Jizan

Researchers are evaluating luspatercept treatment in adults with transfusion-dependent beta-thalassemia in the Middle East. This observational study aims to understand real-world use and outcomes of luspatercept, a drug given according to its product label, in people aged 18 and older who require regular blood transfusions due to beta-thalassemia. Participants in this study have started luspatercept treatment within the past 12 months and continue therapy as prescribed in routine care. The study collects data retrospectively and prospectively, observing changes in transfusion needs, pre-transfusion hemoglobin levels, and iron levels up to 144 weeks. Researchers also monitor transfusion-related visits, treatment duration, dose modifications, medical encounters, and reasons for treatment changes. During the study, participants medical records are reviewed to assess transfusion burden, hemoglobin levels, iron chelation therapy, hospital visits, and other health outcomes. The study tracks long-term treatment patterns and safety events while collecting demographic and disease information. Participation involves ongoing treatment with luspatercept and regular data collection through week 144 to understand treatment effects and patient experience over time.

Researchers are evaluating the long-term safety and effects of etavopivat, a new oral medicine being developed for inherited blood disorders such as sickle cell disease and thalassaemia. These conditions affect haemoglobin, the protein responsible for carrying oxygen in the blood. This phase 3 study involves participants who have already completed treatment in a prior etavopivat study and aims to understand how etavopivat performs over an extended period of up to 264 weeks, though the study may end earlier if the drug gains approval locally. Participants will receive oral doses of etavopivat, with different forms A, B, or C given based on their age and condition. Those aged 12 years and older will receive etavopivat A or C, while children under 12 years will receive etavopivat B. The study includes several groups covering various sickle cell disease and thalassaemia categories, including transfusion-dependent and non-transfusion-dependent cases. Treatment is continuous during the study period. Throughout the study, participants will undergo regular monitoring for side effects and treatment responses, including tracking treatment emergent adverse events, hospitalizations, vaso-occlusive crises, hemoglobin concentration changes, and blood transfusion needs. These assessments will help evaluate the drugs safety and efficacy across age groups and disease types. The study is open-label and non-randomized, with follow-up lasting up to about six years.

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.

Researchers are investigating whether a hands-on therapy called Mobilization with Movement MWM can reduce pain and inflammation in adults with moderate to severe knee osteoarthritis OA. This study compares MWM to a sham treatment that looks like mobilization but does not include the specific therapeutic movements. Both groups also receive standard care, including an exercise program and education about managing knee OA. Participants will be randomly assigned to either the MWM group or the sham group. Each group receives treatment twice a week for four weeks. The MWM involves continuous gliding movements applied to the tibia during active knee flexion and extension, while the sham treatment simulates MWM by gently rotating the skin without affecting the joint. Both groups will follow a standard exercise program designed to strengthen the knee muscles. During the study, participants will have blood tests to measure inflammation markers such as C-Reactive Protein and Erythrocyte Sedimentation Rate at the start and after four weeks. Pain intensity and knee function will also be assessed at these times. The study aims to see if MWM leads to lower inflammation and less pain compared to the sham treatment. The total participation lasts for the four-week intervention period.