Sousse

A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants: * Participants receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the investigator, may derive clinical benefit from continuing treatment with luspatercept * Participants in the follow-up phase previously treated with luspatercept or placebo in the parent protocol will continue into long-term post-treatment follow-up in the rollover study until the follow-up commitments are met * The study design is divided into the Transition Phase, Treatment Phase and Follow-up Phase. Participants will enter transition phase and depending on their background will enter either the treatment phase or the Long-term Post-treatment Follow-up (LTPTFU) phase * Transition Phase is defined as one Enrollment visit * Treatment Phase: For participants in luspatercept treatment the dose and schedule of luspatercept in this study will be the same as the last dose and schedule in the parent luspatercept study. This does not apply to participants that are in long-term follow-up from the parent protocol * Follow-up Phase includes: \- 42 Day Safety Follow-up Visit * During the Safety Follow up, the participants will be followed for 42 days after the last dose of luspatercept, for the assessment of safety-related parameters and adverse event (AE) reporting \- Long-term Post-treatment Follow-up (LTPTFU) Phase * Participants will be followed for overall survival every 6 months for at least 5 years from first dose of luspatercept in the parent protocol, or 3 years of post-treatment from last dose, whichever occurs later, or until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. Participants will also be monitored for progression to AML or any malignancies/pre-malignancies. New anticancer or disease related therapies should be collected at the same time schedule Participants transitioning from a parent luspatercept study in post-treatment follow-up (safety or LTPTFU) will continue from the same equivalent point in this rollover study. The ACE-536-LTFU-001 rollover study will be terminated, and relevant participants will discontinue from the study when all participants fulfill 5 years on the study, including treatment and follow-up.

Researchers are evaluating the efficacy and safety of inavolisib combined with Phesgo compared to placebo with Phesgo as maintenance therapy in participants who have previously untreated HER2-positive advanced breast cancer with PIK3CA mutations. This Phase III, multicenter, randomized, double-blind, placebo-controlled study focuses on participants with locally advanced or metastatic breast cancer, aiming to understand the treatment impact after initial induction therapy. Participants will receive inavolisib orally once daily on Days 1 to 21 of each 21-day cycle, starting on Day 1 of Cycle 1 during maintenance treatment. Phesgo will be administered subcutaneously every three weeks on Day 1 of each cycle. The study includes an induction therapy phase where taxane-based chemotherapy is given after Phesgo. Optional endocrine therapy such as tamoxifen, aromatase inhibitors, or fulvestrant may be used based on the investigator's choice, with luteinizing hormone-releasing hormone agonists administered according to local guidelines. During the study, participants will be monitored for progression-free survival for up to approximately 40 months. Assessments include evaluation of heart function, organ function, and overall health status. Researchers will track the safety and effectiveness of the treatment combination through regular clinical evaluations and laboratory tests. The total duration includes maintenance treatment cycles and follow-up to measure outcomes and monitor safety.

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 the safety and clinical performance of the biodegradable polymer-coated Supraflex Cruz Sirolimus-eluting Stent (SES) in patients with multivessel coronary artery disease. This study includes an unselected, all-comer population reflecting daily clinical practice, involving patients who require coronary revascularization with drug-eluting stents. It covers patients with chronic coronary artery disease as well as those with acute coronary syndromes such as STEMI and NSTEMI. The study is a prospective, observational, single-arm, multi-center registry where all patients receive the Supraflex Cruz SES. The device is a cobalt-chromium stent coated with a biodegradable polymer that releases sirolimus. Patients with two or more affected vessels are treated exclusively with this stent. Follow-up is conducted via routine clinical practice and includes telephonic or clinical visits at 30 days, 6 months, and 12 months after the initial procedure. Participants will be monitored for safety and clinical outcomes, with the primary outcome measure being Target Lesion Failure (TLF) at 12 months. The study also allows for subgroup analyses based on clinical presentation, lesion type, and use of atherectomy. The total participation involves follow-up visits and data collection over a one-year period to assess the device's performance in real-world settings.

Researchers are studying the effects of cancer diagnosis and treatment during pregnancy on both mothers and their children. The aim is to understand the overall survival of mothers diagnosed with cancer during pregnancy and to test whether children exposed to cancer treatments like chemotherapy or radiation in the womb develop normally, particularly focusing on neurological and heart health. The study includes several parts addressing maternal health during and after pregnancy and long-term follow-up of children exposed to cancer treatments before birth. The study involves registering mothers diagnosed with cancer during pregnancy and collecting maternal blood samples, umbilical cord blood, and tissue samples from the placenta and umbilical cord. Mothers complete questionnaires about anxiety and emotional needs related to their diagnosis. Children who were exposed to cancer treatments in the womb will have regular health check-ups at ages 6 months, 18 months, 3, 6, 9, 12, 15, and 18 years. After age 18, cardiologic assessments and questionnaires will continue every five years, with optional MRI scans at several ages during childhood and adolescence. Participants will be monitored through neurological and cardiological exams performed by specialists to assess the children's development. Mothers’ outcomes will be tracked prospectively from diagnosis through treatment and delivery. The study includes long-term follow-up for both mothers and children, with comprehensive assessments to evaluate health effects over many years. Participants provide informed consent, and children older than 12 provide assent with consent from parents or themselves once they reach adulthood.

