Behcet Disease

Researchers are evaluating the long-term safety of apremilast in children aged 2 years or older with oral ulcers linked to Behçet's disease, and in children aged 5 years or older with active juvenile psoriatic arthritis who have completed prior related studies. This phase 3, multicenter, open-label extension study aims to monitor safety outcomes over an extended period. Participants receive apremilast orally in doses based on body weight: those weighing between 12 kg and less than 20 kg receive 10 mg twice daily, those between 20 kg and less than 50 kg receive 20 mg twice daily, and those weighing 50 kg or more receive 30 mg twice daily. The study continues treatment and observation over approximately four years, focusing on long-term effects. During the study, researchers will monitor participants for adverse events, changes in vital signs, laboratory parameters, growth measures such as height, weight, and BMI, and assess suicide risk using the Columbia-Suicide Severity Rating Scale. Tanner staging will also be evaluated to assess physical development. Participants are expected to attend scheduled visits and adhere to protocol requirements throughout the study period, which may last up to around four years.

Researchers are evaluating the efficacy and safety of Hemay005 tablets in patients with Behçet's disease through a phase 3, multi-center, randomized, placebo-controlled, double-blind study. The trial compares high and low doses of Hemay005 against placebo to better understand treatment effects in this condition. The study includes adults aged 18 to 75 years diagnosed with Behçet's disease and experiencing oral ulcers. Participants are randomly assigned to one of three groups: Hemay005 60 mg twice daily, Hemay005 45 mg twice daily, or placebo, during a 12-week core treatment phase. After this, all participants enter a 40-week extension phase where those initially receiving placebo are re-randomized to one of the two Hemay005 doses. Dose escalation occurs during the first week of treatment periods to reduce side effects. Following treatment, a 4-week observation period monitors drug discontinuation effects. Throughout the study, participants undergo regular assessments including oral and genital ulcer evaluations, pain scales, joint and skin lesion assessments, gastrointestinal activity monitoring, eye symptom checks, quality of life questionnaires, and laboratory tests. Safety is closely monitored by tracking adverse events and lab results over the course of treatment and follow-up. The primary outcome focuses on the efficacy related to oral ulcers at week 12, with continued efficacy and safety evaluations throughout the study duration.

Researchers are studying the natural progression of Interstitial Lung Disease (ILD) and related conditions such as Idiopathic Pulmonary Fibrosis, Sarcoidosis, and Connective Tissue Disorder. This observational study aims to collect data and blood samples from patients receiving care for ILD, as well as from a control group, to better understand the different expressions of these diseases. Participants include patients diagnosed with ILD and a control group without the disease. The study involves gathering data and blood samples during their care without any specific drug or treatment interventions. The observational nature means participants continue their usual medical care while contributing information to the research. During the study, researchers will monitor the natural history and course of ILD yearly through data collection and blood sampling. This includes tracking disease progression and characteristics over time. Participation involves regular assessments as part of routine clinical care, with no experimental treatments given. The study is sponsored by the University of Chicago and is ongoing through December 2030.

Researchers are studying the effects of arginine supplementation on immune function in infants who have undergone major bowel surgery or have necrotising enterocolitis. This exploratory study will compare gene expression related to immune response in preterm and term infants receiving different nutrition regimens. The goal is to understand how arginine affects genes involved in nutrition and infection in postoperative babies. The study involves 48 infants divided into three groups: one receiving standard parenteral nutrition without added arginine, one receiving parenteral nutrition with added arginine plus oral arginine supplementation, and one receiving standard parenteral nutrition with oral arginine supplementation only. Arginine dosing will begin with the first enteral feeds and adjust based on feeding progress. Treatment lasts up to 30 days post-surgery or diagnosis. Participants will have blood samples collected at specified intervals up to 30 days after surgery to analyze gene expression, amino acid levels, ammonia, and metabolomics. Routine nutritional intake and clinical lab data will also be recorded. Researchers will assess changes in immune-related gene activity, metabolomic profiles, and growth during the study period. The study is conducted across two neonatal centers under a neonatal partnership.

Researchers are developing and managing the AnovaOS Network Powered Patient Registry to collect real-world patient data across various diseases globally. This registry aims to capture meaningful clinical information on diagnosis, infection course, treatments, and outcomes to enhance understanding and support future clinical trials and observational studies. The registry serves as a resource to better understand, prevent, diagnose, and treat diverse health conditions. Participants' data will be gathered through this registry, which can also be used to recruit individuals for clinical trials and observational studies on promising therapies. The registry collects ongoing information on patients' health status and treatments, enabling long-term monitoring and analysis. This observational study does not involve administering treatments but focuses on data collection and management. Participants will provide information through questionnaires or instruments, either personally or via an informed proxy, with an expected follow-up once per year. The research team will assess natural history, clinical effectiveness, safety, and quality of care over a period of five years. The registry includes patients with a wide range of conditions, and participation requires informed consent and the ability to complete follow-up data collection.

