Sousse

This research aims to evaluate the long-term safety of luspatercept in participants who have previously taken part in other luspatercept clinical trials for conditions such as Myelodysplastic Syndromes MDS, Beta-thalassemia, and Myeloproliferative Neoplasm-associated Myelofibrosis. It is a Phase 3b, open-label, single-arm rollover study designed to continue monitoring participants who tolerated previous luspatercept treatment and may benefit from ongoing therapy, as well as those in post-treatment follow-up phases. Participants transitioning from prior luspatercept studies will enter a Transition Phase defined by an enrollment visit. Those continuing treatment will receive luspatercept injections subcutaneously at the same dose and schedule as their parent trial, administered by study staff at clinical sites. The study includes a Treatment Phase for ongoing luspatercept administration and a Follow-up Phase comprising a 42-day safety follow-up after the last dose and a long-term post-treatment follow-up phase lasting at least 5 years to monitor overall survival and progression to malignancies. Throughout the study, participants will be regularly assessed for adverse events, progression to high-risk MDS or AML, and development of other malignancies or treatment-related masses. Safety parameters are evaluated during the 42-day follow-up, while long-term survival and disease progression are monitored every six months for at least five years. The study will conclude when all participants have completed five years of combined treatment and follow-up.

Researchers are evaluating the effectiveness and safety of inavolisib combined with Phesgo compared to a placebo combined with Phesgo as maintenance treatment for participants with previously untreated HER2-positive advanced breast cancer that has a PIK3CA mutation. This Phase 3 study focuses on participants with locally advanced or metastatic breast cancer who have completed induction therapy. Participants first receive induction therapy with Phesgo plus taxane-based chemotherapy. Following this, they enter the maintenance phase where they are randomly assigned to receive either inavolisib tablets taken orally once daily for 21 days of each 21-day cycle along with Phesgo administered subcutaneously every 3 weeks, or a matching placebo tablet with Phesgo on the same schedule. Optional endocrine therapy may be given based on the investigators choice according to standard care. Throughout the study, participants undergo regular monitoring including tumor assessments, quality of life questionnaires, and safety evaluations lasting up to approximately 111 months. Key outcomes measured include progression-free survival assessed by investigators, overall survival, response rates, duration of response, and adverse event rates. Plasma concentrations of inavolisib are also measured at specific timepoints. Participants will be followed closely during and after treatment to assess these outcomes over an extended period.

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 observing the safety and clinical performance of the biodegradable polymer-coated Supraflex Cruz Sirolimus-eluting Stent in patients with multivessel coronary artery disease who need coronary revascularization using drug-eluting stents. This study includes a broad group of patients reflecting everyday clinical practice, including those with chronic coronary artery disease and acute coronary syndromes such as STEMI and NSTEMI. It aims to gather real-world data on this stents use in multiple vessel treatments. The study involves the use of the Supraflex Cruz Sirolimus-eluting Coronary Stent System, which is a biodegradable polymer-coated cobalt-chromium stent releasing sirolimus. All treated vessels will exclusively receive this stent during the procedure. Patients are followed according to routine clinical practice, with scheduled follow-ups either by phone or in person at 30 days, 6 months, and 12 months after the initial procedure. The study also plans subgroup analyses based on clinical presentation and lesion characteristics. Participants will undergo standard clinical evaluations and follow-up visits over one year to monitor outcomes such as Target Lesion Failure, mortality, myocardial infarction, revascularization, stent thrombosis, and target vessel failure. Follow-ups are designed to align with regular care schedules and include telephonic or clinical assessments. The total participation time for each patient is approximately 12 months, allowing researchers to collect comprehensive data on stent performance and patient safety in a real-world setting.

Researchers are investigating the outcomes for mothers diagnosed with cancer during pregnancy, focusing on overall survival and the long-term development of children exposed to cancer or cancer treatments before birth. The study aims to understand how prenatal exposure to therapies like chemotherapy, radiation, or targeted treatments may affect childrens neurological and heart health as they grow. The study includes several parts initially, it registers and tracks maternal and neonatal health during pregnancy and delivery, including collecting blood samples and tissue biopsies. Emotional needs of parents facing a cancer diagnosis are assessed through questionnaires. The second part follows children exposed in utero to cancer treatments with regular check-ups at various ages up to adulthood, including neurological and cardiological exams, and optional MRI sessions. Participants will undergo scheduled assessments including blood sample collection, placental biopsies, and questionnaires for parents. Children will have neurological and heart evaluations at set ages from 6 months through 18 years, with further cardiologic monitoring every five years into adulthood. The research team monitors overall survival in mothers and developmental outcomes in children, ensuring comprehensive long-term follow-up extending potentially into the fourth decade of life for offspring.

