Active clinical trials for "Syndrome"

Anti-CD20 monoclonal antibodies are emerging as the steroid-sparing therapy of choice for nephrotic syndrome.This Randomized Clinical Trial seeks to evaluate whether Rituximab biosimilar maintains drug-free disease remission in patients with steroid-dependent nephrotic syndrome for 12-24 months and verify its superiority vs. mycophenolate mofetil (1,200 mg/m2 orally in 2 daily doses). The investigators will compare the risk of relapse to test this hypothesis (primary outcome). Secondary objectives will include assessing short- and long-term side-effects and developing specific biomarkers of sensitivity to therapy. Patients will be recruited, treated and followed at IRCCS G Gaslini and IRCCS Bambino Gesù where laboratory studies will be performed at in-site facilities.

Infants with Down syndrome (DS) develop slower than their typically developing peers. Physical therapist (PT) supervised home programs have the potential to optimize gross motor development in a financially feasible way. An inexpensive orthotic garment (Hip Helpers®) is commonly employed by PTs as a home program supplement, but its effectiveness has not yet been investigated. The garment is worn as pliable shorts over a child's lower extremities to keep upper legs together, promoting a narrow base of support. This encourages activation of upright postural muscles to improve gross motor skill development. The purpose of this randomized controlled study is to investigate the impact of a home program using the Hip Helpers® orthotic garment on gross motor skill acquisition in infants with DS. We hypothesize that the addition of a structured home program using Hip Helpers®, supervised by a PT and implemented by parents, will increase the rate at which infants with DS acquire gross motor skills. Thirty-four participants, consisting of children who are at least three-months-old and are not yet able maintain sitting independently, will be randomly assigned to a control (n=17) or intervention group (n=17). PTs at pediatric therapy agencies will initiate the home program and administer the Gross Motor Function Measure-88 (GMFM-88) at regular intervals to monitor gross motor skill acquisition until the child is able to take three independent steps. Groups will be compared on the length of time elapsed between the acquisition of identified gross motor skills using independent t-tests. GMFM-88 scores will be compared between the two groups at different ages to identify trends using independent t-tests. The contribution of this project will be significant by informing physical therapists about the effectiveness of an inexpensive orthotic garment used in a supervised home program on gross motor outcomes in infants with DS.

More than 25% of patients referred for diagnostic coronary angiography and percutaneous coronary intervention (PCI) due to acute coronary syndrome (ACS) suffer from non-valvular atrial fibrillation (AF). In this particular setting, balancing between the prevention of thrombosis and the risk of bleeding remains challenging. Oral anticoagulation (OAC) prevents stroke and systemic embolism, but has not been shown to prevent stent thrombosis (ST). Dual antiplatelet therapy (DAPT) reduces the incidence of recurrent ischemic events and ST, but is less effective in reducing the incidence of cardioembolic stroke associated with AF. A common guideline-supported practice is to combine three drugs (OAC, aspirin and clopidogrel) in a triple therapy, which is associated with high annual risk (up to 25%) of major bleeding. Thus, new therapeutic strategies are urgently needed to maintain the efficacy while improving the safety of treatment in patients with AF and ACS undergoing PCI. This is a prospective, randomized, open-label, blinded-endpoint, non-inferiority trial. 2230 patients with non-valvular AF that had undergone successful PCI due to an ACS within the previous 72 hours will be randomized in 1:1 ratio to receive one of the two treatments: dual therapy with dabigatran (150 mg twice daily or 110 mg twice daily) and ticagrelor (90 mg twice daily for 1 month, followed by 60 mg twice daily up to 12 months), or standard therapy according to current guidelines triple therapy with dabigatran (150 mg b.i.d. or 110 mg b.i.d.) plus clopidogrel (75 mg o.d.) plus aspirin (75 mg o.d.) followed by double therapy depending on the bleeding and ischaemic risk. Study treatment will be continued for 12 months. The primary study end-point is the first major or clinically relevant non-major bleeding event (per ISTH), in a time-to-event analysis. The main secondary end-point is a composite efficacy end-point of thromboembolic events (myocardial infarction, stroke, or systemic embolism), death, or unplanned revascularization (PCI or coronary artery bypass grafting) at 12 months. We expect that dual antithrombotic therapy including reduced dose ticagrelor and dabigatran is at least non-inferior regarding bleeding risk and ischaemic protection, compared to the standard triple therapy in patients with AF and after ACS, treated with PCI.

This study is a multi-center, randomized, partially double-blind, and placebo-controlled Phase Ib clinical trial of inhaled CO (iCO) for the treatment of sepsis-induced acute respiratory distress syndrome (ARDS). The purpose of this study is to evaluate the safety and accuracy of a Coburn-Forster-Kane (CFK) equation-based personalized iCO dosing algorithm to achieve a target carboxyhemoglobin (COHb) level of 6-8% in patients with sepsis-induced ARDS. We will also examine the biologic readouts of low dose iCO therapy in patients with sepsis-induced ARDS.

