RCT2100-101
Research type
Research Study
Full title
A Phase 1/2, Multicenter Study 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 Cystic Fibrosis
IRAS ID
1010013
Contact name
Heather Clark
Contact email
Sponsor organisation
Recode Therapeutics Inc.
Clinicaltrials.gov Identifier
Research summary
The study is being conducted in people with cystic fibrosis (CF), caused by specific mutations (changes) in the CF transmembrane conductance regulator (CFTR) gene that leads to blockage of the airways in the lung. There is currently no cure for CF. ReCode Therapeutics Inc. has developed a potential new treatment (RCT2100) for CF.
RCT2100 is an inhaled treatment 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. There are three parts to this study. Part 1 tested RCT2100 in healthy people to determine an acceptable dose. This part of the study, Part 2, will test the safety of several different dose levels of RCT2100 in people with CF to determine how much RCT2100 can be administered. In Part 2, up to 24 people with CF may be able to participate in one of several dose groups. The main goal of Part 2 is to find out whether RCT2100 is well tolerated, as well as how the body uses RCT2100, and how RCT2100 affects the body.
Part 2 of the study will involve approximately 20 centres in the United Kingdom, United States, Netherlands and France. It is open to adults with CF aged 18 or older, meeting the study criteria. Study participation will last up to 8 months. During the study treatment period, all participants will receive RCT2100, via a nebuliser. Participants will stay overnight in hospital for observation after having the first dose of RCT2100, and may be asked to return to the hospital for another overnight stay on Day 8 of the treatment period for the second dose of RCT2100. Further doses will be administered 3 times per week for the next 21 days until the end of the study treatment period. During the study treatment period, all participants will receive 10 total doses of RCT2100. There are either 15 or 17 total study visits, depending on assigned dose group. Some of these visits may be performed at the participant’s home by a nurse.REC name
North East - York Research Ethics Committee
REC reference
24/NE/0096
Date of REC Opinion
20 Jul 2024
REC opinion
Further Information Favourable Opinion