44 Clinical Trials for Various Conditions
Wegener's granulomatosis (WG) is a rare disease that causes inflammation of blood vessels, or vasculitis. It may involve many different parts of the body, but typically affects the upper and lower respiratory tract and kidneys. The purpose of this study is to determine the safety and effectiveness of the medication abatacept in treating adults with mild relapsing WG.
Granulomatosis with polyangiitis (Wegener's) (GPA) and microscopic polyangiitis (MPA) are two rare immune system disorders that cause the inflammation of blood vessels, or vasculitis. In order to properly treat these diseases, it is critical that the level of disease activity can be determined over the course of the disease. The purpose of this study is to determine new biological markers, or biomarkers, that may be used to assess the severity of disease in people with GPA or MPA.
Multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy of abatacept to achieve sustained glucocorticoid-free remission in patients with relapsing non-severe granulomatosis with polyangiitis (Wegener's) (GPA) . Participants will be randomized 1:1 to receive either abatacept 125 mg or placebo administered by subcutaneous injection once a week. Participants will continue on study treatment for a minimum of 12 months unless they experience a disease relapse or disease flare. Participants who experience a non-severe disease relapse, non-severe disease worsening, or who have not achieved remission by month 6 will have the option of entering an open-label trial period whereby they would receive open-label abatacept.
This Phase IIa international multicenter, open-label, uncontrolled study will evaluate the safety and pharmacokinetics of rituximab (MabThera/Rituxan) in pediatric participants with severe granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). Participants will receive rituximab 375 milligrams per square meter (mg/m\^2) intravenously (IV) on Days 1, 8, 15 and 22.
This prospective observational study will evaluate the long-term safety of MabThera/Rituxan (rituximab) in participants with granulomatosis with polyangiitis (Wegener's) or microscopic polyangiitis. Data will be collected for a maximum of 4 years from participants initiated on MabThera/Rituxan therapy by their physician according to prescribing information.
Antineutrophil cytoplasmic antibodies (ANCA)-associated vasculitis is the most common type of small blood vessel inflammation in adults. ANCA-associated vasculitis includes Wegener's granulomatosis (WG) and microscopic polyangiitis (MPA). Rituximab is a man-made antibody used to treat certain types of cancer. The purpose of this study is to determine the effectiveness of rituximab in treating patients with WG and MPA. Study hypothesis: Rituximab is not inferior to conventional therapy in its ability to induce disease remission by Month 6.
This study will examine the use of rituximab in patients with Wegener's granulomatosis (WG) who have experienced a relapse of the disease through standard therapies. Rituximab is an antibody directed against the human protein called CD20, found on the surface of normal and abnormal B lymphocytes. Rituximab decreases the number of B lymphocytes. This study will examine the safety of rituximab in WG and rituximab's ability to reduce the level of circulating antineutrophil cytoplasmic antibodies (ANCA), which are antibodies that react to substances found in white blood cells. ANCA have been found to be strongly associated with WG. The study will also explore whether rituximab can reduce the occurrence of disease relapse. WG is a disease marked by inflammation of blood vessels. It can involve many different parts of the body, including the sinuses, lungs, kidneys, brain, nerves, eyes, intestinal tract, skin, joint, heart, and others. Before the use of cytotoxic drug therapy, WG was almost always fatal if untreated, with a mortality rate of 93% within 2 years. Patients 18 to 75 years of age who have a history of at least one relapse of the disease despite standard treatments, who have had active WG within the previous 12 months and are in remission, who are receiving either methotrexate or azathioprine for remission maintenance, and who have circulating ANCA, may be eligible for this study. A minimum of 22 visits to the clinic will be required to complete the entire study. Patients will undergo a comprehensive medical evaluation, with laboratory studies and x-rays. There may also be consultations and possible biopsies of affected organs only if medically indicated for diagnosis and treatment of the disease. In the 4-week period that patients will receive rituximab infusions, the methotrexate or azathioprine will be continued at the same dosage unless there are side effects that requite the medication to be temporarily stopped or the dosage reduced. Patients will receive four doses of rituximab, at 375 mg per meter squared of body surface area, once a week. It will be infused into a vein, through an intravenous catheter. For the first dose, patients will be admitted as inpatients for at least 24 hours, for monitoring during the infusion and for any reactions associated with it. The second, third, and fourth rituximab infusions may be given either on an inpatient or outpatient basis to be decided on how the patient tolerates the first infusion. Following the four infusions, there will be blood tests to monitor the safety of the medication and the status of the disease, to be done at home every week for 4 weeks. Results will be sent to the researchers by fax. Patients will be asked to return to the clinic 1 month after the fourth infusion and every 1 to 3 months afterward. If there are no side effects or a relapse of the disease, the methotrexate or azathioprine will be continued for 2 years past remission. If by then the disease then remains in remission, the dose of either medication will be gradually decreased and eventually stopped. The usual schedule is to reduce methotrexate by 2.5 mg per month and to reduce azathioprine by 25 mg per month. If at that point there are no signs of active disease, the patients' illness will be considered to be in continued remission and no further treatment will be necessary. If relapse does occur, treatment would be different than previously. In most cases, treatment would involve prednisone and cyclophosphamide or methotrexate If the ANCA finding is negative after rituximab treatment and again becomes positive, and there is evidence of a return of B lymphocytes, patients may receive a second course of four rituximab infusions.
