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Prospective validation and clinical evaluation of a new posaconazole dosing regimen for children and adolescents with cystic fibrosis and Aspergillus infection

- In silico definition of the most optimal posaconazole dose for children and adolescents with CF aged 8 to 17 years.- Assess the prevalence of Aspergillus infection in children and adolescents with CF aged 8 -17 years.- An intensive sampling…

Validation of bi-dimensional (2D) and three-dimensional (3D) Fourier Decomposition (FD) as Magnetic Resonance Imaging (MRI) technique to assess lung ventilation and perfusion compared to computed tomography (CT), Hyperpolarised gases (HP-MRI) and Contrast-Enhanced MRI (CE-MRI)

The final goal of this validation plan is to develop an MRI platform that can provide information about ventilation, inflammation, perfusion and structure (VIPS-MRI) in a single MRI examination of 30 minutes for safe and efficient monitoring of CFLD…

Early identification of progressive pulmonary fibrosis
Precision Medicine for more Oxygen - ILD extension

1. Identify biomarkers and risk factors that associate with fibrosis progression or predict transformation into a rapid progressive (fibrosing) phenotype and acute exacerbations in IPF and FPF patients.2. Identify biomarkers and risk factors that…

The Origins of Pediatric Respiratory diseases: laying groundwork for genetic, cellular and immunological analyses. (OPeRA)

The main objective of this proposal is to establish ex-vivo models of bronchial epithelial cells from children. Use of (1) epithelial organoid cultures derived from bronchial or nasal epithelial cells; and of (2) bronchial brushings; (3) nasal…

A Natural History Study of Exocrine Pancreatic
Function in Infants with Cystic Fibrosis Less Than
12 Months of Age

To characterize the natural history of exocrine pancreatic function as measured by FE-1 in infants with CF <12 months of age

TAAI Erasmus Research Initiative to Fight CF: Monitoring Inflammation in CF Lung disease into a new Era

The overall aim of the study is to develop innovative minimally invasive monitoring techniques that can identify lung inflammation in pwCF when using highly effective modulators, compared to patients whom are not eligible for CFTR modulators (…

Performance and patient acceptance of a commercially available beverage as compared to an oral glucose solution for oral glucose tolerance tests in cystic fibrosis (CF) patients who are screened for CF-related diabetes.

Primary Objective: To compare the performance of a glucose tolerance test (*AATT*) with a commercially available beverage to the results of the conventional OGTT with respect to diagnosing IGT and CFRD in patients with CF. Secondary Objective(s): To…

A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes

This study has been transitioned to CTIS with ID 2024-515637-14-00 check the CTIS register for the current data. To evaluate the long-term safety and tolerability of elexacaftor(ELX)/tezacaftor (TEZ)/ivacaftor (IVA)

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

This study has been transitioned to CTIS with ID 2024-514173-22-00 check the CTIS register for the current data. Primary ObjectiveTo evaluate the long-term safety and tolerability of VNZ/TEZ/D-IVA in subjects with CFSecondary ObjectiveTo evaluate…

I-BALL 2: Non-invasive Disease Monitoring in Infants with Cystic Fibrosis

To validate our previous findings, and to continue and deepen our exploration of biomarkers in BALF, providing endpoints that correlate with disease progression. We will do this through in-depth cellular and molecular profiling of BALF and blood,…

Growing up after surgery for congenital diaphragmatic hernia (CDH): Problems for life? A study to evaluate optimal medical and psychosocial care in CDH up till adulthood (CDH-FU)

Primary Objective: To evaluate persisting pulmonary morbidity and echocardiographic signs of pulmonary hypertension in young adults born with CDH that interfere with physical fitness and participation in society.Secondary Objective(s): To evaluate…

Congenital Lung Abnormalities on MRI

Our main objective is to validate a MRI scan protocol for imaging parenchymal abnormalities in CPAM and describe the appearance of these abnormalities on MRI. Furthermore, we want to image postoperative changes in developing lungs of patients…

Identification of new genetic defects in telomeropathies

To identify new genetic defects in telomeropathies.

Getting insight in the exposure effect relationship of Elexacaftor/ Tezacaftor/Ivacaftor treatment in people with cystic fibrosis

Primary objective: To compare the exposure of ETI in a group of pwCF with the highest decrease in sweat chlorid and the lowest decrease in sweat chloride as result of ETI therapy.

Pilot: Feasibility of a partially supervised conditioning program in patients with CF: ACTIVATE- CF.

The primary aim of this pilot study is to evaluate the feasibility of the study protocol. The ACTIVATE-CF protocol will be judged feasible when at least 75% of the included patients can comply to the study protocol (with regard to both the…

Experimental CFTR correctors for the treatment of Cystic fibrosis.

The objective of the present application is to obtain cell material in the form of blood, from two patients homozygous for the trafficking mutation N1303K rare mutations for which no effective treatment is available, to generate induced pluripotent…