Behcet Disease

Researchers are evaluating the long-term safety of apremilast in children aged 2 years or older with oral ulcers linked to Behets 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 Behets 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 Behets 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. The goal is to collect detailed data and blood samples from patients receiving care in the ILD program as well as from a control group to better understand the different ways these diseases can appear and progress over time. Participants in this observational study will not receive a specific treatment but will contribute data and blood samples over time. This approach allows researchers to compare disease characteristics in affected patients with those in healthy controls, aiming to describe the phenotypic expression of these lung diseases. Throughout the study, participants will have yearly evaluations to track the natural history of their lung disease. Data collection will include clinical assessments and blood samples to monitor disease progression. The study is ongoing, with participation potentially lasting for many years, allowing for long-term observation of the disease course.

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.

This trial evaluates the effects of apremilast compared to placebo in children aged 2 to under 18 years who have active oral ulcers caused by Behets disease. The goal is to understand how well apremilast works in treating these oral ulcers during a 12-week period. This study is a Phase 3, multicenter, randomized, placebo-controlled trial designed to provide important information about treatment options for pediatric patients with this condition. Participants are randomly assigned to receive either apremilast or a matching placebo orally for the first 12 weeks in a double-blind phase. After this, all participants receive apremilast for an additional 40 weeks in an active treatment phase. This design allows researchers to compare the initial effect of apremilast to placebo and then monitor longer-term treatment effects in all participants. Throughout the study, participants will be assessed for the number of oral ulcers over 12 weeks, pain levels, complete response rates, and changes in disease activity. Safety is closely monitored up to 56 weeks, including adverse events and growth measurements such as height, weight, and body mass index. Researchers also track plasma levels of apremilast and acceptability of the treatment. The total participation lasts up to 56 weeks to capture both short- and long-term outcomes.

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 conducting a long-term observational study called ATHN Transcends to better understand non-cancerous blood disorders. This study follows participants with various hematologic conditions, including hemophilia, von Willebrand Disease, congenital platelet disorders, rare bleeding disorders, thrombosis, and other related conditions. The aim is to collect uniform and high-quality data on the safety, effectiveness, and treatment practices of new and existing therapies over many years, addressing gaps beyond initial clinical trials and real-world treatment outcomes. Participants are assigned to specific cohorts based on their diagnosis, such as Hemophilia, Congenital Platelet Disorders, and others. Within these cohorts, there are multiple study arms and modules focusing on different therapies or disease aspects, including gene therapy outcomes and product-specific data collection. Data is gathered through scheduled assessments at enrollment, every six months, annually, and as needed, with follow-up planned for a minimum of 15 years. The study also includes a biorepository for collecting biological samples to support current and future research. Throughout the study, participants provide information during regular visits, and data is collected about their medication use, bleeding events, and treatment effectiveness. Researchers monitor safety and treatment outcomes continuously to better understand long-term effects. The study involves comprehensive evaluations but does not involve administering treatments instead, it observes participants receiving usual care. Participants may remain in the study for many years, contributing to a valuable resource that helps improve understanding of blood disorders and their management.

Researchers are exploring the natural history and genetics of autoinflammatory and autoimmune diseases, including VEXAS syndrome and related disorders. This study aims to discover genetic factors contributing to these inflammatory conditions and to enhance understanding of their immune system mechanisms. It also seeks to describe clinical features of poorly characterized diseases through chart reviews of routine medical care. Participants include individuals with known or suspected autoinflammatory diseases, family members related by blood or marriage, and healthy volunteers. Samples such as blood, saliva, hair, nails, or buccal swabs may be collected for genetic and immunologic studies. Some participants might undergo skin biopsies. The study involves genetic analysis and functional immunologic tests to identify disease-associated variants and features. During the study, participants provide samples for laboratory analysis and may have their medical records reviewed. Researchers measure the number and types of genetic variants linked to inflammatory diseases over up to five years. Study involvement includes mail-in sample collection and possible clinical evaluations. The total participation time can extend up to the study end date in 2032, with ongoing data collection and analysis to improve understanding of these conditions.

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 Behets disease, Still disease, Schnitzlers 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.