Autoinflammatory syndrome

This Phase 3 trial tested a drug called efzofitimod in 268 adults with pulmonary sarcoidosis, a condition that causes lung inflammation. Participants received either the drug or a placebo every 4 weeks for 48 weeks. The main goal was to see if the drug could help people safely re…

This study tested a program to help veterans with heart disease or rheumatic conditions take their medications as prescribed. Over 5,000 participants received reminders via text or phone calls, with extra support if needed. The goal was to improve medication adherence and reduce …

This phase 2 trial tested the drug crizanlizumab in 18 people with RVCL, a rare and fatal genetic condition that damages small blood vessels in the brain and eyes. There is currently no treatment for RVCL. The study measured changes in brain lesions on MRI scans to see if the dru…

This study tested whether H.P. Acthar Gel can improve symptoms and quality of life in people with CNS sarcoidosis, a rare condition where inflammation affects the brain and spinal cord. Four adults with the disease received the gel to see if it could control disease activity and …

This study tested canakinumab, an anti-inflammatory biologic, in 14 Japanese adults with Adult-Onset Still's Disease (AOSD), a rare condition causing fevers and joint pain. Participants received injections every 4 weeks for at least 48 weeks. The goal was to see if the drug could…

This study tested a drug called anakinra in 24 children and young adults (ages 2-20) with severe systemic juvenile idiopathic arthritis that did not respond well to steroids. For the first month, half received anakinra and half received a placebo, then all received anakinra for 1…

This study tested a drug called emapalumab in 33 children and adults with macrophage activation syndrome (MAS), a life-threatening immune overreaction, linked to Still's disease or lupus. The goal was to see if the drug could safely control the condition and reduce the need for h…

This study tested an inhaled medication called treprostinil in 5 people with sarcoidosis who also have lung scarring and high blood pressure in the lungs. The goal was to see if the drug could improve blood flow and heart function. Participants took the drug and were monitored wi…

This phase 2 trial tested baricitinib (Olumiant), a drug used for rheumatoid arthritis, in 54 people with Aicardi Goutières Syndrome (AGS), a rare genetic disorder that causes brain inflammation and developmental problems. The goal was to see if the drug could stabilize or improv…

This study looked at how well flu vaccines (seasonal and H1N1) work in 234 adults with autoimmune diseases like lupus or vasculitis, some of whom were on steroids or immunosuppressants. Researchers measured antibody levels three weeks after each shot to see if the vaccine provide…

This small pilot study tested whether drugs normally used for HIV could help children with Aicardi-Goutières syndrome (AGS), a rare genetic disorder that causes severe brain inflammation. The trial gave 11 children a combination of three reverse transcriptase inhibitors to see if…

This study tested whether a special lung MRI can replace CT scans for diagnosing idiopathic pulmonary fibrosis (IPF), a scarring lung disease. Researchers compared MRI to standard CT in 60 patients to see if MRI could spot the same key signs of IPF without using radiation. The go…

This study tested whether adding a tiny forceps biopsy to the standard needle biopsy during a lung ultrasound procedure helps doctors diagnose sarcoidosis more often. Fifty-five adults with enlarged lymph nodes in the chest took part. The goal was to see if the combined approach …

This study tested a device called SUDOSCAN, which measures sweat function in just two minutes, to see if it can detect small fiber nerve damage in people with autoimmune diseases (like lupus or rheumatoid arthritis) or unexplained pain. Researchers enrolled 139 adults and compare…

This study looked at whether a pulmonary rehabilitation program helps people with Stage IV pulmonary sarcoidosis (advanced lung scarring) become more active in their daily lives. 38 adults took part and were followed for one year. The program aimed to improve exercise ability, re…

This study tested two different exercise programs in 28 children and teens with rheumatic diseases like juvenile arthritis and familial Mediterranean fever. One program used external focus (thinking about the movement's effect), the other used internal focus (thinking about the b…

This study tested three different drug combinations to keep patients comfortable and still during a heart procedure called pulsed field ablation for atrial fibrillation. 240 adults were randomly assigned to one of three sedation groups. The goal was to see which method achieved t…

This study tested a pure cannabidiol (CBD) solution called CardiolRx in 27 adults with recurrent pericarditis, a condition causing repeated inflammation and pain around the heart. Participants had not responded well to standard treatments or needed long-term steroids. The goal wa…

This study looked at how stretchy the skin and blood vessels are in people with Williams syndrome, a genetic condition that can cause heart problems. Researchers measured skin and pulse speed in 43 participants aged 5 to 70. The goal was to learn how these tissues change over tim…

This study looked at 719 people, including patients with autoimmune connective tissue diseases like myositis, their blood relatives, and healthy volunteers. Researchers collected blood samples and medical histories to find genetic and environmental factors that may trigger these …

