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NCT ID: NCT04470713 Completed - Sandhoff Disease Clinical Trials

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

RETRIEVE
Start date: July 31, 2019
Phase:
Study type: Observational

This study is being conducted to better understand the natural course of GM1 gangliosidosis, GM2 gangliosidoses and Gaucher disease Type 2 (GD2). Information is planned to be gathered on at least 180 patients with GM1 gangliosidosis, GM2 gangliosidoses, and Gaucher Disease type 2. Retrospective data collection is planned for at least 150 deceased patients (Group A). Group B is for patients alive at the time of enrollment. In Group B it is planned to prospectively collect more comprehensive data from at least 30 patients. The purpose of this study is to collect relevant information for a adequate design of a potential subsequent research program in these diseases. In this study no therapy is being offered.

NCT ID: NCT04469985 Completed - Clinical trials for Allogeneic Stem Cell Transplantation in Elderly Patients

Allogeneic Stem Cell Transplantation (ALLO-SCT) in Elderly: 17-years Retrospective GITMO Survey

Start date: February 1, 2020
Phase:
Study type: Observational

The age of patients' population is increasing every year, as the life expectancy. Thus, the incidence and prevalence of hematological cancers is increasing. In particular, acute leukemias and myelodisplastic syndromes are the most frequent diseases of the hematopoietic system. Allogeneic stem cell transplantation (allo-SCT) is the main therapeutic options for these diseases, and the number of allo-SCTs in the elderly is currently in progressive growth. This is partially due to the improvement of the allo-SCT procedure over the years, with the amelioration of donor selection, HLA typing, graft versus host disease (GVHD) and infections prophylaxis and treatment. Nevertheless, there is still an unmet clinical need, represented by the lack of extensively and detailed outcome analysis of elderly patients addressed to allo-SCT. With this aim, we planned this retrospective analysis of allo-SCTs in patients older than 60 years, within the GITMO transplant activity of the last 17 years. This study will help us in designing future prospective trials, including a comprehensive geriatric assessment of frailty, in order to address to allo-SCT those elderly who have the highest probability to achieve the best long term outcome with the lowest transplant-related mortality and morbidity.

NCT ID: NCT04469465 Completed - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)

Start date: December 16, 2020
Phase: Phase 3
Study type: Interventional

The main objective of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH.

NCT ID: NCT04467840 Completed - COVID-19 Clinical Trials

Opaganib, a Sphingosine Kinase-2 (SK2) Inhibitor in COVID-19 Pneumonia

Start date: August 21, 2020
Phase: Phase 2/Phase 3
Study type: Interventional

A phase 2/3 multi-center randomized, double-blind, parallel arm, placebo- controlled study in Adult Subjects Hospitalized with Severe SARS-CoV-2 Positive Pneumonia to determine the potential of opaganib to improve and/or stabilize the clinical status of the patient.

NCT ID: NCT04466592 Completed - Clinical trials for Esophageal Neoplasms

Health and Employment After Gastro Intestinal Surgery - HEAGIS1

HEAGIS1
Start date: December 13, 2019
Phase: N/A
Study type: Interventional

By using the M.A.D.I.T. methodology and the Dialogics science, SALVO Project aims to develop operational guidelines to support oncological target patients in the resumption of their daily post-operative activities. The research will implement an instrument for the purpose of measuring the health need of participants who are admitted to the surgical ward. Therefore, targeted interventions will be implemented with participants, and efficacy will be evaluated in order to define treatment guidelines. The principal aim of this study is to create a validated and replicable intervention model for supporting patients who undergone surgery for esophagus and gastro intestinal cancer.

NCT ID: NCT04465149 Completed - Clinical trials for Local Anesthetic Efficacy

Efficacy of Differents Anaesthetics in Mandibular Third Molar Germectomy

Start date: June 30, 2018
Phase: N/A
Study type: Interventional

Aim: To compare the clinical efficacy of local anaesthetics with articaine 4% or mepivacaine 2% (both with epinephrine 1:100.000) using different anaesthetic techniques to perform germectomy of lower third molars and to assess patients' feelings and pain during surgery. Methods: 50 patients (ranged 11-16 years) who required germectomy of mandibular third molars were recruited. Each patient received local anaesthesia on one side with articaine inoculated with plexus technique while on the other side with mepivacaine using inferior alveolar nerve block technique. The patients' evaluation was performed on pre and intraoperative tactile-pressure feelings and intraoperative pain with four levels on the analogic visual scale (VAS).

