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Active, not recruiting Phase 2

A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF

NCT06237335 · tracked via the Priya Life Science France tracker
Sponsor
ReCode Therapeutics
Phase
Phase 2
Started
2024-02-01
Last updated
2026-08-19

Condition(s) studied

Cystic Fibrosis

Investigational drug(s) / intervention(s)

RCT2100PlaceboRCT2100RCT2100Ivacaftor →RCT2100

RCT2100: RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer

Placebo: Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer

RCT2100: RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer for 4 weeks

RCT2100: RCT2100 supplied at a single dose strength administered via oral inhalation using nebulizer for 12 weeks

Ivacaftor: ivacaftor administered orally for 6 weeks

RCT2100: RCT2100 supplied at varying dose strengths. Co- administered via oral inhalation using nebulizer for 4 weeks with ivacaftor after initial 2 weeks of ivacaftor dosing run in period

Study summary

This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.

Eligibility

Sex
ALL
Min age
18 Years
Max age
60 Years
Healthy volunteers
Accepted
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.

Primary outcome measure(s)

  • 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.

Trial sites (23)

FacilityCityRegionStatus
The University of Alabama at Birmingham Birmingham Alabama
University of Arizona Tucson Arizona
Stanford University Palo Alto California
UCSD San Diego California
National Jewish Health Denver Colorado
Emory University Atlanta Georgia
Boston Children's Hospital Boston Massachusetts
New York Medical College Valhalla New York
The University of North Carolina at Chapel Hill Chapel Hill North Carolina
Oregon Health & Science University Portland Oregon
University of Pittsburgh Pittsburgh Pennsylvania
UT Southwestern Medical Center Dallas Texas
University of Washington Seattle Washington
Centre Hospitalier Régional Universitaire de Montpellier - Hôpital Arnaud de Villeneuve Montpellier France
Hôpital Necker Enfants Malades Paris France
UMC Utrecht Utrecht Netherlands
New Zealand Clinical Research (Part 1 Only) Auckland New Zealand
University Hospitals Birmingham Birmingham United Kingdom
Royal Papworth Hospital Cambridge United Kingdom
Leeds Teaching Hospitals Leeds United Kingdom
King's College Hospital London United Kingdom
Nottingham University Hospitals Nottingham United Kingdom
University Hospital Southampton Southampton United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06237335 on ClinicalTrials.gov ↗ ← All trials in France