This study will examine the safety and effectiveness of daclizumab (also called Zenapax or anti-CD25) in patients with Wegener's granulomatosis, a type of vasculitis (blood vessel inflammation). Wegener's granulomatosis can affect many parts of the body, including the brain, nerves, eyes, sinuses, lungs, kidneys, intestinal tract, skin, joints, heart, and other sites. Standard treatment is a combination of prednisone and a cytotoxic agent (a drug that interferes with cell growth), usually cyclophosphamide or methotrexate. However, many patients treated with this regimen have a disease relapse, and others cannot take these drugs because of severe side effects. This study will focus on the effectiveness of daclizumab in preventing disease relapse. The Food and Drug Administration approved daclizumab in 1997 for preventing kidney transplant rejection, and the drug has also been studied in people with an eye infection called uveitis. The drug works by binding to a protein on T lymphocytes (white blood cells of the immune system) called CD25. This prevents another protein, called interleukin-2, from binding to this site, thereby preventing a series of events that normally results in inflammation. Patients between 10 and 75 years of age with Wegener's granulomatosis may be eligible for this study. Participants will have a medical history review and physical examination, including laboratory studies. If medically indicated, x-rays, consultations and biopsies (surgical removal of a small tissue sample) of affected organs will also be conducted. All patients will begin treatment with prednisone and cyclophosphamide daily. Those who improve on this regimen will reduce the prednisone gradually and continue with cyclophosphamide until their disease is in remission. While taking cyclophosphamide, patients must have blood and urine tests done every 1 to 2 weeks. Those who achieve disease remission will stop cyclophosphamide and start taking methotrexate once a week, usually by mouth but possibly by injection into the muscle or skin. Blood and urine tests will be conducted once a week for 4 weeks while the dosage is being adjusted and then once a month for the duration of treatment. Patients on methotrexate whose prednisone dose is reduced to 10 to 30 mg every other day will be randomly assigned either to receive or not receive daclizumab in addition to the methotrexate. Daclizumab is given intravenously (through a plastic tube inserted into a vein) the day after the randomization, then again in 2 weeks, 4 weeks, and once a month for 18 months. All patients will continue to taper their prednisone dose until it is stopped. Methotrexate will continue for 2 years. Patients whose disease remains in remission at this time will decrease the methotrexate dose. If there is no active disease when both prednisone and methotrexate have been stopped, no further treatment will be given. If disease recurs at a later time, treatment will be reinstituted. The treatment will be determined by the severity of disease, other medical conditions, and history of side effects. Patients not randomized to daclizumab who relapse while still taking methotrexate may be offered re-treatment with daclizumab. Patients will be evaluated in the outpatient clinic every 4 to 8 weeks until randomization. Patients not taking daclizumab will be followed every 4 to 12 weeks; those taking the drug will be seen every 2 weeks for the first month, every month after that during the 18-month treatment period, and every 4 to 12 weeks until all medications stop. Follow-up evaluations include a physical examination, blood draws and, if medically indicated, X-rays. The total study duration is 60 to 70 months.
