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.
Sanfilippo syndrome Type A, or Mucopolysaccharidosis (MPS) IIIA, is a rare lysosomal storage disease caused by deficiency of the enzyme heparan N-sulfatase (sulfamidase). In the absence of this enzyme, there is an accumulation of the glycosaminoglycan, heparan sulfate, resulting in progressive neurodegeneration. Symptoms are usually first noted in the 1st or 2nd year of life, although definitive diagnosis is often delayed, with an average age of diagnosis of 4.5 years. The disease is characterized by developmental delays initially, followed by neurological developmental arrest, then regression. These developmental deficits are typically associated with severe behavioral disturbances. Patients have a significantly reduced lifespan, with few surviving beyond the 2nd or 3rd decade. The purpose of this study is to evaluate the safety and efficacy of recombinant human heparan-N-sulfatase (rhHNS) in pediatric patients with Early Stage Mucopolysaccharidosis Type III A Disease.
The purpose of this study is to evaluate whether endocavitary intramyocardial injection of autologous bone-marrow-derived CD133+ cells is safe on the basis of number of adverse events, with follow-up assessments extending up to 1 year after enrolment.
This study is to determine whether canakinumab is able to induce and maintain a clinically meaningful reduction of disease activity in participants with Hereditary Periodic Fevers (HPF) compared to placebo.
The purpose of this study is to evaluate the immunogenicity and safety of GSK Biologicals' HZ/su vaccine administered on a 0- and 1- to 2-months schedule in adults 18 years of age or older who are receiving chronic immunosuppressive therapy.
Primary Objective: To compare the insulin glargine/lixisenatide fixed ratio combination to lixisenatide alone and to insulin glargine alone (on top of metformin treatment) in HbA1c change from baseline to week 30. Secondary Objective: To compare the overall efficacy and safety of insulin glargine/lixisenatide fixed ratio combination to insulin glargine alone and to lixisenatide alone (on top of metformin treatment) over a 30 week treatment period in patients with type 2 diabetes
This is a prospective, non-interventional, longitudinal study of the natural history and function of approximately 60 patients with MTM from the United States, Canada and Europe. The duration of the study, including the enrollment period, will be 36 months. Data from the study will be used to characterize the disease course of MTM and determine which outcome measures will be the best to assess the efficacy of potential therapies.
The purpose of this study is to evaluate the impact of Advagraf, prolonged-release, once daily tacrolimus formulation, on long-term graft survival in kidney and liver allograft recipients. This study will also evaluate the overall long-term impact of Advagraf on kidney and liver allograft recipients.
Diseases do not only have a physical role in people's live, but they usually involve changes in life as whole. They may modify the structure of the conjunction with life setting, thus, deeply impacting relationships with others. While clinical results of new therapies for hematological diseases are well documented in scientific literature in terms of prolonged life expectancy or remission from disease, less is known about problems and barriers preventing the return of patients with a chronic blood ailment to everyday life. Indeed, there are no published data on this topic within the Italian context. The present explorative study aims at identifying the main problems with which patients affected by a Chronic Hematological Disease (CHD) deal when returning to everyday working life, factors associated with work reintegration and, finally, to understand the need for facilitators enhancing reintegration outcomes. Results from this study will be also helpful to raise consciousness about the problem of reintegration into the labour market of workers with CHD and to call for awareness campaigns for the general public and health professionals.
The primary objective of this study is to evaluate sustained clinical remission (for the definition see below) in patients with inflammatory bowel disease receiving long-term (> 2 years) scheduled treatment with infliximab. Secondary objectives include: - to identify predictors of sustained clinical remission during long-term infliximab scheduled treatment - to identify predictors of loose of response during infliximab scheduled maintenance treatment - to identify predictors for maintaining clinical remission in patients who discontinue infliximab because of long-lasting steroid-free clinical remission - to evaluate percentage of surgery during and after treatment (total follow-up) - to evaluate safety of long-term infliximab scheduled treatment List the clinical hypotheses Infliximab is indicated and recommended in moderate to severe inflammatory bowel disease patients who not tolerate or are not responsive to conventional therapies. Most of randomized clinical trials about the use of infliximab in inflammatory bowel diseases are limited to 52 weeks and very few data come from some observational studies about results of prolonged (over one year) treatment with infliximab. No validated predictors of sustained clinical remission or loss of response are available so far. Moreover, few data are available about the hypothetical reduction of IBD related surgery in the "biological era". In this proposal we suggest the following hypotheses: - infliximab scheduled treatment may be efficacious in maintain long-term clinical remission; - among clinical, laboratory and endoscopic data some predictors of sustained clinical remission during infliximab long-term scheduled treatment may be found; - among clinical, laboratory and endoscopic data some predictors of loss of response during infliximab long-term scheduled treatment may be found; - among clinical, laboratory and endoscopic data some predictors of sustained clinical remission after infliximab discontinuation because of long-lasting (> 6 months) steroid-free clinical remission may be found; - maintenance of remission with infliximab may reduce rates of surgery over time; - long-term scheduled treatment with infliximab may be safe and well tolerated. Results from this study may really help clinicians to make practical decisions in these particular clinical settings.
Open-label phase Ib/II, multicenter, international non-comparative trial. This study is designed to determine the safety and efficacy of the novel salvage regimen (CBd) followed by a carfilzomib maintenance in patients with relapsed or refractory multiple myeloma. Patients will be evaluated at scheduled visits in up to 4 study periods: pretreatment, treatment, maintenance and long-term follow-up (LTFU).