Phase 2 Study of RCT2100 for Cystic Fibrosis
This study is testing a new inhaled medication called RCT2100 for cystic fibrosis (CF). It's the first time RCT2100 is being given to people, and the main goal is to see how safe it is and if people can tolerate it well. The study will involve healthy volunteers and people with CF. Some participants with CF will also take RCT2100 along with an existing CF medicine called ivacaftor. The study aims to enroll 192 participants aged 18 to 60 years old. The current recruitment status is unclear.
- Study design
- This is a multi-part study with an estimated enrollment of 192 participants. It will assess single and multiple doses of RCT2100, and also RCT2100 given with ivacaftor.
- What's involved
- Participants will receive RCT2100 or a placebo (an inactive substance) via an oral inhaler (nebulizer) for 4 or 12 weeks. Some participants will also take ivacaftor orally for 6 weeks.
- Compensation
- Not stated in the trial record.
- Follow-up
- Healthy participants will be followed for safety through day 29. Participants with CF will be followed for safety through week 24.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT06237335
A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
Active, Not Recruiting
PHASE2Ages 18–60InterventionalTreatmentReCode Therapeutics
~128 participants
Updated 2026-08-19 on ClinicalTrials.gov
What's tested:RCT2100PlaceboIvacaftor
At a glance
Recruiting sites
0 of 23 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Part 1: The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).
Measured over From Baseline Through Day 29
+2 more outcomes measured
Conditions
Where it's being run
23 sites across 16 statesUnited Kingdom6
California2
France2
Alabama1
Arizona1
Colorado1
Georgia1
Massachusetts1
Study leadership
- John Matthews, MBBS, MCRP, PhD · STUDY_CHAIR · ReCode Therapeutics, Inc.
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Healthy, adult, male or female, 18-55 years of age, inclusive, at screening.
Body weight greater than or equal to 50 kg and body mass index (BMI) between 16-32 kg/m2, inclusive
The participant has a forced expiratory volume in one second (FEV1) of at least 80% predicted
The participant is considered by the investigator to be in good general health as determined by medical history, clinical laboratory test results, vital sign measurements, 12-lead ECG results, and physical examination findings at screening.
Understands the study procedures in the informed consent form (ICF), and is willing and able to comply with the protocol.
Confirmed diagnosis of CF
Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height
a) Not eligible for CFTR modulators based on having mutations of CFTR gene on both alleles that are not responsive to CFTR modulator therapy OR
b) Eligible for CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications
Confirmed diagnosis of CF
Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height
a) Not eligible for CFTR modulators based on having mutations of CFTR gene on both alleles that are not responsive to CFTR modulator therapy OR
b) Eligible for dual or triple CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications
Exclusion
History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease.
The participant has supine blood pressure (BP) \>150 mm Hg (systolic) or \>90 mm Hg (diastolic), following at least 5 minutes of supine rest.
The participant has abnormal clinical laboratory tests at screening, as assessed by the study-specific laboratory.
The participant is a smoker or has used nicotine or nicotine-containing products 6 weeks before the first dose of study drug. Former smokers with greater than 10 pack years of smoking history are excluded.
Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for sinopulmonary disease within 4 weeks before the first dose of study drug
Lung infection with organisms associated with a more rapid decline in pulmonary status
Arterial oxygen saturation on room air less than 94% at screening
Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, Orkambi, or Alyftrek) within 12 weeks of Screening
Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for sinopulmonary disease within 4 weeks before the first dose of study drug
Lung infection with organisms associated with a more rapid decline in pulmonary status
Arterial oxygen saturation on room air less than 94% at screening
Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, Orkambi, or Alyftrek) within 12 weeks of Screening
What this trial measures
- Part 1: The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).From Baseline Through Day 29
Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
- Part 2: The number of participants with CF with AEs and SAEs.From Day 1 through Safety Follow-up, Week 24
Safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered to participants with CF
- Part 3: The number of participants with CF with AEs and SAEs.From Day 1 through Safety Follow-up, Week 24
To assess the safety and tolerability of RCT2100 co-administered with ivacaftor in participants with CF.