Cystic Fibrosis Mucoviscidosis
Conditions
Interventions
After baseline measurements all patients will be assigned to receive oral
Salbutamol, during 8 weeks up untill 24 hours prior to the second study visit.
Sponsors
Universitair Medisch Centrum Utrecht
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: - CFTR genotype compound/A455E or compound/R117H - Already had a rectal biopsy to produce an organoid - Males and females, aged 18 years or older on the date of informed consent - Signed informed consent form (ICF)
Exclusion criteria
Exclusion criteria: - Severe acute exacerbation or pulmonary infection during last four weeks (needing intravenous treatment and/or systemic corticosteroids) - Known cardiovascular medical history like cardiac failure, arrhythmias, ischemic cardiac disease, long QT interval syndrome and hypertension - Known hyperthyroidism, thyrotoxicosis, galactose intolerance, lactase deficiency or glucose-galactose malabsorption - Haemoglobin A1C (HBA1C) > 45 mmol/mol - Use of oral B2-agonist one week prior to the start of the study (V1) - Use of: heart glycoside, high dose sympathomimetics, theophylline, thiazide diuretics or non-selective beta-blockers - Pregnancy or breastfeeding - Participation in another drug-investigating clinical study at the start - Inability to follow instructions of the investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Pulmonary function (spirometry and airway resistance measured with the bodybox and Rint) * Before and after treatment with salbutamol | — |
Secondary
| Measure | Time frame |
|---|---|
| * Fraction exhaled Nitric Oxide (FeNO) and Nasal Nitric Oxide (nNO) before and after the use of salbutamol; * BMI (=weight (in Kg)/Length2 (in cm)) before and after the use of salbutamol; * Quality of life (measured with Cystic Fibrosis Questionnaire (CFQ)) before and after the use of salbutamol * Bile salt measurements in plasma and the feces before and after the use of salbutamol * Elastase measurements in the feces before and after the use of salbutamol * SCC measurements before and after the use of salbutamol * Upper and lower airway microbial profiles (microbiome) before and after treatment of the use of salbutamol (conventional culturing, high throughout pyrosequencing (16S rRNA) for bacterial diversity and relative abundance). * Correlation between individual salbutamol induced CFTR function in vitro (organoid-based measurements) and the in vivo treatment effect; * The CFTR stimulating ability of the concentration of salbutamol in the patient*s blood samples, examined by in vitro testing (in the organoid model); | — |
Countries
Netherlands
Outcome results
None listed