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NCT ID: NCT05712577 Recruiting - Rare Diseases Clinical Trials

Psychological Support in Adult Patients With Marfan Syndrome

MARFANPSY
Start date: January 9, 2023
Phase:
Study type: Observational

The goal of this observational study is to identify key factors leading to psychotherapy or psychological support in adult subjects with Marfan syndrome (MSF). the main questions it aims to answer are: - Understand and define which internal variables of the individual drive this request for psychological support; - To learn about verify the impact that the clinical manifestations of MSF have on psychosocial aspects, self-esteem, subjective perception of the disease and coping mechanisms.

NCT ID: NCT05712512 Recruiting - Clinical trials for Autism Spectrum Disorder

Experimental Techniques for Early Identification of Autism

Start date: July 19, 2021
Phase:
Study type: Observational

This project deals with essential challenges in the context of Autism Spectrum Disorder, benefiting from a longitudinal design in infancy and a cutting-edge electroencephalogram/eye-tracking integrated approach. The investigators will focus on multisensory audiovisual integration to identify early markers of autism in infants at-risk for autism. The investigators will characterize early derailments from the typical developmental trajectories to identify critical "time windows" and better describe the heterogeneity of autism.

NCT ID: NCT05712369 Recruiting - Nephrotic Syndrome Clinical Trials

B Cells in Idiopathic Nephrotic Syndrome

BLADE
Start date: April 2, 2019
Phase: N/A
Study type: Interventional

The role of the immune system in Idiopathic Nephrotic Syndrome (INS) of Minimal Change Disease (MCD), Mesangial proliferative Glomerulonephritis (MesGN) or Focal and Segmental Glomerulosclerosis (FSGS) has been widely investigated. However, among immune cell populations, a major player in disease pathogenesis was never found. The efficacy of B cell depleting therapy with anti-CD20 monoclonal antibodies suggests that B lymphocytes may play the pivotal role. Preliminary data suggest that memory B cells may be the responsible of the Nephrotic Syndrome (NS) relapse after rituximab treatment in in children with Steroid Dependent Nephrotic Syndrome (SDNS) or Frequently-Relapsin gnephrotic Syndrome (FRNS), enforcing the role of the B cell lineage in the disease pathogenesis. NS is a severe glomerular disease affecting more frequently children and young adult. It is characterized by edema, heavy proteinuria and hypoalbuminemia, the clinical counterpart of the alteration of the selective glomerular permeability barrier. Despite extensive investigation, the mechanism and the immune cell population responsible for the disruption of glomerular filtration barrier and, consequently, of the development of proteinuria is still not clearly defined. However, the efficacy of the different immunosuppressive approaches including prednisone and anti-CD20 antibodies in the treatment of NS strongly suggests a central role of the immune system, in particular the role of B cells in the pathogenesis SDNS. Recent evidence indicates that, after B cell depletion, the delayed reconstitution of the switched memory B cells in children with SDNS was significantly and independently protective against relapse. These results suggest that recovery of switched memory B-cells after anti-CD20 therapies could be a useful predictor of subsequent relapse of the NS in SDNS and FRNS patients, and that memory B-cells may play a role in the pathogenesis of SDNS or FRNS in children. The main aim of the present study is to determine whether reconstitution of different B-cell subpopulations can predict relapse after treatment with B-cell depleting antibodies in adult with NS, and whether specific B- or T-cell anomalies (as well as dysregulation of other circulating immune cell subsets) may play a role in the disease pathogenesis of SDNS and FRNS

NCT ID: NCT05712200 Recruiting - Clinical trials for Atrial Fibrillation (AF)

Study to evaLuate the effIcacy and Safety of abeLacimab in High-risk Patients With Atrial Fibrillation Who Have Been Deemed Unsuitable for Oral antiCoagulation (LILAC-TIMI 76)

LILAC-TIMI 76
Start date: December 27, 2022
Phase: Phase 3
Study type: Interventional

A study to evaluate the effect of abelacimab relative to placebo on the rate of ischemic stroke or systemic embolism (SE) in patients with Atrial Fibrillation (AF) who have been deemed by their responsible physicians or by their own decision to be unsuitable for oral anticoagulation therapy.

NCT ID: NCT05711992 Recruiting - Embryonal Tumor Clinical Trials

Rare Embryonal Tumors of the Central Nervous System: International Registry

Start date: February 1, 2023
Phase:
Study type: Observational

Central nervous system (CNS) tumors are the most common solid malignancies among children. Although some types of CNS tumors like medulloblastomas and low-grade gliomas are widespread and well-studied, there is a huge number of rare diseases that need further research. This international registry aims to establish a large multicenter database of pediatric and young adult patients with rare embryonal tumors of the central nervous system and describe the clinical presentations, diagnostics, treatment regimens, and outcomes. Embryonal tumors with multilayered rosettes (ETMR), FOXR2-activated CNS neuroblastoma, cribriform neuroepithelial tumor, and CNS tumor with BCOR internal tandem duplication are extremely rare embryonal tumors some of which were first described in the last edition of the World Health Organization (WHO) Classification of Tumors of the Central Nervous System. Objectives of the registry are 1) to evaluate prognostic factors, 2) to identify diagnostic and treatment gaps, 3) to investigate the characteristics and outcome of the disease with different treatment regimens, and 4) to generate data-based prospective diagnostic and treatment recommendations.

