Great Neck

This research aims to evaluate the effects of litifilimab (BIIB059), a monoclonal antibody, in adults with active subacute or chronic cutaneous lupus erythematosus (CLE), with or without systemic lupus erythematosus (SLE). Participants have active skin symptoms of CLE that have not improved with antimalarial therapy or had difficulties continuing that treatment. The study focuses on reducing skin disease activity using several scores including CLA-IGA-R and CLASI, while also assessing safety, immune response, and quality of life. Participants will be randomly assigned to receive either litifilimab or a placebo injection under the skin every four weeks during a 24-week double-blind period where neither participants nor researchers know which treatment is given. After this, all participants will receive litifilimab injections every four weeks for an additional 28 weeks. Those who complete the treatment may join a long-term extension study or enter a follow-up safety period lasting up to 24 weeks. Total participation may last up to 80 weeks. Throughout the study, researchers will monitor skin disease activity using the CLA-IGA-R erythema score and the CLASI-A activity score to see how many participants improve. They will also assess safety, tolerability, immune system effects, and participants' quality of life using questionnaires. These evaluations occur regularly during both treatment periods and follow-up to understand the impact of litifilimab on CLE symptoms and overall health.

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 studying the effects of NEU-411 in men and women aged 40 to 80 years who have early Parkinson's Disease (PD) with increased activity in the LRRK2 pathway, identified through a genetic test. This Phase 2 trial aims to evaluate how well NEU-411 works and its safety in this specific group compared to a placebo. The study involves participants with clinically established or probable PD and focuses on those with LRRK2-driven PD. Participants will be randomly assigned to receive either NEU-411, a brain-penetrant oral drug that inhibits LRRK2 activity, or a matching placebo once daily for 52 weeks. After completing this treatment phase, participants will have a safety follow-up visit within two weeks. Those eligible may join an open-label extension to receive NEU-411 for an additional 26 weeks. During the study, researchers will monitor changes in a digital biomarker score using a Parkinson's Disease app from enrollment through 52 weeks, along with recording any treatment-emergent or serious adverse events until the study ends at 54 weeks. Assessments include genetic testing, clinical evaluations of PD status, safety monitoring, and tracking of side effects to understand the drug's effects and tolerability over time.

Researchers are investigating the safety, tolerability, and effectiveness of two dosing regimens of itepekimab compared to placebo as an add-on to intranasal corticosteroids in adults with chronic rhinosinusitis with nasal polyps (CRSwNP) that is not well controlled. This multinational Phase 3, randomized, double-blind, placebo-controlled trial involves male and female participants aged 18 years and older living with CRSwNP. Participants are randomly assigned to one of three groups receiving either itepekimab injections or placebo injections, both administered subcutaneously, alongside mometasone furoate nasal spray delivered intranasally. The study includes a 4-week screening period, followed by a 52-week treatment phase, and a 20-week safety follow-up, totaling up to 76 weeks. Participants transitioning to an extension study (LTS18420) will have a total duration of 56 weeks. Study visits include nine site visits and 20 phone or home visits. During the trial, participants will undergo assessments including endoscopic Nasal Polyp Scores (NPS) and Nasal Congestion Scores (NCS) measured from baseline to week 24 to evaluate changes. Researchers will monitor safety and tolerability throughout, with regular evaluations involving symptom severity, treatment adherence, and adverse events. The study aims to understand how well itepekimab works and is tolerated as an additional treatment for CRSwNP over the study duration.

Researchers are evaluating the long-term safety of avacopan in adults with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis, including granulomatosis with polyangiitis or microscopic polyangiitis. This phase 4 randomized, double-blind, placebo-controlled trial focuses on participants newly diagnosed or experiencing a relapse who require induction treatment with cyclophosphamide or rituximab and have specific antibody positivity and disease activity criteria. Participants will receive oral avacopan or placebo alongside standard of care immunosuppressive therapy for induction and maintenance, tailored by the investigator according to guidelines and patient needs. The study monitors treatment effects and safety over an extended period, up to 60 months, assessing adverse events and clinical changes. Throughout the study, participants will undergo regular evaluations including vital signs, blood tests, and urinalysis to detect any significant changes from baseline. The main outcomes measured include the percentage of participants experiencing various types of adverse events, serious adverse events, and events leading to withdrawal or death, all tracked up to 60 months to ensure comprehensive long-term safety data.