This completed study looked at how the COVID-19 pandemic and social distancing impacted the mental health of 259 adults with chronic inflammatory rheumatism (like rheumatoid arthritis or ankylosing spondylitis). Researchers used questionnaires to measure stress, depression, anxie…

This study tested a smartwatch and mobile app platform called Pedi@Ctivity to track physical activity in 362 adolescents, including those with chronic rheumatic disease. The goal was to collect big data and use AI to analyze activity patterns and create personalized exercise prog…

This study collected blood, saliva, and eye fluid samples from 343 people with uveitis, an inflammatory eye disease that can cause vision loss. Researchers aimed to learn which genes and other factors might lead to the disease and affect treatment response. Participants were foll…

This study looked at over 13,000 people with chronic inflammatory rheumatism or autoimmune diseases who had or were suspected to have COVID-19. Researchers tracked how many developed severe COVID-19 requiring intensive care or leading to death. They also examined risk factors lik…

This study looked at over 2,400 people with rheumatic diseases who take methotrexate. Researchers compared those who had standard lab monitoring to those who had fewer blood tests. The goal was to see if reduced monitoring is as safe in terms of preventing organ damage or death.

This study looks at why some people develop hives from physical triggers like cold, pressure, or sunlight. Researchers will test participants' skin reactions and collect blood and genetic samples to understand the role of immune cells and genes. The goal is to uncover the underly…

This study tested whether using an indoor air purifier changes health measures like heart rate, sleep, and oxygen levels. 42 healthy adults wore Apple Watches while using a real or fake (sham) air purifier at home. The goal was to see if cleaner air makes a short-term difference …

This study aimed to create and test a new questionnaire called CARD-SARC that measures how cardiac sarcoidosis affects patients' daily lives and well-being. Researchers worked with 130 adults diagnosed with cardiac sarcoidosis at two NHS specialist centres. The goal was to develo…

This completed study looked at anxiety about COVID-19 in 200 adults with rheumatic diseases who were already taking either biologic or conventional disease-modifying drugs. Researchers called participants and asked them to fill out a questionnaire about their coronavirus-related …

This study looked at whether video or phone visits (tele-rheumatology) work as well as in-person visits for people with rheumatic diseases like arthritis. Over 650 adults took part, and researchers measured how satisfied they were with each type of visit and which they preferred.…

This study tested whether coaching sessions are practical for young people with rheumatic diseases moving from pediatric to adult care. 26 participants received up to 8 coaching sessions covering self-advocacy, medication management, and future planning. The main goal was to see …

This completed registry study in Japan looked at how doctors treat palmoplantar pustulosis (PPP), a skin condition causing pus-filled blisters on hands and feet. Researchers collected data from 276 patients' medical records and clinic visits to understand which treatments are use…

This study measured two newer blood markers (calprotectin and serum amyloid protein) alongside standard tests in 20 children with juvenile arthritis and related conditions. The goal was to see if these markers could better track disease flare-ups in everyday clinic visits. The st…

This completed study from Alexandria University looked at 40 adults with lung sarcoidosis who were receiving or about to start immunosuppressive therapy. Researchers tracked them for 12 months to see if any developed lymphoma, confirmed by lymph node biopsy. The goal was to under…

This study collected information from 2,200 people with sarcoidosis around the world using online surveys. The goal was to learn more about how the disease affects different people, not just those seen at major medical centers. Participants also had the option to provide a DNA sa…

This completed study reviewed the medical charts of 32 people with Still's disease who developed a serious complication called macrophage activation syndrome (MAS). The goal was to learn more about their symptoms, how long it took for lab values to return to normal, and what trea…

This study looked at whether the seasonal flu vaccine works as well in people with sarcoidosis as it does in healthy people. Researchers gave the flu shot to 190 adults with sarcoidosis and compared their immune response to a group of healthy volunteers. The goal was to see if th…

This study looked at whether whole genome sequencing (a complete read of a person's DNA) can help diagnose leukodystrophies, a group of rare brain diseases that are hard to identify. Researchers enrolled 236 children with white matter abnormalities on brain scans but no known gen…

This study involved 39 adults and children with autoinflammatory diseases like FMF and Still's disease. Researchers conducted interviews to understand how these conditions affect quality of life. The goal was to create a tool that doctors can use to better assess and address pati…

This study tested a simple approach where doctors review pharmacy refill data during clinic visits and talk with patients about their medications. The goal was to see if this method is practical and acceptable for improving medication adherence in people with chronic rheumatic di…

This completed study looked at 32 people with known genetic changes in connective tissue genes to better understand how these changes affect overall health. Participants underwent a range of medical tests and exams over 3-5 days. The goal was to gather information, not to test a …