145 results
To assess the PK/PD relationship of prednisolone as either prophylaxis or therapy of GVHD in children after HCT.
Primary Objective: - Achieving an event-free survival, which is not inferior to the ML-DS 2006 trial (87±3%) Secondary Objective(s): - Reduction of toxicity: severe adverse events (CTCAE v4.0 grade III or higher) - Evaluation of…
Primary objectives: Improvement of CR2 rates after induction with ALL R3 with bortezomib versus without bortezomib in HR relapsed ALL patients Secondary objectives: Improvement of Event Free Survival (EFS) and overall survival (OS) rates…
The main objective is to pinpoint the biological pathways that set long-term risk of CVD and NDI when epigenetically disturbed during fetal development by (1) establishing a longitudinal cohort of MC twins, (2) defining epigenetic alterations…
The primary objective is reducing treatment-related morbidity and mortality without adversely impacting DFS in Ph+ ALL patients, classified as Standard Risk (SR) based on low minimal residual disease (MRD) at week 10-12 of therapy.Because there is…
The primary objective is to assess the hypoxic challenge test, combined with echocardiography, as a function test for the pulmonary and pulmonary vascular system in preterm born children with and without bronchopulmonary dysplasia. Secondary…
Primary objective of this study is to significantly reduce relevant (moderate/severe) parenchymatous brain injury on postoperative MRI for the total group (both prenatal and postnatal diagnosis) and the prenatal diagnosis group, which is needed to…
Part I:Primary Objective: Evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, as measured by the Gross Motor Function Measure (GMFM)-88 and Bayley Scales of Infant Development (BSID-III) Gross Motor Skill Domain…
Primary Objectives:• Phase 1 Only: To determine the recommended Phase 2 dose(RP2D) of quizartinib, in combination with chemotherapy, forsubjects in the older (>=1 year old to <=21 years old) and younger(>=1 month old to <…
To evaluate the performance of a *genotype first approach* (WES-based panel analysis) in diagnostics of genetic predisposition in children with cancer or neoplasms, compared to the current *phenotype first approach* (standard of care). In particular…
PRIMARY OBJECTIVES*Phase 1 Dose Finding Studies:-To determine the recommended phase II dose (RP2D) of new systemic therapy regimens. *Frontline chemotherapy questions:-To compare systemic therapy regimens for patients with VHR disease at diagnosis (…
Estimate key transmission parameters of SARS-CoV-2 in Europe from observing within household virus spread and seroconversion of household members, and to characterize the views and experiences of households regarding perceptions, practices regarding…
The main objective of this longitudinal study is to gain insight into the neurodevelopmental trajectories following neonatal critical illness, from the perinatal period to school-age compared to healthy controls. This insight may lead to the…
Primary: To describe safety and tolerability during longer-term administration of AR101 and follow-up observation after the last dose of AR101.Secondary: - To assess the level of desensitization achievable through extended maintenance dosing of…
• Primary Objective: To evaluate the effect of azacitidine treatment in AML subjects at molecular relapse after CR1 with regard to molecular response prior to further treatment (reinduction / HSCT)• Secondary Objectives:o To assess safety of…
Monitoring & evaluation of immune responses induced by COVID-19 vaccines in the general population in the Netherlands.
Determination of the (cost)effectiveness of the replacement of the expensive, risky and time-consuming food challenge test bythe Basophil Activation Test (BAT) for the diagnosis of an IgE-mediated cow*s milk allergy in children.
Assessing the feasibility of the research procedures, the burden on participants, the degree of dropout and reason, the interest in participating.What is the main treatment effect for parents / carers motivating them to participate in this study?•…
To validate biomarkers that are associated with severe RSV infection and respiratory sequelae.
Brain tumors: Characterize and quantify changes in immune parameters during disease in patients with a CNS malignancyAML: Characterize and quantify changes in immune parameters and immunotherapeutic targets during disease in pediatric patients with…