Available clinical trials
CHOICES trial – ex vivo portion of the trial ongoing, clinical portion to start in the second half of 2020
- Crossover trial based on Human Organoid Individual response in CF – Efficacy Study
- European trial designed to support marketing authorization in patients with rare mutations who are responsive in the organoid assay
Personalized medicine in cystic fibrosis
The goal of personalized medicine efforts in cystic fibrosis is to tailor treatment regimens for individuals based on how well they respond to drugs in a laboratory test. We are initiating a clinical trial to demonstrate that an ex vivo response is predictive of clinical efficacy for people with cystic fibrosis treated with our drug combination.
HIT-CF Europe initiative
We are participating in the HIT-CF Europe project to test our investigational drugs in rare mutations.
How does it work?
Participating CF centers across Europe began collecting rectal biopsies from people with CF with rare genetic mutations in January 2019 and completed enrollment of more than 500 patients in February of 2020.
- Participating subjects have provided rectal biopsies that will be used to make organoids (mini intestines) from each person’s biopsy.
- The organoids are currently being tested with our CFTR modulators to measure the potential functional response in the laboratory assay.
- Once the laboratory part of the trial is completed, we will initiate the clinical study with selected participants. The trial is designed to demonstrate that individuals whose organoids show ex vivo responsiveness to investigational drugs are also individuals who experience a clinical benefit when treated with those drugs.
This program aims to pave the way for regulatory approvals so that patients with rare mutations can also access therapeutic interventions, with the option of expansion to include patients with the F508del mutation.