Antiphospholipid 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 evaluating the effects of TAK-330 compared with a standard treatment known as four-factor prothrombin complex concentrate (4F-PCC) for reversing anticoagulation caused by Factor Xa inhibitors in adults needing urgent surgery or invasive procedures. This Phase 3 trial aims to determine which treatment better controls bleeding during and after surgery in patients who require rapid reversal of blood thinning effects. Participants will be randomly assigned to receive either TAK-330 or the standard 4F-PCC treatment before surgery. TAK-330 is given as a single intravenous infusion of 25 international units per kilogram on the day of surgery, with an optional additional dose during surgery if needed, not exceeding a total dose of 50 IU/kg or 5,000 IU. The standard 4F-PCC is administered according to local protocols, with a possible additional dose during surgery under similar dosing limits. During the study, patients will be hospitalized and monitored closely. Researchers will assess bleeding control during surgery, blood product use, and adverse events up to 30 days after surgery. Follow-up contact will be conducted by phone or telehealth 30 days post-surgery. The main outcome measured is the percentage of participants achieving effective bleeding control at the end of surgery.

Researchers are evaluating the long-term safety and effects of belimumab in adults with interstitial lung disease (ILD) linked to systemic sclerosis (SSc) and other connective tissue diseases (CTD). This open-label extension study builds on previous randomized controlled trials to see if belimumab can continue to slow lung function decline, disease progression, and improve quality of life in these patients. Participants in this study will receive belimumab as the investigational treatment. The study continues from prior trials, focusing on long-term use and tolerance. Participants will be monitored over time to assess how well belimumab is tolerated and its impact on lung function and overall disease status. During the study, researchers will track adverse events, including serious and special interest events, for up to approximately five years. They will also measure changes in lung function using forced vital capacity (FVC) at multiple time points: baseline, 12, 26, and 52 weeks. Participants will undergo regular assessments to monitor safety, disease progression, and quality of life throughout the study period.

Researchers are investigating a new treatment for sepsis-induced coagulopathy, a condition where an infection causes an overactive immune response leading to harmful blood clots and low platelet levels. This condition can damage blood vessels and organs, sometimes resulting in death. The study aims to learn about the safety, suitable dose, and effects of the drug BAY 3389934 in patients receiving intensive care for sepsis-induced coagulopathy. Participants will receive BAY 3389934 as a continuous intravenous infusion over up to 96 hours. They will be divided into two groups: the first group receives a low starting dose which may be adjusted based on safety and tolerability, while the second group will receive a higher dose if the first group experiences no serious side effects. This phase 1 study focuses on carefully monitoring how the drug affects participants. During the approximately 28-day study period, doctors will collect blood and urine samples, perform physical exams, and monitor vital signs such as body temperature, blood pressure, and heart rate. Heart health will be assessed using electrocardiograms (ECG). Researchers will track any medical problems that occur during and after treatment to evaluate safety, including treatment-emergent adverse events and their severity. They will also measure blood clotting times from day 1 to day 6 to understand the drug’s effects.

Connective tissue disease (CTD) is an autoimmune and inflammatory condition often linked with lung inflammation and scarring called interstitial lung disease (ILD). The number of people affected and deaths from CTD-ILD have increased recently. While corticosteroids and strong immunosuppressants help some patients, progressive lung fibrosis can lead to respiratory failure and may require lung transplantation. Stem cell therapy is emerging as a new approach for treating CTD-ILD, with promising reports using stem cells for ILD patients. This Phase I/IIa open-label trial involves intravenous transfusion of allogenic adipose tissue-derived mesenchymal stem cells (AD-MSC) in 10 patients with refractory or rapidly progressive ILD due to CTD. The first 3 participants will receive a low-dose infusion once; if no adverse effects occur after 4 weeks, the next 4 to 7 participants will receive medium-dose infusions twice; if still no adverse effects after 4 weeks, the last 2 to 3 participants will receive high-dose infusions three times. The goal is to improve outcomes and find the best dose of AD-MSC therapy. Participants will undergo initial evaluations before treatment, receive the stem cell infusions during admission, and then be followed up for safety and effectiveness for 48 weeks after the last infusion. Researchers will monitor safety throughout and assess lung function and other health indicators to evaluate treatment impact. The total study period includes treatment and nearly a year of post-infusion observation to track outcomes and any adverse effects.

Researchers are evaluating the safety, initial effectiveness, and pharmacokinetic and pharmacodynamic characteristics of YTS109 cell injections in people with recurrent or difficult-to-treat autoimmune diseases. This exploratory clinical trial focuses on patients who have conditions such as systemic lupus erythematosus, Sjogren's syndrome, systemic sclerosis, inflammatory myopathy, ANCA-associated vasculitis, and antiphospholipid syndrome. The study aims to better understand how YTS109 cells behave and impact these diseases when conventional treatments have not worked. Participants will receive a single injection of YTS109 cells at a dose of 3 million STAR+ T cells per kilogram of body weight. The treatment is administered once during the study. The trial does not include a placebo or comparison group and is conducted without masking or blinding. The study covers a treatment assessment period of 3 months, with additional evaluations extending up to 6 months for some measures. During the study, participants will undergo safety monitoring for treatment-emergent adverse events over 3 months. Researchers will measure efficacy outcomes for each autoimmune condition at 3 and 6 months. Blood tests will assess YTS109 cell levels and pharmacodynamics at 3 and 6 months as well. Participants will be closely followed for organ function, immune responses, and disease activity, with a total involvement period spanning up to 6 months after treatment.

