A trial investigating the safety and tolerability of a new mRNA based therapy (RCT2100) in people with CF
Details

- Therapeutic approach
- Genetic Therapy
- Trial status
- Open to recruitment Participating Centres
- Trials Tracker ID
- TT014421
- Last updated
- 21/10/2024
An early phase study evaluating the safety and tolerability of a new mRNA based therapy (RCT2100) with single doses in people without Cystic Fibrosis (Part 1) and multiple doses in people Cystic Fibrosis (Part 2)
Study detailsReCode Therapeutics Inc. has developed a potential new genetic therapy (RCT2100) for cystic fibrosis. 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 functional CFTR protein and help prevent further damage to the lungs.
Part 1 of the study tested RCT2100 in people without CF to determine doses to test in the remainder of the study. We are now recruiting people with CF for Part 2. In Part 2 of the study we will test the safety of different dose levels of RCT2100 in people with CF to determine how much RCT2100 can be administered. People with CF will 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, how the body uses RCT2100, and how RCT2100 affects the body.
If you are eligible and decide to participate in Part 2 of the study, a study doctor will share more information about the time expected from you during the study. There are three parts to the study:
- Screening period: involves a 2–4-hour clinic visit to determine your eligibility to participate.
- Treatment period: will take 4 weeks where you will receive RCT2100.
- Follow-up period: will take 24 weeks.
If you participate, you can expect to be in the study for about 28 weeks (~7 months).
- Phase
- Phase 1
- Length of participation
- Approximately 8 months
- Recruitment target
- 24
- CF sponsor
- ReCode Therapeutics
- CF sponsor type
- Commercial
Who can take part?
- Age range
- 18 years and older
- Including people
- People with cystic fibrosis ages 18 years or older
- Not eligible for CFTR modulators like Kaftrio based on gene mutation OR eligible for CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications
- FEV1 greater than or equal to 40% of predicted mean value for age, sex, and height
- Excluding people
- Taking a CFTR modulator therapy (such as Kalydeco, Trikafta, Symdeko, or Orkambi) within 12 weeks of screening
- With moderate or severe liver problems (hepatic impairment) or complications such as hepatic cirrhosis with portal hypertension or low liver function
- New or serious lung infections or low blood oxygen levels
Additional exclusion criteria may apply, please speak with your local CF team to explore if you are eligible for this study.
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