People with CF who were 16 years of age* or older at the day of signing the informed consent for biopsy taking were enrolled in this European project. They have a confirmed diagnosis of CF and a rare genetic profile (see below: Is my genetic profile rare?). They made one visit to one of the participating hospitals to collect rectal biopsies for making their unique organoids. Also, if they are selected for a clinical trial they will need to visit this hospital for probably 6-8 times.
*18 years or older in some countries because of national laws
Is my genetic profile rare?
Only patients with rare genotypes could participate in the HIT-CF Europe project. Patients with the following genetic profiles could not participate:
- one of the following mutations: F508del, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H, A455E, 3849+10kbC>T, or
- a combination of any two of the following mutations: G542X, 1717-1G>A, 621+1G>T, 3120+1G>A, 1898+1G->A, CFTRdele2,3 and 2183AA->G
What were additional inclusion and exclusion criteria?
Inclusion criteria -> patients participated if:
- They had a confirmed diagnosis of CF by their doctor. CF must have been diagnosed by the following:
- The new-born screen or one or more CF symptoms or sibling(s) with CF
- A sweat test result (SSC) higher than 60 mmol/L
Exclusion criteria -> patients could not participate in this project if:
- They had an additional disease (comorbidity) that increases the risk of administering a drug candidate would they be selected for a clinical trial. For example, they had severe liver disease. During their first visit for the project, the investigator decided whether they had a comorbidity that might put them at risk for this study.
- They have had a lung transplantation.
What is the timeline for this project?
People with CF could apply for participating in this project from March 2018 onwards in one of the participating CF-centers. From April 2018 to February 2020 biopsies were taken from over 500 people with CF .
The subsequent laboratory tests of drug candidates on organoids will be finished by June 2020.
Based on the responses in organoids, 78 patients will be selected for clinical trials. The clinical trials with the drug candidates will start at the second half of 2020.
The HIT-CF Europe project is carried out in specific CF-Centers of the Clinical Trial Network (CTN) of the European CF Society (ECFS) (see map).
Recruitment has ended in February 2020. 47 sites from 16 countries throughout Europe contributed to enroll 502 patients.
For more information, you can also contact HIT-CF Europe directly via HITCF@umcutrecht.nl.
Additional important information
If you sign an additional consent, your mini-intestines will be stored in a biobank for future research.
For more answers to your questions, see our FAQ.
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