Researchers are evaluating the effectiveness of apremilast compared to a placebo for treating oral ulcers in children aged 2 to under 18 years with Behçet's disease (BD). The study aims to measure how well apremilast works in reducing the number and pain of oral ulcers during a 12-week period. This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled clinical trial sponsored by Amgen. Participants will be randomly assigned to receive either apremilast or a matching placebo orally for the first 12 weeks in a double-blind phase. After this period, all participants will continue receiving apremilast for an additional 40 weeks in an active treatment phase. The study monitors both the effects of the drug and safety throughout these phases. During the study, participants will have regular assessments of oral ulcer counts, pain levels, disease activity, and quality of life using tools like the Short Form Survey (SF-10). Safety will be closely monitored by tracking adverse events, suicide-related thoughts or behaviors, and physical growth measurements. Blood samples will be collected to measure apremilast levels. The total study participation lasts up to 56 weeks, including the treatment and follow-up periods.

Researchers are conducting a nationwide registry in Armenia to observe and track systemic autoimmune and autoinflammatory diseases. These diseases involve abnormal immune system activity causing widespread inflammation and organ damage, often influenced by genetics and environmental factors. The study aims to better understand disease progression, which is usually measured by specific activity scores, and to gather detailed epidemiological and clinical data, as current knowledge in Armenia is limited. The study involves usual medical care for patients diagnosed with these systemic diseases, alongside collecting additional blood and stool samples for biobanking. Data collected include clinical exams, lab results, current treatments, and disease-specific activity scores reported by both patients and physicians. This observational registry will continue for about five years, assessing disease manifestations and evolution under treatment. Participants will be regularly evaluated through clinical and laboratory assessments, with data recorded on disease activity and progression. The study monitors systemic symptoms and uses disease-specific scores to track changes over time. Researchers will identify rare clinical forms and prognostic factors. The registry includes informed consent procedures and follows patients longitudinally to improve understanding of these complex diseases and their management.

Researchers are studying people with various non-cancerous blood disorders to better understand the long-term safety and effectiveness of treatments used for these conditions. This observational study aims to collect consistent and high-quality data over time from patients with congenital or acquired hematologic and connective tissue disorders that cause bleeding or clotting problems. It addresses the need for reliable long-term information beyond initial drug approval trials and includes multiple disease-specific groups for comprehensive research. Participants will join one of several cohorts based on their specific blood disorder, such as hemophilia, von Willebrand disease, congenital platelet disorders, rare bleeding disorders, thrombosis/thrombophilia, or other non-neoplastic hematologic conditions. The study includes several arms and modules focusing on different therapies or patient groups, like previously untreated patients, gene therapy outcomes, and treatment with specific drugs (e.g., ALTUVIIIO® or Hemlibra®). Data collection occurs at enrollment and regularly every six months and annually, with additional data gathered as needed. During the study, participants will undergo various assessments including medical history, treatment records, bleeding event tracking, and biospecimen collection for future research. The study will follow participants for at least 15 years to monitor therapy safety and effectiveness. Researchers will analyze dosing patterns, bleeding rates, and real-world treatment outcomes while maintaining long-term participant follow-up and data quality.

Researchers are investigating genetic factors that may contribute to human disorders of inflammation, including conditions like VEXAS syndrome and other autoinflammatory diseases. This exploratory natural history study aims to better understand these diseases by identifying genetic causes, examining immune system features, and describing clinical characteristics through medical record review. Participants include those with known or suspected autoinflammatory diseases, their family members, and healthy volunteers. Samples such as blood, saliva, hair, nails, or cheek swabs may be collected for genetic and immunologic studies. Some participants might also undergo skin biopsies. The study is observational and involves no treatment interventions. During the study, researchers will collect biological samples and review medical histories to discover genetic variants linked to inflammatory diseases. The main outcomes measured are the numbers of newly identified genetic changes that may cause or increase susceptibility to these disorders. Participation involves providing samples, consenting to medical record review, and may include follow-up over several years to monitor findings.

Researchers are investigating autoinflammatory diseases (AID), which involve recurring inflammatory episodes without infection, cancer, or adaptive immune system problems. This study focuses on both monogenic hereditary periodic syndromes and polygenic or multifactorial AID, including rare conditions like Behçet's disease, Still disease, Schnitzler's disease, PFAPA syndrome, chronic recurrent multifocal osteomyelitis, non-infectious uveitis and scleritis, among others. The goal is to gather detailed data to improve understanding and management of these rare diseases through an international collaboration. The study uses the AIDA registry, a secure online platform for collecting demographic, genetic, clinical, laboratory, radiologic, and therapeutic information. Data are gathered retrospectively and prospectively during routine clinical visits scheduled every 3-6 months. Eleven registries focus on different AID conditions, enabling comprehensive data collection to identify clinical patterns, treatment impacts, and long-term outcomes over a period of at least 10 years. Participants provide information during their usual care visits, allowing researchers to track changes in disease activity, organ involvement, inflammatory markers, visual function, pain levels, fatigue, fertility, and socioeconomic factors. The study monitors these outcomes over multiple timepoints up to 120 months. Data privacy is ensured by pseudonymizing patient information. This registry supports multiple clinical studies and aims to enhance knowledge, awareness, and future research on autoinflammatory diseases worldwide.