18 results
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 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…
PHASE 1 OBJECTIVES:Primary:To determine the safety of oral larotrectinib , including dose-limiting toxicity (DLT), in pediatric patients with advanced solid or primary central nervous system (CNS) tumors. PHASE 2 OBJECTIVES:Primary Objective:To…
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…
To investigate the reliability and feasibility of PK-guided prophylactic dosing of factor concentrates in hemophilia A and B patients (predictive performance).
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…
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…
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…
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 <…
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…
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…
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…
Per Health Authority guidelines for gene therapy medicinal products that utilize integrating vectors (eg, lentiviral vectors), long-term safety and efficacy follow up of treated subjects is recommended.The primary objectives of this study are as…
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 <=10 years with recurrent RTIs, when…
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…
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…
To assess the pharmacokinetics of various cytotoxic agents (carboplatin, cisplatin, cytarabine, dactinomycin, daunorubicin, doxorubicin, etoposide, methotrexate and vincristine) and tyrosine kinase inhibitors (ALK inhibitors, MEK inhibitors, BCR-ABL…