Sjogren's Syndrome

Researchers are studying pulmonary arterial hypertension PAH, including its common subtypes idiopathic PAH IPAH and connective tissue disease-associated PAH PAH-CTD. The study aims to develop pulmonary vascular biomarker signatures using hyperpolarized 129Xe MRI to differentiate these subtypes and to evaluate the ability of this imaging to monitor disease progression and treatment response over time. Additional assessments such as laboratory tests, echocardiography, and six-minute walk distance 6MWD are also used to support these evaluations. The study is observational and enrolls 20 subjects divided into two groups 10 with IPAH and 10 with PAH-CTD. Participants will undergo hyperpolarized 129Xe MRI and MR spectroscopy at baseline, 3 months, 6 months, and 12 months. Alongside imaging, data from standard clinical assessments including labs, echocardiography, and 6MWD will be collected at these same timepoints to track changes. Participants will be monitored for changes in pulmonary vascular remodeling, red blood cell oscillation amplitude, 6MWD, NTproBNP levels, and WHO functional class over one year. Safety is assessed by tracking adverse events, serious adverse events, withdrawals, and specific examinations such as electrocardiograms and vital signs. The total follow-up period for each participant is one year, with multiple scheduled evaluations to comprehensively assess disease status and response.

Researchers are studying the immunologic changes on the surface of the eye in cancer patients, focusing especially on those with ocular graft-versus-host disease oGVHD. This observational, natural history study aims to understand risk factors, mechanisms of eye damage, drug targets, and effects of oGVHD on the eye over a period of up to four years. Two groups will participate cancer patients scheduled for bone marrow transplant, who will be evaluated every three months for two years, and healthy control patients without eye disease or cancer history, who will be evaluated every six months. During each visit, standard eye exams will be performed alongside collection of biological specimens such as ocular surface wash, mucocellular material, corneal filaments, impression cytology of conjunctiva, blood, or serum. Participants will attend periodic study visits for clinical exams and specimen collection while receiving their usual care. Researchers will assess various factors including eye disease risk, mechanisms of eye damage, and ocular effects of graft-versus-host disease. The study involves repeated evaluations and biological sampling to track changes over time, with a maximum follow-up of four years.

Researchers are investigating the specific targets of T cells involved in autoimmune diseases by studying tissues from patients with active organ inflammation caused by autoimmune conditions. The study aims to identify which T cells are activated and expanding in diseased tissues compared to blood or normal tissues. This information will help discover new peptide targets and their associated T cell receptors TCRs to develop potential new therapies for autoimmune diseases. Participants will provide tissue samples and matched blood samples during clinical procedures such as endoscopy, arthrocentesis, lumbar puncture, skin biopsy, bronchoscopy, or surgery, depending on their autoimmune condition. The study includes several groups covering diseases like Crohns disease, ulcerative colitis, celiac disease, ankylosing spondylitis, multiple sclerosis, scleroderma, systemic sclerosis, and other autoimmune diseases. Samples may come from excess clinical materials or research-specific biopsies, with the possibility of serial sampling over time. During the study, participants will undergo standard clinical procedures with collection of additional tissue or fluid samples and companion blood draws. Researchers will analyze these samples to identify peptide targets linked to disease-reactive T cells over a period of up to three years. The study includes comprehensive assessments of tissues and blood to understand T cell activity in autoimmune disorders, with monitoring of participant safety and no interventions beyond routine clinical care.

Researchers are studying the safety and effectiveness of CAR NK cells for treating relapsed or refractory B-cell related autoimmune diseases. This early phase 1 pilot study plans to enroll 15 patients to evaluate the safety, feasibility, and potential benefits of this treatment. The study also explores how well the CAR NK cells expand, persist, and deplete B cells in these patients. Participants will receive a pre-treatment of Fludarabine and Cyclophosphamide on days -5, -4, and -3, followed by an infusion of CAR NK cells. This is a single-group, open-label trial where all participants receive the study treatment. The trial monitors patients for adverse events and dose-limiting toxicities within the first 4 weeks and up to 52 weeks after infusion. During the study, participants will be closely monitored through clinical assessments and laboratory tests at multiple time points up to one year after treatment. Researchers will measure safety outcomes including adverse events and toxicities, as well as effectiveness outcomes like disease control and remission rates. The total study duration may last up to approximately three years, including follow-up to assess long-term safety and treatment effects.

Researchers are evaluating the safety, tolerability, and initial effectiveness of CD19BCMA-targeted CAR-T cell therapy in patients with refractory autoimmune diseases. This early exploratory, open-label, single-arm clinical study focuses on adults aged 18 to 70 who have autoimmune conditions that have not responded to standard treatments. The study aims to explore whether this new in vivo CAR-T therapy approach can control disease activity without prior lymphodepletion. Participants will receive an intravenous infusion of the CD19BCMA-targeted CAR-T lentiviral vector drug. After infusion, the CAR-T cells are produced directly inside the patients body. No lymphodepleting chemotherapy will be given before treatment. Following the infusion, participants will be monitored for safety and efficacy over a 24-month period to assess disease control and treatment tolerability. During the study, participants will undergo regular safety assessments to monitor adverse events at multiple time points including Day 28, and Months 2, 3, 6, 12, 18, and 24. Researchers will track the maximum tolerated dose within 28 days after infusion. Additional evaluations will include clinical and laboratory tests to assess disease response and patient health. The total participation length can be up to two years, allowing long-term observation of treatment effects and safety.

