Little Silver

Researchers are evaluating the effects and safety of efgartigimod given intravenously in adults with primary immune thrombocytopenia ITP, a condition where the immune system attacks platelets. This phase 3 clinical trial aims to determine how well efgartigimod controls platelet counts compared to a placebo during a 24-week double-blinded treatment period. The study is sponsored by argenx and includes follow-up periods to monitor ongoing effects and safety. Participants will be randomly assigned in a 21 ratio to receive either efgartigimod IV or placebo IV during the double-blinded treatment period. After this, all participants will receive efgartigimod IV during a first 52-week open-label treatment phase, followed by an optional second 52-week open-label phase. After completing these treatment periods, participants will have an approximately 8-week follow-up while off the study drug, with total study participation lasting up to 138 weeks. During the study, participants will have regular blood tests to measure platelet counts and assess disease control. Researchers will track the number of weeks participants maintain platelet counts above specified levels and monitor any bleeding events or adverse effects. Safety is closely observed throughout the trial, including antibody responses to the drug. Participants will have scheduled visits and assessments throughout treatment and follow-up to gather comprehensive data on efgartigimods impact.

Researchers are evaluating the effectiveness of remibrutinib compared to dupilumab as add-on treatments for adults with moderate to severe chronic spontaneous urticaria CSU that is not well controlled by second generation H1-antihistamines sgH1-AH. This Phase 3b, multi-center, randomized, double-blind, double-dummy study focuses on early treatment effects within 4 weeks. The study addresses the need for better management of CSU symptoms such as hives and itch. Participants will be assigned to one of two treatment groups one group will receive remibrutinib tablets twice daily plus placebo injections, while the other group will receive dupilumab injections with matching placebo tablets. Both groups continue their stable background therapy of sgH1-AH daily. The study includes a screening period up to 4 weeks, a 12-week core double-blind treatment period, and an optional 12-week open-label extension where all participants may receive remibrutinib if it is not commercially available. After treatment, safety follow-up occurs for up to 12 weeks, with phone calls and possible site visits. Participants will be monitored through regular assessments including symptom severity scores, urticaria activity scores, and daily diaries. Safety follow-up includes phone calls and visits depending on treatment continuation. The main outcome is the change in weekly urticaria activity score at Week 4. Other measures include severity of hives and itch at Weeks 1 and 4. Total study participation may last up to 24 weeks, including optional extension and follow-up phases.

Researchers are observing how avacincaptad pegol is used in routine clinical practice for people with geographic atrophy caused by age-related macular degeneration AMD. Geographic atrophy is an advanced form of AMD where retinal cells waste away, leading to worsening central vision and potential permanent vision loss. This observational study collects information on treatment and medical events related to avacincaptad pegol without influencing the doctors treatment decisions. Participants in this study have chosen to begin treatment with avacincaptad pegol, delivered as intravitreal injections into the eye. The study records how often and how long the treatment is given, along with reasons for stopping treatment. It monitors participants receiving this treatment in one or both eyes as part of their regular medical care, without additional interventions from the study. During the study, participants will have eye exams as part of their usual care and will complete surveys about their eye health at the start of treatment, every six months for two years, and then yearly. Researchers will gather data from medical records to track treatment usage, vision changes, and any adverse events for up to about five years. This long-term observation helps understand real-world treatment patterns and safety.

Neovascular age-related macular degeneration nAMD, also called wet AMD, involves abnormal growth of new blood vessels in the retina, the light-sensitive tissue at the back of the eye. This study aims to evaluate the safety and effectiveness of Surabgene Lomparvovec ABBV-RGX-314, an investigational gene therapy, in adults aged 50 and older with previously treated nAMD. Approximately 561 participants will be enrolled worldwide to compare this gene therapy with standard treatment. Participants will be randomly assigned to one of three groups. Two groups will receive a single subretinal injection of Surabgene Lomparvovec at different doses, and the third group will receive Ranibizumab injections as needed through an intravitreal route. The study includes an assessment period starting two weeks before treatment and continues for up to five years. Participants will attend monthly visits at hospitals or clinics for treatment and evaluation. During the study, participants will undergo medical assessments, blood tests, side effect monitoring, and complete questionnaires to track treatment effects and safety. The primary outcomes include the rate of intravitreal anti-VEGF injections up to week 54 and adverse events observed up to five years. Secondary outcomes focus on changes in visual acuity and injection rates over three years. This long-term follow-up will help understand the treatment impact and preservation of vision.