Great Neck

Researchers are investigating new treatments for neovascular age-related macular degeneration NVAMD, a condition that affects vision. This study aims to learn if a medicine called tiespectus also known as MK-8748 or EYE201 can treat NVAMD as well as the standard treatment called aflibercept. The trial is a pivotal Phase 23 study that compares these treatments in people newly diagnosed with NVAMD. Participants are randomly assigned to one of three groups one group receives a low dose of tiespectus, another receives a high dose of tiespectus, and the third group receives aflibercept. Those in the tiespectus groups get three initial injections every 4 weeks, then continue injections every 8 weeks until week 48, followed by treatments at intervals based on their individual response up to week 92. The aflibercept group receives three initial injections followed by injections every 8 weeks until week 92. During the study, participants are regularly assessed for changes in their best-corrected visual acuity using ETDRS letters from baseline to one year. Other evaluations include eye imaging to measure retinal thickness and monitoring for any adverse events up to approximately 96 weeks. The study lasts over one year with ongoing visits to track treatment response and safety.

Researchers are evaluating the safety, tolerability, and effectiveness of two drugs, inebilizumab and blinatumomab, in adults with active and refractory autoimmune diseases such as systemic lupus erythematosus SLE with nephritis and rheumatoid arthritis RA. This phase 2, open-label, multicenter trial aims to better understand how these drugs work in these conditions and their impact on disease activity and kidney health. Participants will receive inebilizumab through intravenous IV infusions in different dosing schedules, or blinatumomab through subcutaneous SC injections at varying doses depending on the subprotocol group. The study includes several parts focusing on different autoimmune conditions and treatment regimens, with some parts no longer recruiting new participants. During the trial, participants will undergo regular assessments including monitoring for adverse events, evaluation of disease activity using specific clinical scores, kidney function tests, and antibody levels. Researchers will track safety and treatment responses over a period of up to 52 weeks, with visits occurring at scheduled intervals to collect data and monitor participant health.

Researchers are investigating the effects of APL-3007 combined with SyfovrePegcetacoplan APL-2 in patients with geographic atrophy caused by age-related macular degeneration AMD. This Phase 2 randomized, placebo-controlled study aims to assess the efficacy, safety, tolerability, and pharmacodynamics of these treatments in this eye condition. The study involves multiple centers and uses a masked design to ensure unbiased results. Participants will be assigned to one of three groups two receiving different doses or frequencies of APL-3007 in combination with pegcetacoplan APL-2, and one receiving a placebo along with pegcetacoplan APL-2. The study will evaluate the treatments given as multidose regimens. The treatments focus on complement C3 inhibition to potentially impact disease progression. Throughout the study, participants will undergo assessments including artificial intelligence-based imaging to measure retinal pigment epithelium lesion area and photoreceptor degeneration, safety evaluations through adverse event reporting and visual acuity tests, and blood tests to assess serum markers. These evaluations occur over 12 months to monitor changes from baseline. Participants involvement includes regular visits for these assessments, with the study tracking treatment effects and safety over the duration.

Researchers are evaluating the long-term safety of avacopan in adults with antineutrophil cytoplasmic antibody ANCA-associated vasculitis AAV, a condition requiring immunosuppressive therapy. This Phase 4 clinical trial aims to assess how participants tolerate avacopan combined with standard care over an extended period. The study involves participants diagnosed with granulomatosis with polyangiitis or microscopic polyangiitis who need induction treatment with cyclophosphamide or rituximab. Participants are randomly assigned to one of three groups avacopan 30 mg twice daily for five years plus standard care, avacopan 30 mg twice daily for one year followed by placebo twice daily for four years plus standard care, or placebo twice daily for five years plus standard care. Standard care involves background immunosuppressive therapy guided by current guidelines and tailored to each participants needs. Treatments are administered orally, and the study is double-blind to ensure objective assessment. During the study, participants will be monitored regularly for treatment-emergent adverse events, serious adverse events, and changes in vital signs and laboratory tests over up to 60 months. Researchers will also evaluate remission rates, relapse timing, kidney function, health perception scores, and medication use. Safety and efficacy data will be collected through clinical assessments, laboratory evaluations, and questionnaires, with follow-up continuing for the full duration of the trial.

Researchers are evaluating targeted therapies to treat adults with moderately to severely active Rheumatoid Arthritis RA, a chronic inflammatory condition causing joint pain, stiffness, swelling, and loss of function. This Phase 2 study involves three substudies focusing on different drug treatments to assess their effectiveness and safety for participants who have not responded to one or two prior biologic or targeted synthetic DMARD therapies. Participants will be randomly assigned to receive one of several treatments lutikizumab alone, ravagalimab alone, a combination of lutikizumab and ravagalimab, or matching placebos. These drugs are given by subcutaneous injection. The study involves regular visits at hospitals or clinics where participants receive the assigned treatment and are monitored closely. The treatment period and detailed dosing schedules are part of the studys design. During the trial, participants will undergo medical assessments, blood tests, and questionnaires to monitor treatment effects, side effects, and disease activity. The main outcomes measured include the percentage of participants achieving a 50% improvement according to the American College of Rheumatology criteria by Week 12 and the number of adverse events up to approximately Week 22. Participants will attend regular visits for evaluations throughout the study period, which is expected to complete by November 2027.

