Takayasu Arteritis

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.

Takayasu arteritis (TAK) is a rare chronic inflammatory disease affecting arteries, and there is no widely accepted effective treatment strategy. Researchers classify TAK patients into three severity levels: mild, moderate, and severe. This study focuses on severe TAK patients and compares two biological agents, tocilizumab and adalimumab, to find which treatment may provide better intervention outcomes in this group. The study randomly assigns severe TAK patients to receive either tocilizumab at a dose of 8mg/kg intravenously every four weeks or adalimumab at 40mg biweekly by injection for 24 weeks. Patients start with prednisone at 40mg daily, tapered gradually over months, and treatment adjustments are made if disease relapse occurs. After 24 weeks, if remission is not achieved, patients switch to the other biological agent for an additional 24 weeks. Participants are monitored throughout treatment and follow-up for disease remission using clinical and laboratory assessments. Researchers track disease relapse, vascular progression through angiographic exams, prednisone dose reduction, and quality of life changes using questionnaires over a total of 48 weeks. The primary outcome is disease remission at 24 weeks. Safety and disease activity are closely observed during the study.

Researchers are evaluating the effectiveness and safety of a precision treatment plan tailored to clinical-molecular phenotypes compared with a conventional treatment approach in adults with active Takayasu's arteritis. This randomized, controlled, open-label, multicenter study aims to provide clearer insights into managing this condition by assessing outcomes over a 14-month period, including a 6-month induction remission phase and an 8-month maintenance phase. Participants will be randomly assigned to either the precision therapy group or the conventional therapy group. The precision group receives treatments based on their clinical-molecular phenotype: constitutional type patients get glucocorticoids combined with tocilizumab and methotrexate; vascular inflammation type patients receive combinations involving glucocorticoids plus tofacitinib, tocilizumab, adalimumab, and methotrexate depending on specific cytokine elevations. The conventional group receives traditional treatments based on clinical-molecular phenotypes. Prednisone dosing starts based on weight and is gradually tapered over the study. During the study, participants undergo evaluations including effectiveness rates at 6 and 12 months, relapse rates, safety monitoring, hormone dosage tracking, vascular imaging, and cytokine profile changes. Patient-reported outcomes like pain scores, quality of life surveys, and fatigue assessments are also collected. The study includes monitoring for adverse events and continues follow-up through the full 14-month period to assess long-term treatment effects and disease control.

Researchers are gathering detailed real-world information on people with chronic wounds and ulcers to better understand their medical journey and treatment outcomes. This observational registry collects data from over 1,400 healthcare providers across the USA and Puerto Rico using a specialized electronic health record system. It includes all major wound types such as diabetic foot ulcers, venous leg ulcers, pressure ulcers, arterial ulcers, surgical wounds, traumatic wounds, and inflammatory ulcers. Participants receive care as usual from their clinicians, who use standard and advanced wound treatments like specialized dressings, compression therapy, offloading devices, cellular and tissue-based products, negative pressure therapy, ultrasound therapy, topical oxygen, hyperbaric oxygen, growth factors, debridement, and topical antibiotics. Data on treatment practices, wound characteristics, patient health conditions, and complications are collected continuously during routine care. Patients may be followed for over five years to track long-term outcomes. During the study, researchers collect information on patient demographics, wound details, treatment methods, visit frequency, and complications. They monitor healing progress, amputations, deaths, wound recurrence, hospitalizations, and other outcomes. The registry uses a wound healing index to adjust for risk and supports quality improvement and research by linking clinical data with insurance claims for comprehensive analysis. This ongoing effort helps to better understand wound healing and treatment effectiveness in everyday healthcare settings.

