No registrations found.
ID
Source
Health condition
Cystic Fibrosis CF
Sponsors and support
Intervention
Outcome measures
Primary outcome
Intestinal organoid response of 500 subjects to three drug products of
different pharmaceutical companies, ranked by best response per drug product.
Secondary outcome
Not applicable
Background summary
The organoid study is part of the HIT-CF project. The aim of the Hit-CF project is to develop ‘personalized treatments’ for patients with Cystic Fibrosis (CF) and uncommon genetic profiles throughout Europe. It consist of 2 studies which will run after one another. In the first part, the organoid study, we will identify subjects who could potentially benefit from specific drugs. We will do this by using mini-intestines and test drugs on those mini-intestines. The second part of the project is a clinical trial. In that trial we will investigate whether or not the subject really benefits from the drugs that are identified in the organoid study.
This Organoid Study is the first part of the HIT-CF project. The purpose of this study is to investigate if CF-patients with rare mutations can benefit from a treatment with a CFTR-modulating drug. We will assess this by taking a small tissue sample (rectal biopsy) from 500 patients across Europe. In the laboratory, we will generate mini-intestines (called intestinal organoids) from the tissue. Using the organoids we have made we can test which drugs can repair the disturbed salt transport caused by CF.
We will test drugs on organoids of 500 CF-patients across Europe. The 100 patients whose organoids show the best response to the drugs will be asked to participate in a new clinical trial testing that specific drug.
Study objective
We can identify the predicted best clinical responders and predicted low-responders (based on amount of organoid swelling) to new CFTR-modulators out of 500 unique
patient-specific intestinal organoids screened for in-vitro drug efficacy.
Study design
- Biopsy collection
Intervention
Intestinal (rectal) biopsy by forceps or rectal suction device
Huispostnummer KH 01.419.0
Postbus 85090
S. Michel
Utrecht 3508 AB
The Netherlands
+31 (0)88 75 537 25
s.michel-2@umcutrecht.nl
Huispostnummer KH 01.419.0
Postbus 85090
S. Michel
Utrecht 3508 AB
The Netherlands
+31 (0)88 75 537 25
s.michel-2@umcutrecht.nl
Inclusion criteria
–Male or female with confirmed diagnosis of CF
–Adult age on the date of informed consent
–An increased sweat chloride concentration (above 60 mmol/L) by pilocarpine iontophoresis (documented in patient records)
–Subject has signed and dated an ICF
Exclusion criteria
–Subject has at least one of the following CFTR-mutations:
•F508del, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H, A455E, 3849+10kbC>T
–Subject has a combination of any two of the following mutations:
•G542X, R553X, W1282X, R1162X, E60X, Q493X, 1717-1G>A, 621+1G>T, 3120+1G>A, 1898+1G->A, CFTRdele2,3 and 2183AA->G
–History of any comorbidity that might pose an additional risk in administering study drug to subject
–History of Lung Transplantation
Design
Recruitment
Followed up by the following (possibly more current) registration
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL7304 |
| NTR-old | NTR7520 |
| CCMO | NL65123.041.18 |
| OMON | NL-OMON48963 |