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

Details

CTAP badge
Therapeutic approach
Genetic Therapy
Trial status
Open to recruitment Participating Centres
Trials Tracker ID
TT014421
Last updated
21/10/2024

Full title

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 details

ReCode 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:

  1. Screening period: involves a 2–4-hour clinic visit to determine your eligibility to participate.
  2. Treatment period: will take 4 weeks where you will receive RCT2100.
  3. 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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CF centres running this trial

Recruiting

Birmingham Heartlands Hospital (Adults)

CTAP centre
NHS Trust
University Hospitals Birmingham NHS Foundation Trust
Address
Bordesley Green
B9 5SS
Recruitment starts
Recruitment ends
Trial Coordinators
Gurcharan (Robbie) Singh
Recruiting

Cambridge Royal Papworth Hospital (Adults)

CTAP centre
NHS Trust
Royal Papworth Hospital NHS Foundation Trust
Address
Papworth Road
CB2 0AY
Recruitment starts
Recruitment ends
Trial Coordinators
Victoria Christenssen
In Setup

King's College Hospital (London) - Adults

CTAP centre
NHS Trust
King's College Hospital NHS Foundation Trust
Address
King's College Hospital
Denmark Hill
SE5 9RS
Recruitment starts
Recruitment ends
Trial Coordinators
Mélanie Le Sayec
Recruiting

Nottingham City Hospital (Adults)

CTAP centre
NHS Trust
Nottingham University Hospitals NHS Foundation Trust
Address
City Hospital, Wolfson Cystic Fibrosis Centre
Hucknall Road
NG5 1PB
Recruitment starts
Recruitment ends
Trial Coordinators
Fozia Naushahi
Recruiting

Southampton General Hospital (Adults)

CTAP centre
NHS Trust
University Hospital Southampton NHS Foundation Trust
Address
Tremona Road
SO16 6YD
Recruitment starts
Recruitment ends
Trial Coordinators
Ben Wilson
Lorraine Hewitt
Recruiting

St James's University Hospital - Leeds (Adults)

CTAP centre
NHS Trust
The Leeds Teaching Hospitals NHS Trust
Address
St James's University Hospital
Beckett Street
LS9 7TF
Recruitment starts
Recruitment ends
Trial Coordinators
Helen Chadwick