Cryoglobulinemia

Researchers are establishing a prospective group of patients with plasma cell disorders (PCDs) to better understand these conditions and their treatments. This study focuses on various PCDs including multiple myeloma, amyloidosis, and other related disorders. It aims to explore how minimal residual disease (MRD) status relates to patient prognosis and investigate the role of the tumor microenvironment (TME) in the disease's development and progression. Patients with confirmed PCDs who are hospitalized and willing to participate will be followed over time. Biological samples such as peripheral blood, bone marrow aspirate, and urine will be collected before and after treatment to support future research. The study does not involve any intervention and participants will be regularly assessed at baseline, then at 1, 3, 6, and 12 months after treatment, followed by yearly visits. During the study, clinical and laboratory data along with biological samples will be gathered to analyze disease features and treatment responses. Follow-up visits will monitor patient progress and outcomes for up to ten years. The main outcome is to maintain a comprehensive cohort and understand the relationship between MRD and prognosis in PCD patients, while also supporting further studies on tumor clone evolution and disease mechanisms.

This research aims to better understand cutaneous vasculitis by studying skin lesions in patients with various types of idiopathic vasculitis. It involves a multi-center approach with both primary vasculitis care providers and dermatologists working together to select suitable patients and collect lesion samples for analysis. The study focuses on evaluating the histopathology and gene expression profiles (transcriptome) of these skin lesions. Participants with active vasculitis skin lesions will undergo a punch biopsy to collect tissue samples. These samples will be examined using standardized methods to assess the histopathology and transcriptomic characteristics of the cutaneous vasculitis. The study does not involve any experimental treatments but gathers detailed clinical data and biopsy specimens for analysis. Participants will be evaluated by specialists, who will collect clinical information and perform skin biopsies at affected sites. The study will review clinical data linked to biopsy samples over one year. Researchers will monitor the samples to better characterize the disease at the tissue level. The total participation time varies, with follow-up focused on the evaluation of biopsy results and clinical data.

Researchers are conducting a natural history study to collect blood samples from healthy adults to support research on diseases involving blood, blood vessels, and the immune system. The study aims to establish normal physiological parameters and validate technical tests for vascular inflammation and thrombosis. This research will help improve understanding and testing methods for vascular and immunologic diseases. Participants will provide blood samples during visits to the NIH Clinical Center, where about 10 tablespoons of blood will be drawn from a vein. Blood tests may include complete blood count and blood typing, and the samples will be used for genetic studies and other research assays. Some participants may return for repeat visits over a period of up to 10 years to provide additional samples and updated medical history. During the study, participants will have their medical history reviewed remotely via telehealth or telephone and in person during visits. The research team will monitor the collected blood samples to optimize assays and study vascular biomarkers. Participants will receive $50 for each blood collection visit. The study is designed to follow Good Clinical Practices and does not have a specific outcome measure but supports ongoing and future research.

Cryoglobulinemia vasculitis (CV) is a systemic immune-related small blood vessel inflammation that can relapse in many patients even after treatment with rituximab, which helps most but not all. Researchers are studying whether belimumab, compared to a placebo, is safe and effective for treating non-infectious active CV. This is a phase 2 multicenter randomized controlled trial sponsored by Assistance Publique - Hôpitaux de Paris. Participants are randomly assigned to receive either belimumab or placebo injections under the skin once a week for 24 weeks. Both groups follow the same plan to gradually reduce corticosteroid (prednisone) doses over 12 weeks, starting at 30 mg per day and tapering down if the disease remains inactive. After 12 weeks, stopping corticosteroids is decided by the doctor. This study is double-blinded, meaning neither participants nor researchers know who receives belimumab or placebo. During the study, participants undergo assessments at various time points including weeks 5, 13, 25, and 48 to measure clinical response, safety, disease activity, quality of life, and laboratory markers like cryoglobulins, rheumatoid factor, and complement levels. The main outcome is the rate of complete clinical response at week 25 after corticosteroid withdrawal. Participants are monitored for infections and relapses throughout the study, which helps researchers understand the treatment's effects and safety over nearly a year.

Researchers are collecting information from patients with rare kidney diseases to support research and improve care. This National Registry of Rare Kidney Diseases (RaDaR) aims to develop clinical guidelines, audit treatments and outcomes, and help develop future therapies by gathering comprehensive data. Rare kidney diseases often have genetic causes and affect patients from childhood into adulthood, but their rarity makes research and treatment development challenging. The registry gathers clinical data and biological samples from various rare kidney disease groups, each focusing on conditions like Alport Syndrome, APRT Deficiency, Polycystic Kidney Disease, and many others. It connects patients and clinicians and allows patients to contribute information about their quality of life. This infrastructure enables identification of patient groups for clinical trials, biomarker development, and genotype-phenotype studies. Participants provide clinical and disease-specific information over time, which supports epidemiological and translational research. The registry facilitates patient recruitment for studies, improves patient and clinician education, and provides access to current knowledge about rare kidney diseases. The registry is ongoing and primarily includes UK patients but also accepts international participants consented through UK NHS hospitals.

Researchers are studying patients with amyotrophic lateral sclerosis (ALS) who have an autoantibody against NRIP, which may cause harmful effects depending on its levels. This study evaluates the potential benefits of plasmapheresis, a procedure that removes this autoantibody from the blood, in this specific group of ALS patients. The trial is interventional and sponsored by National Taiwan University Hospital. Participants will receive regular plasmapheresis treatment aimed at removing the anti-NRIP autoantibody. The study focuses on ALS patients with different antibody titers and monitors changes in their condition before and after the intervention. There is no placebo group, and the treatment is delivered directly through the plasmapheresis procedure. During the study, participants will undergo serial examinations and follow-up visits to assess various measures including ALS functional rating scale scores over several time points, lung function, muscle nerve responses, and antibody levels. Safety is also monitored by recording any adverse effects during and after the plasmapheresis treatment, with the overall participation spanning at least six months.

Researchers are creating a registry of patients with plasma cell disorders (PCDs), such as multiple myeloma, to collect detailed assessments over time. The study aims to understand patient care patterns and satisfaction with these assessments, and to support future research in this patient group. An optional blood sample may be collected to study a genetic aging marker called p16INK4a. Participants complete a comprehensive assessment at the start and at regular intervals afterward. This assessment covers their ability to live independently, other medical conditions, cognitive and psychological health, social support, medications, and nutrition. Researchers also review patients' medical records to gather more information. During the study, patients are asked to complete questionnaires and assessments repeatedly to track changes over time. The primary outcomes include building the registry and completing these assessments over ten years. Secondary outcomes focus on patient response rates to the assessment and their satisfaction. The study is observational and involves no experimental treatments, with participation lasting up to ten years.

This research aims to understand the experiences of women with vasculitis who become pregnant. It focuses on assessing the severity of their vasculitis, their pregnancy-related experiences, and the outcomes of their pregnancies. The study is observational and seeks to gather detailed information through surveys over time. Women aged 18 to 50 years who have a diagnosis of vasculitis and are currently pregnant will be invited to participate. They will complete online questionnaires at several points: at study entry, during the second trimester, the third trimester, and after giving birth. These surveys take about 20 minutes each and are available in multiple languages including Spanish, Portuguese, Italian, and Turkish. Participants will provide information through these surveys, which will be securely stored and later analyzed by the study team. The main outcome measured is the unique pregnancy characteristics among women with vasculitis over a four-year period. This research involves no in-person visits and allows participants to contribute from home through their online responses.