Researchers are evaluating the long-term safety and tolerability of dazodalibep in people with Sjgrens Syndrome. This phase 3 open-label study extends previous trials by continuing to monitor participants who completed 48 weeks of treatment with dazodalibep or placebo. The study is sponsored by Amgen and aims to better understand the safety profile of dazodalibep over an extended period. Participants who finished the initial 48-week trials HZNP-DAZ-301 or HZNP-DAZ-303 will receive an assigned dose of dazodalibep intravenously for an additional 132 weeks. This extension study involves a single treatment group receiving dazodalibep without placebo, focusing on ongoing treatment effects and participant safety. During the study, participants will be monitored for treatment-emergent adverse events for up to 152 weeks. Researchers will also measure the presence of anti-drug antibodies and plasma concentrations of dazodalibep for up to 132 weeks. Participants need to be available for all study visits and procedures, with safety assessments conducted regularly throughout the long-term extension period.

Researchers are studying the use of unlicensed cryopreserved cord blood units CBUs for transplantation in both children and adults with blood cancers and other related disorders. This observational study involves patients with hematologic malignancies and various inherited and acquired disorders affecting the blood and immune system. The main goal is to monitor how well neutrophil recovery occurs after transplantation using these unlicensed CBUs in multiple institutions. Participants receive unlicensed cryopreserved CBUs as part of their transplant treatment. The study includes patients of any age receiving these CBUs for approved indications. The protocol focuses on the access and distribution of these unlicensed units rather than a specific treatment intervention. The study gathers data from recipients who receive these CBUs, tracking outcomes after transplantation. Participants are monitored for neutrophil recovery at 60 and 100 days after transplant, defined by a neutrophil count of at least 500mm3. Researchers also collect information on infection transmission, infusion reactions, survival rates at one year, and incidence of acute and chronic graft versus host disease. Platelet recovery is also evaluated. Safety and efficacy outcomes are followed over time to better understand the effects of unlicensed CBUs in this patient population.

Researchers are studying XmAb657 to evaluate its safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity in healthy adults and adults with autoimmune diseases such as idiopathic inflammatory myopathies, systemic sclerosis, and Sjogrens disease. This Phase 1, first-in-human study aims to understand how the drug behaves in the body and its potential effects. Participants will receive XmAb657 by subcutaneous injection under the skin in a dose-escalation design. The study includes both healthy volunteers and individuals diagnosed with specific autoimmune diseases. The treatment phase involves administering XmAb657 and monitoring responses over time. During the study, participants will undergo various assessments including safety monitoring for treatment-emergent and serious adverse events, blood tests to measure drug concentration, and evaluations of pharmacodynamic effects. Researchers will track these outcomes through Week 52. Participants are expected to follow contraception requirements and attend scheduled visits for monitoring throughout the study duration.

Researchers are evaluating anitocabtagene autoleucel anito-cel, a BCMA-directed CAR-T cell therapy, in adults with generalized myasthenia gravis GMG classified as MGFA Grade 2 to 4a. This Phase 1 open-label, multi-center study aims to assess the safety, tolerability, and preliminary efficacy of anito-cel in patients who require immunosuppressive therapy as determined by their neurologist. The study focuses on identifying dose-limiting toxicities DLTs, the maximum tolerated dose MTD, and selecting the recommended Phase 2 dose RP2D for treatment. The study includes sequential phases screening, enrollment with leukapheresis to collect cells, pretreatment with a standard lymphodepletion chemotherapy regimen for 5 days, and a single intravenous infusion of anito-cel cells. Optional bridging therapy may be given while the CAR-T cells are prepared. Following infusion, participants will be closely monitored for safety and efficacy outcomes over time. Participants will undergo safety monitoring for at least 28 days after infusion, with safety data collected throughout the study duration of up to 24 months. Clinical effects will be evaluated using measures including the Myasthenia Gravis Activities of Daily Living MG ADL score, Quantitative Myasthenia Gravis QMG score, and Myasthenia Gravis Composite MGC scale. Additionally, blood tests will monitor autoantibody levels and pharmacokinetics of the therapy. This comprehensive follow-up ensures careful assessment of treatment impact and participant health.

Researchers are evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and early clinical effects of CND319 in healthy adults and patients with rheumatic diseases such as rheumatoid arthritis and Sjogrens syndrome. This Phase 1, first-in-human study consists of two parts a dose escalation phase and an open-label expansion, aiming to better understand how CND319 behaves in the body and its initial impact on these conditions. Participants will receive CND319 according to the dosing schedule assigned to their cohort. The study includes single ascending dose SAD and multiple ascending dose MAD parts, with the open-label expansion providing further evaluation. The dose escalation part assesses increasing doses to understand safety and drug behavior, while the expansion phase allows additional participants to receive the drug openly. During the study, participants will be closely monitored through various assessments including vital signs body temperature, heart rate, respiratory rate, blood pressure, pulse oximetry, ECG parameters, and laboratory safety tests from baseline through up to 12 weeks for SAD or 24 weeks for MAD and open-label parts. Researchers will also track the presence of anti-drug antibodies and pharmacokinetic measures such as drug concentration and clearance. Total participation duration depends on the study part but may last up to 24 weeks.