This study will determine if the drug etanercept, also called Enbrel, is effective in producing and maintaining remission (reduction of disease symptoms) of Wegener's granulomatosis (WG). Etanercept blocks the action of tumor necrosis factor-alpha, a substance that may be involved in inflammatory conditions such as WG. Eight clinical centers around the United States will enroll 181 people who have WG. Patients will have an equal chance to receive either etanercept or placebo (inactive treatment). We will treat patients with standard medications for WG in addition to either etanercept or placebo. We will treat all patients with tapering doses of corticosteroids. After the patients' disease is controlled (in remission), we will reduce the dosages of the standard medications to lower the risk of side effects associated with these drugs. During the study, we will collect and save blood and tissues samples from patients and use the samples to address other medical questions, such as the cause of WG and factors that lead to disease progression.
This study will compare the safety and effectiveness of two drugs-methotrexate and mycophenolate mofetil (MPM)-in preventing disease recurrence in patients with Wegener's granulomatosis and related inflammatory blood vessel disorders. The standard treatment for these conditions is combination drug therapy with prednisone plus cyclophosphamide. However, although most patients improve on this therapy and achieve disease remission, many experience a relapse (return of the disease) some time after therapy is stopped. Also, these drugs can produce serious side effects during treatment. This study will test a new treatment regimen to try to maintain disease remission in these patients with minimal side effects. Patients with Wegener's granulomatosis or other related blood vessel disorders between 10 and 80 years old will be considered for this study. All participants will start therapy with daily doses of prednisone and cyclophosphamide. Prednisone will be reduced gradually and then stopped after symptoms improve significantly. Cyclophosphamide will continue until the disease is in remission. Patients in remission will then be randomly assigned to continue treatment with either MPM or methotrexate. MPM is taken twice a day by mouth. Methotrexate is taken once a week, usually by mouth, but in some cases, by injection into a muscle or under the skin. Patients who do well and have no side effects will continue treatment for 2 years. Then, the drug will gradually be reduced (usually at monthly intervals) and finally stopped. No further treatment will be given unless a relapse occurs. At that time, the type of treatment will depend on various medical factors, including the severity of the recurrence and the patient's history of drug side effects. Physical examinations and various tests, including blood and urine analyses, and X-rays, will be done periodically to evaluate the response to treatment and monitor drug side effects. The total duration of the study-from the screening evaluation through a 2-year follow up after all medications have been stopped-is about 5 to 6 years.
This study will examine the use of etanercept (also called Enbrel or TNFR:Fc) in patients with Wegener's granulomatosis, a type of vasculitis (blood vessel inflammation). Wegener's granulomatosis may affect many parts of the body, including the brain, nerves, eyes, sinuses, lungs, kidneys, intestinal tract, skin, joints, heart, and other sites. Generally, the greater the disease involvement, the more life-threatening it is. Standard treatment is a combination of prednisone and a cytotoxic agent-usually cyclophosphamide or methotrexate. However, many patients treated with this regimen have a disease relapse, and others cannot take these drugs because of severe side effects. This study will evaluate etanercept's safety and effectiveness, and particularly its value in reducing the need for prednisone and preventing disease relapse. The Food and Drug Administration has approved etanercept for treating rheumatoid arthritis, another inflammatory disease. The drug works by blocking the activity of TNF-a protein made by white blood cells that is involved in the inflammatory process. Since prednisone also affects inflammatory proteins and lowers TNF production, the use of etanercept may reduce the need for prednisone in patients with Wegener's granulomatosis, and thus the risk of its side effects. Patients between 10 and 70 years of age with Wegener's granulomatosis who have never taken prednisone, methotrexate or cyclophosphamide, or have taken these drugs for less than 3 weeks may be eligible for this study. Participants will have a medical history review and physical examination, including laboratory studies. If medically indicated, X-rays, consultations and biopsies (surgical removal of a small tissue sample) of affected organs will also be done. All patients will begin treatment with prednisone, methotrexate and etanercept. Those who improve on this regimen will stop prednisone gradually over 3 months. Those who achieve disease remission at the end of another 3 months will be randomly assigned to either continue taking etanercept and methotrexate for another 12 months or to stop etanercept and continue only methotrexate for the next 12 months (after which methotrexate will gradually be stopped). Patients who are not in remission by the 6-month point will continue taking etanercept until they go into remission, when they will be assigned to stop or not stop etanercept, as described above. Patients who do not achieve remission within 12 months of beginning treatment will be taken off the study. Patients who have a disease relapse while on the study will likely be switched to treatment with prednisone and either methotrexate or cyclophosphamide. Patients randomized to stop etanercept and who have a relapse within a year of stopping the drug may be offered re-treatment on this protocol, but with continuing etanercept for a full year after remission. Patients will be evaluated in the outpatient clinic every 2 to 4 weeks for the first 4 months and every 1 to 3 months after that. Patients whose disease is in remission and who stop all medications will be followed every 3 to 6 months for 2 years. Follow-up evaluations include a physical examination, blood draws and, if medically indicated, X-rays. The total study duration is 60 to 70 months.
