Granulomatosis with Polyangiitis (Wegener's)

Researchers are investigating antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), a condition where lung and kidney involvement often leads to worse outcomes. This study aims to evaluate a new PET imaging tracer, 68Ga-Fibroblast Activation Protein Inhibitor (FAPI), which targets activated fibroblasts to detect disease activity and lesion distribution in AAV patients. Early and accurate diagnosis using this novel tracer could help personalize treatment and better assess disease severity and prognosis. The study will observe patients with AAV, including those with or without interstitial lung disease and renal dysfunction, using the 68Ga-FAPI PET/CT scan. This noninvasive imaging technique allows whole-body disease localization in a single exam. The study will compare disease severity and activity as measured by the PET/CT scan within four weeks after enrollment. No experimental treatments are administered since this is an observational study. Participants will undergo clinical evaluations and the PET/CT imaging to assess disease activity and severity. Researchers will collect clinical information and imaging data to provide insights on lesion distribution and organ involvement. The study requires informed consent and cooperation throughout the process. Total participation time varies, with the primary outcome measured within four weeks post-enrollment. Safety and compliance will be monitored during the study period, which runs until July 2028.

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 NKX019, an investigational allogeneic CAR NK cell therapy targeting CD19, in adults with various autoimmune diseases. This Phase 1/2 open-label, multi-center study aims to assess the safety and tolerability of NKX019, as well as preliminary efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity. The study includes participants with systemic sclerosis, idiopathic inflammatory myopathies, ANCA-associated vasculitis, and rheumatoid arthritis. Participants undergo a dose escalation phase following a "3+3" design to find the recommended dose for further study. Treatment cycles include lymphodepletion with fludarabine and cyclophosphamide, or cyclophosphamide alone for those with low blood cell counts, followed by three doses of NKX019. The study will expand to enroll more participants across different autoimmune conditions based on dose findings. During the study, participants will be monitored closely for adverse events and dose-limiting toxicities, especially within the first 28 days after the first NKX019 dose. Follow-up evaluations include laboratory tests, assessments of lung and muscle function, and immune response tracking for up to two years post-infusion. Researchers will also assess the impact of treatment on background therapies and measure drug levels and immune responses over time.

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.

Researchers are evaluating the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and effectiveness of a new CAR-T cell therapy called RD06-05. This therapy targets CD19 and BCMA markers and is being studied for several autoimmune diseases, including systemic lupus erythematosus (SLE) with lupus nephritis (LN), ANCA-associated vasculitis and glomerulonephritis, anti-glomerular basement membrane (GBM) disease, membranous nephropathy (MN), systemic sclerosis (SSc), and idiopathic inflammatory myopathy (IIM). The study is an early phase 1, exploratory, single-arm, open-label dose-escalation trial led by Nanjing Bioheng Biotech Co., Ltd. Participants will receive RD06-05 CAR-T cell therapy intravenously after a preparatory treatment with fludarabine and cyclophosphamide to reduce lymphocytes. The trial involves dose escalation to assess safety and tolerability with a focus on adverse events during treatment. The study treatment is given once, and patients will be monitored for two years to observe effects and side effects. During the study, participants will be closely monitored for adverse events, serious adverse events, and specific safety concerns related to the therapy. Researchers will assess blood tests, organ functions, and disease activity scores relevant to each autoimmune disease. Women of childbearing potential and their partners must use effective contraception during the study and for six months afterward. The total participation time includes the treatment and a two-year follow-up period to evaluate safety and treatment impact.

Researchers are evaluating the safety, tolerability, pharmacokinetics, and early effects of OL-108, an allogeneic CAR-T cell therapy, in adults with relapsed or refractory autoimmune diseases such as systemic lupus erythematosus (SLE), idiopathic inflammatory myopathy (IIM), systemic sclerosis (SSc), and ANCA-associated vasculitis (AAV). This open-label, single-arm clinical trial aims to understand how OL-108 works in these conditions and to assess any side effects. Participants will receive OL-108 through an intravenous bolus at increasing dose levels to find the maximum tolerated dose. The study includes a phase of lymphodepletion before OL-108 administration. The trial monitors the treatment from the start through up to two years to evaluate safety, immune response, and drug levels in the body. No placebo or comparison group is involved. During the study, participants will undergo regular assessments including monitoring for dose-limiting toxicities and treatment-related adverse events up to 30 days and two years respectively. Researchers will measure overall response rates, drug concentration peaks and durations, serum cytokines, immunogenicity, and replication-competent retrovirus levels. Safety and treatment effects will be carefully tracked throughout the study period, which may last up to two years after dosing.

Researchers are evaluating the long-term safety of avacopan in people with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), a condition involving inflammation of blood vessels. This phase 4 clinical trial focuses on assessing the safety and efficacy of avacopan over an extended period to understand its impact when combined with standard immunosuppressive treatments. Participants are randomly assigned to one of three groups: one group receives 30 mg of avacopan twice daily for 5 years along with standard care; another group takes avacopan for 1 year followed by placebo for 4 years, plus standard care; the third group receives placebo twice daily for 5 years combined with standard care. Standard care includes background immunosuppressive therapy tailored by the investigator according to guidelines and individual patient needs. Throughout the study, participants undergo regular monitoring for treatment-emergent adverse events, serious adverse events, and changes in vital signs, blood, urine, and serum chemistry over 60 months. Researchers also measure remission rates, relapse times, kidney function, quality of life, and glucocorticoid or immunosuppressant use. The study spans up to 5 years, with ongoing assessments to evaluate long-term safety and health outcomes.

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 evaluating the use of pioglitazone to improve kidney outcomes in patients with ANCA-associated vasculitis who have confirmed kidney involvement. This phase 3 multicenter randomized controlled trial aims to see if adding pioglitazone to standard immunosuppressive therapy can reduce kidney damage. The study also looks at whether pioglitazone affects blood pressure, metabolic side effects of steroids, disease activity, and its safety in this patient group. Participants will be randomly assigned to receive either pioglitazone 30 mg daily or a placebo for 26 weeks, along with standard care. Standard treatment includes rituximab infusions given weekly for four weeks, then every six months, plus a steroid tapering schedule starting with intravenous pulses followed by oral prednisone. The study includes a follow-up period lasting 52 weeks with scheduled visits at weeks 1, 2, 3, 4, 8, 12, 26, 38, and 52. Biological samples such as plasma, serum, and urine will be collected at several visits for analysis. Participants will have regular assessments including kidney function and protein levels in the urine, disease activity scores, quality of life questionnaires, and safety monitoring throughout the study. The main measurement is improvement in serum creatinine and urine protein by week 26. Safety and side effects, especially related to glucocorticoid use, will be followed until week 52. The full study duration for each participant is about one year from treatment start to final follow-up.

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.