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The effect of Ivacaftor in CF patients with a class III mutation

Primary objective is to objectively investigate the therapeutic potential of Ivacaftor in Dutch CF patients carrying a mutation associated with residual CFTR function. A secondary objective is to evaluate the correlations between individual…

A Phase 1, Randomized, Double blind, Placebo Controlled, Single and Multiple Dose Escalation Study Evaluating Safety and Pharmacokinetics of VX 152 Alone and in Combination with VX 661/Ivacaftor, and Bioavailability and Food Effect of VX 152 in Healthy Adult Subjects

The purpose of Part A is to investigate how safe the study compound VX-152 is and how well the study compound is tolerated. The study will also investigate how quickly and to what extent the compound is absorbed into and eliminated from the body (…

A Phase 1, Randomized, Double-blind, Placebo-Controlled, Single- and Multiple-Dose Escalation Study Evaluating Safety and Pharmacokinetics of VX-440 Alone and in Combination with VX-661/Ivacaftor, and Bioavailability and Food Effect of VX-440 in Healthy Adult Subjects

The purpose of Part A is to investigate how safe the study compound VX-440 is and how well the study compound istolerated. The study will also investigate how quickly and to what extent the compound is absorbed into and eliminatedfrom the body (this…

Assessment of real life and trained inhalation of Tobramycin Inhalation Powder (TIP) and Tobramycin Inhalation Solution (TIS) TIPTIS study.

Primary objective of the study is to record TIP inhalations on video at home to study the association between inhalation manoeuver and cough. Secondary objectives are: To study the inhalation maneuvers of patients using TIP in the home situation; to…

Long Term Safety and Tolerability Study of Open-Label Liposomal Amikacin for Inhalation (Arikace*) in Cystic Fibrosis Patients with Chronic Infection due to Pseudomonas aeruginosa

To evaluate the long-term safety, tolerability and efficacy of ArikaceTM 590 mg for a maximum 12 cycles administered once daily, where each cycle consists of 28 days on treatment followed by 28 days off treatment.

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.…

A randomised, multicentre, double-blind, placebo-controlled, crossover trial determining the efficacy of dry powder mannitol in improving lung function in subject with Cystic Fibrosis aged six to seventeen years.

The purpose of this trial is primarily to assess the efficacy of inhaled mannitol compared with a true placebo in subjects with cystic fibrosis aged 6 to 17 years.We hypothesize that inhaled mannitol will improve the overall health and hygiene of…

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…

Hypertonic saline in primary ciliary dyskinesia: a pilot study

We hypothesize that hypertonic saline nebulizations improve respiratory symptoms and quality of life in PCD patients.

The effect of curcumin and genistein in CF patients with a class III mutation

Primary objective:To investigate the clinical response to treatment with curcumin and genistein in CF-patients with a class III S1251N mutation.. Secondary objectives:1.To evaluate the correlations between individual curcumin+genistein induced CFTR…

Gene expression during ciliagenesis: Towards a novel candidate gene list for Primary Ciliary Dyskinesia

We aim to assess gene expression patterns in cilia-producing cells from healthy controls to develop a candidate gene list for PCD.

Validation of *in vitro counterpart* models to enable personalized care in CF

The specific objectives are:1. What is the difference of in vitro CFTR function in CF patients with either mild (compound heterozygote A455E) mutations or severe (homozygote F508del) mutations with mild or severe clinical disease? 2. What is the…

Rhinosinusitis and nasal polyps in adults with Cystic Fibrosis

Investigate the prevalence of rhinosinusitis and/or nasal polyps in adult patients with Cystic Fibrosis. Also several aspects of sinonasal disease are investigated; quality of life, correlation between phenotype and genotype, microbiology, anatomy…

A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis

The primary objective of this Phase 3 extension study will be to obtain essential long term safety data to augment the overall safety database. The secondary objective will be to augment the efficacy information collected in the double-blind study (…

Biomarkers in acute and chronic bacterial endobronchial infections in Cystic Fibrosis
A Pilot study

Primary Objective: The purpose of this pilot study is to determine cut offs for both Procalcitonin and MR-proADM in stable CF patients. These measurements will be compared with Procalcitonin and MR-proADM values in CF patients with a pulmonary…

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…

High frequency oscillation versus conventional mechanical ventilation in newborns with congenital diaphragmatic hernia: an international randomized controlled trial

Primary objective: -to determine if there is a significant difference in the incidence of oxygen dependency at day 28 and/or death within the first 28 days of life between newborns with congenital diaphragmatic hernia treated with high frequency…

Pharmacokinetics of nasally administered tobramycin and colistin in Cystic Fibrosis

To investigate the clinical pharmacokinetics of tobramycin and/or colistin after nasal administration. With this pharmacokinetic parameters the safety of this treatment can be investigated.

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.