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 Crohn's 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 evaluating the safety and effectiveness of anti-CD19 CAR NK cells (KN5501) in patients with relapsed or refractory B-cell related autoimmune diseases. This early phase 1 pilot study plans to enroll 15 patients to assess the treatment's safety, feasibility, and effectiveness. The study also explores how well KN5501 expands, persists, and depletes CD19 positive B cells in these patients. Participants will receive a preparatory treatment with Fludarabine and Cyclophosphamide on three days before the infusion of anti-CD19 CAR NK cells. The trial includes two dose levels (6x10^9 and 9x10^9 cells) for dose escalation. This is a single-arm, open-label study without a placebo group. During the study, researchers will monitor participants closely for dose limiting toxicities and adverse events within the first 4 weeks and at 12, 24, 36, and 52 weeks after infusion. Effectiveness will be evaluated by disease control and remission rates at the same time points. Participants will undergo regular assessments including clinical evaluations and laboratory tests, with follow-up lasting up to one year after treatment.

Dry Eye Disease (DED) is a long-term condition caused by insufficient lubrication in the eyes due to low tear production or poor tear quality. This study aims to compare the safety and effectiveness of a new artificial tear formulation called ABBV-444 with Refresh Optive UD over a 90-day period in adults diagnosed with DED. The research involves around 250 participants across approximately 20 sites in the US to evaluate these treatments for managing dry eye symptoms. Participants first undergo a 7-day run-in period using REFRESH PLUS® eye drops. Those who qualify are then randomly assigned to receive either ABBV-444 or REFRESH OPTIVE® Unit Dose eye drops, both given as needed but at least twice daily for 90 days. This double-masked, randomized trial compares these two treatments during the study period to assess their impact on dry eye disease. Throughout the study, participants will attend multiple visits at the study sites for medical assessments and complete questionnaires to monitor treatment effects and side effects. Researchers will measure changes in the Ocular Surface Disease Index (OSDI) score and track any adverse events from baseline to day 90. Additional evaluations include tear breakup time and ocular surface staining to assess changes in eye surface health during the treatment period.

Researchers are evaluating the long-term safety and tolerability of dazodalibep in people with Sjögren's 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 pediatric and adult patients with various blood-related cancers and other disorders affecting the blood-forming system. This observational study aims to evaluate outcomes such as the recovery of a certain level of white blood cells after transplantation, as well as the incidence of infections, infusion reactions, survival rates, and graft-versus-host disease over time. The study involves patients receiving unlicensed CBUs at multiple U.S. transplant centers. These CBUs are used for patients with hematologic malignancies and other blood disorders. The protocol collects data on patients who receive these unlicensed transplant units, without administering a new treatment but observing the outcomes after transplantation. Participants will be monitored for neutrophil recovery at 60 and 100 days post-transplant, along with assessments of infection transmission, infusion reactions, survival one year after transplant, and occurrences of acute and chronic graft-versus-host disease. Platelet engraftment levels will also be tracked. The study includes patients of any age and follows them through the transplantation and recovery process to gather information on these key outcomes.

Researchers are studying the safety, tolerability, how the body processes, and immune response to XmAb657 in healthy adults and adults with autoimmune diseases such as Idiopathic Inflammatory Myopathies, Systemic Sclerosis, and Sjogren's Disease. This is a Phase 1, first-in-human, open-label clinical trial evaluating the effects of XmAb657 given by injection under the skin. The study aims to better understand the potential effects of XmAb657 in these populations. Participants will receive XmAb657 as a subcutaneous injection in a dose-escalation format. The study includes healthy adults and adults diagnosed with specific autoimmune diseases based on established classification criteria. The treatment is administered under medical supervision, and dosing levels may increase over time to monitor safety and immune responses. During the study, participants will be monitored for any side effects or adverse reactions, including serious or treatment-emergent events, through week 52. Blood samples will be collected to measure the levels of XmAb657 and assess its effects on the immune system. The study involves regular visits for safety assessments and pharmacokinetic and pharmacodynamic evaluations. Participants may be involved for up to a year to ensure thorough monitoring.

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.

This research aims to evaluate the safety and effectiveness of a topical treatment called recombinant human proteoglycan 4 (rhPRG4) in people with Sjögren's related Dry Eye Disease. The study is a phase II, prospective, randomized, and double-masked trial conducted across multiple centers in Australia. It focuses on improving symptoms and healing the eye surface in adults diagnosed with Sjögren's Syndrome who experience dry eye symptoms. Participants will be randomly assigned to receive either rhPRG4 at a concentration of 450 micrograms per milliliter or a placebo vehicle control. The treatment is applied topically to the eyes, and the study lasts 28 days. The trial compares the effects of rhPRG4 with the placebo to assess its impact on corneal damage and dry eye symptoms. During the study, participants will attend scheduled clinic visits where researchers will examine their eyes using fluorescein staining to measure corneal healing and perform other eye assessments such as slit lamp examination and intraocular pressure checks. Symptoms like dryness, burning, itching, and blurred vision will be recorded using questionnaires and visual scales. Safety will be closely monitored by observing any adverse events and changes in vision. The total study duration for each participant is 28 days from baseline to end of treatment.