Tunis

Researchers are conducting an observational study to better understand fucosidosis, a rare disease, its symptoms, and how it changes over time. The study aims to gather detailed information about the diseases natural history and progression, including patients who have or have not undergone stem cell transplant. There is currently no approved treatment for fucosidosis, so this study focuses on observing the condition without introducing new medications. The study is divided into two parts Part A involves collecting past medical data from up to 57 participants, and Part B involves following up with up to 31 participants prospectively, many of whom may also be in Part A. Participants will continue to receive their usual medical care throughout the study, with no additional treatments or interventions provided by the study team. Participants will provide medical history and health information that will be reviewed over time. The main focus is to track disease progression in individuals untreated by investigational products for up to four years. This includes gathering retrospective data and ongoing prospective observations to aid future research and potential treatment development. The study is expected to run until January 2031.

Researchers are evaluating orforglipron to measure its effects on cardiovascular outcomes in adults aged 50 and older who have atherosclerotic cardiovascular disease ASCVD andor chronic kidney disease CKD. This phase 3 study aims to compare orforglipron with a placebo to better understand its impact on major cardiovascular events over about five years. Participants will be randomly assigned to receive either orforglipron orally along with standard care or a placebo orally along with standard care. The study is double-blinded, meaning neither participants nor researchers will know who receives the active drug or placebo during the trial period. During the study, participants will be followed for around five years, with researchers monitoring the time to the first major cardiovascular event and additional outcomes such as cardiovascular and kidney events, changes in kidney function measured by eGFR, and the onset of type 2 diabetes. The study includes regular assessments to track these outcomes and ensure participant safety throughout the long-term follow-up.

Researchers are evaluating MB12, a proposed pembrolizumab biosimilar, compared to Keytruda in combination with pemetrexed-platinum chemotherapy as the first treatment for patients with advanced metastatic non-squamous non-small cell lung cancer NSCLC. This randomized, double-blind, multicenter study aims to compare the pharmacokinetics, efficacy, safety, and immune response of MB12 and Keytruda in this patient population. Participants will be assigned to one of three groups MB12 with pemetrexed and carboplatin or cisplatin, European Union-sourced Keytruda with the same chemotherapy, or US-sourced Keytruda with the same chemotherapy. MB12 and Keytruda are given intravenously at 200mg every three weeks on Day 1. Pemetrexed is given at 500 mgm2 IV every three weeks on Day 1, while carboplatin or cisplatin is administered every three weeks for four cycles. During the study, participants will be monitored from Week 1 to Week 52 for drug levels in the body, treatment effectiveness, safety, and immune response. Key assessments include measuring pharmacokinetic bioequivalence and efficacy equivalence within the first 24 weeks, along with longer-term safety and immune monitoring. The study is led by mAbxience Research S.L. and is expected to continue until September 2027.

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.

Pediatric patients undergoing spine surgery to correct scoliosis face significant risks related to blood loss during the procedure. This research aims to identify factors before and during surgery that increase the chance of needing transfusions of donor red blood cells. The study also evaluates how using a device called Cell Saver, which recycles the patients own blood during surgery, may reduce the need for these transfusions. The study looks at a group of children under 18 who had posterior instrumented spinal fusion surgery for scoliosis between April 2023 and March 2026. Researchers retrospectively analyze these cases to find risk factors for donor blood transfusions and assess the impact of reinfusing blood collected by the Cell Saver device during surgery. The surgical correction is done under general anesthesia using standard techniques. Participants data from the start of surgery until three days after are reviewed to measure how many required donor red blood cell transfusions and how much blood was saved by the Cell Saver device. The primary outcome is the proportion of patients needing donor blood within this period. Secondary outcomes include the rate of blood conservation achieved during surgery and total transfusion volume. This observational study is led by Mehdi Trifa and is expected to complete by the end of 2026.

Shoulder tendinopathy is a common condition causing pain and limiting daily activities due to damage or overuse of the rotator cuff tendons. Researchers are comparing two injection treatments for this condition corticosteroid injections, which reduce inflammation and provide quick pain relief but may have short-term effects, and Platelet-Rich Plasma PRP injections, made from the patients own blood, which may promote longer-term healing. This trial involves adult patients diagnosed with simple shoulder tendinopathy confirmed by clinical examination and imaging. The study randomly assigns 60 adult participants to receive either a single corticosteroid injection or a single PRP injection into the affected shoulder tendon. Corticosteroid injections aim to quickly reduce pain and inflammation, while PRP injections focus on promoting tissue healing and longer-term recovery. Both injections are given under sterile conditions by trained physicians. Patients will be monitored for changes in pain and shoulder function at one week and three months after the injection. Participants will undergo evaluations before the injection and at follow-up visits one week and three months later. Pain levels will be measured using a visual analog scale VAS, and shoulder function will be assessed with the Disabilities of the Arm, Shoulder and Hand DASH score and the Shoulder Pain and Disability Index SPADI. These assessments will help determine which treatment provides better pain relief and functional improvement over time. The study is conducted at the Rheumatology Department of Charles Nicolle Hospital in Tunis, Tunisia, with a total participation duration of about three months post-injection.

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 evaluating whether delayed umbilical cord clamping improves early health outcomes compared with immediate clamping in preterm infants born between 32 and 37 weeks. Preterm babies are at increased risk of complications, and the timing of cord clamping may affect blood volume, iron levels, and neonatal adaptation after birth. This study is conducted in a tertiary maternity center in Tunisia to better understand these effects. Participants are assigned to one of two groups delayed cord clamping, where the umbilical cord is clamped at least 30 seconds after birth allowing continued placental blood flow or immediate cord clamping, where the cord is clamped within seconds after birth without delay. Both procedures are done right after delivery with standard neonatal stabilization and care provided accordingly. During the study, newborns will be monitored for early outcomes such as respiratory status, need for resuscitation, hemoglobin levels, and neonatal morbidities including anemia, necrotizing enterocolitis, and intraventricular hemorrhage. Researchers will track neonatal mortality within 7 days or until hospital discharge, length of NICU stay, and other health indicators. Participation lasts through early neonatal hospitalization and follow-up assessments may occur up to several months after birth.

Sepsis is a serious condition caused by a severe infection leading to widespread inflammation and organ failure. This research evaluates the early use of norepinephrine NE alongside standard treatment to manage low blood pressure and support organ function in patients with severe sepsis. The study aims to determine if starting NE earlier can better restore blood pressure and improve outcomes compared to standard care alone. Participants are randomly assigned to one of two groups. One group receives low-dose norepinephrine infusion early after observing low blood pressure due to sepsis, in addition to usual treatments following the 2021 Surviving Sepsis Campaign guidelines. The other group receives a placebo infusion plus standard therapy. The norepinephrine is given intravenously at a dose adjusted by body weight to maintain target blood pressure. The study monitors treatment over the initial hours and days after sepsis diagnosis. During the study, participants will be closely monitored for blood pressure control within 6 hours, changes in blood lactate levels, the volume of fluids administered within 48 hours, and mortality at 28 days. Researchers will assess organ function and overall response to treatment, following patients throughout their ICU stay and beyond. The trial aims to provide detailed information on the safety and impact of early norepinephrine use in sepsis management.