Bad Bramstedt

Researchers are evaluating the effect and safety of efgartigimod PH20 SC compared to placebo in adults with systemic sclerosis, a chronic autoimmune disease. This phase 2, randomized, double-blinded, placebo-controlled study aims to assess how well the treatment works and its safety profile. Participants have systemic sclerosis with specific skin involvement and meet classification criteria, with the study sponsored by argenx. Eligible participants will be randomly assigned in a 21 ratio to receive either subcutaneous efgartigimod PH20 SC or a placebo, both given by prefilled syringe. The treatment period lasts up to 48 weeks, followed by a safety follow-up period. The total duration of the study can be up to approximately 15 months, including screening, treatment, and follow-up. Participants will undergo screening to confirm eligibility and then receive regular doses of the study drug or placebo during the treatment phase. Assessments include changes in skin thickness measured by the modified Rodnan Skin Score mRSS at 24 and 48 weeks, safety monitoring for adverse events, and evaluations of disability, patient and clinician assessments, lung function, and antibody levels. The study also tracks pharmacokinetics, immunogenicity, and antibody responses throughout the treatment and follow-up periods.

Researchers are evaluating a medicine called nerandomilast for adults with systemic sclerosis, a condition affecting the skin and internal organs. The study focuses on people with limited or diffuse cutaneous systemic sclerosis who have had symptoms for less than 7 years. The goal is to see if nerandomilast helps improve symptoms and slows disease progression while assessing how well the medicine is tolerated. Participants are randomly assigned to one of two groups one takes nerandomilast tablets, and the other takes placebo tablets that look identical but do not contain medicine. Tablets are taken twice daily. The study lasts from 1 to about 4 years, with regular visits to the study site and phone check-ins. During visits, participants have blood tests and assessments of skin thickening, lung function, and organ health to monitor changes and treatment effects. Throughout the study, doctors monitor participants overall health and any side effects. They regularly assess safety and tolerability of the treatment. The main outcome measured is the time to disease progression or death over up to 4 years. Other outcomes include changes in skin scores, lung capacity, physical function, and disease impact questionnaires. Participants health is closely followed during the study period.

Researchers are evaluating depemokimab in adults with hypereosinophilic syndrome HES who have uncontrolled disease despite standard care therapy. This phase 3, randomized, placebo-controlled, double-blind, parallel group, multicenter study focuses on adults with confirmed HES diagnosis, a history of repeated flares two or more in the past 12 months, and a blood eosinophil count of at least 1,000 cellsL. The goal is to assess the efficacy and safety of depemokimab in reducing HES flares compared to placebo while continuing standard care treatment. Participants will be randomly assigned in a 21 ratio to receive either depemokimab or a matching placebo. Both groups will continue their stable standard of care HES therapy during the 52-week treatment period. Depemokimab or placebo is administered under blinded conditions, and participants will be monitored throughout the study to evaluate treatment effects and safety. Throughout the 52 weeks, participants will have regular assessments including monitoring the frequency and timing of HES flares, fatigue levels measured by the Brief Fatigue Inventory, and other safety evaluations. The primary outcome is the frequency of HES flares up to 52 weeks. Secondary outcomes include time to first flare, number of participants experiencing flares, and changes in fatigue scores. Safety and tolerability will also be closely monitored during the study period.

Researchers are evaluating the real-world clinical outcomes and patient-reported experiences of adults with systemic lupus erythematosus SLE who start treatment with subcutaneous anifrolumab. This prospective, single-arm, multicenter, non-interventional study focuses on patients who have not previously used anifrolumab subcutaneously or intravenously. The study aims to describe disease activity, including remission and lupus low disease activity states, quality of life, medication patterns, flares, and organ damage over time. Participants will receive subcutaneous anifrolumab as part of their routine care, with treatment decisions made independently of the study. The study follows patients for 24 months to capture long-term disease control, medication use, and organ damage progression. Data collected include clinical assessments and patient-reported outcomes in a real-life setting without altering usual treatment. During the 24-month observation period, participants will be evaluated at routine visits where disease activity, treatment patterns, quality of life, fatigue, flares, and organ damage are measured. Researchers will review outcomes such as remission rates, time to remission, and sustained disease control. Patient questionnaires and clinical assessments will help track these measures, providing insights into long-term disease management and treatment effects in everyday practice.