29 results
This study has been transitioned to CTIS with ID 2024-514073-22-01 check the CTIS register for the current data. To investigate differences in coagulation between (Omniplasma) and FFP in paediatric cardiac patients, who are undergoing cardiac…
This study has been transitioned to CTIS with ID 2022-501456-28-00 check the CTIS register for the current data. Primary objectives Part A:To test the non-inferiority, as evaluated by OS, of three courses of HDCT compared to focal RT plus…
Main Objectives: To collect clinical data and biological samples derived from blood (plasma, serum, cells and DNA) as well as urine and stool to perform multi-omic and functional biological assays. The resulting data will be integrated and analyzed…
The primary objectives of this study are:- Can EEnG measure colonic motility/activity?- Can EEnG be used to distinguish between healthy subjects and patients suffering from a colonic motility disorder.Secundary objective:- What is the…
The primary objective of the study is to develop new sensitive and quantitative outcome measures for clinical trials in ALD. The secondary objective is to identify biomarkers to stratify patients based on disease progression rate.
Our proposal builds on 12 years of sustained EU funding, a consortium covering 11 European countries and 27partners (with non-EU sites in African and two Asian populations to provide a global perspective) and our cuttingedge new research, which has…
This study has been transitioned to CTIS with ID 2023-504593-38-00 check the CTIS register for the current data. The aim of this trial is to infuse multiple doses of human 1st trimester liver-derived MSC for the treatment of severe OI to determine…
To investigate the role of talkers* voices in speech communication in NH, HI, and CI children and adult listeners, and how the perception of talker voice information relates to the processing of indexical and linguistic information among these…
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia…
The primary objective of the cohort study is to test the effectiveness of a complex intervention of paediatric sleep problems on a 24-hour sleep duration (day- and nighttime) of children aged 6-36 months of age with sleep onset and/ or sleep…
Primary objective: To identify rare, high impact genetic variants in immunological genes and pathways in children with a history of MIS-C or pediatric post-COVID condition. Secondary objectives: To analyze the clinical characteristics and long-term…
The main aim is to study early neurocognitive functioning and development in very young children with SCD. The secondary aims are to identify determinants (risk and protective factors) and biomarkers of early neurocognitive deficits in SCD and to…
To validate biomarkers that are associated with severe RSV infection and respiratory sequelae.
PRIMARY OBJECTIVE: -To assess the antibody response after mRNA (Pfizer, Moderna) SARS-CoV-2 vaccination in children with cancerSECONDARY OBJECTIVES:To assess in children with cancer after SARS-CoV 2 vaccination:- durability of the antibody response…
Primary Objectives: 1. To assess the metabolic profile of patients with Dravet syndrome and identify metabolic variations that contribute to the pathophysiology of seizures and developmental delaySecondary Objectives:2. To assess the association…
The main objective of this proposal is to establish ex-vivo models of bronchial epithelial cells from children. Use of (1) epithelial organoid cultures derived from bronchial or nasal epithelial cells; and of (2) bronchial brushings; (3) nasal…
Primary Objective:To investigate efficacy and adverse effects of AP treatment for pregnant women with SMI.Aim 1a: Efficacy of AP treatment for pregnant women with SMI. Hypothesis: Women taking either APs or non-AP mood stabilizers will be less…
Primary objectives: The first aim is to examine the role of microtubule network in electrical conduction, protein localization and function in induced cardiomyocytes from pediatric BrS patients, life threatening ventricular arrhythmia patients and…
The Primary Objectives of the Phase 1b are: • To assess the safety and tolerability of carfilzomib, alone and in combination with induction chemotherapy, for the treatment of children with relapsed or refractory ALL • To determine the maximum…
The aim of the DONOR study is to define if OD pregnancies, specifically those with immunogenetic differences between mother and child, are associated with abnormal placentation and vasculogenesis, resulting in distinct maternal immunomodulation and…