Researchers are evaluating the safety and effects of YTS109 cells in adults aged 18 to 65 with relapsed or refractory autoimmune diseases, including Systemic Lupus Erythematosus (SLE), Lupus Nephritis (LN), Sjogren's Syndrome, Inflammatory Myopathy, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, and Antiphospholipid Syndrome. This phase 1, single-arm, open-label trial aims primarily to assess safety while also studying how well YTS109 cells work and their behavior in the body. About 18 patients will participate in this study conducted at Bengbu Third People's Hospital. Participants will receive a single infusion of YTS109 cells, starting at a dose of 3x10^6 STAR-T cells per kilogram, with dose escalation guided by a 3+3 principle. There are no placebo or comparator groups. The infusion is given once, and patients will be monitored closely for safety and treatment effects over several weeks. The study includes detailed follow-up periods to observe adverse events and treatment efficacy. During the study, participants will undergo evaluations at 2, 4, 8, 12, 24, and up to 52 weeks after treatment. These include assessments of adverse events, measurements of YTS109 cell levels in the blood at multiple time points, cytokine levels, and reconstitution of B cells in peripheral blood. Patients will provide informed consent and comply with study procedures, including use of contraception if applicable. The study also tracks various clinical and laboratory parameters to monitor safety and treatment response over time.

Researchers are investigating the safety and effectiveness of Anti-CD19 IL-10/IL15 CAR-NK cells in people with difficult-to-treat or relapsed autoimmune diseases. These diseases include systemic lupus erythematosus, systemic sclerosis, idiopathic inflammatory myositis, ANCA-associated vasculitis, Sjogren's syndrome, and antiphospholipid syndrome. This is a single-center, open-label trial designed to evaluate these cells in patients who have not responded well to standard treatments. Participants will receive conditioning treatment with Fludarabine and Cyclophosphamide before being given multiple doses of Anti-CD19 IL-10/IL15 CAR-NK cells. These cell infusions occur on Day 0, Day 3, and Day 6. The study includes a dose-escalation phase to assess safety and observe effects at different dose levels. During the trial, researchers will monitor participants for adverse events and dose-limiting toxicities within the first 28 days after infusion and over 12 months. They will also assess disease activity through specific scores and response criteria tailored to each autoimmune condition, such as SLEDAI-2K for lupus and mRSS for systemic sclerosis, over 12 to 48 weeks. Participants will be followed and evaluated regularly to track safety, treatment effects, and disease progression over time.

Researchers are evaluating the safety and effectiveness of an anti-CD38 antibody treatment for patients with antiphospholipid syndrome (APS) who also have secondary thrombocytopenia. This study focuses on patients who have not responded well or have relapsed after first-line treatment and at least one second-line therapy, including rituximab or thrombopoietin receptor agonists. APS is an autoimmune disorder that causes blood clots and pregnancy complications, associated with certain antibodies that increase clotting risk. Thrombocytopenia, a low platelet count, occurs in many APS patients and increases bleeding risk, making treatment challenging. The study involves a single group of 10 participants who will receive intravenous anti-CD38 antibody infusions at a dose of 16 mg/kg once a week for 8 weeks. This initial treatment phase is followed by a 16-week observation period to monitor the ongoing safety and continued effect of the antibody after treatment ends. The anti-CD38 antibody targets plasma cells and has been used in other autoimmune conditions and blood cancers, with early reports suggesting it may reduce antibody levels and improve platelet counts in APS. During the study, participants will be closely monitored for treatment response and safety. Researchers will assess platelet counts, antibody levels, bleeding episodes, and any clotting events. The primary goal is to evaluate how well participants respond to the treatment within 8 weeks and to observe safety for up to 24 weeks. Additional measures include time to platelet improvement, duration of response, and immune cell changes. This study offers an opportunity to explore a new therapeutic option for difficult-to-treat APS with thrombocytopenia over a total participation period of about 24 weeks.

Researchers are studying postpartum pulmonary artery pressure in women who experienced preeclampsia during pregnancy, focusing on those with and without obstetric antiphospholipid syndrome (oAPS). The study aims to understand if the combination of preeclampsia and oAPS increases the risk of long-lasting pulmonary hypertension. This observational study is conducted at a single center and follows women up to three years after delivery to explore cardiovascular outcomes and risks. Women in two groups—those with preeclampsia and oAPS, and those with preeclampsia without oAPS—are followed for three years postpartum. During this period, routine data including echocardiography images and 24-hour ambulatory blood pressure monitoring are collected. These diagnostic tests assess heart function and blood pressure to compare outcomes between the groups. Participants will undergo scheduled evaluations including clinical exams, echocardiography, blood pressure monitoring, and blood sampling. Researchers will analyze changes in systolic pulmonary artery pressure and systemic arterial blood pressure at three months and three years after delivery. The study also seeks to assess the prevalence of arterial hypertension and develop prediction models for cardiovascular risks in women who had preeclampsia. Data collection will continue through 2028, covering retrospective and prospective follow-up periods.