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Evaluating the safety of RCT2100 in healthy individuals and those with cystic fibrosis

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

Phase 2 Interventional ReCode Therapeutics · NCT06237335

This study is testing a new inhaled treatment called RCT2100 to see if it's safe for healthy people and those with cystic fibrosis.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment192 (estimated)
Ages18 Years to 60 Years
SexAll
SponsorReCode Therapeutics Industry-sponsored
Locations23 sites (Birmingham, Alabama and 22 other locations)
Trial IDNCT06237335 on ClinicalTrials.gov

What this trial studies

This Phase 1 study aims to assess the safety and tolerability of RCT2100, an inhaled treatment, in both healthy participants and individuals diagnosed with cystic fibrosis (CF). The study is divided into two parts: the first part involves healthy adults receiving a single ascending dose of RCT2100, while the second part involves participants with CF receiving multiple ascending doses. The study will monitor the biodistribution of the drug and gather data to inform future clinical trials.

Who should consider this trial

Good fit: Ideal candidates include healthy adults aged 18-55 and individuals diagnosed with cystic fibrosis.

Not a fit: Patients with significant medical or psychiatric conditions or those with uncontrolled hypertension may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to a new treatment option for patients with cystic fibrosis, improving their quality of life.

How similar studies have performed: While this is a first-in-human study, similar approaches in drug development for cystic fibrosis have shown promise in previous trials.

Eligibility criteria

Show full inclusion / exclusion criteria 
Part 1 Major Inclusion Criteria:

* 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.

Part 1 Major Exclusion Criteria:

* 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.

Part 2 Major Inclusion Criteria:

* 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

Part 2 Major Exclusion Criteria:

* 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

Other protocol defined Inclusion/Exclusion criteria may apply.

Part 3 Major Inclusion Criteria:

* 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

Part 3 Major Exclusion Criteria:

* 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

Other protocol defined Inclusion/Exclusion criteria may apply.

Where this trial is running

Birmingham, Alabama and 22 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Cystic FibrosisCFmRNA
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.