This research aims to evaluate the CardioStory device in adult patients who come to the emergency department with new shortness of breath, also known as dyspnea. The study focuses on distinguishing heart failure from other causes of dyspnea by collecting data from routine medical assessments and device measurements. The study is observational and involves adults aged 18 years and older. Participants will undergo non-invasive cardiac assessments using the CardioStory device, which quickly measures cardiac filling pressure using fiber-optic sensors and advanced algorithms without invasive procedures. Measurements with the device take about 2 minutes and are compared to standard clinical assessments including medical history, physical exams, laboratory tests, and echocardiography. The study collects data only during the emergency visit. During participation, patients will have routine clinical data recorded alongside the device measurements. Researchers will assess the diagnostic performance, sensitivity, specificity, and time efficiency of the CardioStory device in identifying heart failure. They will also monitor for any adverse events related to device use. The primary and secondary outcomes are measured on the first day of the emergency visit, with no long-term follow-up mentioned.

This research aims to validate the Glasgow-Blatchford Score GBS in adults presenting with non-traumatic upper gastrointestinal bleeding in Tunisia. The study addresses the challenge of routinely hospitalizing all patients for observation by evaluating whether GBS can accurately predict risks like rebleeding, the need for hemostatic treatment, complications, and mortality within 30 days. It is a multicenter, descriptive, and analytical observational study conducted in emergency departments. Adult patients aged 18 years and older with non-traumatic upper gastrointestinal bleeding will be included. Clinical and epidemiological information will be collected using a standard form that incorporates GBS criteria. Follow-up will occur 30 days after initial presentation to track adverse outcomes including rebleeding, need for intervention, complications, and death. The performance of the GBS will be assessed statistically for sensitivity, specificity, and predictive values. Participants will have their clinical data recorded at emergency department visits. A telephone follow-up at 30 days will gather information about any complications or mortality. Researchers will analyze the data to determine how well the GBS predicts outcomes in this population. The total duration of involvement per participant will be approximately 30 days, with confidentiality and informed consent ensured throughout the study.

Axial myopia is a form of nearsightedness caused by the eye being too long from front to back, leading to blurry distant vision. It typically begins in childhood and worsens as the eyes grow longer, potentially causing serious eye issues later in life. Researchers are studying T10430 eye drops to see if they can slow or stop this progression in children aged 6 to 11 years, aiming to prevent more severe complications. This Phase 2 trial also evaluates the safety of T10430 based on encouraging animal study results. Participants will be randomly assigned to receive one of three different strengths of T10430 eye drops or a placebo. Each participant will instill one drop in each eye once daily at around 8 PM for up to 56 weeks, followed by a 4-week follow-up period. The study will include about 200 children with axial myopia, and the treatments will be given under double-blind conditions so neither participants nor researchers know which dose is administered. Throughout the study, children and their guardians will attend visits for physical and eye examinations to monitor safety and treatment effects. Researchers will measure treatment-emergent adverse events during the entire study period of up to 60 weeks. They will also assess changes in axial eye length and spherical equivalent refraction at 6 and 12 months. This careful monitoring aims to understand how T10430 impacts eye growth and vision while ensuring participant safety.

Heart failure is a common and serious condition that leads to many hospital visits and deaths worldwide. Access to specialized heart failure clinics is limited, although such care can improve patient outcomes. This international, multicenter randomized controlled trial called VICTORY-HF is studying whether adding a virtual heart failure clinic to routine care can improve medication use and health outcomes for patients recently discharged after hospitalization or urgent care for heart failure. Participants will be randomly assigned to receive either 90 days of virtual heart failure clinic care plus routine care or routine care alone. The virtual clinic offers structured remote follow-up, monitoring of physiological measures, and optimization of guideline-directed medical therapies. The trial will evaluate the effects of this intervention on medication use, patient-reported health status, mortality, hospital readmissions, and emergency visits. Participants will be followed for 90 days during the virtual clinic intervention, with co-primary outcomes measured at 90 and 180 days. Assessments include monitoring of clinical events, medication optimization, and healthcare utilization. The follow-up period includes clinic visits and data collection to evaluate the impact of virtual care on recovery and health status after heart failure hospitalization.