Cystic Fibrosis Mucoviscidosis
Conditions
Interventions
None listed
Sponsors
Universitair Medisch Centrum Utrecht
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Male or female with confirmed diagnosis of CF. The subject must have of the following: o One or more characteristic phenotypic features, such as chronic cough and sputum production, persistent chest radiograph abnormalities, or airway obstruction manifested by wheezing and air trapping; or a history of CF in a sibling ; or a positive new-born screening test result; 2. An increased sweat chloride concentration (above 60 mmol/L) by pilocarpine iontophoresis (documented in patient records) 3. Adult age* on the date of informed consent for biopsy taking. Younger patients will not be included due to extensive and long-term safety data needed before a trial in younger paediatric patients is considered safe. *16 or 18 years depending on country-specific ethical regulations 4. Subject will sign and date an informed consent form (ICF).
Exclusion criteria
Exclusion criteria: 1. Subject has at least one of the following CFTR-mutations: 508del, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H, A455E, 3849+10kbC>T 2. Subject has a combination of any two (2) of the following mutations: G542X, 1717-1G>A, 621+1G>T, 3120+1G>A, 1898+1G->A, CFTRdele2,3 and 2183AA->G 3. History of any comorbidity reviewed at the Screening Visit that, in the opinion of the investigator, might pose an additional risk in potentially administering study drug to the subject if he or she enters the subsequent clinical study. For example, a history of cirrhosis with portal hypertension. 4. History of lung transplantation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Intestinal organoid response of 500 subjects to three drug products of different pharmaceutical companies, ranked by best response per drug product. | — |
Secondary
| Measure | Time frame |
|---|---|
| NVT | — |
Countries
Netherlands
Outcome results
None listed