This study will examine the safety and effectiveness of the drug mycophenolate mofetil (MPM) in treating Wegener's granulomatosis and related inflammatory vessel diseases. Blood vessel inflammation in these patients may involve different parts of the body, including the brain, nerves, eyes, sinuses, lungs, kidneys, intestinal tract, skin, joints, heart, and other sites. The more severe the involvement, the more likely the disease will be life-threatening. Standard treatment consists of combination drug therapy with prednisone and a cytotoxic agent-usually cyclophosphamide or methotrexate. However, some patients in whom this treatment is initially successful have a disease relapse; other patients cannot take the medications because of other health problems or because of severe side effects of the drugs. MPM is approved by the Food and Drug Administration to prevent kidney transplant rejection. It is chemically similar to another cytotoxic drug called azathioprine, which has been beneficial in maintaining remission in patients with Wegener's granulomatosis who have been treated successfully with cyclophosphamide. Because MPM is more effective than azathioprine in preventing organ rejection, it may also prove beneficial as a second-line treatment for Wegener's granulomatosis. Patients with Wegener's granulomatosis or related inflammatory vessel diseases who have had a relapse following treatment with cyclophosphamide and methotrexate or who cannot take one or both of these drugs may be eligible for this study. Only patients who have been treated at NIH in the methotrexate protocol or the cyclophosphamide switching to methotrexate protocol, or who have received the exact same treatment from their own physician may participate. Participants will have a complete medical evaluation including laboratory studies. Consultations, X-rays and biopsies of affected organs may also be done if indicated for diagnosis or treatment. Patients with active disease will be given MPM and prednisone, both in tablet form. Patients with inactive disease will receive only prednisone if they are already taking it. In both cases, the prednisone will be reduced gradually and discontinued if the disease improves significantly. MPM therapy will continue for at least 2 years. If after 2 years the disease remains in remission, the MPM dose will be gradually reduced and then stopped. If active disease recurs while on MPM therapy, the treatment plan will likely be changed. The new regimen will be determined by the severity of disease, other medical conditions, and history of side effects to previous medications. Patients will be followed at the NIH clinic every month for the first 3 months on MPM and then every 3 months for another 18 months. Those whose disease has remained in remission and have stopped all medications will then be followed every 6 months for 4 visits. The follow-up visits will include a physical examination, blood draws, and, if needed, X-rays. Visits may be scheduled more frequently if medically indicated.
The purpose of this study is to assess the safety, tolerance, and immunologic effects of interleukin-10 (IL-10), in patients with Wegener's granulomatosis. A secondary objective is to determine if IL-10 demonstrates sufficient anti-inflammatory activity in the treatment of Wegener's granulomatosis to warrant further study in a larger trial. In this study, IL-10 will be given either alone or in combination with standard therapeutic agents, usually consisting of cyclophosphamide, methotrexate, and/or prednisone. Patients will be eligible to receive IL-10 when there is evidence of active disease. IL-10 will be administered by subcutaneous injection at a dose of 4 µ (Micro)g/kg/day for 28 days.
