Evaluating the Safety, Tolerability, and Biodistribution of RCT2100 with Single-Ascending Doses in Healthy Participants and Multiple-Ascending Doses and Proof-of-Concept in Participants with CF

We have recently commenced a new study funded and sponsored by ReCode Therapeutics.

The study is being led at Nottingham University Hospitals by Dr Helen Barr of the University of Nottingham and NIHR Nottingham BRC.

What is the purpose of this study?

This study is being conducted and sponsored by ReCode Therapeutics, Inc., a pharmaceutical company based in the United States.

ReCode Therapeutics, Inc. is developing the experimental drug RCT2100 as a potential new treatment for patients who have CF due to specific mutations in their genetic code. Because of these mutations, the CFTR protein doesn’t work well and that leads to blockage of the bronchial tubes (airways in the lung) with thick mucus and inflammation. RCT2100 is a treatment you breathe in that mimics a natural substance in the body that makes the CFTR protein and may lead to the production of normal CFTR protein and help prevent further damage to the lungs.

RCT2100 is an experimental drug. This means that it is not an approved treatment in the United Kingdom In order to be approved, RCT2100 must be tested to see if it is a safe and effective treatment for people with CF.

There are three parts to this study. Part 1 tested RCT2100 in healthy people to determine how much RCT2100 can be administered and determined to be an acceptable dose. Part 2 will test the safety of RCT2100 in people with CF to determine how much RCT2100 can be administered and at what doses.  This RCT2100 Patient Information Sheet is only for Part 2 of the study.

The main aim of Part 2 of the clinical research study is to find out whether the study drug is well tolerated, as well as how your body uses the study drug and how the study drug affects your body. Part 2 will also provide information in order to be decided what dose will be used in Part 3 of the study, and possibly other studies.

In Part 2 up to 30 people with CF may be able to participate.  This study will involve approximately 20 Study Sites in, United States, the United Kingdom, Netherlands, and France.  

The expected number of participants to be enrolled in the UK is about 6.

To find out more, visit clinicaltrials.gov/study/NCT06237335