A phase 2a study evaluating the safety and tolerability of a new mRNA therapy (RCT2100) in combination with ivacaftor in people with CF

Full title

A phase 2a study to evaluate inhaled mRNA-based therapy RCT2100 in combination with ivacaftor in adults with CF who are not eligible for or not taking CFTR modulator therapy (ReCode Phase 2a IVA Combination Cohort)

Study details

This phase 2a study follows a phase 1 study, which looked at the safety and tolerability of a new inhaled mRNA therapy, RTC2100, when taken by itself. 

This phase 2a study will evaluate the safety and tolerability of RTC2100 in combination with ivacaftor, in adults with CF who are not eligible for CFTR modulator therapy or who are not able to take CFTR modulator therapy.

Ivacaftor is a CFTR modulator therapy. In people with CF, the CFTR protein doesn’t properly regulate the flow of water and chloride in and out of the cells lining the lungs and other organs. Ivacaftor works by binding to the CFTR protein at the cell surface and opening the chloride channel so that chloride can flow through.

An mRNA therapy delivers the correct genetic instructions to cells by providing a new, functional copy of the CFTR mRNA. It is hoped that this will result in a functional CFTR protein. 

Pre-clinical lab studies looking at cells from people with CF show that RTC2100 and ivacaftor work better together. Based on these lab studies, combining RCT2100 with ivacaftor will allow better movement of chloride through the channel and potentially improve CFTR function in the lungs.

Find out more about CF genetic therapies and trials.

This study is an open-label study, meaning everyone taking part will receive RTC2100 in combination with ivacaftor. They will receive doses of the study drug based on results of Phase 1b of this study. Researchers will assess safety and tolerability by monitoring for any changes in their health.

People may be asked to provide blood samples, lung function tests, electrocardiograms, and/or other measurements.

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