This study will collect fluid and tissue samples from the bronchi (air passages to the lungs) of patients with Wegener's granulomatosis for laboratory examination. Wegener's granulomatosis is a type of vasculitis (blood vessel inflammation) that can affect many parts of the body, including the brain, nerves, eyes, skin, sinuses, kidneys, intestinal tract, joints, heart, lungs and other sites. About 85 percent of patients have lung involvement. The lining of the bronchi (bronchial mucosa) is composed of cells that produce mucus, inflammatory cells, and inflammatory mediators (chemicals produced in response to inflammation). Analysis of these various substances may provide insight into what causes different types of lung problems in Wegener's granulomatosis. Patients between 18 and 75 years of age with Wegener's granulomatosis who require bronchoscopy to evaluate the cause of their lung problem may be eligible for this study. Participants will undergo a bronchoscopy in the hospital intensive care unit (ICU). For this procedure, the mouth and throat are numbed with lidocaine jelly and spray. If needed, a sedative is given for comfort. A small plastic tube (intravenous catheter) is placed in a vein to give medications. A pencil-thin tube is then placed through the nose or mouth into the lung airways to examine the airways carefully. At the time of the bronchoscopy, patients in this study will undergo the following additional procedures: * Bronchoalveolar lavage - Saline (salt water) is injected through the bronchoscope into the air passage, acting as a rinse. A sample of the fluid is then withdrawn and examined for infection, inflammatory cells and inflammatory chemicals. (This may be done as part of the standard medical care procedure.) * Bronchial lavage - This procedure is similar to bronchoalveolar lavage, but less fluid is used to rinse larger airways. * Bronchial mucosal biopsies - A small wire is inserted through the bronchoscope next to the bronchial lining. Forceps at the end of the wire pinch off a small piece of tissue for withdrawal and examination. The patient's heart rhythm and rate and oxygen levels are monitored during the procedure. When the procedures are finished, the patient is monitored in the ICU until the numbing effect of the anesthetic has worn off and then moves to a regular hospital bed for overnight. Patients whose test results show an isolated infection or isolated Wegener's lung tissue involvement and who are being treated or are eligible for treatment under another NIH protocol will be followed by X-ray for improvement of their infection or other lung involvement with treatment. Patients whose infection or lung tissue involvement improves may be asked to undergo a second bronchoscopy as described above, but for research purposes only.
The purpose of this protocol is to continue to treat patients with Wegener's granulomatosis who have already entered the study, and to treat new patients. The investigators will attempt to correlate the clinical response with specific immunosuppressive effects of drug administration. The investigators are accumulating data on the optimal duration and side effects of therapy.
The purpose of this study is to determine whether plasma exchange as well as immunosuppressive therapy are effective in reducing death and end-stage renal disease (ESRD). The trial will also study whether a reduced cumulative dosing regimen of glucocorticoids is as effective as a standard disease regimen. The FDA-OOPD is one of the funding sources for this study.
This is a phase IV, single-center, randomized, placebo-controlled pilot study that will evaluate the efficacy of rituximab at inducing otolaryngologic remission in GPA patients with active otolaryngologic disease.
The purpose of this study is to learn about how patients with vasculitis think about their illness and to assess to what extent patient perceptions of illness are associated with physical, mental, and social functioning
Childhood chronic vasculitis describes a group of rare life-threatening diseases that have in common inflammation of blood vessels in vital organs such as kidneys, lungs and brain. Most knowledge about them comes from adult patients. Severe disease requires aggressive life-saving treatments with steroids and some cancer drugs which can themselves cause damage, and increase risks of cancer and severe infections. Conversely, milder disease can be treated with less toxic drugs. Different classification and "scoring tools" are used to define the types and severity of vasculitis and to measure damage caused by disease or drugs. These in turn help direct how aggressively to treat a patient and to measure outcome. None of these tools however have been assessed in children and the best balance of disease and treatment risks against outcome for children is not known. Although causes of these diseases in children and adults are probably the same, the effects of the disease and the response (good and bad) to drugs will differ in growing children. Because specialists may see only one new child with vasculitis each year, obtaining enough information to learn about childhood vasculitis requires cooperation. We will use an international web-based registry to which doctors from 50 or more centers can contribute patient data. We will determine the features which help better classify and diagnose children compared to adults. Through the web we will collect and analyze information on patients similarly classified and "scored" so that most successful treatments can be identified. Children with vasculitis are less likely to have diseases associated with aging, alcohol and smoking etc., and therefore may be a better group in whom to study the underlying biology of vasculitis. We will use this opportunity and collect spit, blood and tissue from registry patients for laboratory study with an aim to find biomarkers to better classify, define and direct optimal treatment and outcomes.
