Cystic Fibrosis Mucoviscidosis
Conditions
Interventions
All patients will be randomly assigned to receive four times daily 200 mcg
Salbutamol per inhalation (treatment 1) or four times daily 4 mg Salbutamol per
os (treatment 2), during four days. After a
Sponsors
Universitair Medisch Centrum Utrecht
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: * Signed informed consent form (ICF); * Males and females, aged 18 years or older on the date of informed consent; * CFTR genotype compound/A455E or compound/R117H.
Exclusion criteria
Exclusion criteria: * Severe acute exacerbation or pulmonary infection (needing intravenous treatment and/or systemic corticosteroids); * Uncontrolled CF related Diabetes; * Participation in another drug-investigating clinical study at the start or within 1 month prior to the start; * Treatment with B2 agonist at the start of the study or a week prior to the start of the study; * Inability to follow instructions of the investigator; * Increased risk on side-effects of Salbutamol * Use of medication that are known to potentially interact with Salbutamol * (Potential) pregnancy or breastfeeding.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main study parameters will be the nasal potential difference (NPD) and sweat chloride concentration (SCC) before and after treatment with Salbutamol. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary endpoints will include: * Difference between change in NPD and SCC when treated with B2-agonist per inhalation and oral B2-agonist * Correlation between individual B2-agonist-induced CFTR function (organoid-based measurements) and in vivo treatment effect (NPD,SCC). * The CFTR stimulating effect of patients* blood samples in vitro, on autologous organoid cultures. | — |
Countries
Netherlands
Outcome results
None listed