There are about 21062 clinical studies being (or have been) conducted in Italy. The country of the clinical trial is determined by the location of where the clinical research is being studied. Most studies are often held in multiple locations & countries.
Children with congenital myotonic dystrophy (CDM) present at birth with respiratory insufficiency, talipes equinovarus, feeding difficulties and hypotonia. There is a 30% mortality rate in the first year of life. Children with childhood onset myotonic dystrophy present with symptoms later on but soon develop behavioural difficulties and learning difficulties and are at risk for autistic features and gastrointestinal symptoms. The ability to conduct a therapeutic trial in children with CDM or ChDM is directly limited by the lack of available data regarding appropriate clinical endpoints and biomarkers. Whereas there is an active Italian collaboration recruiting adults with DM1 to study muscle and multisystem aspects in this population, there is no active network in Italy involved in the pediatric population with DM1. Though the underlying mechanism is the same in adult DM1, in CDM and ChDM there are specific challenges to the pediatric population. The aim of this project is to coordinate the Italian Child Neurologist actively involved with CDM and ChDM in a common effort of standardizing protocols and procedures to be applied in the care of these patients. Specific aims are to collect functional measures and clinical information over time to define clinically meaningful endpoints and outcome measures in preparation for international therapeutic clinical trials. This project will contribute to the ongoing international study in CDM by recruiting additional patients from all over Italy and will extend the investigations to the childhood onset forms as an additional add-on pilot study in view of potential treatment options. The investigators expect that the Italian network, with Telethon support, will provide the necessary backbone for trial readiness in the pediatric population both at the national and international levels.
Autoimmune cytopenias resistant to treatment are among the most common clinical manifestations observed in patients with congenital alterations of the immune system, such as primary immunodeficiencies (PI). The exact contribution of immune system alterations to the pathogenesis of autoimmune cytopenias has not yet been fully elucidated. Moreover, conventionally employed therapeutic strategies often fail, leading to increased healthcare costs, high morbidity, and even mortality. Therefore, there is a need to establish clinical guidelines for diagnosis and to identify early biomarkers capable of identifying individuals responsive to therapy. Thus, a systematic approach to the study of such pathologies will allow for the identification of early biomarkers and facilitate the development of targeted therapeutic strategies
The sagittal alignment of Adolescent Idiopathic Scoliosis (AIS) curves has attracted growing interest in recent years, to the extent that it has become a pivotal point in the Lenke classification, with the introduction of a sagittal modifier. In particular, thoracic curves, partly due to the theory of anterior overgrowth, are almost invariably characterized by thoracic hypokyphosis, which can be severe (T5-T12 thoracic kyphosis < 10°, that corresponds to a Lenke - sagittal modifier). However, the development of such a severely abnormal sagittal alignment has consequences that are not limited only to the thoracic region, but it rather results in a disruption of the entire sagittal spinal alignment. In fact, thoracic hypokyphosis tends to shift the C7SVA backward and to decrease the T1 slope. As a compensation, this ultimately leads to the development of a cervical kyphosis in order to translate the head forward and maintain global sagittal balance. While the interplay relationship between thoracic hypokyphosis and the development of cervical kyphosis has been well established in modern literature, the results regarding the amount of spontaneous correction of cervical kyphosis achieved after hypokyphotic AIS correction are conflicting. There are several papers in literature that study the complex relationship between AIS and cervical kyphosis, and they did not report any improvement in cervical lordosis after AIS correction, even when successful restoration of thoracic kyphosis (TK) was achieved. Conversely, other authors did report an improvement in cervical sagittal alignment after AIS correction. The aim of the present paper is firstly to assess the amount of spontaneous change in cervical sagittal alignment after correction of AIS with associated severe thoracic hypokyphosis (<10°). Secondly, the aim of the study is to seek for any radiographical parameter able to predict the postoperative cervical sagittal alignment in these patients, via a multivariate regression analysis.
