Validation of in vitro counterpart models to enable personalized care in CF

Validation of *in vitro counterpart* models to enable personalized care in CF - VICI CF study

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON40312

Enrollment

Registered

2013-09-10

Start date

2013-12-12

Completion date

Unknown

Last updated

2024-04-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

cystic fibrosis

Interventions

None listed

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: - Being able to sign informed consent form (ICF). - Males and females aged 18 to 40 years on the date of informed consent and homozygous for the F508del-CFTR mutation (as documented in the subject's medical record) or: - Males and females aged 18 years or older on the date of informed consent and hetrozygous for the compound/A455E mutation (as documented in the subject's medical record)

Exclusion criteria

Exclusion criteria: CF patients with: Instabile CF-related diabetes Active ABPA (allergic bronchopulmonary aspergillosis) Other major CF related complications (CF related liver disease with abnormal coagulation).

Design outcomes

Primary

Measure	Time frame
Primary endpoint: CFTR activity (expressed as absolute swelling of organoids, and % apical CFTR-expressing nasal epithelial cells) measured in intestinal organoids and nasal epithelial cells.	—

Secondary

Measure	Time frame
Secondary endpoints: Clinical correlates: Intestinal current measurement (ICM) in rectal biopsies; Current lung function and at age 12; Slope of lung function decline; Exacerbation rate; Current nutritional status and at age 12; Current chronic infection with Pseudomonas and Staphylococci and at age 12; Current Chest X-ray Chrispin Norman score and at age 12; current Chest CT Brodi score.	—

Countries

Netherlands

Outcome results