Jizan

This research aims to evaluate the use of luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East. The study focuses on people who require regular blood transfusions due to this genetic blood disorder. The goal is to understand how luspatercept, a drug given according to its official guidelines, affects these patients in real-world clinical settings. Participants receive luspatercept following the product's approved label. The study includes adults who have started this treatment within the last 12 months and are continuing therapy. The study is observational, meaning it collects information on patients receiving luspatercept as part of their usual care without altering their treatment. During the study, researchers monitor changes in the participants' need for blood transfusions and their hemoglobin levels before transfusions, from the start of treatment up to week 144. Participants provide informed consent for their medical data to be collected and analyzed. The study observes safety and effectiveness over this extended period to better understand luspatercept's impact on transfusion-dependent beta-Thalassemia.

Researchers are evaluating the long-term safety and effectiveness of etavopivat, a new oral medicine being developed to treat inherited blood disorders such as sickle cell disease and thalassemia. These disorders affect hemoglobin, the protein responsible for carrying oxygen in the body. This phase 3 study aims to monitor how well etavopivat works and its safety profile over an extended period. Participants will receive one of three forms of etavopivat (A, B, or C) as oral doses. The study is open-label and multicenter, involving adults, adolescents, and children who have previously completed treatment in an etavopivat parent study and continue to benefit clinically. The treatment period can last up to 264 weeks but may end earlier if etavopivat is approved in the participant's country. During the study, researchers will track the number of treatment-emergent adverse events and adverse reactions for each participant by indication and age group from baseline through the end of the study, which can last up to 316 weeks. Participants' safety and response to long-term treatment will be closely monitored throughout this period.

This research aims to improve understanding of rare autoinflammatory diseases (AID), which cause repeated inflammatory episodes without infection or cancer. The study focuses on hereditary periodic syndromes (monogenic AID) caused by gene mutations, as well as related polygenic or multifactorial AID like Behcet's disease, Still disease, Schnitzler's disease, PFAPA syndrome, chronic recurrent multifocal osteomyelitis, non-infectious uveitis and scleritis, spondyloarthritis, and Castleman disease. The goal is to gather detailed clinical and therapeutic data to expand knowledge of these rare conditions, which are often difficult to diagnose outside specialized centers. Participants will be enrolled in the AIDA international registry, which uses a secure online platform to collect retrospective and prospective information. Data collected include demographics, genetics, clinical features, laboratory and radiologic results, treatments, and socioeconomic impact. The registry covers multiple specific AID types and will track patients over at least 10 years through routine clinical visits usually every 3-6 months. The platform supports data sharing and analysis to identify disease patterns, treatment responses, and long-term outcomes. During the study, patients' medical records will be regularly updated with clinical and laboratory data. Researchers will analyze changes in patient numbers and disease characteristics over time. The registry also aims to foster international collaboration, improve early diagnosis, assess quality of life and socioeconomic effects, and support future research and clinical trials. Patient data privacy is maintained by using pseudonyms and complying with data protection laws throughout the study.

Researchers are evaluating whether a hands-on therapy called Mobilization with Movement (MWM) can reduce pain and inflammation in adults with moderate to severe knee osteoarthritis (OA). The study compares MWM to a sham treatment that mimics the feeling of mobilization but lacks the therapeutic movements. Both groups also receive standard care, including exercise and education about managing knee OA. The trial aims to clarify the potential of MWM as a treatment option for this condition. Participants will be randomly assigned to receive either MWM or sham mobilization twice a week for four weeks. The MWM involves specific continuous glide movements applied to the tibia during active knee movements, while the sham treatment simulates a slight skin rotation without affecting the joint. Additionally, all participants will engage in a standard exercise program to strengthen the knee muscles and receive education on knee OA management. Throughout the study, participants will undergo assessments before and after the intervention period, including blood tests to measure inflammation markers like C-Reactive Protein (CRP) and Erythrocyte Sedimentation Rate (ESR). Researchers will also evaluate self-reported pain levels and knee function. The study will monitor changes over the four-week treatment period to determine the effects of these interventions on inflammation and pain.