NCT ID: NCT04463849 Completed - COVID19 Clinical Trials

Effects of COVID-19 Infection on Beta-cell Function in Euglycemic Patients

COBETOX
Start date: June 30, 2020
Phase: N/A
Study type: Interventional

In recent months, a new coronavirus, SARS-CoV-2, has been identified as the cause of a serious lung infection named COVID-19 by the World Health Organization. This virus has spread rapidly among the nations of the world and it is the cause of a pandemic and a global health emergency. There is still very little scientific evidence on the virus, however epidemiological data suggest that one of the most frequent comorbidities is diabetes, along with hypertension and heart disease. There is no scientific evidence on the possible effects of this infection on the function of the β cell and on glycemic control. Clinical evidence seems to suggest that COVID-19 infection mostly affects the respiratory system, and an acute worsening of glycemic compensation is not described as generally observed in bacterial pneumonia. However, previous work on acute respiratory syndromes (SARS) caused by similar coronaviruses, had described that the infection has multi-organ involvement related to the expression of the SARS coronavirus receptor, the angiotensin 2 converting enzyme, in different organs, especially at the level of endocrine pancreatic tissue. In the population of this previous work, glucose intolerance and fasting hyperglycaemia have been described and in 37 of 39 diabetic patients examined, a remission of diabetes was observed three years after the infection. It is possible that the coronaviruses responsible for SARS may enter the pancreatic islets using the angiotensin 2 converting enzyme receptor, expressed at the level of the endocrine pancreas, thus causing diabetes. Additionally, previous literature on coronavirus infections (SARS and MERS or Middle-East Respiratory Syndrome) suggested that diabetes could worsen the evolution of the disease. In particular, in case of Middle-East Respiratory Syndrome-CoV infection, diabetic mice had a more prolonged serious illness and a delay in recovery regardless of the viremic titer. This could probably be due to a dysregulation of the immune response, which results in more serious and prolonged lung disease. There are currently no data on pancreatic beta cell function in patients with COVID-19.

NCT ID: NCT04463108 Completed - Clinical trials for Depressive Disorder, Major

A Study to Describe Routine Treatment Pathways in Participants With Major Depression and Active Suicidal Ideation With Intent in Italy

ARIANNA
Start date: June 23, 2020
Phase:
Study type: Observational

The purpose of the study is to describe the pharmacological and non-pharmacological treatment utilization pathways in Italian routine clinical practice of participants with Major Depressive Disorder (MDD) and active suicidal ideation with intent.

NCT ID: NCT04462536 Completed - Stroke, Acute Clinical Trials

Efficacy and Safety of Nerinetide in Participants With Acute Ischemic Stroke Undergoing Endovascular Thrombectomy Excluding Thrombolysis

ESCAPE-NEXT
Start date: December 6, 2020
Phase: Phase 3
Study type: Interventional

The primary purpose of this study is to determine if a single dose of nerinetide can reduce global disability in people who have had a stroke and are selected for endovascular therapy without the use of a tissue plasminogen activator (alteplase, tenecteplase, or equivalent).

NCT ID: NCT04462315 Completed - Clinical trials for Cardiovascular Diseases

10-Years Follow-up of the EXAMINATION Trial

EXAMINAT10N
Start date: May 1, 2015
Phase:
Study type: Observational

The EXAMINATION trial was a superiority trial that compared everolimus-eluting stents (EES) versus bare-metal stents (BMS) in an all-comer ST-segment elevation myocardial infarction (STEMI) population. The patient-oriented endpoint was not superior at 1-year, but it was at 5-year. However, very-long term follow-up is unknown. The study had an independent Clinical event Committee (CEC). All events were adjudicated by an independent clinical committee, according to the Academic Research Consortium 1.