Ignite Creation Date:
2024-07-17 @ 11:24 AM
Last Modification Date:
2024-10-26 @ 3:32 PM
Study NCT ID:
NCT06468527
Status:
RECRUITING
Last Update Posted:
2024-06-21
First Post:
2024-06-03
Brief Title:
Clinical Trial to Evaluate the Efficacy and Safety of DirocaftorPosenacaftorNesolicaftor in Adults With CF
Sponsor:
Kors van der Ent
Organization:
UMC Utrecht
Study Overview
Official Title:
A Phase IIb Multicentre Randomised Double-Blind Placebo-Controlled Crossover Study to Evaluate the Efficacy and Safety of DirocaftorPosenacaftorNesolicaftor in Subjects With Cystic Fibrosis Aged 18 Years or Older
Status:
RECRUITING
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
CHOICES
Brief Summary:
CF is caused by mutations in the gene that encodes the Cystic Fibrosis Transmembrane Conductance Regulator CFTR channel To re-establish the function of this complex chloride channel typically two to three drug modes of action are needed To date clinical studies of CFTR modulators have focused on patients carrying the F508del CFTR mutation which is present in approximately 80 of CF patients or gating mutations which are present in 5 of CF patients gating mutations result in a reduced opening of the CFTR-channel at the cell surface which limits the flow of chloride ions through the CFTR channel Although CF is a monogenetic disease the 15 remaining patients represent more than 2000 different rare and mostly uncharacterized CFTR mutations Multiple pharma companies have one or more CF drugs in their developmental pipeline However it is not known which patients may respond to the drugs in the pipeline It is hypothesized that by using individual patients intestinal organoids to screen for drug response a subset of patients with rare CFTR mutations can be identified who will clinically respond to drugs in the developmental pipeline The Human Individualized Therapy of CF HIT-CF project has been designed to further evaluate this hypothesis The project has received funding from the European Unions Horizon 2020 research and innovation program under grant agreement No 755021 The core of the project consists of a two-step approach to identify patients outside the existing drug label who may also benefit from CFTR-modulator treatment In the first step of the project HIT-CF Organoid Study NTR7520 novel CFTR modulators and their combinations were tested on organoids from over 500 European and Israeli CF patients with rare CFTR mutations to identify patients who are predicted to clinically benefit from these treatments The second step will evaluate the predicted clinical effect of the CFTR modulators in subjects identified by their organoid response to investigational products CFTR modulators from the HIT-CF participating pharmaceutical company FAIR Therapeutics will be evaluated in the CHOICES clinical study described in this protocol Data from this clinical study will be compared with the HIT-CF Organoid Study results to validate the organoid model
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?:
None