A B2-agonist as a CFTR activator in CF

Phase 2

Completed

Conditions: Cystic Fibrosis
Mucoviscidosis
10083624

Registration Number: NL-OMON40957

Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 10

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

* 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.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Main study parameters will be the nasal potential difference (NPD) and sweat<br /><br>chloride concentration (SCC) before and after treatment with Salbutamol. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary endpoints will include:<br /><br>* Difference between change in NPD and SCC when treated with B2-agonist per<br /><br>inhalation and oral B2-agonist<br /><br>* Correlation between individual B2-agonist-induced CFTR function<br /><br>(organoid-based measurements) and in vivo treatment effect (NPD,SCC).<br /><br>* The CFTR stimulating effect of patients* blood samples in vitro, on<br /><br>autologous organoid cultures. </p><br>