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ACTIVE NOT RECRUITINGPhase2

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

Sponsor: ReCode Therapeutics

NCT ID: NCT06237335

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
RCT2100 (drug), Placebo (other), RCT2100 (drug), RCT2100 (drug)
How long the study runs
Study runs about 33 months (dates as stated)
About the drug or intervention
RCT2100 — drug: RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer · Placebo — other: Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer · RCT2100 — drug: RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer for 4 weeks · RCT2100 — drug: RCT2100 supplied at a single dose strength administered via oral inhalation using nebulizer for 12 weeks · Ivacaftor — drug: ivacaftor administered orally for 6 weeks · RCT2100 — drug: RCT2100 supplied at varying dose strengths.
Patient visit burden
Not specified by the sponsor
Type of study
Testing a treatment
Ages
18 Years to 60 Years
Who
All
Number of participants
128
Started
2024-02-01
Last checked
2026-08

Plain English Summary

What is this study?

  • • Testing a new treatment for cystic fibrosis
  • • Phase2 - 128 participants
  • • This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies

Who can take part?

  • • Ages 18 Years to 60 Years
  • • Diagnosed with cystic fibrosis

Where?

  • • Birmingham - University Hospitals Birmingham
  • • Cambridge - Royal Papworth Hospital
  • • Leeds - Leeds Teaching Hospitals
  • • London - King's College Hospital
  • • +2 more UK sites

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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

More detail

This is a multi-part study to assess the safety, tolerability, and biodistribution of a single ascending dose of inhaled RCT2100 administered via nebulizer to healthy participants (Part 1), the safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered to participants with CF (Part 2), and the safety and tolerability of RCT2100 co-administered with ivacaftor in participants with CF (Part 3).

Cystic Fibrosis

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 18 Years - 60 Years
  • Who can join: All genders

What the study is looking for

  • ✓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
  • ✓Understands the study procedures in the agreement to take part form (ICF), and is willing and able to comply with the...
  • ✓Part 1 Major

Who cannot take part

  • ✗History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or...
  • ✗The participant has supine blood pressure (BP) \>150 mm Hg (systolic) or \>90 mm Hg (diastolic), following at least...
  • ✗The participant has abnormal clinical laboratory tests at screening, as assessed by the study-specific laboratory.
  • ✗Part 2 Major Inclusion Criteria:
  • ✗Confirmed diagnosis of CF
See the full 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 Is This Study? (6 UK sites)

University Hospitals Birmingham

Birmingham, United Kingdom

Hospital R&D contact (matched)

Sarah Pountain Head of Research Governance

R&D@uhb.nhs.uk0121 371 4185

Royal Papworth Hospital

Cambridge, United Kingdom

Hospital R&D contact (matched)

Vikki Hughes

papworth.randdenquiries@nhs.net01223 638000

Leeds Teaching Hospitals

Leeds, United Kingdom

Hospital R&D contact (matched)

R&I Team

leedsth-tr.researchfacilitation@nhs.net0113 2060469

King's College Hospital

London, United Kingdom

Hospital R&D contact (matched)

Jasmine Palmer

kch-tr.research@nhs.net0203 299 1980

Nottingham University Hospitals

Nottingham, United Kingdom

Hospital R&D contact (matched)

Alison Lloyd

nuhnt.researchsponsor@nhs.net0115 9249924

University Hospital Southampton

Southampton, United Kingdom

Hospital R&D contact (matched)

Dr Mikayala King

researchmanagement@uhs.nhs.uk023 81208215
Data sourced from ClinicalTrials.gov · Last verified: 2026-08