NCT ID: NCT05711966 Recruiting - Clinical trials for Bronchopulmonary Dysplasia

Comparison of INRECSURE and LISA in Preterm Neonates With RDS

INRECLISA
Start date: January 9, 2023
Phase: N/A
Study type: Interventional

The primary hypothesis of this study is that surfactant administration by INtubate-RECruit-SURfactant-Extubate (IN-REC-SUR-E), via a high frequency oscillatory ventilation recruitment maneuver increases survival without BPD at 36 weeks' gestational age in spontaneously breathing infants born at 24+0-27+6 weeks' gestation affected by Respiratory Distress Syndrome (RDS) and failing nasal CPAP or nasal intermittent positive pressure ventilation (NIPPV) during the first 24 hours of life compared to less invasive surfactant administration (LISA).

NCT ID: NCT05711680 Recruiting - HIV Infections Clinical Trials

INTERFACE: hIv patieNts TracER For clinicAl assistanCe and rEsearch During COVID 19

INTERFACE 3436
Start date: January 18, 2021
Phase:
Study type: Observational

The health emergency related to the SARS CoV2 pandemic has highlighted the problems of health management of patients at risk of COVID19 and with chronic diseases, suggesting the need for new methods of patient monitoring. Teleconsultation, and telemedicine in general, are applications that allow, at these times when access to hospitals is by definition limited, to monitor patients with chronic conditions who need regular and consistent care and monitoring. People living with HIV/AIDS (PLWHA) represent a paradigm of chronic patients on whom the development of e-health-based remote monitoring could have a significant impact in terms of maintaining an adequate standard of care. For these reasons, in a large teaching hospital we are planning the creation and use of an application to enable remote monitoring of PLWHA by combining the COVID19 prescreening system and chronicity monitoring.

NCT ID: NCT05711394 Recruiting - Episodic Migraine Clinical Trials

A Study to Assess the Adverse Events and Change in Disease Activity of Oral Atogepant Tablets in Pediatric Participants (6-17 Years of Age) With Episodic Migraine

Kaleidoscope
Start date: May 1, 2023
Phase: Phase 3
Study type: Interventional

A migraine is a moderate to severe headache on one side of the head. A migraine attack is a headache that may be accompanied by throbbing, nausea, vomiting, sensitivity to light and sound, or other symptoms. A number of treatments are available for adults with migraine but there are limited approved treatments available for pediatric participants. The main goal of the study is to evaluate the safety and efficacy (how well treatment works) of a low-dose and high-dose of atogepant in pediatric participants between the ages of 6 and 17. Atogepant is a medicine currently approved to treat adults with episodic migraine (0 to 14 migraine days per month) and is being studied in pediatric participants between the ages of 6 and 17 with a history of episodic migraine. This is a Phase 3, randomized, double-blind study of atogepant in participants with a history of episodic migraine with an open-label pharmacokinetic substudy. Eligible participants will be randomized into 6 different groups. Participants between the ages of 12 and 17 will be randomized to receive placebo, low-dose atogepant, or high-dose atogepant for 12 weeks. Participants between the ages of 6 and 11 will also be randomized to receive placebo, low-dose atogepant, or high-dose atogepant for 12 weeks. The specific atogepant doses to be used in participants between the ages of 6 and 11 will be determined after the PK substudy is complete. Around 450 participants will be enrolled in approximately 100 sites. Placebo, low-dose atogepant, and high-dose atogepant are given as a tablet to take by mouth once a day. At the end of Week 12, participants will either undergo a follow-up visit 4 weeks after last study treatment or join an extension study where they can continue to receive atogepant for another 52 weeks. There may be a bigger responsibility for participants in this study. Participants will attend regular visits during the study at a hospital or clinic. The effects of treatment will be checked by medical assessments, blood tests, checking for side effects, and completing questionnaires.

NCT ID: NCT05710926 Recruiting - Clinical trials for Schizotypal Personality Disorder

Evolutionary Systems Therapy for Schizotypy

ESTS-RCT
Start date: October 5, 2022
Phase: N/A
Study type: Interventional

This study aims at replicating existing preliminary evidence about the effectiveness of Evolutionary Systems Therapy for Schizotypy (ESTS). The present randomized controlled trial (RCT) will compare ESTS with Cognitive Behavioral Therapy (CBT) in treating Schizotypal Personality Disorder (SPD). The main questions our RCT aims to answer are: 1. Is ESTS more effective than CBT in treating SPD? 2. Is ESTS more feasible than CBT in treating SPD? 38 patients diagnosed with SPD will be recruited and randomly allocated to either the experimental group (i.e. ESTS) or the control group (CBT). Primary outcome will be reduction in general symptomatology, whereas secondary outcomes will be changes in target mechanisms (self-criticism and metacognition) and remission from diagnosis.

NCT ID: NCT05710562 Recruiting - Anticoagulants Clinical Trials

Epidemiological Description of Health Determinants in Italian Anticoagulated Population

EDIA
Start date: February 5, 2019
Phase:
Study type: Observational

The primary aim of this study is to describe and develop a cross-sectional profile of the level of the main health determinants in the Italian anticoagulated population (disease knowledge; self-efficacy; health literacy; quality of life). Indeed, evaluate how socio-demographic and clinical data may affect the same relations. Furthermore, the secondary aim is to describe relationships between health determinants and anticoagulation control (i.e., Time in Therapeutic Range, TTR%, or absence of clinical complications). To evaluate the impact of socio-demographic and clinical data on the relationship between health determinants and anticoagulation control.