A B2-agonist as a CFTR activator in CF
Completed
- Conditions
- CFTR residual function, Salbutamol, CFTR activation, personalized medicine
- Registration Number
- NL-OMON21450
- Lead Sponsor
- MCU Utrecht, Prof. dr. C.K. van der Ent
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 10
Inclusion Criteria
CFTR genotype compound/A455E or compound/R117H
-CFTR measurement available in intestinal biopsies
Exclusion Criteria
-Severe acute exacerbation or pulmonary infection (needing intravenous treatment and/or systemic corticosteroids)
-Known cardiovascular medical history like cardiac failure, arrhythmias, ischemic cardiac disease, long QT interval syndrome and hypertension
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Difference in nasal potential difference (NPD) and sweat chloride concentration (SCC) before and after treatment with B2-agonists (per inhalation or oral)
- Secondary Outcome Measures
Name Time Method -Difference between change in NPD and SCC when treated with B2-agonist per inhalation and oral B2-agonist <br /><br>-Correlation between individual B2-agonist-induced CFTR function (organoid-based measurements) and in vivo treatment effect (NPD, SCC)<br /><br>-The CFTR stimulating effect of patients’ blood samples in vitro, on autologous organoid cultures<br>