This phase Ib/II trial best dose, possible benefits and/or side effects of omacetaxine and venetoclax in treating patients with acute myeloid leukemia or myelodysplastic syndrome that has come back (recurrent) or does not respond to treatment (refractory) and have a genetic change RUNX1. Drugs used in chemotherapy, such as omacetaxine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving omacetaxine and venetoclax may help to control the disease.

The aim of this study is to evaluate whether the use of a commercially available standardized olive extract (Tensiofytol) and a commercially available combination preparation (Cholesfytol NG) by individuals with metabolic syndrome Leads to a clinically relevant reduction of blood pressure on the short term, Leads to a clinically relevant reduction of cholesterol levels, especially LDL, Leads to a change in oxidative stress biomarkers. Participants will be stratified by sex and menopausal status before randomization to one of the three treatments for 8 weeks: Tensiofytol: 100 mg oleuropein and 20 mg hydroxytyrosol per day Cholesfytol NG: 2.9 mg monacoline K and 10 mg hydroxytyrosol per day Placebo All treatments have an identical shape and color and should be used in the same way (oral intake; 3 capsules/day during dinner). No dietary instructions are given and participants are asked not to change their dietary habits, nor to start other therapies (medication, supplements, slimming diets, extra physical activity, etc.) during their study period. Standardized questionnaires are used to obtain information on demographics, dietary habits and side effects. At baseline and after 8 weeks, 27 ml blood is drawn for various biological analyses, and blood pressure, BMI and waist circumference are measured.

The project will highlight the potential benefit of endovascular therapy on post thrombotic syndrome reduction after proximal iliac DVT. There is actually not real standard of care for the treatment of this pathology. A clear evidence of efficacy of endovascular therapy will be of great benefit for both the patients and the healthcare system, and will provide new data for further international guidelines

Acute coronary syndromes (ACS) result from coronary plaque(s) disruption, which initiates a thrombotic process leading to partial or complete obstruction of the vessel lumen with subsequent myocardial ischaemia and necrosis. The mainstay of treatment is currently focused on the re-establishment and maintenance of coronary artery patency using anti-platelets and anticoagulants with or without mechanical dilatation and stenting of the culprit artery. Despite important advances in management, ACS still carries a risk of substantial morbidity and mortality. The improved efficacy of novel anti-platelet and anticoagulant agents have been limited by increased risk of haemorrhagic events. Future breakthroughs in management are most likely to arise from targeting other relevant pathophysiological pathways. Particularly, the immune response which is an important process that has been neglected in the management of patients with ACS. In this trial the investigators investigate the efficacy of low dose IL-2 compared with placebo in patients with ACS.

This study will determine the safety profile, maximum tolerated dose (MTD), dose-limiting toxicities (DLT), and recommended Phase 2 dose (RP2D) of SX-682 in the treatment of patients with Myelodysplastic Syndromes (MDS).

Antiphospholipid syndrome (APS) is defined by thrombosis or obstetric complication (≥ 3 spontaneous miscarriages or fetal death or prematurity <34 weeks gestation-related amenorrhea (SA)) associated with antiphospholipid antibodies. The rate of term pregnancies has been improved by conventional treatment (aspirin 100 mg / day with low molecular weight heparin (LMWH) in an isocoagulant dose) to almost 75%. In the PROMISSE study, when considering progressive pregnancies after 20 weeks, 19% of pregnancies presented at least one complication despite the treatment (maternal, fetal or neonatal complications) related to APS. In the European APS register, maternal complications and IUGR were observed in 13% of cases, and prematurity in approximately 14% of cases despite treatment. In a previous study of 72 pregnancies during a LAS, we observed, under aspirin and LMWH, 25% of fetal losses, and 10% of at least one maternal and / or fetal complication or prematurity. The presence of lupus, a history of thrombosis, a circulating anticoagulant (ACC) and a triple positivity of antiphospholipids are considered to be factors associated with a poor obstetrical prognosis. Hydroxychloroquine (HCQ) has anti-inflammatory and anti-thrombotic properties. In vitro studies have shown that HCQ is able to restore the expression of placental annexin V, which has an anticoagulant effect and prevent the attachment of antiphospholipid antibodies to the placenta. HCQ during lupus decreases the thrombotic risk and its usefulness during thrombotic APS has been shown in a French series. In a European study, the addition of the HCQ to conventional treatment improved term pregnancies by 70% in the event of refractory APS. Its use during pregnancies of patients with lupus, the numerous data on tolerance during pregnancy and the follow-up of children born to mothers exposed to the HCQ demonstrates a reassuring tolerance profile for the mother and the fetus. The objective of this clinical trial is to evaluate the benefit of addition or no of hydroxychloroquine to conventional treatment in obstetric APS.