Takayasu arteritis is a rare, chronic inflammation of the large arteries such as the aorta and its main branches. This condition can cause narrowing, blockage, or stretching of these vessels, sometimes leading to aneurysms. It often affects young Asian women and may remain undetected until vascular problems arise. Because many people with Takayasu arteritis show no clear symptoms despite active disease, researchers are seeking new biomarkers to improve diagnosis and monitor disease activity, aiming to better understand its underlying biological processes. This observational study focuses on identifying these biomarkers by enrolling individuals diagnosed with or suspected of having Takayasu arteritis. Participants include cases who meet specific classification criteria and controls. The study collects data retrospectively and prospectively, using imaging techniques like vascular magnetic resonance angiography or computed tomography angiography instead of traditional angiography. The study monitors disease status over time to uncover biochemical markers linked to disease activity. Participants will have their diagnosis and disease activity recorded from medical records within about three to six months after sample collection. These records and follow-up visits help assess the progression and activity of the disease. The study involves reviewing clinical data and imaging results to support biomarker discovery. Participation duration and follow-up depend on individual cases, with researchers closely tracking disease status to enhance understanding of Takayasu arteritis.

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.

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 conducting a nationwide registry in Armenia to observe and track systemic autoimmune and autoinflammatory diseases. These diseases involve abnormal immune system activity causing widespread inflammation and organ damage, often influenced by genetics and environmental factors. The study aims to better understand disease progression, which is usually measured by specific activity scores, and to gather detailed epidemiological and clinical data, as current knowledge in Armenia is limited. The study involves usual medical care for patients diagnosed with these systemic diseases, alongside collecting additional blood and stool samples for biobanking. Data collected include clinical exams, lab results, current treatments, and disease-specific activity scores reported by both patients and physicians. This observational registry will continue for about five years, assessing disease manifestations and evolution under treatment. Participants will be regularly evaluated through clinical and laboratory assessments, with data recorded on disease activity and progression. The study monitors systemic symptoms and uses disease-specific scores to track changes over time. Researchers will identify rare clinical forms and prognostic factors. The registry includes informed consent procedures and follows patients longitudinally to improve understanding of these complex diseases and their management.

Researchers are evaluating the safety and effectiveness of avatrombopag in adult Chinese patients with Immune Thrombocytopenia (ITP) who have autoantibodies and have not responded well or cannot tolerate treatments with eltrombopag or herombopag. This is a phase 2, open-label, single-center, one-arm clinical trial focusing on patients with various forms of ITP, including those linked to connective tissue diseases and Evans syndrome variants. Participants will receive avatrombopag starting at 40 mg once daily. Platelet counts will be monitored weekly during the first four weeks and then every two weeks up to week 12. Doses may be adjusted between 20 mg and 40 mg daily to keep platelet levels within a target range. If platelet counts remain low after four weeks at the initial dose, treatment will be discontinued. Throughout the 12-week treatment period, researchers will assess platelet response, duration, and time to response. Additional measurements include bleeding scores, disease activity in systemic lupus erythematosus, symptom improvements, immune function, glucocorticoid discontinuation, fatigue, and patient-reported outcomes. Safety and efficacy will be closely monitored during this time to understand avatrombopag's impact on these patients.

Takayasu arteritis is a chronic inflammation of large blood vessels, mainly affecting the aorta and its main branches. This research focuses on evaluating the outcomes of stopping biologic treatments in patients who have maintained long-term remission using these therapies. The study aims to understand how safe it is to discontinue biologic drugs like tumor necrosis factor inhibitors and tocilizumab after at least three years of use in patients with stable disease. Participants will undergo a specific 3-month dose tapering of their biologic medication. If they do not experience a relapse during this tapering phase, they will completely stop the biologic treatment. After stopping, patients will be followed for 12 months to monitor their health. Clinical and laboratory assessments will be done one month after stopping treatment and then every three months. Imaging tests will be done as needed based on clinical evaluations. Throughout the study, researchers will measure how many patients maintain remission 12 months after stopping biologic therapy. They will also track the rate and timing of disease relapse during both the tapering and follow-up periods. Additional analyses will explore factors that might predict relapse and differences related to the type of biologic agent used or other treatments. The total participation includes the tapering period plus a year of follow-up to closely observe patient outcomes.