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International phase 3 trial in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) testing imatinib in combination with two different cytotoxic chemotherapy backbones

This study has been transitioned to CTIS with ID 2024-515499-12-00 check the CTIS register for the current data. The primary objective is reducing treatment-related morbidity and mortality without adversely impacting DFS in Ph+ ALL patients,…

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…

A Phase 1/2, Multicenter, Dose-Escalating Study To Evaluate the Safety,
Pharmacokinetics, Pharmacodynamics, and Efficacy Of Quizartinib
Administered in Combination with Re-Induction Chemotherapy, and as a
Single-Agent Continuation Therapy, in Pediatric Relapsed/Refractory AML
Subjects Aged 1 Month to <18 Years (and Young Adults Aged up to 21 Years)
with FLT3-ITD mutations

This study has been transitioned to CTIS with ID 2023-510009-16-00 check the CTIS register for the current data. Primary Objectives:• Phase 1 Only: To determine the recommended Phase 2 dose(RP2D) of quizartinib, in combination with chemotherapy,…

A Multicenter, Open-Label, Longer-Term Study of AR101 Characterized Oral
Desensitization Immunotherapy in Subjects Who Participated in a Prior AR101 Study

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…

International multicenter, open-label, phase 2 study to treat molecular relapse of pediatric acute myeloid leukemia with azacitidine

• 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…

The Amsterdam Infant Microbiome Study

Objective: We propose the Amsterdam Infant Microbiome Study (AIMS). The general objective of AIMS is to establish a research infrastructure that allows for explorative research on the development of the microbiome in relation to healthy growth and…

An open-label, single-arm, multicenter study of intracerebral administration of adeno-associated viral vectors serotype rh.10 carrying the human N-sulfoglycosamine sulfohydrolase (SGSH) cDNA for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA)

Primary: To assess the efficacy of intracerebral delivery of AAVrh.10SGSH gene therapy (LYS-SAF302) in improving or stabilizing the neurodevelopmental status of MPS IIIA patients after 24 months (main cohort), compared to the expected evolution…

The effects of intra uterine exposure to Tumor Necrosis Factor alpha inhibitors in infants on the development of the immune system

Primary ObjectiveIn order to assess the effects of anti TNFα on the development of adaptive and innate immunity, children exposed to anti TNFα (with or without other immunosuppressive drugs) will be compared to children exposed to immunosuppressive…

Antibiotic prophylaxis for children with recurrent respiratory infections: towards evidence-based guidelines

Primary: To determine whether three months of prophylactic treatment with co-trimoxazole causes a reduction in the number of days a child experiences at least two RTI symptoms in children aged 6 months to &lt;=10 years with recurrent RTIs, when…

Creation of disease model systems to understand and correct genetic diseases through gene or other therapy using iPS cells derived from somatic cells.

Primary Objective: to derive iPSCs from patients with a variety of diseases caused by genetic mutations or genetic predisposition to disease or of unknown origin. The cells will be propagated and differentiated into a variety of somatic cell types…

LONG-TERM FOLLOW-UP PROTOCOL FOR SUBJECTS TREATED WITH GENE-MODIFIED T CELLS

This study has been transitioned to CTIS with ID 2023-504201-36-00 check the CTIS register for the current data. Per Health Authority guidelines for gene therapy medicinal products that utilize integrating vectors (eg, lentiviral vectors), long-term…

A CELL-based platform to study the role of the micRotubule network in pediatric inHeritable arrhYTHMia patients and Improve the effiCacy of drug treatment

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…

A Phase 1/2 Study of the Oral TRK Inhibitor larotrectinib in Pediatric patients with Advanced Solid or Primary Central Nervous System Tumors

This study has been transitioned to CTIS with ID 2022-502668-20-00 check the CTIS register for the current data. PHASE 1 OBJECTIVES:Primary:To determine the safety of oral larotrectinib , including dose-limiting toxicity (DLT), in pediatric patients…

Granulosa cell tumours: a step towards targeted therapy

Primary objective:To identify common mutations or other genetic alterations and their potential as therapeutic targets in granulosa cell tumours, by studying human granulosa cell tumour specimens and establishing 3D organoid cell cultures derived…

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)

Primary Objective (Cohort 1)To demonstrate a sustained effect of A4250 on serum bile acids and pruritus in children with progressive familial intrahepatic cholestasis (PFIC) Types 1 and 2.Primary Objective (Cohort 2)To evaluate the effect of A4250…

'Study on infant Adiposity development To Understand the Role of early life Nutrition - the Sophia Saturn Study'

Primary Objective:To investigate change in fat mass index between 6 and 12 months of age in infants receiving Nuturis® formula feeding versus standard formula feeding during the first 6 months of life.Secondary Objectives:- To investigate change in…

Primary pOsterioR TRacheopexy prevents collapse of the trachea in newborns with esophageal AtresIa and Tracheomalacia

The aim of this study is to evaluate if a PPT can significantly decrease - or possibly prevent - the collapse of the trachea in newborns with EA and moderate or severe TM. Additionally, the study aims to determine whether the observed effect of PPT…

Children's Health Assessment and Molecular Pathogen Identification for Optimized Novel Sepsis therapy

The aim of this study is to establish the accuracy of Molecular Culture for prediciting the outcome of the traditional blood culture.

PATIENT VENTILATOR ASYNCHRONY IN CRITICALLY ILL CHILDREN - PROSPECTIVE OBSERVATIONAL STUDY

Specific Aim 1) To identify the frequency and risk factors for PVA and its subtypes in ventilated children, with a specific focus on DC breaths. Hypothesis: PVA subtypes related to inadequate ventilator support (flow undershoot and premature cycling…

A phase I/II post-cord blood HCT dendritic cell vaccination trial directed against WT1 for pediatric and young adult acute myeloid leukemia: the U-DANCE-anti-AML-trial

This study has been transitioned to CTIS with ID 2024-517922-24-00 check the CTIS register for the current data. Although DC vaccinations have been used in allo-HCT settings, no previous studies have been performed using a CBDC vaccine after CBT.…