Researchers are evaluating the effectiveness, safety, and pharmacokinetics of the Port Delivery System (PDS) with ranibizumab compared to standard intravitreal ranibizumab injections in adults with diabetic macular edema (DME). This Phase III, multicenter, randomized study aims to compare PDS treatment every 24 weeks with injections every 4 weeks. A substudy will assess the safety of re-implanting the updated PDS and performing refill-exchange procedures in participants previously enrolled in the main study. Participants will receive either the PDS implant pre-filled with ranibizumab or intravitreal ranibizumab injections according to their assigned group. Treatments will be administered on a set schedule specific to each arm. The substudy involves re-implantation of the updated PDS and monitoring post-procedure. The PDS refill exchange is also part of the treatment plan for some participants. Throughout the study, participants will undergo assessments including vision tests using the ETDRS chart to measure changes in best-corrected visual acuity (BCVA). Safety will be monitored by tracking ocular and systemic adverse events, device-related effects, and any serious complications up to 72 weeks after treatment or re-implantation. The study evaluates both short-term and long-term safety and efficacy outcomes over the full duration of participation.

Researchers are evaluating the effectiveness and safety of KarXT for treating schizophrenia in adolescents aged 13 to 17 years. This Phase 3 study focuses on adolescents who meet diagnostic criteria for schizophrenia and experience symptoms of psychosis. The study aims to better understand how KarXT may impact symptoms as measured by a standard schizophrenia rating scale. Participants will receive either KarXT or a matching placebo at specified doses on specific days. The study is randomized, double-blind, and placebo-controlled, meaning neither the participants nor the researchers know who receives the active drug or placebo during the trial. During the study, researchers will assess changes in schizophrenia symptoms using the Positive and Negative Syndrome Scale (PANSS) after 5 weeks of treatment. Participants will be monitored for safety and symptom changes throughout the study period. The goal is to gather detailed information about KarXT's impact on schizophrenia symptoms in this adolescent population.

Researchers are evaluating the safety and effectiveness of tulisokibart, a humanized monoclonal antibody, in people with moderately to severely active Crohn's disease. The research includes two studies: Study 1, which has induction and maintenance treatment phases, and Study 2, which only includes induction treatment. The main goals are to see if tulisokibart can help participants achieve clinical remission and endoscopic response compared to placebo, measured at 12 and 52 weeks depending on the study and region (US/FDA or EU/EMA).

Researchers are assessing the long-term safety and tolerability of two treatments, KarXT and KarX-EC, for adolescents with schizophrenia and children and adolescents with irritability related to autism spectrum disorder. This Phase 3, multicenter, open-label study includes participants aged 5 to 17 years and aims to monitor how these treatments are tolerated over time in these specific populations. Participants receive KarXT or a combination of KarXT and KarX-EC at specified doses on designated days. The study includes adolescents aged 13 to 17 years with schizophrenia and children and adolescents aged 5 to 17 years with autism-related irritability. Treatment is administered openly, meaning both researchers and participants know the treatment being given. Throughout the study, researchers will evaluate participants for any treatment-emergent adverse events, adverse events of special interest, and serious adverse events for up to 54 weeks. Safety assessments include monitoring physical examinations, vital signs, and ECGs. Participants must have completed earlier related studies without safety concerns to join, and their health will be closely monitored during the study to ensure safety and tolerability.

Researchers are evaluating the safety and effectiveness of subcutaneous anifrolumab compared with placebo in adults with moderate to severe Idiopathic Inflammatory Myopathies (IIM), specifically polymyositis (PM) or dermatomyositis (DM). This multicenter, randomized, double-blind, placebo-controlled Phase III study adds anifrolumab or placebo to participants' standard of care treatment to assess overall disease activity. Participants will receive weekly subcutaneous injections of either anifrolumab or placebo for 52 weeks. After this period, all participants will receive open-label anifrolumab injections once weekly for an additional 52 weeks. This design allows researchers to evaluate initial treatment effects and longer-term outcomes with anifrolumab. During the study, participants will be monitored regularly for disease activity and safety. The main outcome measured is the Total Improvement Score (TIS) with a response defined as a score of 40 or higher at 52 weeks. The total study participation lasts up to 104 weeks, including the double-blind and open-label extension periods, ensuring comprehensive assessment of the treatment's impact and participant safety.