Researchers are evaluating the effectiveness of two pain treatments, dexketoprofen given intravenously and piroxicam given intramuscularly, for managing acute pain caused by traumatic injuries in emergency departments. This Phase 3, randomized, double-blind study aims to compare these medications since the commonly used RICE protocol lacks sufficient evidence of effectiveness and painkillers like paracetamol and NSAIDs are often prescribed or used without prescriptions. Participants will be randomly assigned to receive either an intravenous injection of dexketoprofen with an intramuscular placebo or an intramuscular injection of piroxicam with an intravenous saline infusion. The study lasts for three months and includes an initial treatment in the emergency department followed by a telephone follow-up visit seven days later. During the study, researchers will measure pain levels, blood pressure, heart rate, respiratory rate, and oxygen saturation at admission and discharge from the emergency department. At the 7-day phone call, patients will answer satisfaction questions and complete a quality of life questionnaire. The main outcome measured is the time it takes for pain to resolve within 120 minutes after treatment.

Researchers are evaluating the Glasgow-Blatchford Score (GBS) to see how well it predicts risks in adult patients in Tunisia who experience non-traumatic upper gastrointestinal bleeding. Despite advances in managing these bleeds with endoscopy, early assessment remains important. In Tunisia, all such patients are usually hospitalized for observation, which can be challenging, so a reliable risk tool like the GBS could improve patient care decisions. This multicenter study will include adults aged 18 and older who come to emergency departments with non-traumatic upper gastrointestinal bleeding. Clinical data will be collected using a standardized form including GBS criteria. Patients will be followed up by phone at 30 days to check for adverse outcomes like rebleeding, need for hemostatic intervention, complications, or death. Participants will provide informed consent and have their clinical and epidemiological information recorded. Researchers will analyze the GBS's accuracy by measuring sensitivity, specificity, and predictive values for 30-day outcomes. The study aims to confirm whether the GBS is a useful and valid tool for risk assessment in the Tunisian healthcare setting.

Axial myopia is a form of nearsightedness caused by the eye being too long from front to back, which leads to blurry distance vision. It usually starts in childhood and worsens as the eye continues to grow, potentially resulting in serious eye problems later in life. This research aims to study the safety and possible effectiveness of T10430 eye drops in children with axial myopia to slow or stop this progression and prevent more severe complications. The study is a phase 2 trial including 200 children aged 6 to 11 years with a specific range of nearsightedness. Participants will be randomly assigned to receive one of three different doses of T10430 eye drops or a placebo, with one drop instilled in each eye once daily at 8:00 PM (plus or minus 2 hours) from the first day until the day before the final treatment visit. The treatment period will last up to 56 weeks. The eye drops contain the study drug or a vehicle solution as a control. Throughout the study, safety will be closely monitored by recording any treatment-emergent adverse events from the time participants and their guardians provide consent until about 60 weeks later, which includes a 4-week follow-up after treatment ends. Researchers will assess vision changes, eye pressure, and other eye health indicators to evaluate the drug's effects. Participants will attend scheduled visits for assessments, and their adherence to the treatment will be monitored during the study period.

Heart failure is a major cause of death and hospital visits in Canada, and access to specialized care varies across regions. This research aims to create and test a virtual heart failure care program that allows patients to receive outpatient treatment and medication adjustments remotely. The study compares this virtual care to usual care to see if it improves health and treatment outcomes. A pilot phase was done to test the feasibility and acceptability of the virtual care approach and to finalize study procedures. Participants will receive virtual clinic visits for three months after a hospital or emergency visit for heart failure. During this time, their physiological data will be monitored remotely, and treatments will be adjusted as needed. The trial compares this virtual care to routine heart failure care provided by the treating physician. The virtual care period represents the main follow-up for medication and health status outcomes. Throughout the study, participants will be monitored for up to 180 days to assess clinical outcomes, with primary results collected at 30, 90, and 180 days. Researchers will evaluate a combination of health and treatment measures to understand how virtual care impacts recovery after heart failure hospitalization. The study includes assessments of patient health status and ongoing monitoring of their condition during the follow-up period.