Vasculitis is group of diseases where inflammation of blood vessels is the common feature. Patients typically present with fever, fatigue, weakness and muscle and joint aches. These symptoms are very common among many different diseases, not just vasculitis. A clustering of other symptoms, physical examination findings, blood tests, radiology and biopsy help make the diagnosis. There are currently no criteria to help doctors make a diagnosis of vasculitis when a patient presents with these non specific symptoms and they are reliant on previous experience and disease definitions. One of the aims of this project is to develop diagnostic criteria for the primary systemic vasculitides (granulomatosis with polyangiitis (Wegener's), microscopic polyangiitis, Churg Strauss syndrome, polyarteritis nodosa, giant cell arteritis, Takayasu arteritis). We, the investigators, will do this by studying a large group of patients with vasculitis and comparing them to a large group of patients that present in a similar way, but do not have vasculitis. By comparing the 2 groups we will create a list of items to differentiate between vasculitis and 'vasculitis mimics'. We also aim to update the current classification criteria. Classification criteria are used to group patients into different types of vasculitis, once a diagnosis of vasculitis has been made, and are useful for studying patients in clinical trials with similar or identical diseases. The current classification criteria (American college of Rheumatology 1990 criteria) were developed 20 years ago, before the availability of some important diagnostic tests (e.g. antineutrophil cytoplasmic antibodies \[ANCA\]), and are now not consistent with some of the current disease definitions. Therefore to progress future research in vasculitis, it is important that the classification criteria are updated. We will recruit 260 patients with each of the 6 types of vasculitis and compare them with 1300 controls (patients with the 5 other types of vasculitis), in order to determine the optimal combination of symptoms, signs and investigations that classify each person into the appropriate group.
Patient Power is a patient research network and database (registry) to collect prospective information about demographics, self-reported diagnoses and medications, and willingness to participate in research from participants with rheumatoid arthritis (RA), spondyloarthritis (SpA), other musculoskeletal conditions, chronic neurological conditions like migraine, chronic pulmonary conditions like Chronic Obstructive Pulmonary Disease (COPD), asthma, autoimmune dermatological conditions such as psoriasis, and other chronic inflammatory or immune-mediated conditions. In addition, since patients with chronic conditions often have other co-morbidities like cardiovascular health and obesity-related metabolic disorders, these conditions will also be included. Participants will provide information from their smartphones or personal computers. The information will be used by researchers and clinicians to help patients and their providers make better, more informed decisions about treatment of chronic conditions.
This is a randomized controlled trial in patients with a diagnosis of granulomatosis with polyangiitis (GPA; Wegener's)that are in remission to evaluate the effects of using low-dose glucocorticoids ( 5 mg/day of prednisone) as compared to stopping glucocorticoid treatment entirely (0 mg/day of prednisone)on rates of disease relapse/disease flares. This study is a novel approach to conducting a randomized clinical trial in the community setting. This study is being conducted in parallel with a similar study at established vasculitis institutions. This study will have a patient centric approach to research in that subjects will be recruited online and through social media and vasculitis support networks. Participants will be consented online and will receive care through their regular treating physician so no travel or additional doctor visits are required. Study participants will consent to the study and complete online questionnaires about their prednisone dose and about how they are feeling.
Rituximab is now established as an effective drug for anti-neutrophil cytoplasmic antibody (ANCA) vasculitis following major European and US trials reported in 2010. After a time, its effect wears off and the disease can return. This occurs in at least half of patients within 2 years of receiving Rituximab. A preliminary study in Cambridge has suggested that repeating rituximab every six months stops the disease returning and is safe. The RITAZAREM trial will find out whether repeating rituximab stops vasculitis returning and whether it works better than the older treatments, azathioprine or methotrexate. It will also tell us how long patients remain well after the repeated rituximab treatments are stopped, and if repeated rituximab is safe. We should also learn useful information about the effects of rituximab on quality of life and economic measures. The trial results will help decide the best treatment for future patients who have their vasculitis initially treated with rituximab. RITAZAREM aims to recruit patients with established ANCA vasculitis whose disease has come back 'relapsing vasculitis'. All patients will be treated with rituximab and steroids and we anticipate that most will respond well. If their disease is under reasonable control after four months, further treatment with either rituximab (a single dose ever four months for two years) or azathioprine tablets will be chosen randomly. The patients in the rituximab and azathioprine groups will then be compared. Patients will be in the trial for four years. The study has been designed by members of the European Vasculitis Study group (EUVAS) and the Vasculitis Clinical Research Consortium (VCRC). It will include 190 participants from 30 hospitals in Europe, the USA, Australia and Mexico. RITAZAREM is being funded by Arthritis Research UK, the U.S. National Institutes of Health and by Roche/Genentech.
