Cystic Fibrosis
Conditions
Interventions
CFTR
CFTR correctors
Cystic Fibrosis
induced pluripotent stem cell
Sponsors
Erasmus MC Rotterdam
Eligibility
Age
2 Years to 64 Years
Inclusion criteria
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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The cell material will be used in selection and testing of CFTR correctors and potentiators in established and novel assays in vitro. The primary objective is Identification and testing in advanced cellular model systems in vitro of novel CFTR directed compounds (correctors and potentiators) for the treatment of patients with rare trafficking mutations. | — |
Secondary
| Measure | Time frame |
|---|---|
| Further development of organotypic human and personalized (*organ on a chip*) cell culture systems in which compounds can be tested, to improve the efficacy of pre-clinical research in CF and other chronic lung disease. | — |
Countries
The Netherlands
Outcome results
None listed