The aim of this randomized controlled study is to investigate if the sequential administration of focused shock wave therapy and of a rehabilitation program based on the strengthening of gluteal muscles is more effective than a standard shock wave treatment for greater trochanteric pain syndrome
We envisioned a scenario where the interaction between the ATM-Chk2/ATR-Chk1 pathways and Hippo enables GC cells to overcome chemotherapy-induced death stimuli. First, ATM-Chk2 and ATR-Chk1 were found to be activated across all the GC molecular subtypes. Moreover, a number of genes associated with their basal activation are recurrently mutated or amplified. Thus, we retrospectively characterized a cohort of GC patients treated with first-line therapy for DDR- and Hippo-related markers, identifying a signature predicting inferior PFS and OS. This exploratory analysis provided the necessary information (frequency of candidate biomarkers and effect difference between groups) for a prospective study with validation purposes, which is the main goal of this trial.
Endovascular treatment of superficial femoral artery stenosis/obstruction is still the subject of debate in the scientific literature. Previous clinical studies have in fact reported conflicting data regarding the benefits of implanting self-expanding Nitinol stents in the superficial femoral artery district compared to simple percutaneous transluminal angioplasty. Invariably, patient comorbidities and anatomic characteristics of the lesions appear to be important factors influencing procedural success and one-year patency rates. Additionally, there are concerns regarding the potential clinical impact of stent fractures, reported at rates ranging from 12% to 37.2% at one year. Despite the improved outcomes seen with newer Nitinol stent designs, the primary limitations of stenting in the superficial femoral artery are the use of multiple overlapping stents or long stents and the associated potential rate of stent fracture resulting reocclusion of the treated superficial femoral artery and clinical worsening of patients who in most cases are initially treated for disabling claudication. Being able to preoperatively determine in which patients there are risk factors prognostically associated with a higher rate of fracture/reocclusion could represent a help for the operator in choosing the best therapeutic strategy.
Fever represents the main cause of admission to the emergency room in older people pediatric. It occurs in the presence of a wide range of pathologies, from infectious forms (the most common, viral or bacterial) up to more complex and/or systemic forms (such as inflammatory or neoplastic ones). The drugs currently indicated for the management of fever in children are: paracetamol and ibuprofen. There are no recent studies conducted in pediatric population, who have demonstrated the greater effectiveness of therapy with paracetamol or ibuprofen, The objective of our study is, therefore, to identify which therapy is most appropriate for controlling body temperature e of associated symptoms in pediatric patients arriving in the emergency room with fever.
The aim of the study is to describe the association between the perception of cardiovascular (CV) risk and the actual CV risk and, secondarily, to detect the actual CV risk to assess the prevalence of clinical risk factors, determined by means of appropriate instruments.
Actual indications for stage Ia NSCLC patients suggest lobectomy and lymphadenectomy. On the other hand, recent studies reported non-inferiority of segmentectomy in case of nodule < 2cm or with ground glass appearance at computed tomography. However, most of these studies did not report specifical analysis on kind of segmentectomy (single or multiple) and kind of lobectomy. Moreover, a specific study on solid nodules only is still missing. For these reasons, the effectiveness of segmentectomy instead lobectomy for every kind of segment or tumor is still to be defined. Aim of this study is to analyze survival outcome in patients underwent single or multiple segmentectomy compared to lobectomy in stage I non small cell lung cancer.
Prospective, multicenter, noninterventional, nonprofit study of a cohort of patients with glioma, aimed at validating miRNA-serum signatures associated with IDH1 status and prognosis, as reliable, specific and sensitive circulating diagnostic biomarkers also useful for improve prognostic stratification of patients. The study will be conducted on serum samples at diagnosis, at 4-6 days postoperatively and/or at the first post-surgery follow-up, in a new cohort of glioma patients and representative of different IDH1 mutational statuses. Furthermore, because comparison of miRNA expression profiles in serum and tissue may provide further evidence to support the use of serum miRNAs as reliable biomarkers reliable, their expression will also be analyzed, where possible, in tissue biopsies from the same patient and compared with the expression profiles of serum miRNAs.