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 PETCT 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 PETCT scan within four weeks after enrollment. No experimental treatments are administered since this is an observational study. Participants will undergo clinical evaluations and the PETCT 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 studying NKX019, an investigational allogeneic CD19-directed CAR NK cell therapy, to evaluate its safety and tolerability in adults with various autoimmune diseases. This Phase 12, open-label, multi-center study uses a dose escalation and expansion design to find recommended doses and assess preliminary effects including pharmacokinetics, pharmacodynamics, and immunogenicity. Participants will undergo a treatment cycle starting with lymphodepletion using fludarabine and cyclophosphamide before receiving three doses of NKX019. A modified lymphodepletion regimen using cyclophosphamide alone may be given to participants with low blood cell counts. The study includes dose escalation using a 33 design and subsequent enrollment of more participants at the recommended dose levels. During the study, participants will be closely monitored for side effects, treatment-related toxicities, and immune responses. Researchers will collect data on lung disease, systemic sclerosis, inflammatory muscle diseases, vasculitis, and rheumatoid arthritis up to two years after NKX019 infusion. Safety assessments cover the first 28 days after dosing and continue through 30 days after the last treatment. Total participation duration may extend to two years to observe long-term effects.

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 a new anti-CD19BCMA universal CAR-T cell therapy called RD06-05 in adults with various autoimmune diseases, including systemic lupus erythematosus SLE, lupus nephritis LN, ANCA-associated vasculitis AAV, anti-GBM disease, membranous nephropathy MN, systemic sclerosis SSc, idiopathic inflammatory myopathy IIM, and IgA nephropathy. This early phase 1, open-label, single-arm study aims to explore the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of this investigational cell therapy. Participants will receive the RD06-05 CAR T-cell therapy through an intravenous infusion following a preparatory lymphodepleting regimen using fludarabine and cyclophosphamide. The dose escalation design allows careful assessment of safety and biological effects. The study is planned to last up to approximately three years, with primary safety outcome monitoring over 2 years after treatment. During the study, participants will be closely monitored for adverse events, including treatment-emergent and serious side effects, as well as specific safety concerns related to cell therapy. Researchers will evaluate the therapys impact on disease activity and organ function through clinical assessments and laboratory tests. Participants will be followed for safety and efficacy outcomes until the study ends, expected in July 2028, with regular visits scheduled according to the study protocol.

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 adults with antineutrophil cytoplasmic antibody ANCA-associated vasculitis AAV, a condition requiring immunosuppressive therapy. This Phase 4 clinical trial aims to assess how participants tolerate avacopan combined with standard care over an extended period. The study involves participants diagnosed with granulomatosis with polyangiitis or microscopic polyangiitis who need induction treatment with cyclophosphamide or rituximab. Participants are randomly assigned to one of three groups avacopan 30 mg twice daily for five years plus standard care, avacopan 30 mg twice daily for one year followed by placebo twice daily for four years plus standard care, or placebo twice daily for five years plus standard care. Standard care involves background immunosuppressive therapy guided by current guidelines and tailored to each participants needs. Treatments are administered orally, and the study is double-blind to ensure objective assessment. During the study, participants will be monitored regularly for treatment-emergent adverse events, serious adverse events, and changes in vital signs and laboratory tests over up to 60 months. Researchers will also evaluate remission rates, relapse timing, kidney function, health perception scores, and medication use. Safety and efficacy data will be collected through clinical assessments, laboratory evaluations, and questionnaires, with follow-up continuing for the full duration of the trial.

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 people with ANCA-associated vasculitis, a condition that affects blood vessels and involves kidney inflammation. This multicenter, randomized controlled trial includes patients with biopsy-confirmed kidney involvement of ANCA vasculitis at diagnosis. The study aims mainly to see if pioglitazone can reduce kidney damage by improving protein levels in urine and serum creatinine. It also examines effects on blood pressure, metabolic changes from steroids, vasculitis activity, and safety in this group of patients. All participants receive standard immunosuppressive treatment combining corticosteroids and rituximab. They are randomly assigned to take either pioglitazone 30 mg daily or a placebo for 26 weeks alongside the standard care. Rituximab is given weekly for four weeks as induction, then re-administered every six months. Participants follow a predefined steroid tapering schedule. Biological samples are collected at multiple points throughout the study for analysis. Participants are followed for a total of 52 weeks with visits scheduled at weeks 1, 2, 3, 4, 8, 12, 26, 38, and 52. During these visits, kidney function, proteinuria, vasculitis activity, quality of life, and safety are assessed through clinical exams, blood and urine tests, and questionnaires. The primary outcome is measured at week 26, and secondary outcomes continue through week 52 to monitor effects and safety of pioglitazone over time.

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.