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Neuromonitoring during surgical repair of congenital diaphragmatic hernia and esophageal atresia patients

The overall aim of this observational prospective study is to evaluate the association between a) the trajectory of cerebral oxygenation, activity and perfusion as evaluated by NIRS, aEEG, Zonare Doppler Ultrasound and Cytocam-IDF during and 24…

Pharmacokinetics, safety and efficacy of methylphenidate during pregnancy in women with Attention-Deficit (Hyperactivity) Disorder

Primary objective is assessment of the pharmacokinetic profile of MPD in pregnancy. Secondary objectives are the safety and efficacy of any use of MPD during pregnancy.

Multicenter study of the performance of Fabian-PRICO for saturation targeting routine use in the NICU

Investigate whether, in a routine clinical environment across a number of centers, the fabian-PRICO can adequately maintain oxygen saturation, with minimal staff intervention.

An international prospective umbrella trial for children with atypical teratoid/rhabdoid tumours (ATRT)
including
A randomized phase III study evaluating the non-inferiority of three courses of high-dose chemotherapy (HDCT) compared to focal radiotherapy as consolidation therapy

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…

International collaborative treatment protocol for infants under one year with KMT2A-rearranged acute lymphoblastic leukemia or mixed phenotype acute leukemia

This study has been transitioned to CTIS with ID 2022-502503-30-00 check the CTIS register for the current data. The primary objective is to improve the outcome (in terms of event-free survival (EFS) as the primary endpoint) of newly diagnosed KMT2A…

International cooperative prospective study for children and adolescents with standard risk ALK-positive anaplastic large cell lymphoma (ALCL) estimating the efficacy of Vinblastine.

This study has been transitioned to CTIS with ID 2022-501454-11-00 check the CTIS register for the current data. PRIMARY OBJECTIVE:- To show that it is possible to cure at least 75% of patients belonging to the SR group with Vinblastine-monotherapy…

Feasibility study of an orthosis for treatment of a Clubfoot

Primary ObjectiveThe primary objective of this study is to examine the feasibility of using the clubfoot brace. This will be done by recording the effect of the brace on the clubfoot deformity by determining the improvement of the Dimeglio and…

Pharmacokinetics of tacrolimus during pregnancy in kidney and liver transplant recipients.

The overall aim of the study is to gain more insight in as well as understand and map the pharmacokinetics of tacrolimus during pregnancy. Therefore, we will study tacrolimus concentrations in maternal whole blood (routine care) and via area under…

Effectiveness of L-serine dietary supplementation in children with a GRIN2B loss-of-function mutation: n-of-1 series

Assess the potential efficay of L-serine dietary supplementation in patients children with a GRIN2B LoS mutation.

Treatment study for children and adolescents with Acute Promyelocytic Leukemia

This study has been transitioned to CTIS with ID 2024-518669-83-00 check the CTIS register for the current data. Primary objective:To assess, in an international pediatric study, the efficacy, in terms of event-free survival, of a combination of ATO…

Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II

This study has been transitioned to CTIS with ID 2024-516123-13-00 check the CTIS register for the current data. Part I:Primary Objective: Evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, as measured by the…

CeRebrUm and CardIac protection with Allopurinol in neonates with critical congenital heart disease requiring cardiac surgery with cardiopulmonary bypass

This study has been transitioned to CTIS with ID 2024-513041-37-00 check the CTIS register for the current data. Primary objective of this study is to significantly reduce relevant (moderate/severe) parenchymatous brain injury on postoperative MRI…

An open label, single arm study to evaluate single and multiple dose pharmacokinetics, safety and tolerability, and to explore clinical outcomes of treatment with intravenous |(IV) zanamivir in neonates and infants under 6 months of age with confirmed complicated influenza infection

PrimaryTo characterise the single and multiple dose to steady state pharmacokinetics of IV zanamivirin hospitalised neonates and infants under 6 months of age with influenza infection.SecondaryTo evaluate the safety and tolerability of IV zanamivir…

Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

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…

Multicentre prospective trial for extracranial malignant germ cell tumours including a randomized comparison of Carboplatin and Cisplatin

This study has been transitioned to CTIS with ID 2023-507582-25-00 check the CTIS register for the current data. PRIMARY OBJECTIVESThe primary objective of MAKEI V is to assess in a randomized comparison whether the efficacy of Carboplatin (600 mg/…

Oxytocin and reactivity to infant signals in mothers postpartum depression

In a randomized control trial (RCT) with mothers with postpartum depressive symptoms the following main hypotheses will be tested:• Oxytocin will promote more sensitive caregiving during mother-infant interactions and while interacting with a life-…

The effect of antenatal acetaminophen administration on breathing effort of premature infants at birth: a pilot study

The primary objective of this study is to evaluate the feasibility of antenatal paracetamol/placebo administration to pregnant women 0.5-2 hours prior to birth.The secondary objective of this study is to compare the effect of antenatal acetaminophen…

A phase 3 open-label study of PTC923 (Sepiapterin) in Phenylketonuria

This study has been transitioned to CTIS with ID 2023-509229-31-00 check the CTIS register for the current data. Primary:• To evaluate the long-term safety of PTC923 in subjects with phenylketonuria (PKU)• To evaluate changes from baseline in…

CHIP-AML22 Master protocol: An open label complex clinical trial in newly diagnosed pediatric de novo AML patients - a study by the NOPHO-DBSHIP consortium

This study has been transitioned to CTIS with ID 2023-504999-25-00 check the CTIS register for the current data. To investigate wether the treatment of children and adolescents with AML can be improvedby means of:1) improved risk-group adapted…

An Open-label, Multinational, Multicenter, Intravenous
Infusion Study of the Efficacy, Safety, Pharmacokinetics,
and Pharmacodynamics of avalglucosidase alfa in
Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)

This study has been transitioned to CTIS with ID 2024-513859-33-00 check the CTIS register for the current data. The overall objective is to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and…