Granulomatosis with polyangiitis (GPA; Wegener's) is a multi-organ autoimmune disease characterized by necrotizing granulomatous inflammation and vasculitis. Upper respiratory involvement occurs in up to 90% of patients with GPA and is often the first manifestation of the disease. Patients with upper respiratory tract disease are more at risk of local and systemic relapse. Microbial organisms may be involved in inducing disease activity in GPA. Previous culture-dependent studies found that patients with GPA were more likely to be chronic nasal carriers of Staphylococcus aureus compared to non-GPA chronic rhinosinusitis and healthy controls; additionally, GPA patients with S. aureus colonization are more likely to experience a future relapse. This led to a randomized placebo-controlled trial of trimethoprim-sulfamethoxazole (TMP-SMX) which showed this antibiotic/antifungal was effective in preventing relapse in GPA. Whether the benefits of TMP-SMX are related to its antimicrobial properties versus anti-inflammatory effects is still unknown. The objective of this study is to prospectively evaluate the changes in the nasal microbiome, mycobiome, and host immunity in patients with GPA before, during, and after receipt of TMP-SMX for 4 weeks. The target enrollment number is 30 participants, and the investigators will include patients seen at the Penn Vasculitis Center with GPA (diagnosed according to the American College of Rheumatology Classification Criteria or based on investigator's judgment). To analyze nasal microbiome and host immunity, participants will be swabbed with nasal swab and cytobrush for DNA sequencing and other studies. An optional research blood draw is also included. The investigators and coordinators will follow each patient longitudinally over a 6-month period.
This study seeks to understand the journey that patients eventually are diagnosed with vasculitis experience in the period prior to their formal diagnosis by a healthcare provider. Data elements of interest include average time from the onset of the first symptoms to the time a diagnosis of vasculitis is confirmed. Other aims include identifying factors associated with the time to diagnosis. These factors will be divided into: a) intrinsic factors, or so-called "patient-related factors", such as the type of vasculitis symptoms, patient demographics, socioeconomic status, patients' beliefs regarding the etiology of their symptoms, and other factors, and b) extrinsic factors, or "professional/health system factors", such as healthcare access, referral patterns, testing patterns, and other factors. Understanding such factors can guide future efforts to shorten delays in diagnosis and thereby improve outcomes. All analyses will be done for the population of patients with vasculitis as a whole and by individual types of vasculitis.
The purpose of this study is to collect existing tissue specimens from subjects enrolled in Vasculitis Clinical Research Consortium (VCRC) studies. Analysis of these tissue specimens and linked clinical data collected through VCRC studies may lead to the identification and development of a series of translational research projects. Results of these studies will provide vasculitis researchers with insight into the causes of these diseases and generate new ideas for diagnostic tests and therapies, and will be of great interest to the larger communities of researchers investigating vasculitis and other autoimmune, inflammatory, and vascular diseases.
The purpose of this study is to learn about the experience of women with vasculitis who become pregnant. In particular, the study will consist of several online surveys to assess 1. each woman's vasculitis severity and pregnancy-related experiences, and 2. pregnancy outcomes.
The aim of this project is to develop a disease specific patient reported outcome measure (PROM) for patients with AAV (the AAV-PRO). Investigators are developing and validating a questionnaire to assess quality of life in patients with ANCA-associated vasculitis (AAV). Patients with AAV have inflammation in the small blood vessels leading to involvement of a range of organs and can suffer from ongoing disease activity or treatment side effects. Quality of life can be measured by patient reported outcome measures (PROMs).
The purpose of this study is to learn about the impact of vasculitis on employment and income in patients with different systemic vasculitides. All patients enrolled in the Vasculitis Clinical Research Consortium (VCRC) Patient Contact Registry, living in USA or Canada, and followed for more than 1 year since the vasculitis diagnosis will be invited via email to participate in this study, based on an online survey.
The purpose of this study is to provide validation of patient-reported data in the VCRC Patient Contact Registry by comparing patient-reported data with data provided by the physician who is the primary provider caring for the patient's vasculitis. Patients enrolled in the Patient Contact Registry with Behcet's disease, eosinophilic granulomatosis with polyangiitis (Churg-Strauss) (EGPA), giant cell arteritis (GCA), granulomatosis with polyangiitis (Wegener's) (GPA), microscopic polyangiitis (MPA), polyarteritis nodosa (PAN), and Takayasu's arteritis (TAK) were invited via email to participate in this study.