A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
NCT ID: NCT06237335
Last Updated: 2026-01-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
192 participants
INTERVENTIONAL
2024-02-01
2026-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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RCT2100 (Part 1)
RCT2100 single dose
RCT2100
RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer
Placebo (Part 1)
Placebo single dose
Placebo
Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer
RCT2100 (Part 2) 4 week
RCT2100 multiple dose
RCT2100
RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer for 4 weeks
RCT2100 (Part 2) 12 week
RCT2100 multiple dose
RCT2100
RCT2100 supplied at a single dose strength administered via oral inhalation using nebulizer for 12 weeks
Experimental: RCT2100 (Part 3) 6 week
RCT2100 multiple dose
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
Interventions
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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
Eligibility Criteria
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Inclusion Criteria
* 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 Criteria
* 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
18 Years
60 Years
ALL
Yes
Sponsors
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ReCode Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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John Matthews, MBBS, MCRP, PhD
Role: STUDY_CHAIR
ReCode Therapeutics, Inc.
Locations
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The University of Alabama at Birmingham
Birmingham, Alabama, United States
University of Arizona
Tucson, Arizona, United States
Stanford University
Palo Alto, California, United States
UCSD
San Diego, California, United States
National Jewish Health
Denver, Colorado, United States
Emory University
Atlanta, Georgia, United States
Boston Children's Hospital
Boston, Massachusetts, United States
New York Medical College
Valhalla, New York, United States
The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Oregon Health & Science University
Portland, Oregon, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
UT Southwestern Medical Center
Dallas, Texas, United States
University of Washington
Seattle, Washington, United States
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
Countries
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Central Contacts
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Other Identifiers
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2024-512169-15
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
RCT2100-101
Identifier Type: -
Identifier Source: org_study_id
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