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.

This research aims to evaluate the effects of TAK-330 compared to standard four-factor prothrombin complex concentrate 4F-PCC in adults treated with Factor Xa inhibitors who need urgent surgery or invasive procedures. The study focuses on reversing anticoagulation caused by Factor Xa inhibitors and is a Phase 3, randomized, triple-blind trial sponsored by Takeda. Participants will be randomly assigned to receive either TAK-330 or the standard 4F-PCC. TAK-330 is given as a single intravenous infusion dose of 25 IUkg before surgery, with an optional additional 25 IUkg dose during surgery if needed, not exceeding a total of 50 IUkg or 5,000 IU. The standard 4F-PCC dose and infusion speed follow local protocols, with an additional dose allowed during surgery under the same maximum limits. These treatments are administered during hospitalization prior to surgery. During the study, participants will be hospitalized for the surgery and treatment administration, with assessments of hemostasis effectiveness at surgery end and 24 hours after treatment. Researchers will monitor blood product use, adverse events including thrombotic events, and participant survival up to 30 days after surgery. Follow-up will also include contact via telehealth or phone 30 days post-surgery to evaluate safety and outcomes. Total participation duration covers the hospital stay and the 30-day post-surgery monitoring period.

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 drug called BAY 3389934 to find a better way to treat people with sepsis-induced coagulopathy, a serious condition where an active immune response causes uncontrolled blood clotting, damaging blood vessels and organs. This early-phase study aims to learn about the safety, appropriate dose, and effects of BAY 3389934 in patients receiving treatment for this condition in intensive care units. The research is focused on monitoring medical problems, called adverse events, that occur during and after treatment. Participants will receive BAY 3389934 as a continuous intravenous infusion for up to 96 hours. They will be divided into two groups the first group will receive a low starting dose, and their response will be closely monitored to adjust the dose if needed. If there are no serious side effects, the second group will receive a higher dose. This dose escalation approach helps determine the best dose for future studies. During the approximately 28-day study, participants will have blood and urine samples taken, physical exams, vital signs checked, and heart health monitored with electrocardiograms. Researchers will track the number and severity of any treatment-emergent adverse events within about four days after starting the infusion. They will also measure blood clotting times over the first six days to assess effects on coagulation. The study is designed to carefully observe safety and drug effects while patients receive intensive care.

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 IIIa 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, Sjogrens 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, Sjogrens 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 Peoples Hospital. Participants will receive a single infusion of YTS109 cells, starting at a dose of 3x106 STAR-T cells per kilogram, with dose escalation guided by a 33 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-10IL15 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, Sjogrens 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-10IL15 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 mgkg 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 groupsthose with preeclampsia and oAPS, and those with preeclampsia without oAPSare 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.