Little Silver

Researchers are evaluating the effectiveness and safety of efgartigimod given through intravenous infusion in adults with primary immune thrombocytopenia (ITP), a condition characterized by low platelet counts. This Phase 3 trial includes participants who have had ITP for over a year and have previously received treatments like corticosteroids or immunoglobulins but had insufficient responses. The study aims to measure disease control by tracking the number of weeks during which platelet counts remain at or above 50 x 10^9/L over a 24-week treatment period. After a screening period of up to 2 weeks, participants are randomly assigned in a 2:1 ratio to receive either efgartigimod IV or a placebo IV during a 24-week double-blinded treatment period. Following this, all participants enter a 52-week open-label treatment phase where everyone receives efgartigimod IV. Those who complete this phase may continue for an additional 52 weeks in a second open-label treatment period. After finishing these treatment phases, participants undergo an approximately 8-week follow-up period without the study drug. Throughout the study, participants will have their platelet counts regularly monitored to assess the extent of disease control. Researchers will also evaluate safety and monitor participants for any medical conditions that might affect the study results or their well-being. The total duration of participation can be up to 138 weeks, including screening, treatment, and follow-up periods.

Researchers are evaluating the effectiveness of remibrutinib compared to dupilumab in adults with moderate to severe chronic spontaneous urticaria (CSU) that is not adequately controlled by second generation H1-antihistamines (sgH1-AHs). This Phase 3b, multi-center, randomized, double-blind, double-dummy study is conducted in the US and focuses on early treatment effects at 4 weeks and earlier. The study includes a screening period of up to 4 weeks, followed by a 12-week core treatment period where about 400 participants are randomly assigned to receive either remibrutinib (25 mg twice daily by mouth) with a placebo injection or dupilumab (a 600 mg loading dose followed by 300 mg every 2 weeks by injection) with a placebo tablet. All participants continue their stable dose of sgH1-AH during this period, with the option to add rescue doses if needed, not exceeding four times the standard dose per day. After the core period, participants may join an optional open-label extension to receive remibrutinib for an additional 12 weeks if the drug is not commercially available. Participants will complete daily diaries and regular assessments to track urticaria symptoms and treatment effects. Researchers will measure changes in the Weekly Urticaria Activity Score (UAS7) from the start to Week 4. Safety follow-up will occur for 12 weeks after treatment ends, with phone calls and site visits as needed, continuing longer if participants join the extension. The total study duration includes screening, treatment, optional extension, and safety follow-up phases.

This research aims to understand how avacincaptad pegol, a treatment approved in the US, is used for people with geographic atrophy caused by age-related macular degeneration (AMD). Geographic atrophy is an advanced stage of AMD where cells in the retina waste away, leading to worsening central vision and possible permanent vision loss. The study focuses on observing treatment patterns and safety in routine clinical practice rather than testing new effects. Participants in this study will receive avacincaptad pegol through intravitreal injections, which are injections into the eye. The study collects information from patients who have already been prescribed this treatment by their doctors. There is no experimental intervention from the study team, and treatment decisions are made by the patients' doctors. The study follows patients for up to 3 to 5 years, depending on when they join. While in the study, participants will have regular eye exams as part of their usual care. They will also complete surveys about their eye health at the start of treatment, every 6 months for the first 2 years, then annually afterward. Researchers will track treatment details like the number and dose of injections, treatment duration, reasons for stopping treatment, and patient characteristics. Safety and treatment patterns are monitored through medical records during and after treatment.

Researchers are evaluating ABBV-RGX-314, a novel one-time gene therapy, for treating neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD causes vision loss due to abnormal blood vessel growth in the retina and affects millions in the United States, Europe, and Japan. Current treatments require frequent eye injections, which can be burdensome and may lead to reduced vision over time. This Phase 3 study aims to compare the effectiveness and safety of two doses of ABBV-RGX-314 against the standard anti-VEGF drug, aflibercept, in people with wet AMD. Participants will be randomly assigned to receive one of two doses of ABBV-RGX-314 gene therapy or aflibercept injections. The gene therapy involves a one-time subretinal injection delivering a gene that produces an anti-VEGF protein to help control abnormal blood vessels. In addition, a bilateral treatment substudy will examine safety and effectiveness when both eyes are treated in participants with wet AMD in both eyes. This substudy will enroll up to 15 participants for at least 50 weeks of follow-up. During the study, participants will have their vision measured regularly to assess changes in best-corrected visual acuity (BCVA). Safety will be monitored by recording any eye-related adverse events and serious side effects. Participants will be followed for up to 54 weeks or more to evaluate how well the gene therapy maintains or improves vision compared to aflibercept and to assess overall treatment safety and tolerability.

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 Phase 3b study is evaluating how safe and effective Surabgene Lomparvovec (ABBV-RGX-314), an investigational gene therapy, is in treating adults aged 50 and older who have previously been treated for nAMD. Approximately 561 participants will be enrolled at about 150 sites worldwide to compare different treatment approaches in a real-world setting. Participants will be randomly assigned to one of three groups. Two groups will receive a single subretinal injection of Surabgene Lomparvovec, while the third group will receive Ranibizumab injections as needed. Ranibizumab is given as an intravitreal injection into the jelly-like tissue inside the eye, whereas Surabgene Lomparvovec is injected between the retina and the back of the eye. The assessment period begins two weeks before treatment and continues for up to 5 years, allowing long-term monitoring of treatment effects. Participants will attend monthly visits at a hospital or clinic to monitor their eye health. These visits include medical assessments, blood tests, side effect checks, and questionnaires. Researchers will measure the annualized rate of anti-VEGF injections needed and record any adverse events over the 5-year period. The study involves a higher treatment burden than standard care, but aims to gather detailed information on long-term vision preservation and safety.