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A Multi Center, Prospective, Observational, Open-label, Pharmacokinetic Study of Tacrolimus in Heart and Lung Transplantation Patients during the First Days after Transplantation

Primary objective: To show that the variability of whole blood total and unbound plasma tacrolimus concentrations during the first 6 days post transplantation is larger than the variation of tacrolimus concentrations in stable clinical situation.…

Improvement of physical endurance and motor function performance in children with anatomical congenital anomalies and/or following neonatal extracorporeal membrane oxygenation; a randomized study

The main objective is to improve exercise tolerance. Secondary objectives are: 1) improvement of motor function development, daily physical activity, quality of life, self perception of motor competence, and participation; 2) evaluation of cost…

Early detection of pulmonary exacerbations in mucociliary clearance diseases: Molecular fingerprinting by exhaled breath and microbiome analysis

Objectives: The primary objective is to establish whether expiratory VOC analysis by electronic nose and GC-MS can: Ia) discriminate between patients (CF, PCD) with and without an exacerbation and Ib) discriminate between different microbial species…

Genetic sequencing study in Primary Ciliary Dyskinesia.

Objectives: The aim of this study is to:I Identify pathogenic mutations in novel genes, causing PCDII Develop a diagnostic PCD test, based on MPSIII Validate MPS technique for PCD.

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single- and Multiple-Dose Escalation Study Evaluating Safety and Pharmacokinetics of VX-983 Followed by an Open-Label, Randomized, Crossover Study to Estimate the Effect of Steady State Administration of VX-983 on the Pharmacokinetics of a Single, Oral Dose of Midazolam in Healthy Adult Subjects

The study will consist of three parts, part A, B and C. The main purpose of the study is to investigate to what extent VX-983 is safe and tolerated. In addition, it will be investigated how quickly and to what extent VX-983 is absorbed and…

The half-lives of neutrophils in the blood and lungs of patients with cystic fibrosis

Objective of the study:The results of this study will lead to an improved insight in the lifecycle of these cells in health and diseases, and our results will be of importance for more insight in chronic inflammatory diseases particularly CF. For…

insulin therapy in non-diabetic adults with cystic fibrosis

To investigate the effect of low-dose long acting insulin therapy on nutritional status in adult CF patients without diabetes.