Experimental CFTR correctors for the treatment of Cystic fibrosis.

• Conditions: Cystic Fibrosis; 10038686

• Registration Number: NL-OMON46144
• Lead Sponsor: Erasmus MC Rotterdam

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 28 February 2024

Recruitment & Eligibility

• Status: Pending
• Sex: Not specified
• Target Recruitment: 2

Inclusion Criteria

In order to be eligible to participate in this study, the subjects must meet all of the following criteria:
- Diagnosed with CF, confirmed with 2 known CFTR mutations of interest
- Authorised by informed consent

Exclusion Criteria

A subject who meets any of the following criteria will be excluded from participation in this study: Absence of any of the previous inclusion criteria.

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The cell material will be used in selection and testing of CFTR correctors and<br /><br>potentiators in established and novel assays in vitro. The primary objective is<br /><br>Identification and testing in advanced cellular model systems in vitro of novel<br /><br>CFTR directed compounds (correctors and potentiators) for the treatment of<br /><br>patients with rare trafficking mutations.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Further development of organotypic human and personalized (*organ on a chip*)<br /><br>cell culture systems in which compounds can be tested, to improve the efficacy<br /><br>of pre-clinical research in CF and other chronic lung disease.</p><br>