Researchers are evaluating the efficacy and safety of KarXT for treating schizophrenia in adolescents aged 13 to 17 years. This phase 3, randomized, double-blind, placebo-controlled study aims to better understand how KarXT impacts symptoms of schizophrenia, a serious mental health condition characterized by psychosis and other challenges. The study is sponsored by Bristol-Myers Squibb and focuses on adolescents experiencing active symptoms and meeting diagnostic criteria. Participants are randomly assigned to receive either KarXT or a matching placebo at specified doses on scheduled days. The study uses a parallel design with two groups one receiving the experimental drug KarXT and the other receiving placebo. Treatment and observation last through the study period, with key assessments taking place up to week 5. Participants will undergo evaluations including symptom severity scales such as the Positive and Negative Syndrome Scale PANSS, Clinical Global Impression scales, and the Childrens Global Assessment Scale CGAS. Researchers monitor changes from baseline in these measures to understand the impact of KarXT. Safety and symptom assessments occur throughout the trial, which runs until December 2029, allowing for careful monitoring of participant health and treatment effects.

Researchers are evaluating the efficacy and safety of tulisokibart in participants with moderately to severely active Crohns disease. This program includes two studies Study 1 involves both induction and maintenance treatment phases, while Study 2 focuses only on induction treatment. The main goal is to determine if one or more doses of tulisokibart are more effective than placebo in achieving clinical remission and endoscopic response at various time points up to Week 52. Participants are randomly assigned to receive different dosing regimens of tulisokibart or placebo. These regimens include high or low doses administered intravenously followed by subcutaneous injections, or subcutaneous injections alone. Some participants may continue in an extension phase receiving subcutaneous doses after completing their original treatment arm if they meet specific requirements. The studies use a double-blind design to compare tulisokibarts effects against placebo. During the trial, participants undergo regular assessments to measure clinical remission, endoscopic response, and other health outcomes using tools like the Crohns Disease Activity Index and stool frequency with abdominal pain scores. Safety evaluations include monitoring adverse events and treatment discontinuations. The studies last up to 52 weeks for Study 1 and 12 weeks for Study 2, with multiple visits to assess treatment effects and participant health under medical supervision.

Researchers are studying the long-term safety and tolerability of KarXT and KarX-EC in adolescents with schizophrenia and children and adolescents with autism-related irritability. This Phase 3, open-label study evaluates these treatments to better understand their effects over extended periods in these young populations. The trial is led by Karuna Therapeutics, Inc., a Bristol Myers Squibb company. Participants receive KarXT as the study drug, with dosing specified on certain days. The study includes two groups adolescents aged 13 to 17 years with schizophrenia receiving KarXT alone, and children and adolescents aged 5 to 17 years with irritability associated with autism spectrum disorder receiving KarXT combined with KarX-EC. The treatment period extends up to 54 weeks, during which safety and tolerability are closely monitored. During the study, participants are regularly evaluated for treatment-emergent adverse events, serious adverse events, and adverse events of special interest. Additional assessments include monitoring for procholinergic and anticholinergic symptoms, suicidal ideation and behavior, and movement disorders using validated rating scales. The total participation duration spans up to 54 weeks, encompassing treatment and observation to track long-term effects and safety outcomes.

Researchers are evaluating the effectiveness and safety of subcutaneous anifrolumab compared with a placebo in adults with moderate to severe Idiopathic Inflammatory Myopathies IIM, including polymyositis PM and dermatomyositis DM. This phase III, multicenter, randomized, placebo-controlled, and double-blind study aims to assess how adding anifrolumab to standard care affects overall disease activity in these patients. Participants will receive either anifrolumab or a matching placebo as a subcutaneous injection once a week for 52 weeks, alongside their standard of care treatments. After this initial period, all participants will be offered open-label anifrolumab once weekly for an additional 52 weeks, allowing further evaluation of long-term treatment effects. During the study, participants will be monitored through various assessments including muscle strength tests, disease activity scores, corticosteroid usage, and skin severity indexes over 52 weeks. The main outcome measure is the Total Improvement Score response at 52 weeks. Safety and disease activity will be carefully tracked throughout the treatment and follow-up periods, with study participation potentially lasting up to two years.

This research aims to evaluate how the medicine nerandomilast affects lung fibrosis in adults with systemic autoimmune rheumatic diseases who have lung fibrosis. The study includes adults 18 years and older who have not shown improvement in lung function after standard immunosuppressant treatment. Participants have interstitial lung disease related to rheumatic diseases such as rheumatoid arthritis, systemic sclerosis, idiopathic inflammatory myopathy, Sjgrens disease, or mixed connective tissue disease. Participants are randomly divided into two groups one group receives nerandomilast tablets and the other receives placebo tablets that look identical but contain no medicine. Tablets are taken twice daily for at least 26 weeks and up to 1 year. Participants continue their usual immunosuppressant treatments during the study. Participants stay in the study for about 7.5 to 13 months and visit the study site 9 to 10 times. During visits, lung function tests and chest imaging are performed, and participants complete questionnaires about symptoms and quality of life. Researchers compare results between the groups to assess the effects of nerandomilast while monitoring health and any side effects throughout the study.