately to severely active Crohn's disease who meet specific disease activity and treatment history criteria.