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.
The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics, pharmacodynamics, and early clinical activity of INCB086550 in participants with advanced solid tumors who have failed prior treatments.
This was a Phase 3 randomized, parallel-group, double-blind, placebo-controlled trial (blinded treatment [BT] period) followed by an open-label extension [OLE] period intended to evaluate the efficacy and safety of oral PRN1008 in moderate to severe pemphigus. After completing the open-label extension period, eligible participants might continue in a long term extension (LTE) Period of 48 weeks.
The aim of this clinical trial is to assess the effect of treatment with a monoclonal antibody called atezolizumab in patients diagnosed with a type of lung cancer called malignant pleural mesothelioma. The efficacy (whether the treatment works), safety and tolerability (side effects of treatment) of atezolizumab plus bevacizumab in combination with standard chemotherapy versus bevacizumab in combination with standard chemotherapy will be investigated.
This study will evaluate the efficacy, safety, and biomarker effects of RO7234292 (RG6042) compared with placebo in participants with manifest Huntington's disease (HD)
Friedreich ataxia is the most frequent early-onset autosomal recessive hereditary ataxia. It is caused by a pathological expansion of a GAA repeat in the first intron of the frataxin gene (FXN) and results in decreased levels of FXN protein. FXN deficiency results in a relentlessly progressive neurodegenerative condition which frequently presents around puberty. Patients gradually lose coordination, become dysarthric and are frequently wheel-chair bound as adolescents. There is no disease modifying therapy and many patients die prematurely of cardiomyopathy. It was subsequently found that the FXN gene is silenced at the chromatin level by the formation of heterochromatin and that this heterochromatin formation can be antagonized by histone deacetylase inhibitors (HDACi) (Chan et al., 2013). A recent proof-of-concept clinical study on ten patients with Friedreich ataxia demonstrated that FXN levels can be restored to those seen in asymptomatic carriers using the class III HDACi nicotinamide at a dose that is well tolerated by patients (Libri et al., 2014). Since carriers are asymptomatic, this degree of restoration of FXN expression might be expected to halt disease progression. Nicotinamide readily crosses the blood brain barrier and has previously been given at high doses for long periods to normal individuals without serious adverse effects (Gale et al., 2004; Knip et al., 2000). This study will be the first to provide clinical evidence for the efficacy and safety of nicotinamide in patients with Friedreich´s ataxia.
Study of masitinib plus docetaxel as first-line chemotherapy in men with metastatic castration-resistant prostate cancer.
Sedation management of the critically ill patients is still a challenge for the pediatric intensivists. Worldwide the most common sedation approach includes the concomitant use of opioids and benzodiazepines. The use of these drugs is associated with adverse events contributing with morbidity, such as decreased spontaneous ventilation, withdrawal syndrome and delirium onset. Moreover, benzodiazepine demonstrated a neurotoxic apoptotic effect that could potentially impact neurocognitive outcome. Dexmedetomidine (DEX) is a selective alpha-2-adrenergic agonist with sedative, analgesic and anxiolytic effects. Its unique pharmacological profile allows reaching a conscious sedation state with minimal respiratory depression, promoting faster ventilation weaning and better collaboration with the medical staff. Moreover, DEX seems to present adjuvant properties towards withdrawal syndrome and delirium. Finally, some studies in animals suggested that Dexmedetomidine might have a role of neuro-protection, especially in a contest of cerebral ischemia. Currently, the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved DEX only for the adult population and for sedation lasting not more than 24 hours. The Italian Medicines Agency (AIFA), in January 2016, approve DEX in children for specific indications including difficult sedation in mechanically ventilated critically ill patients. Up to now, few data are still available regarding its efficacy and safety for prolonged sedation in Pediatric Intensive Care Units (PICUs) and no studies have reported the use of DEX after AIFA's approval so far. Aims of the study are: to evaluate the characteristics of DEX use for prolonged sedation ≥24 hours in critically ill children (indication, dosages, time of infusion, time of infusion weaning, association with other drugs); to evaluate its efficacy in terms of comfort and conventional drug sparing, using standardized and validate measures; further, to evaluate its efficacy in terms of reduction of incidence of withdrawal syndrome and delirium; to evaluate its safety profile collecting any adverse event potentially correlated with its administration; to define if efficacy and safety could differ among approved indications versus not-approved ones. Design: Multicenter observational prospective study, involving tertiary-care PICUs. Study period: From January 2016 up to reaching of the calculated sample size (N patients =163). Population: All critically ill patients <18 years who received prolonged sedation including DEX for ≥24 hours. In case of multiple infusions, only data regarding the first infusion will be included. Exclusion criteria: extreme prematurity (<28 weeks of gestational age), hypersensitivity to the active substance, incomplete data form. . Collecting data strategy: Data will be prospectively collected from each Institution by means of a anonymous standardized form completed by two different investigators per center. For each patient, the following variables will be collected: demographics characteristics (age, gender, race, weight) and clinical features (main diagnosis, associated morbidities, PIM3 score ad admission, number of high intensity interventions during PICU-stay, mechanical ventilation features, inotropic drugs use, length of stay, survival at discharge); DEX administration characteristics (indication, loading dose, minimum and maximum dosages, duration of DEX infusion, duration of DEX infusion weaning) and information on concomitant use of analgesics or sedative drugs (name of drugs and respective dosages at DEX starting time and 24 hours later); clinical scores of analgesia and sedation (Comfort Behavior Scale, CBS), withdrawal syndrome (Withdrawal Assessment Toll-1, WAT-1) and delirium (Cornell Assessment of Pediatric Delirium, CAPD) depending on the respective indication, registered immediate pre-DEX and 24 hours later; any adverse event potentially related to DEX administration and any related intervention, if present. In particular, we the investigators evaluate if bradycardia, hypotension (with or without poor perfusion), hypertension, agitations or other events are registered during the infusion. Further, investigators will register if any sign of DEX withdrawal (tachycardia, hypertension, agitation, other) are present immediately after the infusion. Statistical analysis: Descriptive and analytic statistics will be performed according with the variable characteristics. A causal multivariate model will be developed to identify any significant risk or protective factors towards adverse outcomes (ineffective sedation, onset of adverse events).
All patients with atrial fibrillation who are treated with vitamin-k antagonists (warfarin, phenprocoumon) or non vitamin K oral anticoagulants (Dabigatran, Rivaroxaban, Apixaban, Edoxaban) in real world settings will be recorded in this register. Within this register a characterization of patients and therapy (with regard to medication, dose and duration) will be done. On basis of defined clinical relevant end points the long-term efficacy and safety will be evaluated.
This is a controlled, open study designed to compare the effects of dual-release hydrocortisone preparations versus conventional glucocorticoid therapy on clinical, anthropometric parameters, metabolic syndrome, hormonal profile, bone status, quality of life, reproductive, sexual and psychological functions and treatment compliance in patients affected by congenital adrenal hyperplasia due to 21 OH deficiency.
Although some studies have brought some evidences about the efficacy of positive expiratory pressure (PEP)-mask therapy as an airway clearance technique, yet it is not clearly understood what is the contribution of this technique in modifying peripheral ventilation inhomogeneity, a typical feature of patients with Cystic Fibrosis (CF). The aim of this study is to investigate how PEP-MASK affects ventilation inhomogeneity in children and adolescents, with moderate to normal CF lung disease by the change in acinar airways (Sacin), lung clearance index (LCI) and conductive airways (Scond) indexes derived from nitrogen multiple-breath washout test (N2MBW).