Stratifying Cystic Fibrosis Patients Based on Intestinal Organoid Response To Different CFTR-modulators

Recruiting

Conditions: Cystic Fibrosis
Mucoviscidosis
10083624
10010613

Registration Number: NL-OMON48963

Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 75

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

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.

Study & Design

Study Type: Observational invasive

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Intestinal organoid response of 500 subjects to three drug products of<br /><br>different pharmaceutical companies, ranked by best response per drug product.</p><br>

Secondary Outcome Measures